Valneva Advances Booster Phase of Cov-Compare Trial of Its Inactivated COVID-19 Vax

  Valneva SE, a specialty vaccine company, today announced the start of booster vaccinations in adult participants from its Phase 3 pivotal trial, Cov-Compare. This booster extension is intended to provide both homologous and first heterologous booster data to complement previous positive Phase 1/2 booster results. The data are not intended for the initial regulatory approval process which the Company expects to finalize in the coming weeks.

The trial extension will evaluate a booster dose of VLA2001 in adults, aged 18 and above, who received primary vaccination with two doses of VLA2001, as well as participants, aged 30 and above, who received two doses of AstraZeneca’s AZD1222. The VLA2001 booster vaccination will be given at least seven months after completion of the primary vaccination series. The trial is currently ongoing in the UK and is supported by the National Institute for Health Research (NIHR). It is expected to provide topline data during the second quarter of 2022.

https://finance.yahoo.com/news/valneva-advances-booster-phase-cov-171500188.html

Merck's chronic cough med gets a nay from FDA, potentially clearing first-to-market path for Bellus

 The FDA rejected Merck's chronic cough treatment, potentially setting up more breathing room for competitor Bellus Health in a first-to-market battle. 

While the FDA rejected Merck's drug, Japan's health regulators accepted the oral treatment last week for people with refractory or unexplained chronic cough, in which the hacking lasts eight weeks or more. The drug will be marketed as Lyfnua in Japan. 

The FDA said no to gefapixant and requested more information related to the med's efficacy. Safety was not a cause of the regulator's hesitation, the New Jersey pharma said Monday. The drug caused tolerability concerns in two phase 3 trials, in which 15% to 20% of patients on the high dose dropped out due to adverse events. The lowest dose, 15 mg, failed in both trials. At the highest dose of 45 mg, patients experienced taste-related issues.

Merck had been positioned to win the race in bringing a P2X3 receptor antagonist for cough to market after the FDA accepted its application last March. The decision was originally expected by Dec. 21, 2021, but the FDA delayed the date to March 21, 2022, Merck revealed last July. 

The pharma was ahead of Bayer, Bellus Health and Shionogi in the race to the U.S. market, but the FDA's vote of no confidence has helped close the gap for Bellus' BLU-5937, RBC Capital Markets analysts wrote in a note. 

At least one of three doses of BLU-5937 met the threshold for clinical efficacy in a phase 2b trial, the Canadian biotech said in September. Now, RBC analysts place the investigational treatment in a "potentially improved position." 

However, the FDA's complete response letter to Merck could have ripple effects on the four companies in the P2X3 space given all of the drugs in development share endpoints and measuring devices. 

The agency's concerns could be addressed by Bellus when it meets with the FDA to form a pivotal trial design for BLU-5937 next quarter, RBC analysts wrote. Bayer's med, eliapixant, also passed muster in a phase 2b trial in September by significantly decreasing cough frequency in 24-hour cough counts versus placebo after 12 weeks. 

Given no company has received an FDA nod in refractory chronic cough, Merck will "remain committed to advancing gefapixant," said Chief Medical Officer Roy Baynes, M.D., Ph.D., also head of global clinical development, in a statement. 

https://www.fiercebiotech.com/biotech/merck-s-chronic-cough-med-rejected-by-fda-accepted-japan-potentially-paving-way-for-bellus

Aptorum Gets Orphan Drug Tag For Repurposed Compound For Pediatric Cancer

 The FDA has granted Orphan Drug Designation to Aptorum Group Limited (NASDAQ: APM) SACT-1, a repurposed small molecule compound for Neuroblastoma.

• Aptorum Group plans to file an Investigational New Drug Application (IND) to commence a Phase 1b/2a trial for SACT-1 in 2022.

• Neuroblastoma is one of the most prevailing solid tumor cancers in children, representing 8% - 10% of all childhood tumors, accounting for around 15% of all cancer-related deaths in the pediatric population.

• Aptorum received its first US patent regarding SACT-1 repurposed drug for various cancers earlier this week.

• The SACT-1 invention provides a composition and method for treating or preventing the growth of cancerous tumors and/or delaying the onset of cancer from tumor-initiating cells.

https://finance.yahoo.com/news/aptorum-shares-rise-fda-orphan-134714476.html

Allscripts upped to Outperform by Leerink

 From Market Perform

https://finviz.com/quote.ashx?t=MDRX

Sierra Oncology's Bet On Failed Gilead Drug Paid Off Big

 Sierra Oncology (SRRA) reported a late-stage win Tuesday for a bone cancer drug it acquired from an industry giant at a discount three years ago — and the biotech stock catapulted to a month-high.

