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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

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

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

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)

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

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

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

Ventus Medical announces the submission of a novel electronic nicotine replacement therapy in the UK

By Press Release, Private Companies, Ventus Medical
Press Release.

 

A novel NRT for quitting smoking and vaping has been submitted to the UK medicines regulator for approval

LIVERPOOL, UNITED KINGDOM, May 7, 2024 /EINPresswire.com/ — Ventus Medical LTD announces that it has submitted a UK Marketing Authorisation Application for ENHALE™, a next-generation smoking cessation and harm reduction product which will give tobacco product users a safer alternative to both smoking and vaping.

ENHALE™ features a certified medical device and easily replaceable cartridges with novel and proprietary electronic inhalation technology. ENHALE™ meets the rigorous safety standards required of a medicinal product.
• ENHALE™’s proprietary formulation and low-temperature vaporisation technology ensure no thermal degradants and safer nicotine delivery than traditional vapes, an important differentiator from every other product.
• Obtaining UK MHRA approval is the first step in enabling healthcare professionals to prescribe for the first time, an effective vape-like product that is regulated and certified to be safe.
• When approved, ENHALE™ will be available without prescription in the UK.

Commenting on this submission, Kaasim Mahmood, Chairman of Ventus Medical and General Partner at Advent Life Sciences, said, “Smoking remains the most preventable cause of premature death globally. There is an urgent need for regulated, safer alternatives to vaping products and cigarettes. Advent Life Sciences is proud to have supported Ventus Medical, a UK company, on its journey to this important milestone. ENHALE™ has been designed and developed following extensive consultation with physicians, healthcare professionals and smokers who require a medicinal alternative to tobacco products.”

David Lawson, co-founder of Ventus Medical, added, “After nine years of rigorous research and development, we have successfully developed aerosolization technology to the strictest standards meeting pharmaceutical and medical device requirements globally. Clinical data on ENHALE™ confirm it’s ability to reduce the urge to smoke by more than leading NRT’s. This is a great step forward for smoking cessation and tobacco harm reduction and will be the first innovation in NRT for decades.”

ENHALE™ features an intuitive, easy-to-use, certified medical device with replaceable single-dose nicotine cartridges. The product is unique in utilizing low-temperature vaporisation technology, which heats the nicotine formulation to much lower temperatures when compared to vape products. Operating at lower temperatures ensures that ENHALE™ can safely and effectively deliver nicotine without any thermal degradation or metals which are of particular concern in vaping products.

About Ventus Medical
Ventus Medical is dedicated to the development of inhalation technology and is a certified medical device manufacturer based in the UK. The founding team of Ventus Medical were responsible for the first and only approved medical vape product (e-VOKE), which was licensed in 2013 by the MHRA.

David Lawson
Ventus Medical LTD
+44 333 344 0201
david.lawson@ventusmedical.com

AviadoBio Announces First Patient Treated in ASPIRE-FTD Clinical Trial Evaluating AVB-101 for Frontotemporal Dementia with GRN Mutations

By AviadoBio, Press Release, Private Companies
Press Release.

 

AviadoBio Announces First Patient Treated in ASPIRE-FTD Clinical Trial Evaluating AVB-101 for Frontotemporal Dementia with GRN Mutations

AVB-101 is an investigational gene therapy designed to deliver a functional copy of the GRN gene directly to the brain, thus restoring progranulin levels and potentially stopping disease progression

• Milestone marks first-in-human intrathalamic gene therapy delivery for any adult neurodegenerative disease

• ASPIRE-FTD trial will enroll patients at sites in Europe and the United States

LONDON APRIL 15, 2024 AviadoBio, a pioneering gene therapy company dedicated to developing and delivering potentially transformative medicines for neurodegenerative disorders, today announced that the first patient has been treated in the Phase 1/2 ASPIRE-FTD trial evaluating AviadoBio’s investigational gene therapy, AVB-101, in people with frontotemporal dementia (FTD) with progranulin (GRN) gene mutations (FTD-GRN). Clinical trial sites are currently open in Poland, Spain, and the Netherlands with additional sites expected to open in multiple countries, including the United States.

“AviadoBio is committed to bringing forward an innovative gene therapy treatment for FTD-GRN and this moment marks an important milestone for the FTD community and our company,” said Lisa Deschamps, CEO. “We are thankful for the dedication of the ASPIRE-FTD clinical investigators studying AVB-101 and immensely grateful for the families who participate in clinical trials for new treatment options that may change the future for generations of families living with FTD-GRN.”

