uniQure: FDA Removes Clinical Hold on Hemophilia B Gene Therapy

  uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, announced today that the U.S. Food and Drug Administration (FDA) has removed the clinical hold on the Company’s hemophilia B gene therapy program after determining the Company satisfactorily addressed all issues identified by the FDA related to a single patient diagnosed with hepatocellular carcinoma (HCC) in the HOPE-B pivotal trial.

https://finance.yahoo.com/news/uniqure-announces-fda-removes-clinical-110500372.html

Vertex: NaV1.8 Inhibitor Advancing to Phase 2 Clinical

  Proof-of-concept studies of VX-548 in acute pain to initiate in the second half of the year -

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that it will advance the selective NaV1.8 inhibitor, VX-548, into Phase 2 clinical development with clinical trials in patients with both visceral and non-visceral types of acute pain. The decision to move to Phase 2 was based on supportive Phase 1 data in healthy volunteers, including safety, tolerability and pharmacokinetics (PK). In these studies, the molecule exhibited a favorable profile at doses considerably lower than those required with our previous NaV1.8 inhibitors.

NaV1.8 is a voltage-gated sodium channel that plays a critical role in pain signaling in the peripheral nervous system. NaV1.8 is genetically validated as a novel target for the treatment of pain, and Vertex has previously demonstrated clinical proof-of-concept with a small molecule investigational treatment targeting NaV1.8 in multiple pain indications including acute pain, neuropathic pain and musculoskeletal pain.

https://finance.yahoo.com/news/vertex-announces-nav1-8-inhibitor-112900041.html

Samsung Biologics, TG Therapeutics Expand Deal for MS Med Manufacture

 Samsung Biologics (KRX: 207940.KS), the world’s leading contract development and manufacturing organization and TG Therapeutics (NASDAQ: TGTX), today announced an expansion of a large-scale contract manufacturing deal for the supply of TG Therapeutics’ ublituximab, an investigational anti-CD20 monoclonal antibody. TG Therapeutics has completed a rolling submission of a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) requesting approval of ublituximab, in combination with UKONIQ™ (umbralisib), TG Therapeutics’ oral once-daily inhibitor of PI3K-delta and CK1-epsilon, as a treatment for patients with CLL, based primarily on the positive results from the UNITY-CLL Phase 3 trial. Ublituximab was also the subject of two successful Phase 3 trials in patients with relapsing forms of multiple sclerosis (RMS) and a BLA is currently being prepared for this indication.

https://finance.yahoo.com/news/samsung-biologics-tg-therapeutics-expand-110000287.html

Sanofi to help manufacture Moderna COVID-19 vaccine

 Sanofi has entered into an agreement with Moderna, under which Sanofi will help manufacture Moderna´s COVID-19 vaccine, supporting the COVID-19 pandemic and vaccine supply needs.

Sanofi will leverage its established infrastructure and manufacturing expertise at its site in Ridgefield, NJ, to perform fill and finish of up to 200 million doses of Moderna's COVID-19 vaccine, starting in September 2021.

"Since the beginning of the pandemic, we have been mobilizing on multiple fronts and we showed solidarity across the industry", said Paul Hudson, Chief Executive Officer, Sanofi. "We are one of the few pharmaceutical companies to leverage many industrial partnerships to improve global supply and access to COVID-19 vaccines, while in parallel, also continuing to develop our two COVID-19 vaccine programs. I would also like to acknowledge the contributions of the Department of Health and Human Services, and the Biden administration, to facilitate this new partnership."

This marks Sanofi's third commitment to provide manufacturing support. Earlier this year, Sanofi announced the company will provide support to BioNTech for 125 million doses for the European Union. In February, Sanofi announced one of its manufacturing site in France would support Johnson & Johnson for the production of its COVID-19 vaccine at a rate of approximately 12 million doses per month. Sanofi is the only large manufacturer to support all three vaccine efforts.

https://www.prnewswire.com/news-releases/sanofi-to-help-manufacture-moderna-covid-19-vaccine-supporting-global-supply-demands-301276590.html

Inspire Medical Agreement to Commercialize Therapy in Japan

 Inspire Medical Systems, Inc. (NYSE: INSP) ("Inspire"), a medical technology company focused on the development and commercialization of innovative and minimally invasive solutions for patients with obstructive sleep apnea (“OSA”), today announced an exclusive distribution agreement with Japan Lifeline Co., Ltd. (“JLL”), an independent full-service distributor of medical devices, to commercialize Inspire therapy in Japan. The parties entered into this agreement following successful completion of reimbursement review of Inspire therapy by the Minister of Health Labour and Welfare (MLHW). Formal listing of Inspire therapy in the Japan National Health Insurance Payment Listing is expected to occur in June 2021.

Inspire therapy received regulatory approval in Japan in 2018. Inspire and JLL worked closely with the MHLW in order to advance the reimbursement application for Inspire therapy, which was approved by an expert panel this month. The formal listing of Inspire therapy is the final step prior to the training of centers and physicians in Japan.

https://finance.yahoo.com/news/inspire-medical-systems-inc-announces-060500835.html

AstraZeneca, Sanofi: AZN : Phase III Trial On Nirsevimab Meets Primary Endpoint

 AstraZeneca Plc. (AZN.L, AZN) said Monday that the MELODY Phase III trial for nirsevimab met its primary endpoint of a statistically significant reduction in the incidence of medically attended lower respiratory tract infections or LRTI caused by respiratory syncytial virus or RSV compared to placebo in healthy late preterm and term infants (35 weeks or more) during their first RSV season.

Nirsevimab is a long-acting antibody, using AstraZeneca's proprietary YTE technology, and being developed by AstraZeneca and Sanofi, with the potential to provide immunity directly to infants and offer immediate protection against RSV.

Preliminary analysis of the safety profile for nirsevimab was consistent with previous trial data. No clinically meaningful differences in safety results between the nirsevimab and placebo groups have been seen.

Respiratory syncytial virus is a very common, contagious pathogen that causes seasonal epidemics of LRTI, including bronchiolitis and pneumonia.It is the leading cause of hospitalisations in infants worldwide.

https://www.nasdaq.com/articles/azn-%3A-phase-iii-trial-on-nirsevimab-meets-primary-endpoint-in-lower-respiratory-tract

Axsome application gets priority review for depression treatment

 Axsome Therapeutics, Inc. (NASDAQ: AXSM), a biopharmaceutical company developing novel therapies for the management of central nervous system (CNS) disorders, today announced that the U.S. Food and Drug Administration (FDA) has accepted for filing the Company’s New Drug Application (NDA) for AXS-05 for the treatment of major depressive disorder (MDD), and has granted the application Priority Review. AXS-05 (dextromethorphan-bupropion) is a novel, oral, investigational NMDA receptor antagonist with multimodal activity.

Priority Review is granted by the FDA to applications for medicines that, if approved, would provide significant improvements in the effectiveness or safety of the treatment, diagnosis, or prevention of serious conditions when compared to standard applications. In general, the FDA’s Priority Review designation accelerates the review time from 10 months to a goal of six months from the date of acceptance of the filing. The FDA has set a Prescription Drug User Fee Act (PDUFA) target action date for the AXS-05 NDA of August 22, 2021.

The FDA previously granted Breakthrough Therapy Designation for AXS-05 for the treatment of MDD in March 2019. The FDA also granted Breakthrough Therapy Designation for AXS-05, for a second indication, Alzheimer’s disease agitation in June 2020.

https://finance.yahoo.com/news/axsome-therapeutics-announces-fda-acceptance-100000880.html