Orexo acquires US rights to commercialise vorvida

Pharmaceutical Technology | November 28, 2019

Swedish company Orexo has purchased the exclusive US rights to vorvida, a digital therapy to treat alcohol use disorder (AUD), from GAIA, for an undisclosed sum. In August, Orexo signed an agreement with GAIA to secure exclusive secure global commercial rights to OXD01, a digital therapy being developed to treat OUD. Under the deal, Orexo will take responsibility for the regulatory approval and commercialisation of vorvida digital therapy in the US. The deal allows GAIA to secure an upfront payment, milestone payments and royalties. Both firms will also negotiate with the FDA regarding the regulatory pathway of digital therapy to help determine the timing of the therapy’s launch in the US and the need for additional investments. GAIA CEO and founder Dr Mario Weiss said: “Working with the Orexo team on the first agreement showed us their dedication and vision of the potential for digital therapeutics in the field of addiction.”

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VIEWS AND ANALYSIS, PHARMACY MARKET

Vincerx Pharma Presents Preclinical Data on VIP943 in Acute Myeloid Leukemia Models at the 64th American Society of Hematology Annual Meeting 2022

Vincerx Pharma, Inc. | December 12, 2022

Vincerx Pharma, Inc. a biopharmaceutical company aspiring to address the unmet medical needs of patients with cancer through paradigm-shifting therapeutics, today announced a poster presentation of preclinical data of Vincerx’s proprietary payload and linker technology and VIP943, the Company’s internalizing ADC targeting CD123, at the 64th American Society of Hematology Annual Meeting 2022. VIP943 is a novel ADC, which binds to the IL3-receptor alpha chain . VIP943 combines the unique payload class of kinesin spindle protein inhibitors with a proprietary legumain-cleavable linker. Vincerx’s CellTrapper™ modification of the KSPi prevents diffusion out of the cell, allowing intracellular accumulation. The nonpermeability of the payload prevents off-target toxicities, leading to favorable efficacy and safety profiles. “I am truly excited about the preclinical results for VIP943 and our proprietary payload and linker technology presented at ASH,” said Ahmed Hamdy, M.D., Chief Executive Officer of Vincerx. “For the first time, we showed a significant improvement in safety over an approved ADC, demonstrating in non-human primates the benefit of our KSPi payload and CellTrapper technology specifically designed to address some of the well-known challenges of ADCs on the market. The in vivo AML mouse model results also showed improved efficacy and survival for VIP943 in combination with venetoclax and azacitidine. This triple combination resulted in significant tumor regression as demonstrated by five complete responses and significantly prolonged survival time without increased toxicity,” added Dr. Hamdy. “This ADC is innovative and exciting. The target is well understood and the novel payload that targets myeloid cells suggests this could be a valuable agent for patients with AML.” Dr. Anthony Tolcher, M.D., Chief Executive Officer, Founder and Director of Clinical Research at NEXT Oncology “Current standard of care for AML patients is combination therapy with venetoclax and azacitidine, yet most patients eventually relapse with progressive disease. The efficacy and safety data for VIP943 we see in our studies suggest it may be a promising option for treating AML as a monotherapy in relapse/refractory elderly, unfit and high-risk patients as well as in combination with venetoclax and azacitidine. Our results also provide compelling evidence that VIP943 represents a substantial advancement and potential paradigm-shift in ADC technology. We look forward to continuing to advance the IND-enabling studies for VIP943 and expect to file our IND in mid-2023,” concluded Dr. Hamdy. ABOUT VINCERX PHARMA, INC. Vincerx Pharma, Inc. is a clinical-stage life sciences company focused on leveraging its extensive development and oncology expertise to advance new therapies intended to address unmet medical needs for the treatment of cancer. Vincerx has assembled a management team of biopharmaceutical experts with extensive experience in building and operating organizations that develop and deliver innovative medicines to patients. Vincerx’s current pipeline derives from an exclusive license agreement with Bayer and includes a clinical-stage and follow-on small molecule drug program and a preclinical stage modular bioconjugation platform, which includes next-generation antibody-drug conjugates and innovative small molecule drug conjugates.

