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Oct 18
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Multiple Clinical Complete Responses Demonstrated Following Single Low Dose Administration of Bel-sar in Patients with Non-Muscle-Invasive Bladder Cancer (NMIBC) in Ongoing Phase 1 Trial

By Aura Biosciences, Press Release, Private Companies
Press Release.

 

Multiple Clinical Complete Responses Demonstrated Following Single Low Dose Administration of Bel-sar in Patients with Non-Muscle-Invasive Bladder Cancer (NMIBC) in Ongoing Phase 1 Trial

October 17, 2024

Clinical Complete Responses Observed in 4 out of 5 Patients in Subset of Patients with Low Grade Disease; Evidence of Bladder Urothelial Field Effect in Non-Target Tumors

Favorable Safety Profile Observed; Only Grade 1 Drug-Related Adverse Events Reported in Less Than 10% of Patients

Aura Hosting Virtual Urologic Oncology Investor Event with Key Opinion Leaders at 4.30 pm ET Today

BOSTON, Oct. 17, 2024 (GLOBE NEWSWIRE) — Aura Biosciences, Inc. (NASDAQ: AURA), today announced positive early data from an ongoing Phase 1 clinical trial of bel-sar (AU-011) in patients with NMIBC. To date, the trial includes 13 patients, with the primary endpoints of evaluating the safety and feasibility of local administration of bel-sar alone (n=5) and bel-sar with light activation (n=8). The secondary endpoints are to evaluate biological activity and immune mediated changes in the tumor microenvironment (TME). 10 of 13 study participants had low grade disease, approximating the 70% incidence of this patient population among all NMIBC patients. The other 3 study participants had high grade disease. In patients receiving bel-sar with light activation (n=8), 4 out of 5 patients with low grade disease demonstrated a clinical complete response with no tumor cells remaining on histopathological evaluation. 2 out of 3 patients with high grade disease demonstrated visual tumor shrinkage observed on cystoscopy. Aura will host a Virtual Urologic Oncology Investor Event at 4:30 pm ET today.

“We are highly encouraged by this positive early data, which shows that bel-sar has the potential to be a transformative cancer treatment,” said Sabine Brookman-May, MD, FEBU, Senior Vice President, Therapeutic Area Head Urologic Oncology of Aura Biosciences. “A potentially differentiating aspect of this novel treatment is the rapid tumor response accompanied by an immune oncology (IO) effect such as a marked CD8+ T-cell infiltration observed in just a matter of days with a single low dose. We believe this could have the potential to translate into a durable response. In parallel with expanding the ongoing Phase 1 trial, we are preparing for a Phase 2 trial to further evaluate bel-sar’s clinical activity and durability of response.”

“Bel-sar has the potential to change the treatment paradigm for NMIBC,” said Neal Shore, MD, FACS, Medical Director, Carolina Urologic Research Center, AUC Urology Specialists. “Based on this early data, bel-sar’s positive clinical activity and evidence of a bladder urothelial field effect with a single dose, may position bel-sar to be the first immune ablative treatment option for early-stage bladder cancer patients delivered with an in-office procedure.”

Bel-sar is a virus-like drug conjugate, designed to have a dual mechanism of action, that induces direct tumor cell necrosis and elicits a robust and durable anti-tumor immune response.

Trial Design: The ongoing Phase 1 trial (NCT05483868) is a two-part, open-label clinical trial, designed to assess the safety and feasibility of bel-sar as a monotherapy. The study treatment is administered 7 to 12 days before the scheduled transurethral resection of bladder tumor (TURBT), the standard of care procedure. The participants are followed for safety monitoring over a 56-day period. The trial is also evaluating bel-sar’s biological activity with histopathological evaluation of tissue samples collected at the time of TURBT (regardless of tumor response) with evaluation of focal necrosis and immune changes in the tumor microenvironment. Part 1 (n=5) of the trial is complete, with patients receiving a single bel-sar dose without light activation. Part 2 (n=10) of the trial is ongoing. 8 patients with a confirmed tumor at time of treatment have received either 100ug or 200ug of bel-sar as a single dose. Of these 8 patients, 5 had low grade disease and 3 had high grade disease. 7 of these 8 patients had a history of recurrent bladder cancer and had undergone multiple TURBTs and adjuvant treatments such as Bacillus Calmette-Guerin (BCG), mitomycin, gemcitabine, cetrelimab and tamoxifen prior to trial enrollment. In the Phase 1 trial expansion, Aura plans to test additional doses and treatment regimens.

Safety Data: In the safety analysis as of the September 9, 2024 data cut-off date (n=12), bel-sar was well-tolerated, with less than 10% of patients reporting Grade 1 and no Grade 2 or higher drug-related adverse events reported. No serious adverse events have been reported. No significant differences between the light-activated and non-light activated cohorts have been observed.

Biological Activity: The data in these 8 patients receiving bel-sar with light activation showed clinical activity detectable as soon as 7 days after a single low dose of bel-sar with light activation. This was demonstrated by histopathological evidence of clinical complete response, necrosis, immune activation or visual tumor shrinkage observed on cystoscopy. For this analysis, “clinical complete response” was defined as the absence of tumor cells on histopathologic evaluation. Of the patients with low-grade disease, 4 out of 5 exhibited a clinical complete response (1 of 4 based on local pathology with central review ongoing), with no tumor cells detected in histopathological evaluation post-treatment in the target and in several non-target bladder tumors. 2 of 3 of the patients with high grade disease demonstrated visual tumor shrinkage observed on cystoscopy, while tumor cells were still present on histopathological evaluation. Immune activation was noted in all patients in both treated target and untreated non-target bladder tumors with infiltration of effector CD8+ and CD4+ T-cells (where immune staining was available). This data provides evidence of a bladder urothelial field effect with a single low dose of bel-sar with light activation, potentially indicating a broader immune response in the bladder beyond the target tumor in these patients.

Aura Virtual Urologic Oncology Investor Event

Aura will host a Virtual Urologic Oncology Investor Event featuring Max Kates, MD (Johns Hopkins), Joe Jacob, MD (Syracuse University), Neal Shore, MD (Carolina Urologic Research Center) and Gary Steinberg, MD (RUSH University) to discuss the early Phase 1 data on Thursday, October 17, 2024, at 4:30 pm Eastern Time. To register for the event, click here. A live question and answer session will follow the formal discussion.

The live webcast of Aura’s Virtual Urologic Oncology Investor Event will be available on the “Investors & Media” page under the “Events & Presentations” section of Aura’s website at https://ir.aurabiosciences.com/events-and-presentations, where a replay of the webcast will be archived for 90 days following the presentation date.

About Aura Biosciences

Aura Biosciences is a clinical-stage biotechnology company focused on developing precision therapies for solid tumors that aim to preserve organ function. Our lead candidate, bel-sar (AU-011), is currently in late-stage development for primary choroidal melanoma and in early-stage development in other ocular oncology indications and bladder cancer. Aura Biosciences is headquartered in Boston, MA. Our mission is to grow as an innovative global oncology company that positively transforms the lives of patients.

For more information, visit aurabiosciences.com. Follow us on X (formerly Twitter) @AuraBiosciences and visit us on LinkedIn.

Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, and other federal securities laws. Any statements that are not statements of historical fact may be deemed to be forward-looking statements. Words such as “may,” “will,” “could,” “should,” “expects,” “intends,” “plans,” “anticipates,” “believes,” “estimates,” “predicts,” “projects,” “seeks,” “endeavor,” “potential,” “continue” or the negative of such words or other similar expressions can be used to identify forward-looking statements. These forward-looking statements include express or implied statements regarding Aura’s future expectations, plans and prospects, including, without limitation, statements regarding the therapeutic potential of bel-sar for the treatment of cancers including bladder cancer; statements regarding Aura’s plans and expectations for the ongoing Phase 1 and future trials of bel-sar for bladder cancer; statements regarding Aura’s beliefs and expectations for bel-sar’s ability to provide durable responses in bladder cancer patients; statements regarding Aura’s expectations for an improved quality of life of patients after treatment with bel-sar and changes to the treatment paradigm for patients; statements regarding Aura’s beliefs and expectations for the high unmet medical need for an effective local treatment in urologic oncology; and statements regarding Aura’s expectations for the estimated patient populations and related market opportunities for bel-sar.

