FDA Ad Com thumbs down on mortality benefit claim for Glaxo’s Trelegy Ellipta

The FDA’s Pulmonary-Allergy Drugs Advisory Committee voted 14-1 that the results from the IMPACT study evaluating GlaxoSmithKline’s (GSK +0.2%) Trelegy Ellipta (fluticasone furoate, umeclidinium, and vilanterol inhalation powder oral inhalation) in chronic obstructive pulmonary disease (COPD) patients do not provide sufficient evidence to support the proposed claim of a reduction in all-cause mortality.

The FDA approved the product in September 2017 for COPD.

Meeting materials

https://seekingalpha.com/news/3610216-fda-ad-com-thumbs-down-on-mortality-benefit-claim-for-glaxos-trelegy-ellipta

FDA OKs Medtronic automated insulin system for young kids

The FDA approves Medtronic’s (NYSE:MDT) MiniMed 770G System, a hybrid closed-loop diabetes management device, for children ages 2 – 6 with type 1 diabetes, the first such system available in the U.S. for this population of children.

The system, a bluetooth-enabled version of the MiniMed 670G, measures blood sugar levels every five minutes and automatically adjusts insulin delivery.

At mealtime, users must manually request insulin doses to counter carbohydrate consumption.

https://seekingalpha.com/news/3610251-fda-oks-medtronic-automated-insulin-delivery-and-monitoring-system-for-young-kids

AstraZeneca backs safety guides in development of COVID-19 vaccine

In a statement, AstraZeneca (NYSE:AZN) reemphasizes its commitment to the highest safety standards and broad equitable global access to COVID-19 vaccine AZD1222, adding that its marketing applications will meet the stringent requirements by regulators around the world.

Its development plan aims to enroll more than 50K volunteers, including 30K in the U.S., Latin America, Asia, Europe, Russia and Africa.

AZD1221, co-invented by the University of Oxford and its spin-out company Vaccitech, is based on a weakened adenovirus vector containing genetic material of the SARS-CoV-2 virus spike protein.

The intense global race to commercialize a legitimate vaccine against the coronavirus has many observers worried that over-eager governments may sign off based on insufficient supporting data, exemplified by Russia’s approval of a vaccine, dubbed Sputnik V, based on results from less than 60 volunteers.

https://seekingalpha.com/news/3610267-astrazeneca-to-follow-science-in-development-of-covidminus-19-vaccine

Bayer to buy vitamin firm Care/Of in deal valued at $225M

August 31, 2020

Bayer (OTCPK:BAYRY) has agreed to buy 70% of online vitamin and health supplement company Care/of in a deal that values the company at $225M, with an option to buy the rest by 2022, Bloomberg reports.

Bayer believes “this model and product type has the ability to expand into traditional retail channels as we aim to reach new consumers,” the company says, adding it will “grow the Care/of business across new channels, new categories and new markets to deliver even more personalized nutrition.”

The latest deal follows Bayer’s agreement earlier this month to buy British women’s health biotech Kandy Therapeutics in an $875M deal.

https://seekingalpha.com/news/3610291-bayer-to-buy-vitamin-firm-care-of-in-deal-valued-225m-bloomberg

Axsome has development plans on AXS-05 for Alzheimer’s agitation

Axsome Therapeutics (AXSM -2.7%) confirms pivotal development status and plan for AXS-05 in the treatment of Alzheimer’s disease (AD) agitation following a successful Breakthrough Therapy meeting with the FDA.

AXS-05 (dextromethorphan/bupropion modulated delivery tablet) is an oral, investigational NMDA receptor antagonist and sigma-1 receptor agonist. There is currently no approved treatment for AD agitation.

Results of the meeting confirm the pivotal status of the previously completed positive ADVANCE-1 trial, and the establishment of the superiority of AXS-05 over its components.

Consequently, only one additional Phase 3 efficacy trial will be needed to support the NDA filing for approval of AXS-05 in this indication, and only a placebo control will be required.

Axsome is on track to initiate this efficacy trial in Q4. The Company also plans to launch an open-label safety extension trial of AXS-05 in AD agitation patients in Q4 to supplement the existing AXS-05 long-term safety database.

https://seekingalpha.com/news/3610117-axsome-therapeutics-reports-development-plans-of-axsminus-05-for-alzheimer-s-disease

Bluebird bio gene therapy shows sustained benefit in rare metabolic disorder

Bluebird bio (BLUE +2.0%) announces long-term results from a Phase 2/3 clinical trial, STARBEAM, evaluating gene therapy elivaldogene autotemcel (eli-cel, Lenti-D) in patients with cerebral adrenoleukodystrophy (CALD), a rare inherited metabolic disorder in which very long chain fatty acids build up in the brain damaging nerve cells.

Median follow-up was 30.0 months (range: 9.1 – 70.7 months) in 32 patients. 20 of the 32 have enrolled in a long-term follow-up study Nine others will continue to be followed in STARBEAM (not yet reached 24 months post-treatment) and two withdrew.

87% (n=20/23) of treated patients who have or would have reached month 24 of follow-up achieved the primary endpoint of being alive and free of six major functional disabilities (MFDs) at month 24. 14 subjects have at least four years’ follow-up while 10 have reached five years.

97% (n=31/32) experienced stable neurologic function score (NFS) after treatment with eli-cel, including 24 with NFS of 0 (no concerns with neurologic functions).

A 35-subject Phase 3 trial, ALD-104, is underway with an estimated completion date in February 2024.

https://seekingalpha.com/news/3610124-bluebird-bio-gene-therapy-shows-sustained-benefit-in-rare-metabolic-disorder

Provention Bio initiates Phase 2b PROACTIVE study of PRV-015

Provention Bio (PRVB +1.1%) announced the initiation of the Phase 2b PROACTIVE study of PRV-015, an anti-interleukin-15 monoclonal antibody, in adult celiac patients not responding to a gluten-free diet, a condition known as ‘Non-Responsive Celiac Disease‘.

Provention is developing PRV-015 under its 2018 collaboration with Amgen.

“PRV-015 has the potential to be the first-ever approved therapeutic for celiac disease.” stated Francisco Leon, MD, PhD, Chief Scientific Officer and Co-Founder.

https://seekingalpha.com/news/3610125-provention-bio-initiates-phase-2b-proactive-study-of-prvminus-015