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May 16
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Shionogi & Co., Ltd. and F2G Ltd., Enter Strategic Collaboration to Develop and Commercialize the New Antifungal Agent Olorofim in Europe and Asia

By F2G, Press Release, Private Companies
Press Release.

 

  • Shionogi to conduct clinical trials, subsequent registration and commercialization of olorofim for invasive aspergillosis IA in Europe and Asia
  • F2G to receive u pfront payment of $100 m and share development costs
  • F2G will also be eligible for additional regulatory and commercial milestones of up to $380 m , as well as double digit royalties on net sales

OSAKA, Japan, and MANCHESTER, UK, May 16, 2022 – Shionogi & Co., Ltd. (Head Office: Osaka, Japan; President and CEO: Isao Teshirogi, Ph.D.; hereafter “Shionogi”) and F2G Ltd.（Head Office: Manchester, UK; CEO: Francesco Maria Lavino; hereafter “F2G”）today announced that they have entered a strategic collaboration to develop and commercialize the new antifungal agent olorofim for invasive fungal infections in Europe and Asia.

Olorofim is a novel oral antifungal therapy developed by F2G to treat invasive aspergillosis (IA) and other rare mold infections. Olorofim works through a unique mechanism of action, different from existing classes of antifungals, exerting fungicidal activity through inhibition of the pyrimidine synthesis pathway. Olorofim represents the first novel antifungal class developed in the past 20 years and is the only antifungal medication to be awarded a Breakthrough Therapy Designation (BTD) for multiple indications by the FDA.

Under the terms of the agreement, Shionogi will conduct the clinical trials and subsequent registration and commercialization of olorofim for IA in Europe and Asia. Shionogi will make an upfront payment to F2G of $100 million and share development costs in global studies. F2G will also be eligible to receive additional regulatory and commercial milestones of up to $380 million, as well as double-digit royalties on net sales.

 

“We are delighted to announce this partnership. By joining forces with Shionogi, we will be able to progress the development of olorofim with a partner which has a proven track record in both global drug development and business development to effectively deliver a potentially life-saving therapy to patients globally,”

said Francesco Maria Lavino, Chief Executive Officer of F2G.

 

“As F2G prepares to commercialize olorofim in the US, we are excited to work closely together with Shionogi to address the unmet medical need caused by invasive aspergillosis and other rare mold diseases around the world.”

“Shionogi is committed to ‘Protect people worldwide from the threat of infectious diseases’ as our key focus. We are pleased to partner with F2G, which is boldly tackling fungal infections where new drug development is difficult.”

said Isao Teshirogi, Ph.D., CEO of Shionogi.

“We will continue to address unmet medical needs in infectious diseases, and to work towards total care for this area. As part of this mission and through our partnership with F2G, we hope to be able to provide new antifungal drugs to patients to protect people’s health from life-threatening, invasive fungal infections.”

 

Invasive aspergillosis is a rare disease that can be fatal, occurring primarily in immunocompromised patients, including those having received cancer chemotherapy or hematopoietic stem cell transplantation. Other rare fungi, such as scedospurium or lomentospora, may also infect patients with compromised immunity. Existing antifungal therapies may not be appropriate treatments due to drug-drug interactions, tolerability or other serious side-effects, or are not effective due to resistance. For these patients, treatment options are limited.

Olorofim is anticipated to be used to treat patients with serious invasive fungal disease where existing treatments are inappropriate or no longer effective. Both companies believe olorofim provides hope to patients with, and physicians treating, serious invasive fungal infections.

 

About Shionogi

Shionogi & Co., Ltd. is a leading global research-driven pharmaceutical company based in Japan, dedicated to bringing benefits to patients based on its corporate philosophy of “supplying the best possible medicine to protect the health and wellbeing of the patients we serve.”

The company has discovered and developed novel medicines for HIV, influenza and antimicrobial resistance, and currently markets products in several therapeutic areas including anti-infectives with the first siderophore cephalosporin, cefiderocol. For more information, please visit https://www.shionogi.com/global/en

 

About F2G

F2G is a biotech 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 details, please visit the F2G web site.

 

About olorofim

Olorofim (formerly, F901318) is F2G’s leading candidate from the orotomide class and is currently in a Phase 2b open-label study1. F2G is currently initiating a global Phase 3 trial (“OASIS”)2 to compare treatment with olorofim versus AmBisome® followed by standard of care (SOC) in patients with lower respiratory tract invasive fungal disease caused by proven or probable infection with 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, are harmless. 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. Invasive aspergillosis is a rare disease that can occur in over 10% of some high-risk immunosuppressed populations with mortality exceeding 80%.

 

Forward-Looking Statements

This announcement contains forward-looking statements. These statements are based on expectations in light of the information currently available, assumptions that are subject to risks and uncertainties which could cause actual results to differ materially from these statements. Risks and uncertainties include general domestic and international economic conditions such as general industry and market conditions, and changes of interest rate and currency exchange rate. These risks and uncertainties particularly apply with respect to product-related forward-looking statements. Product risks and uncertainties include, but are not limited to, completion and discontinuation of clinical trials; obtaining regulatory approvals; claims and concerns about product safety and efficacy; technological advances; adverse outcome of important litigation; domestic and foreign healthcare reforms and changes of laws and regulations. Also, for existing products, there are manufacturing and marketing risks, which include, but are not limited to, inability to build production capacity to meet demand, lack of availability of raw materials and entry of competitive products. The company disclaims any intention or obligation to update or revise any forward-looking statements whether as a result of new information, future events or otherwise.

