When Brain­Storm Cell Ther­a­peu­tics broke out the abysmal num­bers in its Phase III tri­al, the biotech in­sist­ed that the FDA was ea­ger to re­view the da­ta — giv­en the sig­nal they had iden­ti­fied in a sub­group of amy­otroph­ic lat­er­al scle­ro­sis with less ad­vanced dis­ease.

Three months lat­er, reg­u­la­tors did give a look to the high-lev­el sum­ma­ry of the da­ta. But they didn’t have too many good things to say.

“The FDA con­clud­ed from their ini­tial re­view that the cur­rent lev­el of clin­i­cal da­ta does not pro­vide the thresh­old of sub­stan­tial ev­i­dence that FDA is seek­ing to sup­port a Bi­o­log­ics Li­cense Ap­pli­ca­tion (BLA),” Brain­Storm wrote in a re­lease.

Yet — and this is giv­ing the com­pa­ny rea­son to push ahead — “the FDA ad­vised that this rec­om­men­da­tion does not pre­clude Brain­storm from pro­ceed­ing with a BLA sub­mis­sion.”

Chaim Lebovits

Chaim Lebovits, the CEO, says they will con­sult with prin­ci­pal in­ves­ti­ga­tors, ALS ex­perts, sta­tis­ti­cians, reg­u­la­to­ry ad­vi­sors and ad­vo­ca­cy groups be­fore mak­ing a fi­nal de­ci­sion on whether to file.

While fil­ing af­ter such dis­clo­sure would be risky and high­ly un­ortho­dox, Brain­Storm stress­es that ALS is a dev­as­tat­ing dis­ease with few to no treat­ment op­tions, and com­mits to ful­ly an­a­lyz­ing bio­mark­ers from the Phase III tri­al to de­ter­mine the ef­fects of its NurOwn cell ther­a­py, which con­sists of au­tol­o­gous mes­enchy­mal stem cells that are en­gi­neered to pro­duce neu­rotroph­ic fac­tors.

Be­sides, the ex­ec­u­tive team is no stranger to con­tro­ver­sy.

The New York-based crew ig­nit­ed a ring of crit­i­cism more than two years ago by in­di­cat­ing that it was prepar­ing to sell their drug to ALS pa­tients un­der the “Right to Try” law that was about to be passed. They even­tu­al­ly dropped the plan, but not be­fore Lebovits dis­closed that they were think­ing of charg­ing around $300,000 for the ex­per­i­men­tal treat­ment, which had gen­er­at­ed less than en­cour­ag­ing re­sults in Phase II.

He now notes that Brain­Storm has over $40 mil­lion in cash and plans to an­nounce its next flag­ship prod­uct soon.

It wasn’t enough to stem a plunge for the stock $BCLI, which fell 28.70% to $4.92.

DC District Court Judge Christopher Cooper today granted Vanda Pharma’s request to require the FDA to disclose more info on the complete response letter for its sleep disorder drug Hetlioz.

The melatonin receptor agonist is approved by the FDA to treat non-24-hour sleep-wake disorder, a circadian rhythm disorder. But in 2018 Vanda filed a supplemental application to market Hetlioz as a treatment for jet lag, which the FDA rejected in August 2019, with few details on what Vanda needed to correct course, according to the company.

Republican members of Congress want FDA Commissioner Robert Califf and his agency to provide more information on the research FDA has funded that manipulates certain viruses like the SARS-CoV-2 virus — after the FDA confirmed that certain studies it funded involved viral manipulation, but in standard ways that are common in molecular biology.

The three-page letter, signed Monday by House Energy and Commerce Committee Chair Cathy McMorris Rodgers (R-WA) and Subcommittee Chairs Brett Guthrie (R-KY) and Morgan Griffith (R-VA), kicked things off by saying that the committee was looking into FDA’s oversight of “FDA-funded research that may pose significant biosafety or biosecurity risks.”

The FDA has scheduled or planned for three advisory committee meetings in May: one for an over-the-counter birth control pill, one for a nasal spray version of the EpiPen, and another for  Intarcia Therapeutics, which has all but completely folded already as a company.

Perrigo will bring its daily contraceptive, Opill, to the joint Nonprescription Drugs Advisory Committee and the Obstetrics, Reproductive and Urologic Drugs Advisory Committee on May 9 and 10 for its over-the-counter birth control application.

Republican leaders of the House Committee on Energy & Commerce sent a five-page letter yesterday announcing an investigation into the ongoing drug shortages that have rankled the US during the pandemic and the FDA’s response to it.

The letter, signed by Chair Cathy McMorris Rodgers (R-WA), explains how shortages have become more common over the past decade, while pointing to a report from the National Academies of Science, Engineering and Medicine finding that drug shortages have been “on the rise” over the past several decades and are lasting longer, with new drug shortages in the US seeing a 30% increase from 2021 to 2022.