The forward-looking statements in this press release are neither promises nor guarantees, and investors should not place undue reliance on these forward-looking statements because they involve known and unknown risks, uncertainties and other factors, many of which are beyond Aura’s control and which could cause actual results to differ materially from those expressed or implied by these forward-looking statements, including, without limitation, uncertainties inherent in clinical trials and in the availability and timing of data from ongoing clinical trials; the expected timing for submissions for regulatory approval or review by governmental authorities; the risk that the results of Aura’s preclinical and clinical trials may not be predictive of future results in connection with future clinical trials; the risk that early data from ongoing clinical trials may not be predictive of final data from completed clinical trials; the risk that governmental authorities may disagree with Aura’s clinical trial designs; whether Aura will receive regulatory approvals to conduct trials or to market products; whether Aura’s cash resources will be sufficient to fund its foreseeable and unforeseeable operating expenses and capital expenditure requirements; Aura’s ongoing and planned preclinical activities; and Aura’s ability to initiate, enroll, conduct or complete ongoing and planned clinical trials. These risks, uncertainties and other factors include those risks and uncertainties described under the heading “Risk Factors” in Aura’s most recent Annual Report on Form 10-K and Quarterly Report on Form 10-Q filed with the United States Securities and Exchange Commission (SEC) and in subsequent filings made by Aura with the SEC, which are available on the SEC’s website at www.sec.gov. Except as required by law, Aura disclaims any intention or responsibility for updating or revising any forward-looking statements contained in this press release in the event of new information, future developments or otherwise. These forward-looking statements are based on Aura’s current expectations and speak only as of the date hereof and no representations or warranties (express or implied) are made about the accuracy of any such forward-looking statements.

Investor Contact:

Alex Dasalla

Head of Investor Relations and Corporate Communications

IR@aurabiosciences.com

Media Contact:

Kimberly Ha

KKH Advisors

kimberly.ha@kkhadvisors.com

917-291-5744

Oct 09
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U.S. Food and Drug Administration (FDA) accepts New Drug Application for elinzanetant

By Advent Life Sciences, KaNDy Therapeutics, Press Release
Press Release.

 

U.S. Food and Drug Administration (FDA) accepts New Drug Application for elinzanetant

  • The New Drug Application (NDA) for elinzanetant, an investigational compound for the treatment of moderate to severe vasomotor symptoms associated with menopause, is based on the positive data from three Phase III studies OASIS 1, 2 and 3
  • Elinzanetant is the first dual neurokinin-1 and 3 (NK-1 and 3) receptor antagonist, in late-stage clinical development for the non-hormonal treatment of moderate to severe VMS associated with menopause, administered orally once daily
BERLIN–(BUSINESS WIRE)–Bayer today announced that the U.S. Food and Drug Administration (FDA) has accepted the company’s New Drug Application (NDA) for the investigational compound elinzanetant seeking approval for the treatment of moderate-to-severe vasomotor symptoms (VMS, also known as hot flashes) associated with menopause.

“If approved, elinzanetant will offer a new non-hormonal option to women seeking treatment for moderate to severe vasomotor symptoms and we look forward to the review by the agency.”

Post this

“The NDA acceptance of elinzanetant by the FDA marks a significant milestone in our efforts to advance menopause care for women in the U.S.,” said Christine Roth, Executive Vice President, Global Product Strategy and Commercialization, Member of the Pharmaceutical Leadership Team at Bayer.“If approved, elinzanetant will offer a new non-hormonal option to women seeking treatment for moderate to severe vasomotor symptoms and we look forward to the review by the agency.”

The NDA application is based on the positive results from the OASIS 1, 2 and 3 Phase III studies evaluating the efficacy and safety of the investigational compound elinzanetant versus placebo. Findings from OASIS 1 and 2 were published in August 2024 in JAMA.1 Detailed results of the Phase III study OASIS 3 providing supporting efficacy and additional long-term safety data were presented at The Menopause Society (TMS) annual meeting in September 2024.

Bayer is continuing to submit applications for marketing authorizations of elinzanetant to further health authorities as well.

About the Elinzanetant clinical development program
The Phase III clinical development program of elinzanetant, OASIS, currently comprises four Phase III studies: OASIS 1, 2, 3 and 4. OASIS 1 and 2 investigated the efficacy and safety of elinzanetant administered orally once daily in women with moderate to severe VMS associated with menopause over 26 weeks and randomized 396 and 400 postmenopausal women between 40 and 65 years across 184 sites in 15 countries. Patients in the elinzanetant arm received a 120 mg dose of elinzanetant once daily for 26 weeks and patients in the control arm received a matching placebo once daily for 12 weeks, followed by elinzanetant 120 mg dose for 14 weeks. OASIS 3 investigated the efficacy and safety of elinzanetant for the treatment of vasomotor symptoms over 52 weeks in postmenopausal women and randomized 628 postmenopausal women between 40 and 65 years across 83 sites in 9 countries. The OASIS 4 study is an expansion of the clinical Phase III program and investigates the efficacy and safety of elinzanetant in women with moderate to severe VMS caused by endocrine therapy for treatment or prevention of breast cancer.

In addition to the OASIS program, Bayer is conducting NIRVANA (NCT06112756), an exploratory Phase II randomized, parallel-group treatment, double-blind study. The primary objective is to explore the efficacy of elinzanetant on sleep disturbances associated with menopause (SDM) as determined by polysomnography (PSG). PSG is a validated method to study sleep and underlying causes of sleep disturbances. Additional objectives include exploring the efficacy of elinzanetant on SDM as determined by patient-reported outcomes and further evaluating the safety of elinzanetant.

About Elinzanetant
Elinzanetant is the first dual neurokinin-1 and 3 (NK-1 and 3) receptor antagonist, in late-stage clinical development for the non-hormonal treatment of moderate to severe VMS associated with menopause, administered orally once daily. Elinzanetant may address moderate to severe VMS by modulating a group of estrogen sensitive neurons in the hypothalamus region of the brain (the KNDy neurons) which, with the decrease of estrogen, become hypertrophic and lead to a hyperactivation of the thermoregulatory pathway, consequently disrupting body heat control mechanisms resulting in VMS. Based on key secondary endpoints of OASIS 1 and 2, Elinzanetant may also decrease sleep disturbances associated with menopause.

About Vasomotor Symptoms
Vasomotor symptoms (VMS; also referred to as hot flashes) result from hyperactivation of the thermoregulatory pathway mediated by hypertrophy of the KNDy neurons. This is due to a decrease of estrogen, which can result from the progressive reduction of ovarian function due to natural menopause or medical intervention by bilateral oophorectomy or endocrine therapy.

VMS are reported by up to 80% of women at some point during the menopausal transition and are one of the leading causes for seeking medical attention during this phase of a woman’s life. Over one-third of menopausal women report severe symptoms, which can last 10 years or more after the last menstrual period, with relevant impact on quality of life.

VMS may also be caused by endocrine therapy, for the treatment or prevention of breast cancer, impacting quality of life and treatment adherence. For these women, there are currently no approved treatment options.

About Menopause
By 2030, the global population of women experiencing menopause is projected to increase to 1.2 billion, with 47 million women entering this phase each year. Menopause is a transitional phase in women’s lives, related to the progressive decline of ovarian function. It usually occurs in women during their 40s or early 50s. It can also be the result of surgical or medical treatment such as breast cancer treatment. The hormonal decline can lead to various symptoms which can substantially affect a woman’s health, quality of life, healthcare utilization and work productivity. The most frequently reported and disruptive symptoms during the menopausal transition are VMS, sleep disturbances and mood changes. Addressing these symptoms is key to maintaining functional ability and quality of life in menopause which is highly relevant from both a healthcare and socio-economic perspective.

