Ear Diseases

BOSTON & SAN DIEGO--(BUSINESS WIRE)-- Rapport Therapeutics, Inc., a clinical-stage biotechnology company leveraging the science of receptor-associated proteins (RAPs) to advance precision neuromedicines, today announced the appointment of John Maraganore, Ph.D., to its Board of Directors. Dr. Maraganore, former founding Chief Executive Officer of Alnylam Pharmaceuticals, Inc. (Alnylam), currently serves as Venture Partner at both ARCH Venture Partners and Atlas Ventures, as Senior Advisor at Blackstone Life Sciences, and as Principal and Chief Executive Officer at JMM Innovations, LLC. This press release features multimedia. View the full release here: John Maraganore, Ph.D. “As Rapport sets out to build a leading precision neuroscience company, we are incredibly fortunate to have Dr. Maraganore join our board,” said Abraham Ceesay, Chief Executive Officer of Rapport. “John is among a few visionary leaders who have guided a new modality from concept to a dynamic platform for drug discovery. He has been a driving force for innovation within our industry, and we believe his leadership will be indispensable as we work to advance our pipeline and potentially deliver much-needed new treatment options to patients with neurological disorders.” “I am thrilled to once again work alongside John to help Rapport realize its vision to become a preeminent precision neuroscience company. John has an impressive track record of building great science-based companies. His wisdom and experience will be invaluable as we advance our pipeline of precision neuroscience product candidates to positively impact the lives of those affected by debilitating neurological conditions,” said Steve Paul M.D., Chairman of the Rapport Board of Directors and Venture Partner at Third Rock Ventures. Dr. Maraganore served as founding CEO of Alnylam from 2002 to 2021, where he led the development of a novel class of medicines, referred to as RNAi therapeutics. Under his leadership, Alnylam built over $25 billion in market capitalization, established more than 20 major pharmaceutical alliances and saw the approval and commercialization of four of its RNAi medicines, ONPATTRO®, GIVLAARI®, OXLUMO®, and Leqvio®. Prior to founding Alnylam, Dr. Maraganore held leadership positions at Millennium Pharmaceuticals, Inc. and Biogen, Inc. He also previously served as Chair of the Biotechnology Innovation Organization. Dr. Maraganore received his B.A. in biological sciences and M.S. and Ph.D. in biochemistry and molecular biology from the University of Chicago. “There's a significant need and opportunity for new approaches in the treatment of neurological disorders, and Rapport has the potential to revolutionize care with its transformational science,” said Dr. Maraganore. “I'm honored to work with a team of such talented and accomplished individuals in these exciting times for both the company and the field at large.” About Rapport ( ) Rapport is developing a new class of precision neuromedicines aimed at providing patients with better alternatives to existing treatments for central nervous system disorders. Current options often come with debilitating and intolerable side effects, leading to suboptimal dosing and noncompliance that limit drug efficacy and can result in discontinuation. The company’s platform, built and refined over the past decade by its founding scientific team, is designed to achieve precision through the discovery and targeting of receptor-associated proteins, or RAPs, which display regional or neural circuit-specific expression. By more precisely targeting the diseased brain regions, Rapport believes its platform has the potential to improve tolerability and clinical benefit. Rapport has ongoing clinical trials in epilepsy and discovery-stage programs in neuropathic pain, hearing disorders and bipolar disorder.