FTD is a devastating form of early-onset dementia that typically leads to death within three to 10 years from diagnosis.¹,² People with FTD commonly experience personality changes, behavioral disturbance, loss of language, apathy, and reduced mobility.³ It is a leading cause of dementia in people under the age of 65 and is often misdiagnosed.⁴People with FTD who have disease-causing GRN mutations produce a reduced amount of progranulin protein. AVB-101 is a potential one-time therapy designed to stop disease progression by delivering a functional copy of the GRN gene to restore appropriate progranulin levels to affected areas of the brain.

“There is a critical need for new treatments for people living with FTD-GRN. The lack of effective disease-modifying treatments makes this diagnosis particularly difficult for patients and their families who don’t currently have any available treatment options for this devastating and progressive disease,” said David Cooper, M.D., Chief Medical Officer. “Treating the first patient in ASPIRE-FTD marks an important step forward in understanding AVB-101’s potential, which has already shown promise in preclinical studies.”

AVB-101 is delivered using a minimally invasive, stereotactic neurosurgical procedure directly to the part of the brain called the thalamus. The thalamus has extensive connections to other parts of the brain, including the frontal and temporal lobes, which play a critical role in FTD and the symptoms that impact patients and their families. This targeted delivery method aims to safely and effectively cross the blood-brain barrier, delivering targeted treatment directly to the brain to restore progranulin levels in the frontal and temporal cortex where it is needed most, while at the same time minimizing the dose required and thereby limiting any potential systemic exposure.

The Interventional Neurotherapy Center (INC) at Mazowiecki Szpital Bródnowski Hospital is the first and only center in Europe currently performing MRI-guided infusions of gene therapies. “Our team is excited to participate in this important scientific research and to perform the first intrathalamic administration of AVB-101 in the ASPIRE-FTD global trial,” said Prof. Mirosław Ząbek, M.D., Ph.D., the chairperson of the department of neurosurgery at INC.

“As a neurologist and founder of the Neuro-Care Clinic, I’ve seen the difficult struggles of my patients with dementia and their families,” said Gabriela Klodowska, M.D., Ph.D. “It means a lot to our patients with FTD to bring this type of study offering a potential one-time treatment for patients with a GRN mutation to Poland and to be the first to enroll patients in this global study.”

In November 2023, the U.S. Food and Drug Administration (FDA) granted Fast Track designation to AVB-101. Fast Track is a process designed to facilitate the development and expedite the review of new drugs to treat serious conditions and fill an unmet medical need. This designation is intended to bring new medicines to patients earlier. It has also received orphan drug designation by both the FDA and European Commission.

More information about the ASPIRE-FTD study can be found at https://clinicaltrials.gov/study/NCT06064890.

  1. Onyike CU. Neuroepidemiology. 2011;37:166–167
  2. Riedl L et al. Neuropsychiatr Dis Treat. 2014;10:297–310
  3. Pressman P and Miller BL. Biol Psychiatry. 2014;75(7):574–581
  4. Hendriks S, Peetoom K, Bakker C, et al. Global Prevalence of Young-Onset Dementia: A Systematic Review and Meta-analysis. JAMA Neurol. 2021;78(9):1080–1090. doi:10.1001/jamaneurol.2021.216
  5. Hogan DB et al. Can J Neurol Sci. 2016;43 (Suppl 1):S96–S109
  6. Moore KM et al. Lancet Neurol. 19(2):145–156
  7. Kansal K et al. Dement Geriatr Cogn Disord. 2016;41:109–122
  8. Galvin JE et al. Neurology. 89(20):2049–2056
  9. Young JJ et al. Ther Adv Psychopharmacol. 2018;8(1):33–48
  10. Kuang, L., et. al. Frontotemporal dementia non-sense mutation of progranulin rescued by aminoglycosides. Human Molecular Genetics 2020;29(4):624-634

About ASPIRE-FTD

ASPIRE-FTD is an open-label, multi-center, Phase 1/2 dose-escalation study designed to evaluate the safety and preliminary efficacy of AVB-101 in patients with FTD-GRN. In the study, patients will receive a single administration of AVB-101 delivered as a one-time infusion into the thalamus via a stereotactic neurosurgical procedure at expert neurosurgical centers throughout Europe and the United States.

About AVB-101

An investigational gene therapy, AVB-101 contains a correct (non-mutated) version of the GRN gene. It is designed to restore levels of progranulin in the brain, potentially slowing or stopping the progression of FTD-GRN. AVB-101 will be delivered as a one-time infusion directly into the brain via a minimally invasive surgical procedure, performed by a study neurosurgeon at a specialist neurosurgical center.