About Women’s Healthcare at Bayer
Women’s Health is in Bayer’s DNA. As a global leader in women’s healthcare Bayer has a long-standing commitment to delivering science for a better life by advancing a portfolio of innovative treatments. Bayer offers a wide range of effective short- and long-acting birth control methods as well as therapies for menopause management and gynecological diseases. Bayer is also focusing on innovative options to address the unmet medical needs of women worldwide and to broadening treatment choices such as in menopause. Additionally, Bayer intends to provide 100 million women per year in low-and-middle income countries by 2030 with access to family planning by funding multi-stakeholder aid programs for capacity building and by ensuring the supply of affordable modern contraceptives. This is part of the comprehensive sustainability measures and commitments from 2020 onwards and in line with the Sustainable Development Goals of the United Nations.

About Bayer
Bayer is a global enterprise with core competencies in the life science fields of health care and nutrition. In line with its mission, “Health for all, Hunger for none,” the company’s products and services are designed to help people and the planet thrive by supporting efforts to master the major challenges presented by a growing and aging global population. Bayer is committed to driving sustainable development and generating a positive impact with its businesses. At the same time, the Group aims to increase its earning power and create value through innovation and growth. The Bayer brand stands for trust, reliability and quality throughout the world. In fiscal 2023, the Group employed around 100,000 people and had sales of 47.6 billion euros. R&D expenses before special items amounted to 5.8 billion euros. For more information, go to www.bayer.com.

Find more information at https://pharma.bayer.com/
Follow us on Facebook: http://www.facebook.com/bayer
Follow us on LinkedIn: Bayer | Pharmaceuticals

kw (2024-0157E)

Forward-Looking Statements
This release may contain forward-looking statements based on current assumptions and forecasts made by Bayer management. Various known and unknown risks, uncertainties and other factors could lead to material differences between the actual future results, financial situation, development or performance of the company and the estimates given here. These factors include those discussed in Bayer’s public reports which are available on the Bayer website at www.bayer.com. The company assumes no liability whatsoever to update these forward-looking statements or to conformthem to future events or developments.

_________________________
1 https://jamanetwork.com/journals/jama/fullarticle/2822766

Contacts

Contact for US media inquiries:
Courtney Ambrosi, phone 1 (908) 798-1107
Email: courtney.ambrosi@bayer.com

Contact for global media inquiries:
Katja Wiggers, phone +49 30 221541614
Email: katja.wiggers@bayer.com

Oct 08
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Astellas and AviadoBio Announce Exclusive Option and License Agreement for Gene Therapy AVB-101 Targeting Frontotemporal Dementia and Other Indications

By AviadoBio, Press Release, Private Companies
Press Release.

 

Astellas and AviadoBio Announce Exclusive Option and License Agreement for Gene Therapy AVB-101 Targeting Frontotemporal Dementia and Other Indications

  • Astellas receives an exclusive option to license AVB-101, an investigational, AAV-based gene therapy in Phase 1/2 development
  • AviadoBio receives $20 million equity investment, up to $30 million in upfront payments and is eligible to receive up to $2.18 billion in license fees and milestone payments plus royalties if Astellas exercises its option

LONDON, and TOKYO, – October 8, 2024 – AviadoBio Ltd. (“AviadoBio”) and Astellas Pharma Inc. (TSE: 4503, President and CEO: Naoki Okamura, “Astellas”) today announced an exclusive option and license agreement for AVB-101, an investigational, AAV-based gene therapy in Phase 1/2 development for patients with frontotemporal dementia with progranulin mutations (FTD-GRN).

FTD is a devastating form of early-onset dementia that typically leads to death within three to 13 years from diagnosis. People with FTD commonly experience a rapid decline in executive function (attention control, working memory, problem-solving etc.), uncharacteristic behaviors, loss of language, apathy, and reduced mobility. It is an important cause of dementia in those under the age of 65 and is often underrecognized, and misdiagnosed. ¹,²,³,⁴

Under the terms of the agreement, Astellas will have the option to receive a worldwide exclusive license for the development and commercialization rights to AVB-101 in FTD-GRN and other potential indications. Astellas will make a $20 million equity investment and up to $30 million in upfront payments for the option to license AVB-101. AviadoBio is also eligible to receive up to $2.18 billion in license fees and milestone payments plus royalties if Astellas exercises its option.

Lisa Deschamps, CEO, AviadoBio
“As we complete dosing of the first cohort of patients in our Phase 1/2 ASPIRE-FTD trial of AVB-101, we are excited about the potential of this collaboration to help address the unmet need that exists today in frontotemporal dementia. This strategic collaboration will combine our promising gene therapy candidate for FTD-GRN and delivery expertise with Astellas’ global capabilities in development and commercialization of gene therapies. Together, we can further accelerate delivering this investigational medicine to families around the world who so desperately need treatment options for FTD-GRN and other neurological diseases.”

Adam Pearson, Chief Strategy Officer, Astellas
“We look forward to collaborating with the team at AviadoBio as we expand our gene therapy pipeline to help a broader range of people living with debilitating, neurodegenerative diseases. AVB-101 represents a truly innovative approach to the treatment of FTD-GRN and has the potential to be part of the next generation of gene therapy products through the creation of this agreement. Genetic regulation remains a cornerstone of our primary focus strategy at Astellas and this agreement helps us to continue to provide potential solutions for patients in need.”

 

  1. Onyike CU and Diehl-Schmid J. Int Rev Psychiatry. 2013;25(2):130–137;
  2. Riedl L et al. Neuropsychiatr Dis Treat. 2014;10:297–310; 
  3. Onyike CU. Neuroepidemiology. 2011;37:166–167;
  4. Kansal K et al. Dement Geriatr Cogn Disord. 2016;41:109–122;

About AviadoBio

At AviadoBio, we are relentlessly chasing cures by translating groundbreaking science and precision delivery into life-changing medicines for people living with neurological conditions. With our deep understanding of the brain and suite of proprietary gene therapy platforms and delivery technologies, AviadoBio is working to overcome the challenges of delivering the right drug to the right place. Its innovative, neuroanatomy-led approach is designed to maximize the therapeutic potential of gene therapy to halt or potentially reverse neurodegenerative diseases. AviadoBio was founded on pioneering research from King’s College London and the UK Dementia Research Institute and has a leadership team with extensive gene therapy development, delivery, and commercialization experience which uniquely positions the company for success in bringing transformative medicines to patients.

AviadoBio’s investors include New Enterprise Associates (NEA), Monograph Capital, F-Prime Capital, Johnson & Johnson Innovation – JJDC, Inc. (JJDC), SV Health Investor’s Dementia Discovery Fund (DDF), Advent Life Sciences, EQT Life Sciences (Dementia Fund), and LifeArc Ventures.

For more information, please visit www.aviadobio.com and follow us on X @AviadoBio and LinkedIn at AviadoBio.

About Astellas

Astellas Pharma Inc. is a pharmaceutical company conducting business in more than 70 countries around the world. We are promoting the Focus Area Approach that is designed to identify opportunities for the continuous creation of new drugs to address diseases with high unmet medical needs by focusing on Biology and Modality. Furthermore, we are also looking beyond our foundational Rx focus to create Rx+® healthcare solutions that combine our expertise and knowledge with cutting-edge technology in different fields of external partners. Through these efforts, Astellas stands on the forefront of healthcare change to turn innovative science into VALUE for patients. For more information, please visit our website at https://www.astellas.com/en.

Astellas Cautionary Notes

In this press release, statements made with respect to current plans, estimates, strategies and beliefs and other statements that are not historical facts are forward-looking statements about the future performance of Astellas. These statements are based on management’s current assumptions and beliefs in light of the information currently available to it and involve known and unknown risks and uncertainties. A number of factors could cause actual results to differ materially from those discussed in the forward-looking statements. Such factors include, but are not limited to: (i) changes in general economic conditions and in laws and regulations, relating to pharmaceutical markets, (ii) currency exchange rate fluctuations, (iii) delays in new product launches, (iv) the inability of Astellas to market existing and new products effectively, (v) the inability of Astellas to continue to effectively research and develop products accepted by customers in highly competitive markets, and (vi) infringements of Astellas’ intellectual property rights by third parties. Information about pharmaceutical products (including products currently in development) which is included in this press release is not intended to constitute an advertisement or medical advice.

