FDA gives thumbs-down on hear­ing loss drug, cit­ing man­u­fac­tur­ing is­sues, and sends Fen­nec stock reel­ing

A small, North Car­oli­na-based biotech has run in­to some head­winds in its ef­forts to de­vel­op a com­pound that pre­vents hear­ing loss as­so­ci­at­ed with cis­platin chemother­a­py treat­ments.

The FDA hand­ed down a com­plete re­sponse let­ter to Fen­nec Phar­ma­ceu­ti­cals $FENC for its Ped­mark drug late Mon­day night, cit­ing man­u­fac­tur­ing is­sues with the com­pa­ny’s sup­pli­er. Fen­nec hopes to meet with reg­u­la­tors some­time with­in the next month or two, CEO Ros­ty Raykov said in a call with in­vestors Tues­day morn­ing, in or­der to re­solve the is­sue.

Raykov fur­ther em­pha­sized dur­ing the call that there were no clin­i­cal safe­ty or ef­fi­ca­cy prob­lems and an ad­di­tion­al tri­al was not re­quired.

Nonethe­less, the news rat­tled Wall Street, as the biotech’s stock was down more than 35% in morn­ing trad­ing.

Per Raykov, Fen­nec re­ceived a Form 483 from the agency, which list­ed con­di­tions or prac­tices that are re­quired to be re­solved pri­or to Ped­mark’s ap­proval. When asked by in­vestors what specif­i­cal­ly need­ed to be fixed, Raykov de­murred and re­it­er­at­ed that there were no safe­ty is­sues.

Though the com­pa­ny’s goal is to meet with the FDA some­time soon, Raykov couldn’t pro­vide an ex­act time­line due to the CRL be­ing is­sued late last night, he said, and Fen­nec is still hash­ing out a plan.

De­spite the is­sues cit­ed Mon­day night, Fen­nec is not plan­ning on switch­ing man­u­fac­tur­ers, Raykov said. Giv­en that the same fa­cil­i­ty pro­duces planned Ped­mark batch­es for both the Amer­i­can and Eu­ro­pean mar­kets, Fen­nec aims to iron out the headaches as quick­ly as pos­si­ble. Fen­nec’s Eu­ro­pean ap­pli­ca­tion for Ped­mark is still un­der EMA re­view.

There are cur­rent­ly no ap­proved drugs that pre­vent oto­tox­i­c­i­ty, or hear­ing loss, in plat­inum-based chemother­a­pies. Ped­mark, a for­mu­la­tion of sodi­um thio­sul­fate, was aim­ing for a la­bel in pe­di­atric pa­tients old­er than one month to 18 years old as the hear­ing loss stem­ming from such chemother­a­pies is of­ten long-last­ing and per­ma­nent. Typ­i­cal­ly, pa­tients who end up los­ing their hear­ing must opt for cochlear im­plants.

Ped­mark re­ceived fast track des­ig­na­tion in March 2018 fol­low­ing two Phase III stud­ies in the sur­vival and re­duc­tion of oto­tox­i­c­i­ty in five pe­di­atric can­cers that typ­i­cal­ly re­quire in­ten­sive cis­platin treat­ments.

Raykov reaf­firmed Tues­day that Ped­mark’s main func­tion is to “pre­serve hear­ing with­out re­duc­ing the ef­fec­tive­ness of cis­platin treat­ment,” and he is con­fi­dent the com­pa­ny can ap­pease the FDA in short or­der.

Health­care Dis­par­i­ties and Sick­le Cell Dis­ease

In the complicated U.S. healthcare system, navigating a serious illness such as cancer or heart disease can be remarkably challenging for patients and caregivers. When that illness is classified as a rare disease, those challenges can become even more acute. And when that rare disease occurs in a population that experiences health disparities, such as people with sickle cell disease (SCD) who are primarily Black and Latino, challenges can become almost insurmountable.

Jacob Van Naarden (Eli Lilly)

Ex­clu­sives: Eli Lil­ly out to crash the megablock­buster PD-(L)1 par­ty with 'dis­rup­tive' pric­ing; re­veals can­cer biotech buy­out

It’s taken 7 years, but Eli Lilly is promising to finally start hammering the small and affluent PD-(L)1 club with a “disruptive” pricing strategy for their checkpoint therapy allied with China’s Innovent.

