Epizyme Receives Orphan Drug Designation for Leukemia Med

 Epizyme, Inc., (NASDAQ: EPZM) a clinical stage biopharmaceutical company creating innovative personalized therapeutics for patients with genetically defined cancers, announced today that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation for EPZ-5676. Criteria for orphan drug designation requires that the product be intended for treatment of a rare disease or condition, classified as affecting fewer than 200,000 people in the United States.

https://epizyme.com/pdf/Orphan%20Drug%20Designation%20Release%20-%20DOT1L.pdf

FDA to Review Regeneron Libtayo with Chemo for Lung Cancer

 Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) today announced that the U.S. Food and Drug Administration (FDA) has accepted for review the supplemental Biologics License Application (sBLA) for PD-1 inhibitor Libtayo® (cemiplimab-rwlc) in combination with chemotherapy as first-line treatment in advanced non-small cell lung cancer (NSCLC). The target action date for the FDA decision is September 19, 2022.

The sBLA is supported by results from a randomized, multicenter Phase 3 trial that investigated Libtayo in combination with a physician's choice of platinum-doublet chemotherapy (Libtayo combination), compared to platinum-doublet chemotherapy alone. Enrolled patients (n=466) had locally advanced or metastatic NSCLC, irrespective of PD-L1 expression level or tumor histology, and with no ALK, EGFR or ROS1 aberrations. A regulatory filing has also been recently submitted to the European Medicines Agency.

The Phase 3 trial supporting the sBLA was stopped early after the Libtayo combination demonstrated a significant overall survival improvement compared to chemotherapy alone. Results were presented at the European Society for Medical Oncology Virtual Congress in 2021. Among patients in the Libtayo combination (n=312) and chemotherapy alone (n=154) groups, treatment discontinuations due to adverse events (AEs) occurred in 5% and 3% of patients, respectively. Immune-mediated AEs occurred in 19% of patients in the Libtayo combination group.

Notably, the Phase 3 trial was designed to include baseline characteristics seen in everyday clinical practice. Among those enrolled, 43% had tumors with squamous histology, 67% had tumors with <50% PD-L1 expression, 15% had inoperable locally advanced disease not eligible for definitive chemoradiation, and 7% had pretreated and clinically stable brain metastases. In addition, 84% of patients had an ECOG 1 performance status, which indicates the presence of cancer-related symptoms. ECOG performance status assesses patient ability to conduct daily living activities and prognosis on a scale of increasing severity ranging from 0 (no symptoms) to 5 (death).

https://finance.yahoo.com/news/fda-accepts-review-libtayo-cemiplimab-120000030.html

Tandem Diabetes upped to Overweight from Equal Weight by Wells Fargo

 Target to $160 from $137

https://finviz.com/quote.ashx?t=TNDM&ty=c&ta=1&p=d

White House to make 400 million N95 masks available for free

 The White House announced Wednesday that it is making 400 million N95 masks available for free, part of a string of actions aimed at fighting the surging omicron variant.  

The masks will be available for pickup at “tens of thousands of local pharmacies” as well as thousands of community health centers, the White House said. The masks will begin shipping later this week and will start to become available late next week, before being “fully up and running” in early February.  

Making high-quality masks more available has been one of the areas where President Biden has been under pressure to step up the response to the virus. 

Sen. Bernie Sanders (I-Vt.) and more than 50 Democratic lawmakers introduced a bill last week, for example, to send three N95 masks to every person in the U.S.  

Similar to action on testing, experts say the Biden administration should have acted months ago to make high quality masks more available, but the new steps are still progress.  

Biden first announced last week that an announcement on free high quality masks would be coming this week.  

The masks are being deployed from the Strategic National Stockpile, which currently has more than 750 million N95 masks, triple the number in January 2021, the White House said.  

Experts have been stepping up calls for people to use higher quality masks like N95s, which offer much better protection than simple cloth masks, especially in the face of the highly transmissible omicron variant.  