In the final-phase study, one-quarter of patients who received Sierra Oncology's bone marrow cancer treatment showed an improvement in symptoms. That compares with just 9% of patients given a standard drug. Sierra now plans to ask the Food and Drug Administration for approval in the second quarter.

"These data are extremely exciting and everything we had hoped to see from the (study)," Sierra Chief Executive Stephen Dilly said in a written statement.

In morning action on the stock market today, the biotech stock rocketed 55.1% near 24.10.

Biotech Stock: Succeeding Where Gilead Didn't

Sierra bought the drug, called momelotinib, for just $3 million up front from Gilead Sciences (GILD). Gilead previously spent $510 million to buy YM Biosciences in 2012 for access to momelotinib. But the drug put up several testing failures, leading Gilead to sell it at a discounted price.

Sierra tested momelotinib in patients with myelofibrosis, a type of bone marrow cancer, who were also anemic and had already received another drug, called a JAK inhibitor. Half of all myelofibrosis patients are anemic at the time of diagnosis and "virtually all become anemic over time," said Dr. Ruben Mesa, co-principal investigator of the study.

In addition to a reduction in symptoms, 31% of patients who received momelotinib no longer required blood transfusions, Sierra said in a news release. In comparison, just 20% of patients given the standard drug met the same mark. The clean sweep in test results helped the biotech stock fly.

More patients who received the standard drug reported side effects than those given momelotinib — another boon for the biotech stock.

https://www.investors.com/news/technology/biotech-stock-sierra-big-bet-on-a-gilead-failure-just-paid-off/

J&J expects up to $3.5 bln in COVID vaccine sales this year

 Johnson & Johnson on Tuesday forecast $3 billion-$3.5 billion in revenue for its COVID-19 vaccine this year, compared to $2.39 billion it generated in 2021, even as the drugmaker faces manufacturing issues and uneven demand for the shot.

The single-dose J&J shot, once touted as an important tool for vaccinating people living in hard-to-reach areas, has been behind its schedule for deliveries in the United States and Europe.

The drugmaker last year faced quality problems at a Baltimore manufacturing facility, resulting in wastage of millions of doses.

The forecast comes at a time when the company looks to separate its consumer health unit and focus on its medical devices and pharmaceuticals businesses.

https://www.marketscreener.com/quote/stock/MODERNA-INC-47437573/news/J-J-expects-up-to-3-5-bln-in-COVID-vaccine-sales-this-year-37633079/

Mustang has hold on Phase 2 trial

 Mustang Bio, Inc. (“Mustang”) (NASDAQ: MBIO), a clinical-stage biopharmaceutical company focused on translating today’s medical breakthroughs in cell and gene therapies into potential cures for hematologic cancers, solid tumors and rare genetic diseases, today announced that the U.S. Food and Drug Administration (“FDA”) has issued a hold, pending Chemistry, Manufacturing and Controls (“CMC”) clearance, on the Company’s Investigational New Drug (“IND”) application. Submitted in December 2021, the IND is to initiate a pivotal Phase 2 multicenter study to assess the safety, tolerability and efficacy of MB-207, Mustang’s lentiviral gene therapy for the treatment of patients with X-linked severe combined immunodeficiency (“XSCID”), also known as bubble boy disease, who have been previously treated with a hematopoietic stem cell transplantation (“HSCT”) and for whom re-treatment is indicated. The FDA has previously granted MB-207 Orphan Drug and Rare Pediatric Disease Designations. As such, if an MB-207 Biologics License Application (“BLA”) is approved by the FDA, then MB-207 would be eligible for a rare pediatric disease voucher.

An additional Phase 1/2 clinical trial for XSCID in newly diagnosed infants under the age of two is ongoing at St. Jude, UCSF Benioff Children’s Hospital in San Francisco and Seattle Children’s Hospital. The product candidate in this trial is designated as MB-107. Mustang expects to initiate a multi-center pivotal Phase 2 clinical trial of MB-107 under a Mustang-sponsored IND in newly diagnosed infants with XSCID who are between two months to two years of age in the third quarter of 2022. The trial is expected to enroll 10 patients who, together with 15 patients enrolled in the current multi-center trial led by St. Jude, will be compared with 25 matched historical control patients who have undergone HSCT. The primary efficacy endpoint will be event-free survival. Similar to MB-207, if an MB-107 BLA is approved by the FDA, then MB-107 would be eligible for a rare pediatric disease voucher.

https://finance.yahoo.com/news/mustang-bio-provides-ind-application-220000491.html