 

About Astellas Gene Therapies

Astellas Gene Therapies is an Astellas Center of Excellence developing genetic medicines with the potential to deliver transformative value for patients. Our gene therapy drug discovery engine is built around innovative science, a validated AAV platform, and industry leading internal manufacturing capability with a particular focus on rare diseases of the eye, CNS and neuromuscular system. Astellas Gene Therapies will also be advancing additional Astellas gene therapy programs toward clinical investigation. Astellas Gene Therapies is based in South San Francisco, with manufacturing and laboratory facilities in South San Francisco, Calif., Sanford, N.C. and Tsukuba, Japan.

 

Company and Media Contacts

AviadoBio

Farah Speer
SVP, Head of Communications and External Relations
fspeer@aviadobio.com
+1-312-543-2881

Astellas Pharma Inc.

R&D Communications
Sara Zelkovic
+1-347-226-1459
Sara.zelkovic@astellas.com

Corporate Communications
+81-3-3244-3201 

Sep 12
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F2G Announces $100 Million Financing to Advance Late-Stage Development and Commercialization of Novel Antifungal Drug Candidate Olorofim in the US

By F2G, Press Release, Private Companies
Press Release.

 

F2G Announces $100 Million Financing to Advance Late-Stage Development and Commercialization of Novel Antifungal Drug Candidate Olorofim in the US

Financing is being led by new investor AMR Action Fund with strong participation from new investor ICG Life Sciences and existing investors

MANCHESTER, United Kingdom, September 12, 2024 – F2G Ltd, a clinical-stage biopharmaceutical company focused on the discovery and development of novel therapies to treat life-threatening rare fungal infections with a high unmet medical need, today announced a $100 million financing round. The financing is being led by new investor AMR Action Fund and co-led by ICG, with strong participation from existing investors Novo Holdings, Advent Life Sciences, Sofinnova Partners, Forbion, Blue Owl Healthcare Opportunities, Morningside Ventures, Brace Pharmaceuticals, and Merifin Capital. The proceeds from the financing will enable F2G to complete late-stage development, seek regulatory approval, and prepare for commercialization in the US of olorofim, a novel oral antifungal therapy to treat invasive aspergillosis (IA) and other invasive fungal infections. Henry Skinner, Ph.D., Chief Executive Officer of AMR Action Fund and Toby Sykes Ph.D., Managing Director of ICG Life Sciences, will join the F2G Board of Directors. The financing is subject to customary Foreign Direct Investment (FDI) clearance by local authorities in Austria, where F2G has a 100%-owned subsidiary, and expected to close in the fourth quarter of 2024.

Olorofim is the first orotomide antifungal, an entirely novel class of antifungal agents and is the only antifungal medication to be awarded a Breakthrough Therapy Designation for multiple indications by the US Food and Drug Administration (FDA). Olorofim works through a novel mechanism of action, different from existing classes of antifungals, exerting fungicidal activity through inhibition of the pyrimidine synthesis pathway. It is anticipated to be used to treat patients with a serious invasive, rare fungal disease where existing treatments are inappropriate or no longer effective.

Francesco Maria Lavino, Chief Executive Officer of F2G, said: “We are delighted to attract this additional capital investment into F2G, and we are thrilled to be working with the AMR Action Fund, an important player in the antimicrobial space, and ICG, a leading alternative asset manager. The addition of Dr. Skinner and Dr. Sykes to our Board will be invaluable as we move to our next stage of growth. This is a pivotal period for the Company as we conclude the final stages of development and preparation for commercialization of olorofim in the US and elsewhere. If approved, olorofim is expected to be the first of a new class of antifungal agents with a novel, differentiated mechanism of action and will address genuine unmet needs in conditions with high morbidity and mortality.”

Dr. Skinner commented: “Fungal infections are a growing threat to patients around the world and have a disproportionate impact on vulnerable populations, yet there has been a paucity of innovation in the field of antifungals. For decades, clinicians have relied on a handful of antifungal classes, with few mechanisms of action and significant limitations due to spectrum of activity, drug toxicities, or drug-drug interactions. These therapies are increasingly failing in patients. We are thrilled to support F2G’s team as they develop this urgently needed therapy and seek regulatory approval.”

Dr. Sykes commented: “Fungal pathogens continue to emerge and spread. This evolving threat underscores an urgent unmet medical need for advanced antifungal treatments. By investing in the development of novel antifungal drug candidates like olorofim, we hope to address this critical gap, ultimately making a meaningful and sustainable social impact on patients, their caregivers, and healthcare systems.”

About F2G
F2G is a clinical-stage biopharmaceutical company with operations in the UK, US, and Austria focused on the discovery and development of novel therapies to treat potentially life-threatening invasive fungal infections. F2G has discovered and developed a completely new class of antifungal agents called the orotomides which selectively target a key enzyme in the de novo pyrimidine biosynthesis pathway. This is a completely different mechanism from that of the currently marketed antifungal agents and gives the orotomides fungicidal activity against a broad range of rare and resistant fungal mold infections. For more information, please visit: www.f2g.com

About olorofim
Olorofim (formerly, F901318) is F2G’s leading candidate from the orotomide class and is currently in a Phase 2b open-label study (ClinicalTrials.gov Identifier: NCT03583164) in patients who have limited treatment options for difficult-to-treat invasive, rare fungal mold infections such as azole-resistant aspergillosis, scedosporiosis, lomentosporiosis, and other rare mold infections. F2G has initiated a global Phase 3 trial (“OASIS”) to compare treatment with olorofim versus liposomal amphotericin B followed by standard of care (SOC) in patients with invasive fungal disease caused by proven Invasive Aspergillus or probable lower respiratory tract disease by Aspergillus species. Olorofim has received orphan drug status from the European Medicines Agency for the treatment of invasive aspergillosis and invasive scedosporiosis. Olorofim has also received orphan drug status from the FDA for the treatment of coccidioidomycosis, scedosporiosis, and invasive aspergillosis. Olorofim has been granted Qualified Infectious Disease Product (QIDP) designation for invasive aspergillosis, invasive scedosporiosis, invasive lomentosporiosis, coccidioidomycosis, invasive disease due to Scopulariopsis species, and invasive fusariosis.

About invasive aspergillosis
Aspergillosis is a fungal infection caused by Aspergillus species of mold which are commonly found all over the world. Most of these molds, in most people, do not cause disease. However, Aspergillus is transmitted to humans through inhalation and may cause a broad spectrum of disease ranging from hypersensitivity reactions to direct invasion and destruction of tissue, especially in immunocompromised hosts. Invasive aspergillosis is a rare disease that can occur in over 10% of some high-risk immunosuppressed populations with mortality exceeding 80%.

Media Contact
Kristin Politi, Ph.D.
LifeSci Communications
kpoliti@lifescicomms.com
646-876-4783

Sep 12
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Aura Biosciences Reports Positive Phase 2 End of Study Results Evaluating Bel-sar as a First-Line Treatment for Early-Stage Choroidal Melanoma

By Aura Biosciences, Press Release, Private Companies
Press Release.

 

Aura Biosciences Reports Positive Phase 2 End of Study Results Evaluating Bel-sar as a First-Line Treatment for Early-Stage Choroidal Melanoma

Bel-sar Demonstrated 80% Tumor Control Rate, 90% Visual Acuity Preservation, and a Highly Favorable Safety Profile

Aura to Host a Virtual Ocular Oncology Investor Event Featuring Key Opinion Leaders Today at 8:00 am ET

BOSTON, Sept. 12, 2024 (GLOBE NEWSWIRE) — Aura Biosciences, Inc. (NASDAQ: AURA), a clinical-stage biotechnology company developing precision therapies for solid tumors designed to preserve organ function, today announced positive Phase 2 end of study results evaluating bel-sar (AU-011) for the first-line treatment of early-stage choroidal melanoma (CM), a vision and life-threatening ocular cancer. The results were presented at The Retina Society Annual Meeting, on Thursday, September 12, 2024, in Lisbon, Portugal.

