Slammed by FDA re­jec­tion, Aque­s­tive vows to re­file by end of year

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.

UP­DAT­ED: Mer­ck pulls Keytru­da in SCLC af­ter ac­cel­er­at­ed nod. Is the FDA get­ting tough on drug­mak­ers that don't hit their marks?

In what could be an early shot in the battle against drugmakers that whiff on confirmatory studies to support accelerated approvals, the FDA ordered Bristol Myers Squibb late last year to give up Opdivo’s approval in SCLC. Now, Merck is next on the firing line — are we seeing the FDA buckling down on post-marketing offenders?

Merck has withdrawn its marketing approval for PD-(L)1 inhibitor Keytruda in metastatic small cell lung cancer as part of what it describes as an “industry-wide evaluation” by the FDA of drugs that do not meet the post-marketing checkpoints on which their accelerated nods were based, the company said Monday.

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The 2021 top 100 bio­phar­ma in­vestors: As the pan­dem­ic hit and IPOs boomed, VCs swung in­to ac­tion like nev­er be­fore

The global pandemic may have roiled economies, killed hundreds of thousands and throttled entire industries, but the only effect it had on biopharma venture investing was to help turbocharge the field to giddy new heights.

Below you’ll find the new top 100 venture investors in the industry, ranked by the number of deals they were publicly involved in, as tracked by DealForma chief Chris Dokomajilar. The numbers master then calculated the estimated amount of money they put into each deal — divvying up the cash by the number of players — to indicate how they managed their syndicates.

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Hal Barron, Endpoints UKBIO19

GSK, Vir's hopes for a Covid-19 an­ti­body fall flat in NIH 'mas­ter pro­to­col' with no ben­e­fit in hos­pi­tal­ized pa­tients

GlaxoSmithKline and Vir Biotechnology were hopeful that one of their partnered antibodies would carve out a win after getting the invite to a major NIH study in hospitalized Covid-19 patients. But just like Eli Lilly, the pair’s drug couldn’t hit the mark, and now they’ll be left to take a hard look at the game plan.

The NIH has shut down enrollment for GSK and Vir’s antibody VIR-7831 in its late-stage ACTIV-3 trial after the drug showed negligible effect in achieving sustained recovery in hospitalized Covid-19 patients, the partners said Wednesday.

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As Brain­Storm con­tin­ues to tout ‘clear sig­nal’ on ALS drug, the FDA of­fers a rare pub­lic slap­down on the da­ta

A little more than a week after BrainStorm acknowledged that regulators at the FDA had informed them that the biotech needed more data before it could expect to gain an approval for its ALS treatment NurOwn — while still touting a “clear signal” of efficacy and not ruling out an application — the agency has decided to clarify the record in a most unusual statement.

The FDA statement amounts to a straight slap own, offering a different set of efficacy numbers from the company’s public presentation last November and ruling out any chance of statistical significance.

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Martine Rothblatt, United CEO (Jack Plunkett, AP Images)

Unit­ed ax­es PAH pro­gram Trevyent, once bought for cheap in ac­qui­si­tion of a ri­val, af­ter FDA feed­back

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.

In­tro­duc­ing End­points FDA+, our new pre­mi­um week­ly reg­u­la­to­ry news re­port led by Zachary Bren­nan

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.

Antoine Papiernik, Sofinnova managing director (Business Wire)

Sofinno­va Part­ners stays fo­cused on late-stage deals with a new, $540M crossover fund

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.

Presage teams with Mer­ck on its Phase 0 test­ing; Kem­Pharm AD­HD drug wins ap­proval in chil­dren aged 6 and up

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.

Eli Lil­ly claims suc­cess in a new JAK in­di­ca­tion: hair loss

Over the last decade, drugmakers have proven JAK inhibitors can treat a smattering of immune-related diseases ranging from rheumatoid arthritis to Covid-19. Now Eli Lilly has pulled out a new one.

Lilly and its biotech partner Incyte announced Wednesday that their JAK inhibitor baricitinib effectively regrew patients’ hair in a Phase III trial for alopecia areata, an autoimmune condition that can cause sudden, severe and patchy hair loss. Lilly didn’t break down the results from the 546-patient trial, but the primary endpoint was improvement on a standard score for alopecia symptoms.

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