The Centers for Disease Control and Prevention said last week that N95 masks offer the highest level of protection. Experts have also recommended KN95 masks, another type of high quality mask, though consumers are warned to make sure the masks they are buying are not counterfeit.  

In addition to the mask announcement, a new website to order four rapid tests per residence went live on Tuesday, at covidtests.gov.  

https://thehill.com/policy/healthcare/590292-white-house-to-make-400-million-n95-masks-available-for-free

Qiagen upped to Buy by DZ Bank

 From Hold

https://finviz.com/quote.ashx?t=QGEN&ty=c&ta=1&p=d

Glaxo top scientist to quit, become CEO of anti-ageing start-up

 GlaxoSmithKline’s (GSK) chief scientific officer is set to quit his role and become CEO of a Silicon Valley anti-ageing start-up.

After just four years as GSK’s R&D chief, Hal Barron is leaving the pharma giant to head up Altos Labs, a biotech that aims to reverse disease through cellular rejuvenation programming.

GSK announced that Barron is set to exit the company in August, and will be replaced by scientist Tony Wood, who joined GSK from Pfizer in 2017.

The move is not Barron’s first venture into the anti-ageing space; before joining GSK, he was president of R&D at Calico, a Google-backed, anti-ageing biotech that hopes to extend the human lifespan by treating age-related diseases.

Altos Labs first attracted media attention in September last year after it was reported that Amazon founder and multi-billionaire Jeff Bezos had invested in the secretive new company, which at the time had yet to make a public announcement. Billionaire entrepreneur and tech investor Yuri Milner has also contributed funding to the biotech’s research efforts.

The start-up launched on Tuesday (January 19) with $3bn funding, announcing in a statement its mission to “restore cell health and resilience to reverse disease, injury, and the disabilities that can occur throughout life”.

https://www.pharmaceutical-technology.com/news/gsk-top-scientist-ceo-anti-ageing-altos-labs/

Gamida Cell: Update on Blood Cancer FDA Submission

 

• Planning to initiate rolling BLA submission for omidubicel following positive Type B meeting with FDA
  

• Full BLA submission on track for first half of 2022

Gamida Cell Ltd. (Nasdaq: GMDA), an advanced cell therapy company committed to cures for cancer and other serious diseases, announced that following receipt of positive Type B meeting correspondence from the U.S. Food and Drug Administration (FDA) yesterday, the company plans to initiate a rolling Biologics License Application (BLA) submission for omidubicel, a potentially life-saving treatment for patients with blood cancers in need of stem cell transplant. As previously disclosed, in late 2021 the FDA requested a revised analysis of the manufacturing data generated at Gamida Cell’s wholly owned commercial manufacturing facility to demonstrate the analytical comparability to the Lonza clinical manufacturing site that produced omidubicel for the Phase 3 study. Gamida Cell and the FDA have now reached alignment that analytical comparability has been established between the commercial manufacturing facility and the product that was manufactured for the Phase 3 study. Based on this demonstration of comparability, along with the positive clinical results of the Phase 3 study, the FDA has agreed that the initiation of a rolling BLA submission is appropriate. Additional clinical data will not be required to initiate the BLA submission.

"We are very pleased that our productive interactions with the FDA have resulted in alignment on the omidubicel manufacturing comparability analysis and agreement to initiate a rolling submission of our BLA application," said Julian Adams, Ph.D., Chief Executive Officer of Gamida Cell. "Omidubicel is the first bone marrow transplant product to receive Breakthrough Therapy Designation from the FDA and has the potential to be the first FDA-approved advanced cell therapy for allogeneic bone marrow transplant. Initiating the BLA submission will move us one step closer toward bringing potentially curative therapies to patients. We plan to complete the full BLA submission in the first half of this year, which will be an important achievement for Gamida Cell and the bone marrow transplant community."

https://finance.yahoo.com/news/gamida-cell-provides-omidubicel-bla-120000727.html