The Phase 2 study (NCT04417530) is an open-label, ascending single and repeat dose escalation trial in patients with early-stage CM (small CM and indeterminate lesions) designed to evaluate the safety, tolerability, and efficacy of up to three cycles of bel-sar treatment. The trial included both single and multiple ascending dose cohorts, with a total of 22 patients enrolled. Patients were closely monitored over a twelve-month follow-up period to assess tumor control, visual acuity preservation, and tumor growth rate.

Tumor Control and Visual Acuity Preservation

The Phase 2 results demonstrated that bel-sar achieved an 80% tumor control rate (n=8/10) among Phase 3-eligible patients who received the therapeutic regimen, with complete cessation of growth following treatment among responders (post-treatment average growth rate of 0.011 mm/yr among responders compared to 0.351 mm/yr prior to study entry; p<0.0001). Visual acuity preservation was achieved in 90% of these 10 patients. Importantly, 80% of these 10 patients were at high risk for vision loss with tumors close to the fovea or optic disc, highlighting the potential for vision preservation with this novel class of drugs. Of note, the current standard of care is radiotherapy, which leads to visual acuity of <20/200 (the cutoff for legal blindness) in the treated eye in up to 87% of patients.1 The Phase 2 results are a significant achievement considering the typically poor prognosis associated with choroidal melanoma, a rare and life-threatening ocular cancer, where there are no approved vision-preserving therapies to date.

Highly Favorable Safety Profile with No Dose-Limiting Toxicities

The safety profile of bel-sar was highly favorable in all participants regardless of dose. There were no treatment-related serious adverse events (SAEs) reported. Ocular treatment-related AEs (TRAEs) were mild (Grade 1), included anterior chamber inflammation (18%) or cell (9%) and resolved without sequelae. The vast majority (~70%) of the anterior chamber inflammation/cell events were self-limited, requiring no treatment, and resolved in a median of 6 days. For those events that did require treatment, topical steroid eye drops, administered for a median of 6 days, achieved complete resolution of the inflammation. Eye pain occurred in 9% of patients and was mild (Grade 1). Importantly, no treatment-related posterior inflammation events (no vitritis, choroiditis, retinitis, retinal pigment epithelium changes, or vasculitis) were reported.

“Many patients with early-stage choroidal melanoma currently face the difficult choice of whether to treat the cancer and risk losing their vision in the treated eye, or delay treatment and risk the tumor progressing,” said Dr. Ivana Kim, Director of the Ocular Melanoma Center, Mass Eye and Ear / Harvard Medical School. “The Phase 2 end of study data that I presented at The Retina Society Annual Meeting showed 80% tumor control rate, 90% vision preservation, and a highly favorable safety profile in early-stage CM. Bel-sar has the potential to become the first treatment that achieves the dual goals of treating the tumor while also preserving vision, which could change the treatment paradigm for patients with this disease.”

“We believe these Phase 2 results provide clinical evidence for bel-sar as a potential vision-sparing, first-line treatment option for patients with early-stage CM,” said Dr. Jill Hopkins, Chief Medical Officer and President of Research and Development at Aura Biosciences. “Bel-sar is potentially a first-in-class novel therapy and we are excited to continue to advance this program, which is currently enrolling patients in our ongoing global Phase 3 CoMpass trial.”

Aura received written agreement from the U.S. Food and Drug Administration (FDA) under a Special Protocol Assessment (SPA) for the design and planned analysis of the global Phase 3 CoMpass trial indicating concurrence by the FDA with the adequacy of the study, if successful, to address the objectives necessary to support Aura’s planned biologics license application submission. Aura Biosciences is focused on enhancing treatment options and improving outcomes for patients with CM and other cancers.

Aura Virtual Ocular Oncology Investor Event

Aura will host a virtual ocular oncology investor event featuring Dr. Ivana Kim, MD (Mass Eye and Ear) and Dr. Prithvi Mruthyunjaya, MD, MHS (Stanford University Byers Eye Institute) to discuss the Phase 2 end of study data on Thursday, September 12, 2024, at 8:00 am Eastern Time. To register for the event, click here. A live question and answer session will follow the formal discussion.

The live webcast of Aura’s virtual ocular oncology investor event will be available on the “Investors & Media” page under the “Events & Presentations” section of Aura’s website at https://ir.aurabiosciences.com/events-and-presentations, where a replay of the webcast will be archived for 90 days following the presentation date.

About Aura Biosciences

Aura Biosciences is a clinical-stage biotechnology company focused on developing precision therapies for solid tumors that aim to preserve organ function. Our lead candidate, bel-sar (AU-011), is currently in late-stage development for primary choroidal melanoma, and in early-stage development in other ocular oncology indications and bladder cancer. Aura Biosciences is headquartered in Boston, MA. Our mission is to grow as an innovative global oncology company that positively transforms the lives of patients.

For more information, visit aurabiosciences.com. Follow us on X (formerly Twitter) @AuraBiosciences and visit us on LinkedIn.

Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, and other federal securities laws. Any statements that are not statements of historical fact may be deemed to be forward looking statements. Words such as “may,” “will,” “could,” “should,” “expects,” “intends,” “plans,” “anticipates,” “believes,” “estimates,” “predicts,” “projects,” “seeks,” “endeavor,” “potential,” “continue” or the negative of such words or other similar expressions that can be used to identify forward-looking statements. These forward-looking statements include express or implied statements regarding Aura’s future expectations, plans and prospects, including, without limitation, statements regarding the therapeutic potential of bel-sar for the treatment of cancers including early-stage CM and other oncology indications; statements regarding Aura’s expectations for the Phase 3 clinical trial of bel-sar for early-stage CM; statements regarding Aura’s expectations for an improved quality of life of patients after treatment with bel-sar; statements regarding Aura’s beliefs and expectations for the urgent need for an effective local treatment in ocular and other oncology indications to preserve organ function; statements regarding Aura’s expectations for the estimated patient populations and related market opportunities for bel-sar; and the potential for regulatory approval of bel-sar.

The forward-looking statements in this press release are neither promises nor guarantees, and investors should not place undue reliance on these forward-looking statements because they involve known and unknown risks, uncertainties and other factors, many of which are beyond Aura’s control and which could cause actual results to differ materially from those expressed or implied by these forward-looking statements, including, without limitation, uncertainties inherent in clinical trials and in the availability and timing of data from ongoing clinical trials; the expected timing for submissions for regulatory approval or review by governmental authorities; the risk that the results of Aura’s preclinical and clinical trials may not be predictive of future results in connection with future clinical trials; the risk that interim data from ongoing clinical trials may not be predictive of final data from completed clinical trials; the risk that governmental authorities may disagree with Aura’s clinical trial designs, even where Aura has obtained agreement with governmental authorities on the design of such trials, such as the Phase 3 SPA agreement with the FDA; whether Aura will receive regulatory approvals to conduct trials or to market products; whether Aura’s cash resources will be sufficient to fund its foreseeable and unforeseeable operating expenses and capital expenditure requirements; Aura’s ongoing and planned preclinical activities; and Aura’s ability to initiate, enroll, conduct or complete ongoing and planned clinical trials. These risks, uncertainties and other factors include those risks and uncertainties described under the heading “Risk Factors” in Aura’s most recent Annual Report on Form 10-K and Quarterly Report on Form 10-Q filed with the United States Securities and Exchange Commission (SEC) and in subsequent filings made by Aura with the SEC, which are available on the SEC’s website at www.sec.gov. Except as required by law, Aura disclaims any intention or responsibility for updating or revising any forward-looking statements contained in this press release in the event of new information, future developments or otherwise. These forward-looking statements are based on Aura’s current expectations and speak only as of the date hereof and no representations or warranties (express or implied) are made about the accuracy of any such forward-looking statements.

Investor Contact:
Alex Dasalla
Head of Investor Relations and Corporate Communications
IR@aurabiosciences.com

Media Contact:
Kimberly Ha
KKH Advisors
kimberly.ha@kkhadvisors.com
917-291-5744

Sep 03
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Amphista Therapeutics appoints Antony Mattessich as Chief Executive Officer

By Amphista Therapeutics, Press Release, Private Companies
Press Release.