References
1.  https://clinicaltrials.gov/ct2/show/NCT03583164
2.  https://clinicaltrials.gov/ct2/show/NCT05101187

For Further Information, Contact:
SHIONOGI Website Inquiry Form https://www.shionogi.com/global/en/contact.html.

For F2G, contact Petra Taylor-Stadler
Email: ptaylor@f2g.com
Tel: +43 (0)1 997 4267

Optimum Strategic Communications
Mary Clark/ Charlotte Hepburne-Scott/ Supriya Mathur/ Zoe Bolt
Email: F2G@optimumcomms.com
Tel: +44 (0) 203 882 9621

May 11
Love0

Artax Biopharma Closes Financing to Advance Clinical Stage First-in- Class Oral Immunomodulator to Treat T Cell-Mediated Diseases

By Artax Biopharma, Press Release, Private Companies
Press Release.

 

  • Close of $26 Million Funds Immunomodulation Candidate AX-158 Development
  • Adds New Investors Eli Lilly and Company and Sound Bioventures

 

Cambridge, MA, May 11, 2022 – Artax Biopharma, Inc., a clinical stage
biotechnology company focused on transforming the treatment of T Cell-mediated pathologies, today announces the close of a $26 million financing round to develop AX-158, the Company’s first oral small molecule immunomodulating agent. New investors, Eli Lilly and Company and Sound Bioventures, participated alongside existing investors Advent Life Sciences, Columbus Venture Partners, Belinda Termeer and other Company shareholders.

Artax will use these funds to support the clinical development of AX-158 in
autoimmune diseases, including the addition of key clinical development personnel. AX-158 employs a first-in-class mechanism of action that selectively modulates inappropriate T cell activation, a cause of autoimmune disease. AX-158 has the potential to treat T Cell-mediated diseases without the risk of immunosuppression.

Artax’s ongoing Phase 1 clinical trial is assessing the safety, exposure, and
pharmacokinetics of AX-158 in healthy volunteers and includes ex vivo stimulated measures of its pharmacodynamic activity. In preclinical studies, AX-158 demonstrated potential to treat T Cell-mediated diseases by decreasing key cytokines including INFγ, TNFα, IL-2, IL-1 and IL-6 in whole human blood samples.

“We are pleased to welcome Lilly and Sound Bioventures as new investors as we continue to develop our unique immunomodulating agent for patients with T Cell-driven conditions, including autoimmune diseases. We believe AX-158’s immunomodulating effect has the potential to make a transformative difference for patients living with T Cell-driven conditions, without impacting healthy immune system function,”

said Joseph Lobacki, Chief Executive Officer of Artax Biopharma.

“We are also excited to welcome Casper Breum, Managing Partner of Sound Bioventures, who will join the Artax Board of Directors,”

said Mr. Lobacki.

“Mr. Breum’s experience will be a valuable asset to guide the Company’s strategy.”

 

About Artax Science and Immunomodulation

A healthy immune system eliminates harmful foreign pathogens, while being
tolerant of self-tissues and organs. The T Cell Receptor (TCR) is central to healthy T Cell function and a well-functioning immune system. When TCR signaling becomes dysregulated, T Cells behave abnormally. This behavior results in T Celldriven conditions, including autoimmune diseases, T Cell malignancies (lymphomas), and induced T Cell pathologies in which medical treatments result in immune reactions (such as stem cell transplants resulting in acute graft-versus-hostdisease or immuno-oncology treatments resulting in immune related-adverse events). Artax believes immunomodulation – a mechanism through which our investigational agents assist in rebalancing the immune system and eliminating a cause of T Cell-mediated diseases, while not impacting patients’ ability to effectively fight foreign pathogens – holds great potential.

 

About AX-158

AX-158 is a first-in-class, oral small molecule immunomodulating agent in
clinical development for the treatment of T Cell-mediated diseases. AX-158
employs a novel mechanism of action that selectively modulates, or adjusts, T Cell responses that play a critical role in immune system function. AX-158 is a Nck SH3.1 domain inhibitor which selectively counteracts the role of Nck in T Cells. Nck is a protein that naturally amplifies T Cell signaling directly at the TCR, contributing to T Cell mediated disease. This process of  immunomodulation assists the immune system to maintain healthy control and eliminates a direct contributor to T Cell-mediated diseases. Importantly, Artax believes that preclinical data suggests AX-158 will not be immunosuppressive and so will not impact the immune system’s ability to mount a strong response to foreign pathogens and infections.

 

About Artax Biopharma

Artax Biopharma is a clinical-stage biotechnology company transforming
T Cell-mediated disease treatment by developing innovative small molecules that modulate the immune system. Artax science holds broad potential to treat T Cellmediated diseases such as autoimmune diseases; induced T cell pathologies (such as acute graft-versus-host disease and immune-oncology treatment-related adverse events); and T Cell malignancies, while simultaneously allowing the body to fight foreign pathogens. For more information, please visit: www.artaxbiopharma.com.

 

About Sound Bioventures

Sound Bioventures is a biotherapeutics focused venture fund, with a strong
foundation in the Nordic biotech ecosystem, that invests in clinical stage companies in EU, UK and USA. The fund invests in projects addressing significant unmet medical needs having the potential for a profound impact on human health and healthcare systems. Sound Bioventures was founded by a team of experienced life science investors and is backed by a strong investor syndicate comprising Novo Holdings, Saminvest, Vaekstfonden, the European Investment Fund, Ramsbury Invest and the founders. Sound Bioventures Fund I AB is a registered alternative investment fund, under the Swedish Alternative Investment Funds Managers Act.

For more information, please visit: www.soundbioventures.com.