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BUSINESS INSIGHTS

BioMarin Announces Advancements in FDA Review of ROCTAVIAN™ (Valoctocogene Roxaparvovec) for Adults with Severe Hemophilia A

BioMarin Pharmaceutical Inc. | November 24, 2022

BioMarin Pharmaceutical Inc. announced advancements in the U.S. Food and Drug Administration review of the Biologics License Application of ROCTAVIAN™ for adults with severe hemophilia A. The Company was recently notified by the FDA that after further consideration, at this time, the Agency no longer plans to hold an advisory committee meeting to discuss the BLA for ROCTAVIAN that is currently under review. Previously, the FDA communicated to the Company that it did intend to hold an advisory committee meeting but did not specify a date. The Company also remains on track to host the scheduled FDA Pre-Licensure Inspection (PLI) of BioMarin's gene therapy manufacturing facility located in Novato, CA. "The review of a BLA is a dynamic process, and we appreciate FDA's ongoing engagement as we work toward delivering a potentially transformative treatment choice to those patients with severe hemophilia A. We look forward to further dialogue with the Agency as it reviews our application." Hank Fuchs, M.D., President of Worldwide Research and Development at BioMarin About valoctocogene roxaparvovec The FDA granted Regenerative Medicine Advanced Therapy designation to valoctocogene roxaparvovec in March 2021. RMAT is an expedited program intended to facilitate development and review of regenerative medicine therapies, such as valoctocogene roxaparvovec, that are expected to address an unmet medical need in patients with serious conditions. The RMAT designation is complementary to Breakthrough Therapy Designation, which the Company received for valoctocogene roxaparvovec in 2017. In addition to the RMAT Designation and Breakthrough Therapy Designation, BioMarin's valoctocogene roxaparvovec also received orphan drug designation from the EMA and FDA for the treatment of severe hemophilia A. Orphan drug designation is reserved for medicines treating rare, life-threatening, or chronically debilitating diseases. The European Commission (EC) granted conditional marketing authorization to valoctocogene roxaparvovec gene therapy under the brand name ROCTAVIAN™ on August 24, 2022. Robust Clinical Program BioMarin has multiple clinical studies underway in its comprehensive gene therapy program for the treatment of severe hemophilia A. In addition to the global Phase 3 study GENEr8-1 and the ongoing Phase 1/2 dose escalation study, the Company is also conducting a Phase 3, single arm, open-label study to evaluate the efficacy and safety of valoctocogene roxaparvovec at a dose of 6e13 vg/kg with prophylactic corticosteroids in people with severe hemophilia A. Also ongoing is a Phase 1/2 Study with the 6e13 vg/kg dose of valoctocogene roxaparvovec in people with severe hemophilia A with pre-existing AAV5 antibodies (Study 270-203) and a Phase 1/2 Study with the 6e13 vg/kg dose of valoctocogene roxaparvovec in people with severe hemophilia A with active or prior Factor VIII inhibitors. About Hemophilia A People living with hemophilia A lack sufficient functioning Factor VIII protein to help their blood clot and are at risk for painful and/or potentially life-threatening bleeds from even modest injuries. Additionally, people with the most severe form of hemophilia A often experience painful, spontaneous bleeds into their muscles or joints. Individuals with the most severe form of hemophilia A make up approximately 50 percent of the hemophilia A population. People with hemophilia A with moderate or mild disease show a much-reduced propensity to bleed. Individuals with severe hemophilia A are treated with a prophylactic regimen of intravenous Factor VIII infusions administered 2-3 times per week or a bispecific monoclonal antibody that mimics the activity of Factor VIII administered 1-4 times per month. Despite these regimens, many people continue to experience breakthrough bleeds, resulting in progressive and debilitating joint damage, which can have a major impact on their quality of life. Hemophilia A, also called Factor VIII deficiency or classic hemophilia, is an X-linked genetic disorder caused by missing or defective Factor VIII, a clotting protein. Although it is passed down from parents to children, about 1/3 of cases are caused by a spontaneous mutation, a new mutation that was not inherited. Approximately 1 in 10,000 people have hemophilia A. About BioMarin BioMarin is a global biotechnology company that develops and commercializes innovative therapies for people with serious and life-threatening genetic diseases and medical conditions. The Company selects product candidates for diseases and conditions that represent a significant unmet medical need, have well-understood biology and provide an opportunity to be first-to-market or offer a significant benefit over existing products. The Company's portfolio consists of eight commercial products and multiple clinical and preclinical product candidates for the treatment of various diseases.