About Frontotemporal Dementia (FTD) and FTD with GRN Mutations (FTD-GRN

FTD is a devastating form of early-onset dementia that typically leads to death within seven to 13 years of symptom onset and three to 10 years from diagnosis.¹,²  People with FTD commonly experience personality changes, behavioral disturbance, loss of language, apathy, and reduced mobility.³

FTD is a leading cause of dementia in people under the age of 65⁴ with an estimated prevalence at any one time of up to 4.6 cases per 1,000 people.⁵ FTD typically strikes younger than Alzheimer’s disease and the majority of FTD cases occur between 45 and 68 years of age.⁶,⁷ Given the early onset, FTD can have a substantially greater impact on work, family, and finances than Alzheimer’s disease.⁸ Genetic FTD cases account for about one-third of cases and are associated with autosomal dominant mutations in three genes, including the GRN (progranulin) gene.⁹ Approximately 11,000 people in the U.S. and EU5 are living with FTD-GRN with approximately 2,200 new cases per year.¹,¹⁰ Some FTD cases may be misidentified, and diagnostic delay is common. As disease education, genetics knowledge, and research and treatment options grow, these numbers are expected to increase.

About AviadoBio

At AviadoBio, we are relentlessly chasing cures by translating groundbreaking science and precision delivery into life-changing medicines for people living with frontotemporal dementia (FTD) and amyotrophic lateral sclerosis (ALS). With our deep understanding of the brain and suite of proprietary gene therapy platforms and delivery technologies, AviadoBio is working relentlessly 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.

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

Almirall and Eloxx Pharmaceuticals Enter into Exclusive Agreement to license ZKN-013 for rare dermatological diseases

By Eloxx Pharmaceuticals, Press Release, Private Companies
Press Release.

 

Almirall and Eloxx Pharmaceuticals Enter into Exclusive Agreement to license ZKN-013 for rare dermatological diseases

  • ZKN-013 is a phase I ready oral therapy designed to overcome nonsense mutations that cause a premature stop codon resulting in nonfunctional protein production for example in recessive Dystrophic Epidermolysis Bullosa (RDEB), Junctional Epidermolysis Bullosa (JEB) and familial adenomatous polyposis (FAP)

BARCELONA, Spain and WATERTOWN, Mass., March 13, 2024 (GLOBE NEWSWIRE) — Almirall, S.A. (BME:ALM) and Eloxx Pharmaceuticals, Inc. (OTC: ELOX) announced today that the companies have entered into an exclusive license agreement for the asset ZKN-013. Under the agreement, Almirall obtains global rights to develop and commercialize ZKN-013, including for the use in orphan dermatological diseases. ZKN-013 is a potentially promising oral, nonsense mutation readthrough drug, which enables the host cells to produce functional proteins which counteract the root cause of these rare dermatological and potentially other diseases.

The drug candidate is expected to shortly enter into Phase I development in healthy volunteers.

As part of this agreement, Eloxx will receive an upfront of $3 million, and additional payments throughout the potential development phases, including regulatory and sales milestones of up to $470 million, as well as tiered royalties based on any potential future global sales.

We are very excited about this agreement with Almirall to develop and distribute ZKN-013, our lead TURBO-ZM™ based molecule, as we believe it has the potential to have a significant impact on the treatment of these painful and debilitating diseases,” said Sumit Aggarwal, President and Chief Executive Officer of Eloxx. “In addition to advancing development of ZKN-013, this agreement will allow Eloxx to remain focused on fully maximizing the potential of ELX-02 in rare kidney diseases and continue funded discovery efforts on our TURBO-ZM™ platform.”

This license agreement is aligned with Almirall’s R&D strategy to develop novel treatments to help people with dermatological conditions, including rare diseases,” said Karl Ziegelbauer, Executive Vice President R&D and Chief Scientific Officer of Almirall. “We look forward to progressing the development of ZKN-013 to find a potentially impactful solution for patients suffering from rare and devastating diseases caused by nonsense mutations.”

RDEB /JEB are rare skin diseases characterized by defects in the Collagen7 gene which is essential for the correct formation of the skin structure and barrier function. ZKN-013 has demonstrated robust functional preclinical activity in RDEB/JEB patient cells and in APCmin (multiple intestinal neoplasia) mice. The studies demonstrated that ZKN-013 induces the production of functional, full-length COL7 in RDEB patient cells.

ZKN-013 is also being developed for the treatment of FAP patients with nonsense mutations characterized by proliferation of colon polyps and progression to colon cancer. FAP is a rare GI disease with patients progressing to colon cancer caused by mutations in the APC gene.

ZKN-013 is Eloxx’s lead TURBO-ZM based molecule. Eloxx’s TURBO-ZM platform uses chemistry technology to develop novel Ribosome Modulating Agents to target the human ribosome to develop new potential therapeutics. Ribosomes form the translation machinery that generates functional proteins from genetic sequences; modulating the ribosome subunits provides a therapeutic approach to address a number of different diseases.