 

Contacts:
Karen LaRochelle, MBA
Chief Business Officer
Artax Biopharma
klarochelle@artaxbiopharma.com

May 04
Love0

Amphista Therapeutics Enters Strategic Collaboration with Merck for Discovery and Development of Targeted Protein Degradation Therapeutics

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

 

  • Amphista and Merck will collaborate to leverage Amphista’s proprietary EclipsysTM TPD platform and generate novel protein degrading therapeutics in oncology and immunology
  • Collaboration includes  up to €39 million ($44 million*) in combined upfront and R&D funding payments for an initial three programs
  • Amphista will potentially receive up to €893.5 million ($1.0 billion*) in total payments across three programs plus mid-single digit royalties

 

Cambridge, England, May 4th, 2022 – Amphista Therapeutics, a global leader in the discovery and development of next generation targeted protein degradation (TPD) therapeutics, today announced a strategic collaboration with Merck Healthcare, a division of Merck. Under the terms of the agreement, Merck and Amphista will work collaboratively to discover and develop small molecule protein degraders for an initial three targets in oncology and immunology indications. Amphista will receive an upfront payment, R&D funding and success-based milestone payments of up to €893.5 million ($1.0 billion*) as well as royalties in the mid-single digit range. Completion of the transaction is subject to the parties obtaining any necessary regulatory clearances or approvals.

Nicola Thompson, CEO of Amphista, said,

“We are extremely pleased to enter into this collaboration with Merck. This is a significant validation of the progress we have made in TPD research and the potential of our Eclipsys next-generation TPD platform. We look forward to working with the Merck team, using our combined expertise to develop new TPD therapies to treat cancers and immuno-inflammatory diseases.”

TPD therapies are designed to use physiological mechanisms to remove pathogenic protein from the body, offering the potential to access many disease targets previously considered “undruggable.” Amphista’s technology is specifically designed to develop next generation TPD therapeutics based on mechanistic insights and novel chemistry approaches that enable the company to develop novel protein degrading therapeutics with superior levels of efficacy and broad therapeutic applicability.

About Amphista Therapeutics

Amphista Therapeutics is a global leader in the discovery and development of next generation targeted protein degradation (TPD) medicines, addressing the challenges faced by the field to realise the full potential of this transformational modality.

The company’s proprietary EclipsysTM Platform supports development of multiple innovative therapeutic candidates able to overcome the limitations associated with traditional TPD approaches with superior levels of efficacy and broad therapeutic applicability. The Amphista team includes pioneers and established leaders in TPD research and all phases of drug discovery and development. The company is supported by leading life science investors including Forbion, Gilde Healthcare, Novartis Venture Fund, Advent Life Sciences, BioMotiv and Eli Lilly & Company.

For more information, please visit: http://www.amphista.com/.

Media Contacts:

Amphista Therapeutics
CEO Nicola Thompson
+44 (0) 7436102411
nicki@amphista.com

Berry & Company Public Relations
Doug Haslam
dhaslam@berrypr.com
+1 212 253 8881

May 04
Love0

Amphista Therapeutics Enters Strategic Collaboration with Bristol Myers Squibb for Discovery and Development of Targeted Protein Degradation Therapeutics

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

 

Amphista and Bristol Myers Squibb to collaborate and leverage Amphista’s proprietary Eclipsys™ targeted protein degradation platform to develop novel protein degrading therapeutics
Collaboration includes an upfront payment of $30 million, the potential for up to $1.25 billion in performance-based milestone payments and payments for a limited expansion of the collaboration, as well as royalties on global net sales of products

 

Cambridge, England, May 4th, 2022 – Amphista Therapeutics, a global leader in the discovery and development of next generation targeted protein degradation (TPD) therapeutics, today announced a strategic collaboration and license agreement with Bristol Myers Squibb.

Under the terms of the agreement, Bristol Myers Squibb and Amphista will work collaboratively to discover and develop small molecule protein degraders.  Bristol Myers Squibb will be granted a global exclusive license to the degraders developed and will be responsible for further development and commercialization activities.  Amphista will receive a $30 million upfront payment, the potential for up to $1.25 billion in performance-based milestone payments and payment for a limited expansion of the collaboration, as well as royalties on global net sales of products. The closing of the transaction is subject to the parties obtaining regulatory clearances or approvals.

Nicola Thompson, CEO of Amphista, said,

“Our collaboration with Bristol Myers Squibb is a powerful validation of our advances in TPD research and the capabilities of our Eclipsys next-generation TPD platform.  Combining our expertise with Bristol Myers Squibb’s strong legacy and experience in the protein degradation space brings new promise to the potential of delivering more effective new treatment to patients seeking treatment options.”

TPD therapies are designed to use physiological mechanisms to remove pathogenic protein from the body, offering the potential to access many disease targets previously considered “undruggable.” Amphista’s technology is specifically designed to develop next generation TPD therapeutics based on advanced mechanistic insights and novel chemistry approaches that enable the development of novel protein degrading therapeutics.

“Bristol Myers Squibb continues to build its leadership and scientific expertise in the protein degradation space,”

said Rupert Vessey, M.A., B.M., B.Ch., FRCP, D.Phil., Executive Vice President, Research & Early Development, Bristol Myers Squibb.

“We look forward to collaborating with Amphista and using its TPD platform to potentially develop new targeted protein degradation therapies.”

 

About Amphista Therapeutics

Amphista Therapeutics is a global leader in the discovery and development of next generation targeted protein degradation (TPD) medicines, addressing the challenges faced by the field to realise the full potential of this transformational modality.

