Zo­genix shares are claw­ing back some of the val­ue they lost 2 months ago af­ter the FDA hit the biotech with a refuse-to-file no­tice on their ex­per­i­men­tal ther­a­py for Dravet syn­drome. 

Com­pa­ny ex­ecs said this morn­ing that they worked out reg­u­la­tors’  is­sues with the ap­pli­ca­tion for Fin­tepla, which cen­tered on a pair of big prob­lems: the ab­sence of non-clin­i­cal stud­ies need­ed to al­low as­sess­ment of the chron­ic ad­min­is­tra­tion of fen­flu­ramine and the in­clu­sion of an in­cor­rect ver­sion of a clin­i­cal dataset. Now they plan to re­sub­mit in Q3 af­ter get­ting off the hook on both scores — which trig­gered a sigh of re­lief among in­vestors.

Zo­genix stock climbed 27% be­fore the open­ing bell Thurs­day on hopes of a near-term sec­ond turn at bat. The biotech not­ed:

Based on the fi­nal meet­ing min­utes re­ceived, the FDA has agreed to Zo­genix’s plan to re­sub­mit the NDA for Fin­tepla with­out the in­clu­sion of the new chron­ic tox­i­c­i­ty stud­ies re­quest­ed in the RTF let­ter. With re­gards to the sec­ond is­sue, the com­pa­ny con­duct­ed and dis­cussed with the FDA a root cause analy­sis to ex­plain the in­cor­rect clin­i­cal dataset sub­mit­ted in the orig­i­nal NDA, and the FDA is in agree­ment with the com­pa­ny’s plan for re­sub­mis­sion of the datasets in the NDA.

This time around, though, Zo­genix’s team will not have the ben­e­fit of a break­through ther­a­py des­ig­na­tion to boast about. With 2 drugs on the mar­ket, the FDA no longer felt that their drug qual­i­fied. But the com­pa­ny is still hold­ing out hopes for a pri­or­i­ty re­view.

So­cial im­age: Shut­ter­stock

Biotech is one of the smartest, best educated industries on the planet. PhDs abound. We’ve had a long enough track record to see a new generation of savvy, experienced execs coming together to run startups.

And in these times, they are being tested as never before.

Biotech is going through quite a rough patch right now. For 2 years, practically anyone with a decent resume and some half-baked ideas on biotech could start a company and get it funded. The pandemic made it easy in many ways to pull off an IPO, with traditional road shows shut down in exchange for a series of quick Zoom meetings. Generalist investors flocked as the numbers raised soared into the stratosphere.

While calls to diversify clinical trials have grown louder in recent years — gaining support from federal agencies such as the FDA and NIH — progress has largely stalled, according to a new report from the National Academies of Sciences, Engineering and Medicine.

Swaths of patients in racial and ethnic minority groups, as well as LGBTQIA+, pregnant and older adult populations continue to be left out of clinical trials. While some advances have been made in the last 30 years — women now account for roughly half of clinical trial participants — growth in other areas remains stagnant, according to the report, which was mandated by Congress and sponsored by the NIH.

It seems that smallpox vaccination production is weighing on the mind of the US government. And manufacturer Bavarian Nordic is the latest company to benefit.

Just a few days after Emergent, a company that has made government contracts its lifeblood, acquired the exclusive rights to Tembexa from Chimerix, with a $225 million cash payment and an expected BARDA contract, the agency has offered a contract for smallpox vaccine production.

The House Energy and Commerce Committee on Wednesday offered a rare show of bipartisan support for a bill that would provide the FDA with user fees for the next five years.

The committee voted 55-0 to advance the quinquennial user fee bill to the full House floor, which if approved, will allow the FDA to use biopharma funds to hire new reviewers, and hit new marks as outlined in the user fee deals that the FDA and biopharma companies forged over the past several years.