 

Amphista Therapeutics appoints Antony Mattessich as Chief Executive Officer

  • The appointment will accelerate the Company’s next phase of growth, advancing its next generation Targeted Glue™ protein degraders into the clinic and building a robust pipeline
  • Appointment follows achievement of three key milestones for Amphista in 2024 which demonstrated: in vivo efficacy, ability to target and degrade proteins in the CNS and novel mechanism of action for the degradation of proteins

Cambridge, UK, 3 September 2024 – Amphista Therapeutics (“the Company” or “Amphista”), a leader in next generation targeted protein degradation (TPD) approaches, today announces the appointment of Antony Mattessich as its Chief Executive Officer (CEO) and member of the Board of Directors. The appointment of Antony Mattessich as CEO will steer Amphista through its next phase of growth.

Antony is an experienced CEO bringing an impressive track record progressing programs from discovery, through clinical development and commercial success. He has strong capital markets experience having raised over $750 million for companies throughout his career. Antony has held several senior executive positions where he was instrumental in developing medicines that translate innovative science into meaningful clinical outcomes for patients, with extensive experience in CNS, haemato-oncology, immunology, and metabolics. For the last seven years, Antony was President and CEO of Nasdaq-listed Ocular Therapeutix (OCUL), where he launched the company’s lead asset and brought its potential blockbuster in Wet Age-Related Macular Degeneration (wet-AMD) from pre-clinical development into active enrolment in Phase 3.

Antony Mattessich, Chief Executive Officer of Amphista said: “2024 has been a pivotal year for Amphista and I am thrilled to join a company that has such differentiated technology in the TPD space at a really exciting time in its development. Having worked extensively in bringing therapies to patients, I am excited to focus on building Amphista’s portfolio of first-and best-in-class degraders into medicines that transform the lives of patients. The promising pre-clinical data that continues to mount at Amphista is testament to the strength of its scientific team. I am looking forward to working alongside the exceptional leadership team as well as its talented board of directors.”

Joshua Brumm, Chairman of Amphista’s Board of Directors said: “Antony’s extensive drug development and capital markets expertise are a perfect fit for Amphista as we plan to advance our lead assets into clinical development following the compelling new in vivo efficacy and CNS activity data announced this year. We are delighted to have someone with Antony’s leadership and track record joining at such a transformational time for Amphista.”

In January 2024, the Company announced the achievement of two new data sets with its next generation bifunctional non-cereblon / non-VHL-based protein degraders. This was followed in May 2024 by the unveiling of a new mechanism of action for the degradation of BRD9, an emerging target in oncology, that is differentiated from cereblon- or VHL-based PROTACs.

About Amphista Therapeutics

Amphista Therapeutics is focused on transforming the lives of patients with severe diseases, including cancer and neurodegenerative disease, through the advancement of next generation targeted protein degradation (TPD) medicines. Amphista is applying its proprietary warhead chemistry and mechanistic know-how to generate bifunctional Targeted Glues® with a differentiated mechanism and leading physicochemical properties. Its portfolio offers the potential for first- and/or best-in-class assets and expanding the offering of TPD medicines beyond CRBN and VHL-based agents. Founded by Advent Life Sciences, Amphista is a spin-out of TPD expert Professor Alessio Ciulli’s laboratories at the University of Dundee. Amphista is funded by leading life science investors including Advent Life Sciences, Forbion, Gilde Healthcare, Novartis Venture Fund, SV’s Dementia Discovery Fund as well as Eli Lilly & Company, and has strategic collaborations with Bristol Myers Squibb and Merck KGaA. For more information, please visit: www.amphista.com

 

Amphista, Targeted Glue, Targeted Glues and the Amphista logo are all trademarks or registered trademarks of Amphista Therapeutics Limited.

For more information please contact:

Amphista Therapeutics
John Goodall
Info@amphista.com

ICR Consilium
Amber Fennell, Namrata Taak Emily Johnson
Email: Amphista@consilium-comms.com
Tel: +44 (0)20 3709 5813

Aug 08
Love0

Levicept Announces Positive Results of Phase II Trial of novel Neurotrophin-3 Inhibitor, LEVI-04

By Levicept, Press Release, Private Companies
Press Release.

 

Levicept Announces Positive Results of Phase II Trial of novel

Neurotrophin-3 Inhibitor, LEVI-04, for the Treatment of Patients with Moderate to Severe Osteoarthritis

  • All primary and secondary efficacy endpoints met – significant analgesia across all measures for all doses
  • Well tolerated with no increase in incidence of rapid joint deterioration compared to placebo
  • First in class novel biologic under development as non-addictive pain treatment

Sandwich, UK – 6 August 2024 – Levicept Ltd, a biotechnology company focused on the development of LEVI-04, a first-in-class treatment for osteoarthritis and other pain indications, today announces positive results from its Phase II trial of LEVI-04 a novel and first in class neurotrophin-3 (NT-3) inhibitor, showing that LEVI-04 was highly effective and well tolerated.

The multiple arm, multicentre, randomized, double-blind, placebo-controlled, Phase II study enrolled more than 510 participants with pain and disability due to osteoarthritis of the knee (ClinicalTrials.gov ID: NCT05618782). The trial was conducted at sites across Europe and in Hong Kong, led by Professor Philip Conaghan MD from the University of Leeds and Leeds Teaching Hospitals NHS Trust, UK.

LEVI-04 demonstrated significant differences to placebo at Week 17 for all primary and secondary endpoints for all doses evaluated:

  • The primary endpoint was WOMACi pain assessment (change from baseline at Week 17). The mean reduction in WOMAC pain score from baseline was greater than 50% for all three doses of LEVI-04 and all statistically different to placebo.
  • Secondary endpoints were WOMAC subscales of function and joint stiffness, patient global assessment and daily pain scores and all statistically different to placebo.

Standard safety monitoring plus peripheral nervous system assessments showed LEVI-04 to be well tolerated. There was no increase in incidence of rapidly progressive osteoarthritis (RPOA) as measured via detailed, closely examined, radiographic analysis.

Levicept intends to submit full results for publication in a peer-reviewed journal.

Professor Philip Conaghan MD, Director NIHR Leeds Biomedical Research Centre and Principal Investigator said, “These results are truly exceptional and clinically meaningful in their extent. Safe and effective pain management is of critical importance in arthritis with existing treatments limited by adverse effects, addiction liabilities and poor efficacy. In this trial, LEVI-04 has demonstrated the potential to safely harness the analgesic properties of neurotrophin-3 inhibition and offer a vital new treatment option to millions of patients in huge need. If Phase III trials replicate these results, LEVI-04 would represent a major break-through for osteoarthritis treatment, and with substantial potential in other pain indications.”

Globally, 595 million people had osteoarthritis (OA) in 2020, affecting 7.6% of the population and making it the most common form of arthritisii. It is estimated that the market opportunity for drugs that treat OA is worth in excess of $10 billioniii.

Simon Westbrook, founder and CSO of Levicept, “The discovery of LEVI-04 harnessed the power of

evolution to design a molecule that supplements the endogenous NT regulator, soluble p75NTR. LEVI- 04 provides analgesia via inhibition of neurotrophin-3, while avoiding the use-limiting side effects of anti-NGF antibodies, including rapidly progressive of osteoarthritis. LEVI-04 retains the important trophic effects of the neurotrophins, including joint re-modelling. We want to thank all the clinicians and trial participants and hope that the results bring the prospect of a new treatment for them and many others.”

Kevin Johnson, Chairman of Levicept and Partner at founding investor Medicxi said, “The results from this robust and large-scale Phase II fully validate the belief of Levicept’s founders and investors that LEVI-04 could be a genuine breakthrough in the treatment of osteoarthritis. They also leave Levicept exceptionally well-positioned to consider the next strategic options to advance LEVI-04’s development.”

LEVI-04 is a proprietary p75 neurotrophin receptor fusion protein (p75NTR-Fc) that provides analgesia via inhibition of NT-3 activity and returns neurotrophin homeostasis by supplementing the endogenous p75NTR binding protein to scavenge excess neurotrophins present in chronic pain states.

