Aque­s­tive Ther­a­peu­tics is still reel­ing from a pre­cip­i­tous drop in its stock price af­ter the FDA re­ject­ed its oral film for seizure con­trol.

Liber­vant, a buc­cal film for­mu­la­tion of the gener­ic drug di­azepam, would have been the biotech’s sec­ond com­mer­cial treat­ment for epilep­sy fol­low­ing Sym­pa­zan (clobazam), which is ap­proved specif­i­cal­ly for Lennox-Gas­taut syn­drome.

Ac­cord­ing to a re­lease out Fri­day, reg­u­la­tors are main­ly wor­ried about phar­ma­co­ki­net­ics, cit­ing in the com­plete re­sponse let­ter a study where “cer­tain weight groups showed a low­er drug ex­po­sure lev­el than de­sired.”

While Aque­s­tive said they are clear on safe­ty, clin­i­cal phar­ma­col­o­gy or man­u­fac­tur­ing, reg­u­la­tors did al­so take is­sue with a small num­ber of pro­to­col de­vi­a­tions in blood draws in one of the stud­ies. It’s un­clear how big that con­cern looms.

Kei­th Kendall

Next steps in­clude pro­vid­ing more in­for­ma­tion on PK mod­el­ing to con­vince the FDA that the is­sue can be solved by dose ad­just­ments, but no ad­di­tion­al stud­ies.

Still, it is a no­table set­back for a com­pa­ny that’s built its rep­u­ta­tion on a tech­nol­o­gy plat­form to re­design and de­liv­er med­ica­tions through film.

Of­fer­ing al­ready avail­able drugs in a dif­fer­ent form, the rea­son­ing goes, al­lows them to ad­dress dif­fer­ent pa­tient pop­u­la­tions.

“Epilep­sy pa­tients have been un­der­served for some time with lit­tle choice be­yond de­vice-based prod­ucts such as rec­tal­ly ad­min­is­tered gels and nasal sprays and Liber­vant rep­re­sents a mean­ing­ful and im­proved ther­a­py for pa­tients who can’t or won’t use the al­ter­na­tives,” CEO Kei­th Kendall said in a state­ment.

Sim­i­lar­ly with Exser­van, the film ver­sion of rilu­zole that passed FDA muster last No­vem­ber, Aque­s­tive was tar­get­ing amy­otro­phy lat­er­al scle­ro­sis pa­tients who have trou­ble swal­low­ing pills.

Al­most a year af­ter the Exser­van OK, though, Aque­s­tive has yet to launch the drug.

For Liber­vant, the biotech says it will be re­quest­ing a Type A meet­ing with the FDA in the com­ing weeks and ex­pects to re­file an NDA by the end of the year.

That didn’t stop in­vestors from send­ing the stock down 34% to $4.97 on Mon­day, which went down some more dur­ing the day.

About three years after acquiring SteadyMed and its experimental pulmonary arterial hypertension drug Trevyent, United Therapeutics has decided to kick the program to the curb.

In an 8-K form filed with the SEC on Tuesday, United revealed that they are shutting down development of Trevyent after receiving feedback from the FDA last week. The move marks the end of a long and bumpy road for the program, which had previously earned an RTF in 2017 and a CRL last April, as United moves forward with new Tyvaso formulations.

CRLs. 483s. CBER, CDER and RWE. For biopharma professionals, these acronyms command attention because of the fundamental role FDA plays in drug development. Now Endpoints is doubling down on regulatory coverage, and launching a weekly report focusing on developments out of White Oak, with analysis and insight into what it all means.

Coverage will be led by our new senior editor, Zachary Brennan. He joins Endpoints from POLITICO, where he covered pharma. Prior to that he was the managing editor for Regulatory Focus, a news publication from the Regulatory Affairs Professionals Society.

One of Europe’s most high-profile biopharma investors is getting $540 million to invest in new crossover deals for late-stage companies.

The Paris-based VC says the fresh Sofinnova Crossover Fund raise positions them as the “largest crossover investor in Europe dedicated to late-stage biopharma and medtech investments.”

They got a leg up in France after winning a special “Tibi” designation from the French government, giving them access to a pool of €6 billion that helped them gain an edge with institutional investors. Since they were founded close to 50 years ago, the venture group has backed more than 500 companies and currently has more than €2 billion under management.

Seattle-based Presage Biosciences, which approaches drug development through its microdosing platform, has some new partnerships and cash to come with them.

Presage closed a $13 million financing round Tuesday, aiming to expand its network of clinical trial sites and advance development of its microdosing injection devices. They also closed partnership deals with Merck and Maverick Therapeutics.

The financing included $7 million from new investors, including the LabCorp Venture Fund, Bristol Myers Squibb, and InHarv Partners. An additional $6 million convertible note from Takeda Ventures will convert to equity.