The company’s proprietary EclipsysTM Platform supports development of multiple innovative therapeutic candidates able to overcome the limitations associated with traditional TPD approaches with superior levels of efficacy and broad therapeutic applicability. The Amphista team includes pioneers and established leaders in TPD research and all phases of drug discovery and development. The company is supported by leading life science investors including Forbion, Gilde Healthcare, Novartis Venture Fund, Advent Life Sciences, BioMotiv and Eli Lilly & Company.

For more information, please visit: http://www.amphista.com/.

 

Media contacts:

Amphista Therapeutics
CEO Nicola Thompson
+44 (0) 7436102411
nicki@amphista.com

Berry & Company Public Relations
Doug Haslam
dhaslam@berrypr.com
+1 212 253 8881

May 04
Love0

AviadoBio to Present Pre-clinical Data on its Gene Therapy Candidate for Frontotemporal Dementia at ASGCT 2022

By AviadoBio, Press Release, Private Companies
Press Release.

 

  • Intrathalamic delivery of AVB-101 shows promising efficacy in a pre-clinical disease model
  • Gene therapy candidate also demonstrated broad cortical expression in a large animal biodistribution study

 

London, UK, May 4th, 2022 – AviadoBio, a pioneering, pre-clinical stage gene therapy company focused on developing and delivering transformative medicines for people with neurodegenerative disorders, announces that it will be presenting preclinical data at the upcoming ASGCT 25th Annual Meeting (American Society of Gene & Cell Therapy) on 16-19 May, 2022. The data are from studies it has conducted with its investigational, one-time, adeno-associated virus (AAV) gene therapy, AVB-101 (AVB-PGRN), for the treatment of frontotemporal dementia with GRN mutations (FTD-GRN).

The oral presentation, entitled “Intrathalamic Delivery of AVB.PGRN Rescues Pathology in Grn Null Mice and Achieves Widespread Cortical Expression in a Large Animal Model without Expression in the Liver”, will be presented by AviadoBio Co-Founder and Chief Scientific and Clinical Advisor, Professor Chris Shaw, on Tuesday 17 May at 3:45pm (EST). The presentation will form part of the session “Applications of Improved Gene Therapy Methods in Neurologic Disorders” and will be followed by a Q&A discussion.

The abstract number 468 can be viewed here.

In addition, AviadoBio CEO Lisa Deschamps will be presenting at the conference, on Monday 16 May at 12:00pm (EST), in a session entitled: “Startup Showcase: AviadoBio – A revolution in gene therapy for neurodegenerative disorders”.

ABOUT AVIADOBIO

At AviadoBio, our mission is to transform the lives of people living with neurodegenerative disorders by developing and delivering transformative gene therapies for diseases including frontotemporal dementia (FTD) and amyotrophic lateral sclerosis (ALS). The Company’s technology is based on pioneering research from King’s College London and the UK Dementia Research Institute. AviadoBio’s unique platform combines next-generation gene therapy design with deep neuroscience expertise and a novel neuroanatomy-led approach to drug delivery. AviadoBio’s investors include New Enterprise Associates (NEA), Monograph Capital, Advent Life Sciences, EQT Lifesciences, Dementia Discovery Fund (DDF), F-Prime Capital, Johnson & Johnson Innovation – JJDC, Inc. (JJDC), and LifeArc.

The company is developing AVB-101 for patients with FTD-GRN. AVB-101 is an investigational AAV gene therapy designed to slow or stop disease progression by delivering a functional copy of the GRN gene throughout the central nervous system to restore progranulin levels.

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

About Frontotemporal Dementia and AVB-101

Frontotemporal dementia (FTD) is of the second most common form of dementia in people under the age of 65 after Alzheimer’s disease. It affects 50,000 to 60,000 patients in the U.S. and over 100,000 in the E.U. Approximately one third of FTD cases are familial and linked to autosomal dominant mutations in three genes including the granulin gene (GRN) and FTD-GRN represents 5-10% of all patients with FTD. Progressive degeneration of the frontal and temporal lobes of the brain is characteristic of FTD, and is associated with progressive decline of behaviour, decision-making, language and emotion, typically leading to death within 7-10 years of diagnosis. There are currently no approved treatments to stop or slow the progression of FTD or FTD-GRN.

References:

  • Boxer AL. Miller BL.  Alzheimer Dis Assoc Disord. 2005;19 Suppl 1:S3-6
  • Hogan DB, et al. Can J Neurol Sci. 2016;43 Suppl 1:S96-S109
  • Olney NT, et al. Neurol Clin. 2017;35(2): 339–374
  • Greaves CV, et al. J Neurol. 2019;266(8):2075–2086

CONTACT

For media enquiries:
Consilium Strategic Communications
Chris Gardner, Angela Gray, Isabelle Abdou
+44 (0) 20 3709 5700
AviadoBio@consilium-comms.com

May 03
Love0

Aura Biosciences Presents Preclinical Data Highlighting AU-011’s Anti-Tumor Activity in Choroidal Metastasis, an Additional Ocular Oncology Indication at the 2022 ARVO Annual Meeting

By Aura Biosciences, Press Release, Publicly Listed
Press Release.

 

Preclinical studies support further development of AU-011 for choroidal metastasis

CAMBRIDGE, Mass.–(BUSINESS WIRE)– Aura Biosciences Inc. (NASDAQ: AURA), a clinical-stage biotechnology company developing a novel class of virus-like drug conjugate (VDC) therapies for multiple oncology indications, today announced the presentation of preclinical data for its first VDC product candidate, AU 011. AU-011 is in development for the treatment of multiple ocular oncology indications, including primary choroidal melanoma and choroidal metastasis. The results presented today highlight AU-011’s targeted cytotoxicity towards tumor cells derived from the most common cancer types known to metastasize to the choroid. The results were presented as part of the 2022 Association of Research in Vision and Ophthalmology (ARVO) Annual Meeting taking place in Denver, CO on May 1 – 4 and virtually on May 11 – 12.