Lilly in-licensed global rights to sintilimab a year ago, building on the China alliance they have with Innovent. That cost the pharma giant $200 million in cash upfront, which they plan to capitalize on now with a long-awaited plan to bust up the high-price market in lung cancer and other cancers that have created a market worth tens of billions of dollars.

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So what hap­pened with No­var­tis' gene ther­a­py group? Here's your an­swer

Over the last couple of days it’s become clear that the gene therapy division at Novartis has quietly undergone a major reorganization. We learned on Monday that Dave Lennon, who had pursued a high-profile role as president of the unit with 1,500 people, had left the pharma giant to take over as CEO of a startup.

Like a lot of the majors, Novartis is an open highway for head hunters, or anyone looking to staff a startup. So that was news but not completely unexpected.

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Who are the women su­per­charg­ing bio­phar­ma R&D? Nom­i­nate them for this year's spe­cial re­port

The biotech industry has faced repeated calls to diversify its workforce — and in the last year, those calls got a lot louder. Though women account for just under half of all biotech employees around the world, they occupy very few places in C-suites, and even fewer make it to the helm.

Some companies are listening, according to a recent BIO survey which showed that this year’s companies were 2.5 times more likely to have a diversity and inclusion program compared to last year’s sample. But we still have a long way to go. Women represent just 31% of biotech executives, BIO reported. And those numbers are even more stark for women of color.

David Meek, new Mirati CEO (Marlene Awaad/Bloomberg via Getty Images)

Fresh off Fer­Gene's melt­down, David Meek takes over at Mi­rati with lead KRAS drug rac­ing to an ap­proval

In the insular world of biotech, a spectacular failure can sometimes stay on any executive’s record for a long time. But for David Meek, the man at the helm of FerGene’s recent implosion, two questionable exits made way for what could be an excellent rebound.

Meek, most recently FerGene’s CEO and a past head at Ipsen, has become CEO at Mirati Therapeutics, taking the reins from founding CEO Charles Baum, who will step over into the role of president and head of R&D, according to a release.

When ef­fi­ca­cy is bor­der­line: FDA needs to get more con­sis­tent on close-call drug ap­provals, agency-fund­ed re­search finds

In the exceedingly rare instances in which clinical efficacy is the only barrier to a new drug’s approval, new FDA-funded research from FDA and Stanford found that the agency does not have a consistent standard for defining “substantial evidence” when flexible criteria are used for an approval.

The research comes as the FDA is at a crossroads with its expedited-review pathways. The accelerated approval pathway is under fire as the agency recently signed off on a controversial new Alzheimer’s drug, with little precedent to explain its decision. Meanwhile, top officials like Rick Pazdur have called for a major push to simplify and clarify all of the various expedited pathways, which have grown to be must-haves for sponsors of nearly every newly approved drug.

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FDA hands ac­cel­er­at­ed nod to Seagen, Gen­mab's so­lo ADC in cer­vi­cal can­cer, but com­bo stud­ies look even more promis­ing

Biopharma’s resident antibody-drug conjugate expert Seagen has scored a clutch of oncology approvals in recent years, finding gold in what are known as “third-gen” ADCs. Now, another of their partnered conjugates is ready for prime time.

The FDA on Monday handed an accelerated approval to Seagen and Genmab’s Tivdak (tisotumab vedotin-tftv, or “TV”) in second-line patients with recurrent or metastatic cervical cancer who previously progressed after chemotherapy rather than PD-(L)1 systemic therapy, the companies said in a release.

The biggest ques­tions fac­ing gene ther­a­py, the XLMTM com­mu­ni­ty, and Astel­las af­ter fourth pa­tient death

After three patients died last year in an Astellas gene therapy trial, the company halted the study and began figuring out how to safely get the program back on track. They would, executives eventually explained, cut the dose by more than half and institute a battery of other measures to try to prevent the same thing from happening again.

Then tragically, Astellas announced this week that the first patient to receive the new regimen had died, just weeks after administration.

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Lat­est news: It’s a no on uni­ver­sal boost­ers; Pa­tient death stuns gene ther­a­py field; In­side Tril­li­um’s $2.3B turn­around; and more

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