Designation grants access to enter the Innovative Licensing and Access Pathway (ILAP) Five patients treated with FBX-101 show positive safety and efficacy results, including significant improvements in motor function Forge’s CMO, Dr. Maria Escolar, to present updated FBX-101 clinical data during the Advanced Therapies 2024 conference in London March 19-20 Designation provides opportunities for further global acceptance of Forge’s manufacturing process and facility through additional reviews and inspections COLUMBUS, Ohio--(BUSINESS WIRE)-- Forge Biologics, a member of Ajinomoto Bio-Pharma Services and a leading manufacturer of genetic medicines, today announced that the Medicines and Healthcare Products Regulatory Agency (MHRA), the healthcare regulatory body of the United Kingdom (UK), granted Innovation Passport designation to the Company’s novel AAV gene therapy program, FBX-101, to enter the Innovative Licensing and Access Pathway (ILAP). FBX-101 was created for the treatment of patients with Krabbe disease, a rare neurodegenerative disease that is usually fatal in untreated patients by age two. The ILAP is intended to accelerate regulatory and market access interactions in the UK. Maria Escolar, M.D., Forge’s Chief Medical Officer, will discuss the significance of achieving this regulatory designation and present a poster on updated data from REKLAIM, an early phase clinical trial for FBX-101, during the Advanced Therapies 2024 conference being held in London, March 19-20, 2024. “Considering the rapid progression of Krabbe disease in young patients and the positive results we’ve observed in the REKLAIM trial, we are pleased to receive the Innovation Passport designation which will enable Forge to further accelerate the clinical development of FBX-101,” stated Dr. Escolar. “ILAP was created to provide access to tools to decrease the time to market and facilitate patient access to medicines in the UK for life-threatening or seriously debilitating conditions.” The REKLAIM clinical trial is evaluating the safety and efficacy of FBX-101 in patients with infantile and late infantile Krabbe disease. Children assessed in REKLAIM have received intravenous FBX-101 in addition to the current standard of care, hematopoietic stem cell transplantation (HSCT). Clinical data demonstrate that FBX 101 has been well tolerated and has an excellent safety pro the treatment of five patients. Results show improvements in motor function in all patients, the most meaningful clinical outcome for this disease. FBX-101 has been granted Orphan Drug Designation and Priority Medicines (PRIME) designation by the European Medicines Agency (EMA), allowing Forge to advance and expedite the development of FBX-101 as the leading worldwide gene therapy for patients with Krabbe disease. In the US, the FDA has granted FBX-101 Fast Track Designation, Orphan Drug Designation, and Rare Pediatric Disease Designation. ILAP is intended to accelerate regulatory and market access interactions in the UK for patient populations with significant need, leading to accelerated patient access to novel therapies. It also provides access to a range of UK development services and tools, including frequent MHRA interactions, accelerated Marketing Authorization Application (MAA) assessment, a Target Development Profile, expanded licensing routes, and more, creating a roadmap for advancement of therapies through regulatory approval. Regulatory review by the MHRA through ILAP provides opportunities for further global acceptance of Forge’s manufacturing process and facility though additional reviews and inspections, including Compliance Readiness Inspections that provide custom and pragmatic supervisory and licensing inspections specific to the GMP guidelines. Through this designation Forge has expanded global regulatory experience to include interactions with the MHRA in the UK. This access to global health authority resources will enable an added level of assurance to clients utilizing Forge’s CDMO services with integrated regulatory support. About Krabbe Disease Krabbe disease is a rare neurodegenerative disease affecting about 1-2.5 in 100,000 people in the US. Krabbe disease is caused by autosomal recessive mutations in the galactocerebrosidase (GALC) gene, an enzyme responsible for the breakdown of certain types of sphingolipids, such as psychosine. Without functional GALC, psychosine accumulates to toxic levels in cells, specifically in cells insulating the nerves in the brain and peripheral nervous system, causing rapid demyelination. Krabbe disease initially manifests in young patients as irritability, developmental delay, and progressive muscle weakness. Symptoms rapidly advance to difficulty swallowing, breathing, and regression of neurodevelopment followed by seizures, vision and hearing loss. Infantile Krabbe disease (0-12 months of age at onset) usually leads to death in untreated patients by two years of age. Late Infantile patients (12-36 months of age at onset) usually die by the age of six. The current standard of care, hematopoietic stem cell transplantation (HSCT), has been shown to stabilize cognitive decline and significantly improve long-term neurological outcomes. However, HSCT does not correct the peripheral neuropathy that is progressive as the patient grows, leading to loss of gross motor skills and eventually death. About FBX-101 FBX-101 was developed to treat children with Krabbe disease. FBX-101 is an adeno-associated viral serotype rh10 (AAVrh10) gene therapy that is delivered intravenously after HSCT infusion. The vector delivers a functional copy of the GALC gene to cells in both the central and peripheral nervous system and has shown to functionally correct the central and peripheral neuropathy, improve myelination and gross motor function, and significantly prolong lifespan in animal models. This approach also has the potential to overcome some of the immunological safety challenges observed in traditional AAV gene therapies and extend the duration of gene transfer. About the REKLAIM Trial REKLAIM is a nonblinded, non-randomized, early phase dose escalation clinical trial currently enrolling children with asymptomatic infantile and symptomatic late infantile Krabbe disease to investigate the safety and efficacy of a single intravenous infusion of FBX-101 administered more than 21 days after HSCT, the current standard of care. Data from extensive natural history subjects will be used to compare as the control group. More information on the REKLAIM trial can be found online at . About Forge Biologics Forge Biologics, a member of Ajinomoto Bio-Pharma Services, is a hybrid gene therapy contract manufacturing and clinical-stage therapeutics development company, enabling access to life-changing gene therapies by bringing them from concept to reality. Forge’s 200,000 square foot facility, the Hearth, is headquartered in Columbus, Ohio, and houses 20 custom-designed cGMP suites with 200,000L of manufacturing capacity. Forge’s end-to-end, scalable plasmid and AAV manufacturing services include research-grade manufacturing, process and analytical development, cGMP manufacturing, fill and finish, and integrated regulatory support to help accelerate the timelines of transformative medicines for patients with genetic diseases. To learn more, visit .

RESEARCH TRIANGLE PARK, N.C., March 19, 2024 (GLOBE NEWSWIRE) -- Fennec Pharmaceuticals Inc. (NASDAQ: FENC; TSX: FRX), a commercial stage specialty pharmaceutical company, today announced that the Company will release its full year and fourth quarter 2023 financial results before the opening of the U.S. financial markets on Thursday, March 21, 2024. Management will host a conference call and webcast that day to discuss the Company’s financial and business results. Conference Call & Webcast Detail: Date: Time: Link: Thursday, March 21, 2024 8:30 a.m. Eastern Time To access the conference call, please register using . Upon registration, a dial-in number and unique PIN will be provided to join the call. To access the live webcast link, log onto and proceed to the News & Events / Event Calendar page under the Investors & Media heading. Please connect to the company’s website at least 15 minutes prior to the conference call to ensure adequate time for any software download that may be required to listen to the webcast. A webcast replay of the conference call will also be archived on for thirty days. About Fennec Pharmaceuticals Fennec Pharmaceuticals Inc. is a specialty pharmaceutical company focused on the development and commercialization of PEDMARK® and Pedmarqsi to reduce the risk of platinum-induced ototoxicity in pediatric patients. Further, PEDMARK received FDA approval in September 2022 and European Commission Marketing Authorization in June 2023 for Pedmarqsi. PEDMARK has received Orphan Drug Exclusivity in the U.S. for seven years of market protection and Pedmarqsi has received Pediatric Use Marketing Authorization in Europe which includes eight years plus two years of data and market protection. For more information, please visit . Corporate and Media: Lindsay Rocco Elixir Health Public Relations +1 862-596-1304 lrocco@elixirhealthpr.com