“The data presented at ARVO demonstrates AU-011’s potent cytotoxicity in human cancer cell lines as well as in multiple in vivo tumor models including breast, renal and colon cancer which are well known to metastasize to the choroid,” said Elisabet de los Pinos, Ph.D., Chief Executive Officer of Aura. “These results support our commitment to build our ocular oncology franchise and provide a potential novel treatment for patients with cancers in the eye. We look forward to submitting the IND in choroidal metastases in the second half of this year.”

In vitro potency was observed across a panel of human cancer cell lines. Cells were treated with AU-011 followed by light activation, and dose-dependent cell binding and cytotoxicity were noted. Addition of heparin to the binding studies prevented AU-011 association with the tumor cells demonstrating AU-011’s specific targeting of tumor associated heparan sulfate proteoglycans (HSPGs). Syngeneic murine models of breast, colon, and renal cancer were employed to evaluate AU-011’s anti-tumor activity in vivo and dose-dependent efficacy was observed across all models. The studies herein support further development of AU-011 for choroidal metastasis.

Details for the presentation are as follows:

Title: A first in class Virus-Like Drug Conjugate (VDC) shows anti-tumor activity in Cancers that Commonly Metastasize to the Choroid
Presentation Number: 2616
Presenter: Anneli Savinainen, Aura Biosciences
Session Title: Where art thou tumor? – Ocular tumor physiology and metastases
Date and time: Tuesday, May 3 at 3:00 – 3:17 PM ET
Location: 1AB Mile High Ballroom (Denver Convention Center)

The presentation can be accessed by visiting the “Scientific Presentations” section of “VDC Platform” page of the Aura Biosciences website.

About Aura Biosciences

Aura Biosciences, Inc. is a clinical-stage biotechnology company developing virus-like drug conjugates (VDCs), a novel class of therapies, for the treatment of multiple oncology indications. Aura’s lead VDC candidate, AU-011 (belzupacap sarotalocan), consists of a virus-like particle conjugated with an anti-cancer agent. AU-011 selectively targets and destroys cancer cells and activates the immune system with the potential to create long-lasting anti-tumor immunity. AU-011 is currently in development for ocular cancers, with an ongoing Phase 2 dose escalation clinical trial evaluating first-line treatment of choroidal melanoma, a vision- and life-threatening form of eye cancer where standard of care with radiotherapy leaves patients with severe comorbidities, including major vision loss. Aura plans to develop AU-011 across its ocular oncology franchise including for the treatment of patients with choroidal metastases. In addition, leveraging Aura’s technology platform, Aura is developing AU-011 more broadly across multiple cancers, starting with a planned Phase 1 clinical trial in patients with non-muscle invasive bladder cancer. Aura is headquartered in Cambridge, MA.

For more information, visit aurabiosciences.com, or follow us on Twitter and LinkedIn.

Forward Looking Statement

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 AU-011 for the treatment of choroidal metastasis, and expectations with respect to the anticipated timing of the submission of an IND in choroidal metastases.

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 clinical trials may not be predictive of future results in connection with future clinical trials; 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; risks, assumptions and uncertainties regarding the impact of the continuing COVID-19 pandemic on Aura’s business, operations, strategy, goals and anticipated timelines; Aura’s ongoing and planned pre-clinical 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 filed with the U.S. 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.

View source version on businesswire.com: https://www.businesswire.com/news/home/20220503005212/en/

Investor and Media:
Matthew DeYoung
Argot Partners
212-600-1902 | aura@argotpartners.com

 

May 02
Love0

Orphalan announces FDA approval of Cuvrior™ for the treatment of adult patients with stable Wilson’s disease who are de-coppered and tolerant to penicillamine

By Orphalan, Press Release, Private Companies
Press Release.

 

Paris, France 2 May 2022 – Orphalan SA (“Orphalan” or “the Company”), an international orphan drug development and commercialisation company, today announces approval of Cuvrior™, a new salt of trientine (trientine tetrahydrochloride) by the United States Food and Drug Administration (FDA). Cuvrior™ is approved for the treatment of adult patients with stable Wilson’s disease who are de-coppered and tolerant to penicillamine. Penicillamine is currently approved as a first-line treatment of Wilson’s disease in the US with about one third of patients developing intolerance1.

Orphalan recently completed a global phase III trial, CHELATE, which met its primary efficacy endpoint by demonstrating that Cuvrior™ was non-inferior to penicillamine as measured by non-ceruloplasmin copper (NCC). In consultation with the FDA, an assay based on total serum copper protein speciation was used for measuring this primary efficacy endpoint.

Wilson’s disease is a rare inherited disorder of copper transport primarily affecting the liver and brain. Orphalan commercializes its trientine tetrahydrochloride product in Europe under the name of Cuprior® and expects to launch Cuvrior™, which has been granted Orphan Drug Designation by the FDA, in the US by early 2023. The approval follows the New Drug Application (NDA) submission for the company’s product last year.

Dr. Naseem Amin, Chief Executive Officer at Orphalan, commented:

“We are delighted with the approval of our product, Cuvrior™, which provides a well-tolerated and effective option for Wilson’s disease patients. At Orphalan, we are committed to delivering innovative therapies, with our drug Cuprior® already approved and launched in European countries, and we look forward to launching Cuvrior™ in the United States. We also plan further national submissions to make our product available to patients globally.”