ENDS

Levicept

Eliot Forster, CEO – eliot@levicept.com

Media Enquiries Charles Consultants

Sue Charles – Sue@charles-consultants.com +44 (0)7968 726585

Chris Gardner – Chris@CGComms.onmicrosoft.com +44 (0)7956 031077

About Levicept – www.levicept.com

Levicept Ltd is a UK-based biotechnology company developing the first in a new class of novel, safe

and efficacious biological therapies, LEVI-04 [p75NTR-Fc], for the treatment of osteoarthritis and chronic pain. LEVI-04 inhibits NT-3, one of the neurotrophin family of proteins. LEVI-04 has completed a Phase II clinical trial in more than 500 patients with osteoarthritis. It is estimated that the market opportunity for drugs that treat osteoarthritis is worth in excess of $10 billion. LEVI-04 was discovered by Levicept’s founder, Simon Westbrook. Levicept’s investors include Medicxi, Advent Life Sciences, Gilde Healthcare and Pfizer Ventures.

Follow us on LinkedIn – https://www.linkedin.com/company/levicept-ltd

i The Western Ontario and McMaster Universities Osteoarthritis (WOMAC) pain scale – a recognised standard

pain scale

ii https://www.thelancet.com/journals/lanrhe/article/PIIS2665-9913(23)00163-7/fulltext

iii Global Neurotrophins Market- Analysis, Size, Forecast 2033 (futuremarketinsights.com)

Jul 25
Love0

Beacon Therapeutics Appoints Lance Baldo, MD as Chief Executive Officer and Thomas Biancardi as Chief Financial Officer

By Beacon Therapeutics, Press Release, Private Companies
Press Release.

 

CEO Dave Fellows named non-Executive Chairman of the Board

Executive appointments follow $170 million Series B fundraise

London, UK and Cambridge, Mass., July 24, 2024 – Beacon Therapeutics Holdings Limited (‘Beacon’ or ‘the Company’), a leading ophthalmic gene therapy company with a mission to save and restore the vision of patients with blinding retinal diseases, today announced the appointment of Lance Baldo, MD as Chief Executive Officer, effective August 12, 2024, and Thomas Biancardi as Chief Financial Officer, effective August 1, 2024.

Dr. Baldo brings more than 20 years of experience in biopharmaceuticals including the successful launch of two new indications and a new formulation for Lucentis while at Genentech. Most recently, he served as Chief Medical Officer at Freenome, an early cancer detection company, where he led the design and execution of the Company’s medical strategy to support its pipeline, from clinical trials through registration and commercialization. Prior to Freenome, Dr. Baldo was the Chief Medical Officer at Adaptive Biotechnologies, serving as a member of the senior leadership team through the transition from a private to publicly traded company. He has also held numerous roles within the Roche Group and its affiliates, including Senior Vice President and Head of U.S. Medical Affairs of Genentech and Franchise Head for Ophthalmology.

Dr. Baldo succeeds David Fellows, who has served as CEO since Syncona Limited’s acquisition of AGTC in November 2022. Mr. Fellows will assist during the transition and assume the role of non-Executive Chairman of the Board in January 2025. During his tenure, Mr. Fellows oversaw the launch of Beacon Therapeutics in June 2023, initiation of the Phase II DAWN trial and registrational VISTA trial for AGTC-501 for the treatment of X-Linked Retinitis Pigmentosa (XLRP), and the recent closing of Beacon’s $170 million Series B.

“I am honored and incredibly excited to join Beacon at this important juncture for the Company. Dave and the team have guided Beacon to the forefront of ophthalmic gene therapy with a robust clinical pipeline of candidates poised to meaningfully improve the treatment paradigms for patients with both rare and prevalent blinding diseases,” said Dr. Baldo. “With AGTC-501 in a registrational study, world class science and incredible financial backing, we are poised to make a run at a devastating inherited retinal disease.”

Beacon also appointed Thomas Biancardi as the Chief Financial Officer, who assumes the role from interim CFO Andrew Prosser, effective August 1, 2024. Mr. Biancardi is a biopharmaceutical industry veteran with over 25 years of financial and operational leadership experience, predominantly within ophthalmology. During his career, he has assisted numerous companies in raising capital, and establishing clinical and commercial operations. As one of the first employees of Ophthotech Corporation, he helped the Company evolve from a pre-clinical venture backed startup to a publicly traded biotech company. He also played a crucial role in the successful launch of the first pharmacologic treatment for macular degeneration at Eyetech Pharmaceuticals.

“I am excited to serve as Beacon’s CFO and collaborate with Lance, the Board and Beacon’s experienced team to support the Company’s mission to fight blinding diseases,” shared Mr.
Biancardi. “Beacon has a strong operational foundation and the partnership of a global investor syndicate; I am looking forward to supporting the Company’s growth and development through market entry.”

“We are fortunate to bring in two experienced executives who share our passion to develop treatments for sight-threatening diseases. The addition of Lance and Tom will accelerate Beacon’s growth as a leading ocular gene therapy company focused on bringing transformative gene therapies to patients,” said Mr. Fellows.

Beacon has raised approximately $290 million in funding to date. In July, Beacon announced a $170 million Series B fundraise following several clinical milestones, including the first patient dosed in the VISTA registrational trial for AGTC-501, the initiation of the Phase II DAWN trial and the presentation of positive 12-month interim results of the Phase 2 SKYLINE trial at the 47th Annual Macula Society Meeting demonstrating the precision, effectiveness and safety of Beacon’s lead development candidate, AGTC-501.

About Beacon Therapeutics

Beacon Therapeutics is an ophthalmic gene therapy company founded in 2023 to save and restore the vision of patients with a range of prevalent and rare retinal diseases that result in blindness.

The Company has an established scientific foundation that combines a late-stage development candidate to treat X-linked retinitis pigmentosa (XLRP), as well as two preclinical programs, one targeting dry age-related macular degeneration (AMD) and another targeting an inherited cone-rod dystrophy (CRD).

Lead development candidate AGTC-501, is a gene therapy program currently being investigated for the treatment of XLRP, an inherited monogenic recessive disorder that causes progressive vision loss in boys and young men. XLRP is predominantly caused by mutations in the retinitis pigmentosa GTPase regulator (RPGR) gene. AGTC-501 expresses the full length RPGR protein, thereby addressing the full complement of photoreceptor damage caused by XLRP, including both rod and cone loss.

Beacon Therapeutics also has access to a target generation technology platform that will identify, screen, and search secreted proteins in the ophthalmology space.

The Company is supported by funds from Syncona Limited, Forbion, Oxford Science Enterprises, TCGX, Advent Life Sciences, and additional investors.

Find out more about Beacon Therapeutics at beacontx.com.

Contact:

info@beacontx.com

Media:

beacontherapeutics@edelman.com

Jul 03
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Beacon Therapeutics Raises $170 Million in Series B Funding to Advance Development of Ophthalmic Gene Therapies

By Press Release, Private Companies
Press Release.

 

  • Financing led by life sciences venture capital firm Forbion, alongside existing investors Syncona and Oxford Science Enterprises, as well as new investors TCGX and Advent Life Sciences
  • Round will accelerate the development of lead asset AGTC-501 and broader pipeline
  • Beacon appoints Dmitrij Hristodorov, Wouter Joustra, and Cariad Chester to Board of Directors; Dominic Schmidt joins as a Board Observer

London, UK and Cambridge, Mass., July 3, 2024 – Beacon Therapeutics Holdings Limited (‘Beacon Therapeutics’ or ‘the Company’), a leading ophthalmic gene therapy company with a mission to save and restore the vision of patients with blinding retinal diseases, today announced it has raised $170 million in Series B funding.

Forbion led the financing, which included existing investors Syncona Limited, Oxford Science Enterprises and the University of Oxford, as well as initial investments from TCGX and Advent Life Sciences. The funds will be used to support the continued clinical development of Beacon’s lead asset, AGTC-501 for X-Linked Retinitis Pigmentosa (XLRP) and generate Phase 1/2 clinical trial data for the Company’s Dry Age-related Macular Degeneration (dAMD) program.