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BUSINESS INSIGHTS, PRACTICE MANAGEMENT

Telix Announces Executive Leadership Appointments

Telix Pharmaceuticals Limited | December 05, 2022

Telix Pharmaceuticals Limited announces several key executive leadership appointments and promotions, reflecting the increased commercial focus of the Company and ongoing succession planning. Richard Valeix promoted to Group Chief Commercial Officer Richard Valeix, who has been in the role of Chief Executive Officer of Telix's Europe Middle East and Africa operations since joining Telix in May 2021, has been appointed to the newly created role of Group Chief Commercial Officer. This key global leadership role will oversee commercialisation strategy and activities for Telix's late-stage pipeline assets including the global rollout of Illuccix and launch of TLX250-CDx, Telix's kidney cancer imaging agent for which the Company recently reported positive Phase III data. Richard is a highly experienced business leader, with marketing and sales and general management experience in large pharmaceutical companies, having previously held senior roles at Advanced Accelerator Applications a Novartis company, Ipsen and Roche. Raphael Ortiz has been promoted to Chief Executive Officer - EMEA. He has held the position of Chief Operating Officer - EMEA since joining in January 2022. Raphael has more than 20 years of pharmaceutical industry experience across finance, business development, marketing, sales and general management and has previously held senior roles at AAA, where he established radioligand therapy operations for the Asia Pacific region. Genevieve holds Honours degrees in Science and Law from Monash University and a Graduate Diploma of Applied Corporate Governance from the Governance Institute of Australia. Genevieve is a Solicitor of the Supreme Court of Victoria and a Fellow of the Governance Institute of Australia. She replaces Melanie Farris who has retired from the role of Group Company Secretary to take on a broader portfolio as Senior Vice President Global Governance, Risk and Compliance. Melanie has served as Group Company Secretary since March 2017, prior to the Company's listing on the ASX, and has over 18 years' experience in governance and corporate operations. "These leadership appointments are a result of ongoing succession planning to ensure Telix has an optimal mix of skills and experience as we prepare to enter a new phase with a portfolio of multiple commercial products. I congratulate Richard, Raphael and Melanie on their new roles and am pleased that we are able to identify and develop talent to support the Company as it continues to grow. I'm also pleased to welcome Genevieve Ryan as Group Company Secretary, who brings a new depth of experience working within ASX-200 companies." Dr. Christian Behrenbruch, Telix Group CEO and Managing Director About Telix Pharmaceuticals Limited Telix is a biopharmaceutical company focused on the development and commercialisation of diagnostic and therapeutic radiopharmaceuticals. Telix is headquartered in Melbourne, Australia with international operations in the United States, Europe and Japan. Telix is developing a portfolio of clinical-stage products that aims to address significant unmet medical need in oncology and rare diseases. Telix is listed on the Australian Securities Exchange.

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BUSINESS INSIGHTS, PHARMACY MARKET

Annogen Announces Research Project with Pfizer to Test Non-coding Genetic Sequences

Annogen | December 14, 2022

Annogen, the Amsterdam based biotech company behind the SuRE™ technology for the functional annotation of the non-coding part of the genome, today announced it has begun a research project with Pfizer to functionally test tens of thousands of disease-related non-coding sequence variants for their effect on gene regulation. The results may contribute to drug discovery by identifying functionally relevant non-coding variants that play a central role in diseases. More than 95 percent of disease- and trait-related variants are found in the non-coding genome. However, identifying the important causal variants amongst the thousands of non-functional ones is a major challenge, as non-coding variant functionality cannot be deduced from sequence alone. Using its SuRE™ methodology, Annogen can obtain a functional read-out for up to millions of non-coding variants in parallel. “We are very proud of this research project with Pfizer. We believe the non-coding part of the genome represents a huge opportunity for drug discovery that has been largely overlooked. We are now working with several top-tier biopharma companies on a broad array of projects, which demonstrates the interest in the SuRE™ platform and the AIM™ service for gene & cell therapy, drug discovery, and recombinant protein production”. Joris van Arensbergen, Annogen’s founder and CEO, is pleased to establish another project in this field About Annogen At Annogen we use our SuRE™ technology to identify regulatory DNA elements to be used for controlled gene expression valuable for gene & cell therapy, as well as for recombinant protein production. In addition, we offer the AIM™ service to identify favorable gene insertions and their expression levels for more than 100,000 integrations in parallel. These approaches enable researchers to qualitatively interpret the non-coding genome in humans, animals and plants.

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