Mary L Graper, Vice President of Scientific Affairs, Wilson Disease Association, added:

“Wilson’s disease is a devastating disorder affecting patients worldwide and for which there has remained a significant need for innovative new treatments. The approval of Orphalan’s Cuvrior™ is extremely promising and is reflective of Orphalan’s patient-driven approach. This marks the culmination of many years of work and is an important moment, offering new hope for patients affected by this disease.”

Professor Michael Schilsky, MD, Director, Center for Excellence for Wilson Disease at Yale University, said:

“As a physician, I have seen first-hand how Wilson’s disease impacts the lives of patients and, until now, there have been few effective long-term treatment options available. The approval of Orphalan’s Cuvrior™ by the FDA is backed by positive data from Orphalan’s multicenter, multinational CHELATE trial – the first head-to-head controlled study of a new trientine salt versus penicillamine. For patients in need, Cuvrior™ represents a well-tolerated and effective alternative to penicillamine, the current standard of care.”

1 Weiss KH, et al. Efficacy and Safety of Oral Chelators in Treatment of Patients With Wilson Disease. Clin Gastroenterol Hepatol. 2013 Aug;11(8):1028-35.e1-2.
– ENDS –

About Trientine Tetrahydrochloride
Trientine tetrahydrochloride is an oral trientine formulation. In the US, trientine tetrahydrochloride has been granted with Orphan Drug Designation for the treatment of Wilson’s disease excluding patients intolerant of penicillamine. It has been approved under the 505(b)(2) pathway for the treatment of adult stable Wilson’s disease patients who are successfully de-coppered and tolerant to penicillamine. The 505(b)(2) regulatory pathway is a type of New Drug Application (NDA).

About Orphalan
Orphalan is an international orphan drug development and commercialisation company. The company delivers worldwide innovative therapies for people living with orphan diseases and is a pioneer in the space. Orphalan was founded in 2011 and has launched Cuprior®. across Europe with its own commercial organization. For more information visit www.orphalan.com and follow us on LinkedIn.

For more information, please contact:
Orphalan Tel: +33 (0)1 42 49 82 64
info@orphalan.com

Consilium Strategic Communications:
Mary-Jane Elliott, Davide Salvi, Genevieve Wilson
Tel: +44 (0) 203 709 5700
orphalan@consilium-comms.com

Apr 27
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Argá Medtech Shows Promise for New Cardiac Ablation Technology at 2022 Heart Rhythm Society Annual Meeting

By Argá Medtech, Press Release, Private Companies
Press Release.

 

  • Argá Medtech’s next-generation non-thermal cardiac ablation system shows promise in improving precision, speed and flexibility in treating atrial fibrillation
  • Preliminary feasibility study results demonstrate the company’s Coherent Sine-Burst Electroporation (CSE) Pulsed Field Ablation platform’s ability to titrate lesion depth using sinusoidal waveforms

Lausanne, Switzerland and San Diego, CA, USA — April 27, 2022 —Argá Medtech, a company developing Coherent Sine-Burst Electroporation (CSE), a next-generation non-thermal cardiac ablation system for treating cardiac arrhythmias, announced a featured poster presentation on preliminary findings for its innovative technology at the 2022 Annual Meeting of the Heart Rhythm Society (HRS), April 29 to May 1 in San Francisco. The company’s CSE Pulsed Field Ablation (PFA) offers unmatched flexibility in treating atrial fibrillation (AF) with its proprietary CSE generator and a multi-configurable catheter.

“AF affects 37.5 million people worldwide1, significantly raising their chances of a stroke or heart attack and greatly diminishing their quality of life,” said David Neale, CEO and co-founder of Argá Medtech. “While the cardiac ablation field has achieved major progress in treating AF, outcomes have only marginally improved in the past 15 years, and costs have increased. It is imperative to develop new tools that improve success rates while lowering procedure costs.”

Argá Medtech’s unique CSE PFA platform offers several advantages over other PFA technologies, which are powered mostly by a square wave energy source. Argá’s new CSE system allows physicians to select all bipolar, all unipolar or combinations of bipolar and unipolar energy delivery modes to titrate the lesion to the needs of the patient.

Argá Medtech’s catheter also offers unmatched flexibility in its ability to perform any lesion type necessary for the patient’s treatment of AF, eliminating the need to use additional ablation catheters. Its design enables the catheter to take different shapes allowing for the creation of circular, linear or focal lesions. These advantages offer the potential to improve precision, speed, safety and efficiency in cardiac ablation procedures while reducing procedure costs.

“Argá Medtech gives physicians an unparalleled ability to perform much more flexible, elegant ablation procedures, likely improving outcomes and reducing the need for repeat surgeries and saving costs for both hospitals and the healthcare system,” said Neale. “We are very optimistic about our technology’s promise and plan to launch our first in-human trial in 2022.”

The HRS presentation reports findings from an animal feasibility study on using multi-programmable CSE waveform to achieve a titratable range of lesion depths that address the variability of tissue thickness within the atrium and ventricle.

Presentation Information
Multi-programmable Coherent Sine Burst Electroporation Waveform for Atrial and Ventricular Catheter Ablation
Date: Friday, April 29, 2022
Time: 1:30 – 1:45 p.m. PT
Location: Abstract Pavilion Podium 11
Authors: Micah Lee BS, Ricardo Roman BS, Ale Lopez BS, Mark Zurcher MS, Deborrah Hunt BS, Randy Werneth MS, Kurt S Hoffmayer MD.