About Eloxx Pharmaceuticals

Eloxx Pharmaceuticals, Inc. is engaged in the science of ribosome modulation, leveraging its innovative TURBO-ZM™ chemistry technology platform in an effort to develop novel Ribosome Modulating Agents (RMAs) and its library of Eukaryotic Ribosome Selective Glycosides (ERSGs). Eloxx’s lead investigational product candidate, ELX-02, is a small molecule drug candidate designed to restore production of full-length functional proteins. ELX-02 is in Phase 2 clinical development for the treatment of Alport syndrome in patients with nonsense mutations.

For more information, please visit www.eloxxpharma.com.

About Almirall  

Almirall is a global pharmaceutical company dedicated to medical dermatology. We closely collaborate with leading scientists, healthcare professionals, and patients to deliver our purpose: to transform the patients’ world by helping them realize their hopes and dreams for a healthy life. We are at the forefront of science to deliver ground-breaking, differentiated medical dermatology innovations that address patients´ needs.

Almirall, founded in 1944 and headquartered in Barcelona, is publicly traded on the Spanish Stock Exchange (ticker: ALM). Almirall (total revenue in 2023: €898.8 MM, 1900 employees globally) has a direct presence in 21 countries and affiliates in over 70 others.

For more information, please visit almirall.com

Corporate Communications contact Almirall: Investors’ Relations contact
Corporate communications team Pablo Divasson del Fraile
corporate.communication@almirall.com investors@almirall.com
Phone: (+34) 659 614 173 Phone: (+34) 93 291 30 87
Eloxx Contacts:
Investors:
John Woolford
john.woolford@westwicke.com
443.213.0506
Media:
Laureen Cassidy
laureen@outcomescg.com

Eloxx Forward-looking Statements
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements other than statements of historical facts contained in this press release, including without limitation, statements regarding the potential future payments and future benefits under the license agreement, achievement of key milestones under the license agreement and the expected timeline for clinical development and efficacy of ZKN-013 are forward-looking statements. Forward-looking statements can be identified by the words “aim,” “may,” “will,” “would,” “should,” “expect,” “explore,” “plan,” “anticipate,” “could,” “intend,” “target,” “project,” “contemplate,” “believe,” “estimate,” “predict,” “potential,” “seeks,” or “continue” or the negative of these terms similar expressions, although not all forward-looking statements contain these words. Forward-looking statements are based on management’s current plans, estimates, assumptions and projections based on information currently available to us. Forward-looking statements are subject to known and unknown risks, uncertainties and assumptions, and actual results or outcomes may differ materially from those expressed or implied in the forward-looking statements due to various important factors, including, but not limited to: our ability, or our licensees’ ability, to progress any product candidates in preclinical or clinical trials; the uncertainty of clinical trial results and the fact that positive results from preclinical studies are not always indicative of positive clinical results; the scope, rate and progress of our, and our licensees’ preclinical studies and clinical trials and other research and development activities; the competition for patient enrollment from drug candidates in development;; our ability to obtain the capital necessary to fund our operations; the cost of filing, prosecuting, defending and enforcing any patent claims and other intellectual property rights; our ability to obtain financing in the future through product licensing, public or private equity or debt financing or otherwise; our ability to meet the continued listing requirements of the Nasdaq Capital Market; general business conditions, regulatory environment, competition and market for our products; and business ability and judgment of personnel, and the availability of qualified personnel and other important factors discussed under the caption “Risk Factors” in our Quarterly Report on Form 10-Q for the quarterly period ended September 30, 2023, as any such factors may be updated from time to time in our other filings with the SEC, accessible on the SEC’s website at www.sec.gov and the “Financials & Filings” page of our website at https://investors.eloxxpharma.com/financials-filings.

All forward-looking statements speak only as of the date of this press release and, except as required by applicable law, we have no obligation to update or revise any forward-looking statements contained herein, whether as a result of any new information, future events, changed circumstances or otherwise.

Almirall Legal warning
This document includes only summary information and is not intended to be exhaustive. The facts, figures and opinions contained in this document, in addition to the historical ones, are “forward-looking statements”. These statements are based on the information currently available and the best estimates and assumptions that the Company considers reasonable. These statements involve risks and uncertainties beyond the control of the Company. Therefore, actual results may differ materially from those declared by such forward-looking statements. The Company expressly waives any obligation to revise or update any forward-looking statements, goals or estimates contained in this document to reflect any changes in the assumptions, events or circumstances on which such forward-looking statements are based, unless required by the applicable law.

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