Beacon Therapeutics also appointed Dmitrij Hristodorov and Wouter Joustra, General Partners of Forbion, and Cariad Chester, Managing Partner of TCGX, to its Board of Directors. Dominic Schmidt, General Partner of Advent Life Sciences, joins as a Board Observer.

Beacon is focused on both orphan and prevalent diseases, including XLRP, a blinding orphan disease for which there is no available treatment, as well as dAMD. AGTC-501, the Company’s lead asset, is currently in a registrational clinical trial for the treatment of XLRP. AGTC-501 expresses the full length RPGR protein, thereby addressing all photoreceptor damage caused by XLRP, including both rod and cone loss.

David Fellows, Chief Executive Officer of Beacon Therapeutics, said, “We are focused on progressing our pipeline of ophthalmic gene therapies to save and restore the vision of patients with a range of prevalent and rare retinal diseases that result in blindness. I am confident that along with the addition of Dmitrij Hristodorov, Wouter Joustra and Cariad Chester to the Beacon Board, these funds will support the ongoing development of our late-stage and pre-clinical pipeline and enable acceleration of the development of AGTC-501 as we progress through the clinic and toward commercialization.”

“Forbion is pleased to support Beacon Therapeutics at this critical juncture in the development of its pipeline of pioneering ophthalmic gene therapies. Beacon’s deep expertise and renowned management bench give us confidence in the plan to build a leading retinal gene therapy company,” shared Dmitrij Hristodorov, General Partner at Forbion.

Elisa Petris, Lead Partner at Syncona Investment Management Limited and Board Director of Beacon Therapeutics, said, “Syncona’s continued backing of Beacon Therapeutics is a testament to the Company’s proven leadership team and innovative approach to developing gene therapies for retinal diseases. This financing and the partnership of this high-quality syndicate will propel Beacon’s pipeline and enable the Company to advance its programs for both rare and prevalent ophthalmic diseases.”

Beacon has raised approximately $290 million in funding to date. This funding round follows several clinical milestones for the company, including the first patient dosed in the VISTA registrational trial for AGTC-501, the initiation of the Phase II DAWN trial and the presentation of positive 12-month interim
results of the Phase 2 SKYLINE trial at the 47th Annual Macula Society Meeting demonstrating the precision, effectiveness and safety of Beacon’s therapeutic interventions.

J.P. Morgan acted as sole placement agent to Beacon Therapeutics for this transaction.

About Beacon Therapeutics
Beacon Therapeutics is an ophthalmic gene therapy company founded in 2023 to save and restore the vision of patients with a range of prevalent and rare retinal diseases that result in blindness.

The Company has an established scientific foundation that combines a late-stage development candidate to treat X-linked retinitis pigmentosa (XLRP), as well as two preclinical programs, one targeting dry age-related macular degeneration (AMD) and another targeting cone-rod dystrophy (CRD), an inherited retinal disease.

Lead development candidate AGTC-501, is a gene therapy program currently being investigated for the treatment of XLRP, an inherited monogenic recessive disorder that causes progressive vision loss in boys and young men. XLRP is predominantly caused by mutations in the retinitis pigmentosa GTPase regulator (RPGR) gene. AGTC-501 expresses the full length RPGR protein, thereby addressing the full complement of photoreceptor damage caused by XLRP, including both rod and cone loss.

Beacon Therapeutics also has access to a target generation technology platform that will identify, screen, and search secreted proteins in the ophthalmology space.

Find out more about Beacon Therapeutics at beacontx.com.

Contact:
info@beacontx.com

Media:
beacontherapeutics@edelman.com

May 24
Love0

Amphista Therapeutics unveils new differentiated mechanism of action for BRD9 degradation at 2024 Protein Degradation in Focus Symposium

By Amphista Therapeutics, Press Release, Private Companies
Press Release.

 

Amphista Therapeutics unveils new differentiated mechanism of action for BRD9 degradation at 2024 Protein Degradation in Focus Symposium

  • First presentation of a novel mechanism of action for the degradation of BRD9 – completely differentiated from cereblon- or VHL-based PROTACs
  • Amphista’s bifunctional BRD9 degraders selectively induce BRD9 to DCAF16 proximity and serve as molecular glues, leading to strong and rapid degradation of BRD9
  • Discovery further underscores Amphista’s proprietary chemistry and high-quality science which is being applied to the discovery and development of additional targeted glues beyond BRD9

Cambridge, UK, May 23, 2024 – Amphista Therapeutics (“Amphista”), a leader in next generation targeted protein degradation (TPD) approaches, today announces the unveiling of a new mechanism of action for the degradation of BRD9, an emerging target in oncology, that is differentiated from cereblon- or VHL-based PROTACs, during an oral presentation at the 2024 Protein Degradation in Focus Symposium held in Dundee, UK.

This significant news for the TPD field builds upon an earlier announcement by Amphista of two compelling data sets demonstrating in vivo efficacy and central nervous system (CNS) activity of its mechanistically differentiated protein degraders. At today’s presentation titled “Degradation of BRD9 by a novel targeted glue”, Dr Andrea Testa, Scientific Co-Founder and Senior Director, Discovery Chemistry showed data from proteomic and genetic validation studies which demonstrate that Amphista’s bifunctional degraders induced degradation of BRD9 by target-assisted E3 ligase recruitment. This novel mechanism selectively induces the proximity of BRD9 to DCAF16 and serves as a molecular glue. DCAF16 is a cullin-RING E3 ligase which can facilitate degradation of proteins of interest via ternary complex formation. Induction of this complex leads to deep and rapid degradation of BRD9 in vivo.

Louise Modis, Chief Scientific Officer of Amphista Therapeutics, said: “The discovery of a novel mechanism for BRD9 degradation, differentiated from cereblon- or VHL-based PROTACs, is a testament to Amphista’s proprietary chemistry and know-how. Our ability to translate this novel approach into high-quality, drug-like compounds with oral bioavailability and activity in vivo is truly exciting. As we continue to spearhead our efforts to advance new TPD approaches, our goal remains clear – to expand the offering of TPD medicines beyond CRBN and VHL-based agents and bring effective treatments to patients in areas of high unmet need.”

In the results presented, Amphista’s sub-nanomolar BRD9 degraders showed activity in both solid and liquid cancer cell lines and demonstrated a high degree of selectivity over 8,000 proteins quantified by proteomics, including the closely related proteins BRD7 and BRD4. Notably, these compounds induced BRD9 degradation in a mouse xenograft model illustrating Amphista’s BRD9 degraders are orally bioavailable and active in vivo. This is the first-time in vivo degradation of BRD9 has been demonstrated via DCAF16, underscoring the potential therapeutic application of Amphista’s bifunctional degraders.

Building on this knowledge, Amphista is applying its proprietary scientific know-how and expertise to the development of targeted glues which induce degradation of different targets, expanding the opportunity beyond BRD9. Future announcements will provide details of additional pipeline targets as part of Amphista’s ambitions to develop a first- and/or best-in-class portfolio of degraders with leading physicochemical properties.

About Amphista Therapeutics

Amphista Therapeutics is focused on transforming the lives of patients with severe diseases, including cancer and neurodegenerative disease, through the advancement of next generation targeted protein degradation (TPD) medicines. Amphista is applying its proprietary warhead chemistry and mechanistic know-how to generate bifunctional targeted glues with a differentiated mechanism and leading physicochemical properties. Its portfolio offers the potential for first- and/or best-in-class assets and expanding the offering of TPD medicines beyond CRBN and VHL-based agents. Founded by Advent Life Sciences, Amphista is a spin-out of TPD expert Professor Alessio Ciulli’s laboratories at the University of Dundee. Amphista is funded by leading life science investors including Forbion, Gilde Healthcare, Novartis Venture Fund, Advent Life Sciences, Eli Lilly & Company and The Dementia Discovery Fund, and also has strategic collaborations with Bristol Myers Squibb and Merck. For more information, please visit: www.amphista.com

Amphista and the Amphista logo are all trademarks or registered trademarks of Amphista Therapeutics Limited.

 

For more information please contact:

ICR Consilium
Amber Fennell, Namrata Taak
Email: Amphista@consilium-comms.com
Tel: +44 (0)20 3709 5813