About Argá Medtech
Argá Medtech SA (Argá) is a privately held medical device company based in Switzerland founded by a seasoned team of leaders in the medical device industry. Argá is developing an innovative non-thermal energy-based cardiac ablation system for the treatment of cardiac arrhythmias. With the use of Pulsed Electrical Fields to create irreversible electroporation of cardiomyocytes, Argá is developing a safer, faster, cheaper and more effective cardiac ablation treatment for the benefit of millions of people affected by cardiac rhythm disorders and atrial fibrillation. To learn more, visit https://argamedtech.com.

Lippi G, Sanchis-Gomar F, Cervellin G. Global epidemiology of atrial fibrillation: An increasing epidemic and public health challenge. Int J Stroke. 2021 Feb;16(2):217-221. doi: 10.1177/1747493019897870. Epub 2020 Jan 19. Erratum in: Int J Stroke. 2020 Jan 28;1747493020905964. PMID: 31955707

 

Apr 25
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Novel Cryptigen™ Technology unlocks full potential of Tumour Specific Antigens

By Epitopea, Press Release, Private Companies
Press Release.

 

CAMBRIDGE, UK and MONTREAL, CANADA, 25 April 2022 – Epitopea, a transatlantic cancer immunotherapeutics company and global leader in exploiting a new class of untapped tumour-specific antigens (TSAs), announces a $13.6M (£10.3M) seed investment from a transatlantic syndicate of top-tier life sciences investors, including Advent Life Sciences, CTI Life Sciences, Cambridge Innovation Capital (CIC) and Fonds de solidarité FTQ. The seed round was also supported by Novateur Ventures and the Harrington Discovery Institute/University Health Holdings. The funding will leverage Epitopea’s ground-breaking Cryptigen™ approach to create transformational immunotherapies that target broad cancer patient populations in both solid and haematological cancers.

The company’s proprietary technology provides an innovative approach to identifying shared, aberrantly expressed tumour specific antigens, known as Cryptigens™, that it has exclusively licensed from the Université de Montréal (UdeM). The licensing deal was catalysed by IRICoR, a Canadian Centre of Excellence in Commercialization and Research, which supported and incubated the development of the technology. Cryptigen™ TSAs are uniquely and broadly presented across cancer types, providing tumour-specific targets for immunotherapies that are predicted to kill malignant cells while sparing non-cancerous cells, potentially delivering significant therapeutic benefit across broad patient populations with minimal side effects.

The seed round funding will be used to build the company’s executive team, advance further research on this new class of antigens, and catalyse their translation into novel cancer immunotherapeutics, including therapeutic vaccines, cell therapies, and TCR-based biologics.

Dr Jon Moore, CEO, Epitopea and Operating Partner at Advent Life Sciences, said

 “The outstanding work of Epitopea’s co-founders, Université de Montréal scientists Drs. Claude Perreault, and Pierre Thibault, has opened a tremendous and potentially transformative opportunity for future cancer patients. With this significant seed financing by a syndicate of world-class investors from Canada and the UK, we can start translating these discoveries into novel therapeutics. We will be guided by science, deploying the best modalities available to help cancer patients achieve durable benefits.”

Dr Laurence Rulleau, Partner, CTI Life Sciences, said

 “CTI is delighted to co-lead the seed round of Epitopea, which is the first investment from our third fund, CTI LSF III.  We are convinced that Epitopea has the potential to fundamentally change the paradigm of how cancer patients can be treated with therapeutics vaccines and other immunotherapies to significantly improve their quality of life. Epitopea will explore a variety of early and longer-term value creation strategies including partnerships with third parties as well as developing its own therapeutic modalities based on its Cryptigen™ TSAs.”

Notes To Editors

Contact information

Epitopea
Dr Jon Moore, CEO
jon.moore@epitopea.com

Scius Communications
Katja Stout
Tel: +44 7789 435990
katja@sciuscommunications.com

About Epitopea

Epitopea is a transatlantic biotechnology company developing transformational immunotherapies to treat cancer by targeting a new class of antigens that are broadly shared between patients with the same cancer indication. Cryptigen™ TSAs are discovered by a proprietary approach deploying immunopetidomics, mass spectrometry, genomics, and bioinformatics, which allows the identification of conserved, aberrantly-expressed, tumour-specific antigens, hidden in cancer’s ’junk’ DNA. These hidden Cryptigen™ TSAs have been identified by research led by Drs. Claude Perreault and Pierre Thibault at the Institute for Research in Immunology and Cancer at Université de Montréal.

The company has an extensive proprietary library of Cryptigen™ TSAs that will drive the development of transformational off the shelf cancer immunotherapies. Epitopea is backed by leading life science investors including Advent Life Sciences, CTI Life Sciences, Cambridge Innovation Capital, Le Fonds de Solidarité FTQ, the Harrington Discovery Institute and Novateur Ventures. The company has raised over US$13.6 million in seed financing. Epitopea was founded in 2021 and consists of sister companies based in Cambridge, UK and in Montreal, Canada. Please see www.epitopea.com for more information and follow us on LinkedIn.

For more information about our investors please see the following links:

Advent Life Sciences: www.adventls.com

CTI Life Sciences: https://www.ctisciences.com

Cambridge Innovation Capital: www.cic.vc

Fonds de solidarité FTQ: https://www.fondsftq.com

Novateur Ventures: https://www.novateur.ca

Harrington Discovery Institute/University Health Holdings: https://www.harringtondiscovery.org

For more information about the Université de Montréal, please see here: https://www.umontreal.ca/en/

For more information about IRICoR, please see here: https://www.iricor.ca

Apr 14
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Highlight Therapeutics announces follow-up results from Phase 2b study of BO-112 + anti-PD1 in confirmed anti-PD1 progressor melanoma patients at AACR

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

 

BO-112 demonstrates potential as new and highly effective second line therapy for melanoma

  • BO-112 demonstrates potential as best-in-class therapy to overcome anti-PD1 resistance in melanoma patients whose disease has progressed on prior anti-PD-1 treatment
  • Primary endpoint met with a 30% Response Rate, 15% Complete Responses (CR) and 65% Disease Control Rate (DCR) substantially exceeding current standard of care
  • Further improvements anticipated over one year follow up
  • Hard-to-treat mucosal melanoma patients achieved 66% Overall Response Rate (ORR) and 100% DCR
  • Durable responses and manageable safety profile, with no patients discontinuing due to adverse events
  • Potential for use in multiple solid cancers resistant to anti-PD1 inhibitors, and with different anti-PD1 combinations

Madrid, Spain, 13 April, 2022 – Highlight Therapeutics (“Highlight”), a clinical-stage biopharmaceutical company developing RNA-based therapies against cancer, today announced positive results of a Phase 2 study of intratumoral administration of BO-112 with pembrolizumab in patients with advanced melanoma whose disease had progressed on first-line anti-PD1-based therapy. BO-112 is a dsRNA agonist targeting anti-PD1 resistance, which has demonstrated anti-cancer activity in previous Phase 1b studies.

Results of the study were presented at the plenary session at the American Association for Cancer Research (AACR) Annual Meeting 2022 in New Orleans, Louisiana by Iván Marquez-Rodas (CT014. Efficacy of intratumoral BO-112 with systemic pembrolizumab in patients with advanced melanoma refractory to anti-PD-1-based therapy: Final results of SPOTLIGHT203 phase 2 study. 10:15 –12:15 PM CT)

Sales of anti-PD1 therapies are valued at approximately $24 billion1 a year and they are used to treat most solid tumors. However, currently fewer than 20% of all cancer patients benefit from first-line anti-PD1 treatment. BO-112 in combination with anti-PD1 therapy is designed to resensitize tumors to anti-PD1 treatment through improved antigen presentation, enhanced T-cell infiltration and increased MHC-1 and PDL1 expression by the tumor itself.

“This remarkable data confirms that BO-112 is effective at resensitizing tumors and helping to overcome anti-PD1 resistance, opening up the potential to treat many more patients with anti-PD1 therapies. Analysis of the data showed that most patients achieved a response rate of 40%, although patients with very high LDH levels and acral melanoma did not obtain clinical benefit.” Dr. Marisol Quintero, CEO of Highlight Therapeutics, commented. “Importantly, BO-112 was able to achieve improvements even in patients who had initially responded to anti-PD1 therapy before regressing and in hard-to-treat mucosal melanoma, overall response rates of 66% and disease control rates of 100% were seen, greatly exceeding current standard of care.”

Highlight Therapeutics and Merck, known as MSD outside the United States and Canada, conducted an open-label, single arm study to evaluate the efficacy & safety of intra-tumoral administration of BO-112 + pembrolizumab in mucosal, acral and cutaneous melanoma patients whose disease had progressed, confirmed by two consecutive CT scans. The study recruited 42 patients in France and Spain, with recruitment completed by August 24, 2021. Patients included those with high LDH levels, which are often associated with poor response rates and have been excluded from comparable clinical trials.

The analysis shows:

  • Primary endpoint (ORR by independent reviewer) has been met
  • With a median follow up of seven months, there is a clear clinical benefit in patients with confirmed anti-PD1-resistant melanoma, with a 30% ORR and a 65% DCR: superior to 2nd line Standard of Care in stage III/IV melanoma of ~8% (continuing with anti-PD1 Ab) or 13% (second line ipilimumab).
  • Three hard-to-treat mucosal melanoma patients have achieved an ORR of 66% and DCR of 100%
  • High baseline LDH levels (>3xULN) predict progressive disease
  • Responses and stable diseases are durable
  • Study treatment has a manageable safety profile, with no patients discontinuing due to adverse events

Next steps in the development of BO-112 include:

  • Initiation of a randomized Phase 2 study in 2nd-line melanoma is planned in 2023
  • Highlight Therapeutics has initiated strategic partnerships discussions with anti-PD1 companies interested in enhancing their anti-PD1 market potential

1. IQVIA Global Oncology Report 2020

For more information, please contact:

Highlight Therapeutics S. L.             info@highlighttherapeutics.com
Marisol Quintero, CEO

Mo PR Advisory                             Tel: +44 (0) 7876 444977 / 07860 361746
Mo Noonan/Jonathan Birt

Notes to editors

About Highlight Therapeutics

Highlight Therapeutics, formerly known as Bioncotech Therapeutics, is a private, clinical-stage company dedicated to unlocking the full potential of immuno-oncology. Our lead drug candidate BO-112 is a best-in-class RNA-based therapy which has been demonstrated to initiate a powerful immune response, leveraging a unique multi-target approach to turn ‘cold’ tumors ‘hot’ and therefore visible to the immune system. It has the potential to rescue patients who are resistant to current checkpoint inhibitor therapy, a very large market opportunity. BO-112 is currently being investigated in a range of clinical trials as a monotherapy and in combination with checkpoint inhibitors. In addition to in-house research, Highlight Therapeutics has a number of external collaborators, including Merck & Co and UCLA.

For more information, please visit www.highlighttherapeutics.com