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.

Secretary of health and human services Alex Azar speaking in the Rose Garden at the White House (Photo: AFP)

Trump’s HHS claims ab­solute au­thor­i­ty over the FDA, clear­ing path to a vac­cine EUA

The top career staff at the FDA has vowed not to let politics overrule science when looking at vaccine data this fall. But Alex Azar, who happens to be their boss’s boss, apparently won’t even give them a chance to stand in the way.

In a new memorandum issued Tuesday last week, the HHS chief stripped the FDA and other health agencies under his purview of their rule making ability, asserting all such power “is reserved to the Secretary.” Sheila Kaplan of the New York Times first obtained and reported the details of the September 15 bulletin.

President Donald Trump (via AP Images)

Signs of an 'Oc­to­ber Vac­cine Sur­prise' alarm ca­reer sci­en­tists

President Donald Trump, who seems intent on announcing a Covid-19 vaccine before Election Day, could legally authorize a vaccine over the objections of experts, officials at the FDA and even vaccine manufacturers, who have pledged not to release any vaccine unless it’s proved safe and effective.

In podcasts, public forums, social media and medical journals, a growing number of prominent health leaders say they fear that Trump — who has repeatedly signaled his desire for the swift approval of a vaccine and his displeasure with perceived delays at the FDA — will take matters into his own hands, running roughshod over the usual regulatory process.

#ES­MO20: Push­ing in­to front­line, Mer­ck and Bris­tol My­ers duke it out with new slate of GI can­cer da­ta

Having worked in parallel for years to move their respective PD-1 inhibitors up to the first-line treatment of gastrointestinal cancers, Merck and Bristol Myers Squibb finally have the data at ESMO for a showdown.

Comparing KEYNOTE-590 and CheckMate-649, of course, comes with the usual caveats. But a side-by-side look at the overall survival numbers also offer some perspective on a new frontier for the reigning checkpoint rivals, both of whom are claiming to have achieved a first.

UP­DAT­ED: Two wild weeks for Grail end in $8B Il­lu­mi­na buy­out

Grail’s whirlwind two weeks have ended in the wealthy arms of its former founder and benefactors.

Illumina has shelled out $8 billion to reacquire the closely-watched liquid biopsy startup they spun out just 5 years ago and sold off much of its shares just 3 years ago. The deal comes nearly two weeks after the well-heeled startup filed for a potentially massive IPO — one that was disrupted just a week later when Bloomberg reported that Illumina was in talks to buy their former spinout for up to $8 billion.

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Roche vaults to the front of the NL­RP3 clin­i­cal race, pay­ing $448M up­front to bag In­fla­zome

Roche is going all in on NLRP3.

The pharma giant is putting down $448 million (€380 million) upfront to snatch Novartis-backed Inflazome, which makes it a clinical player in the space overnight.

Dublin and Cambridge, UK-based Inflazome is the second NLRP3-focused biotech Roche has acquired in less than two years, and although no numbers were disclosed in the Jecure buyout, this is almost certainly a much larger deal.

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Eli Lilly CSO Dan Skovronsky (file photo)

UP­DAT­ED: #ES­MO20: Eli Lil­ly shows off the da­ta for its Verzenio suc­cess. Was it worth $18 bil­lion?

The press release alone, devoid of any number except for the size of the trial, added nearly $20 billion to Eli Lilly’s market cap back in June. Now investors and oncologists will get to see if the data live up to the hype.

On Sunday at ESMO, Eli Lilly announced the full results for its Phase III MonarchE trial of Verzenio, showing that across over 5,000 women who had had HR+, HER2- breast cancer, the drug reduced the odds of recurrence by 25%. That meant 7.8% of the patients on the drug arm saw their cancers return within 2 years, compared with 11.3% on the placebo arm.

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#ES­MO20: Am­gen team nails down sol­id ear­ly ev­i­dence of AMG 510’s po­ten­tial for NSCLC, un­lock­ing the door to a wave of KRAS pro­grams

The first time I sat down with Amgen’s Greg Friberg to talk about the pharma giant’s KRAS G12C program for sotorasib (AMG 510) at ASCO a little more than a year ago, there was high excitement about the first glimpse of efficacy from their Phase I study, with 5 of 10 evaluable non-small cell lung cancer patients demonstrating a response to the drug.

After decades of failure targeting KRAS, sotorasib offered the first positive look at a new approach that promised to open a door to a whole new approach by targeting a particular mutation to a big target that had remained “undruggable” for decades.

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#ES­MO20: Out to beat Tagris­so, J&J touts 100% ORR for EGFR bis­pe­cif­ic/TKI com­bo — fu­el­ing a quick leap to PhI­II

J&J’s one-two punch on EGFR-mutant non-small cell lung cancer has turned up some promising — although decidedly early — results, fueling the idea that there’s yet room to one up on third-generation tyrosine kinase inhibitors.

Twenty out of 20 advanced NSCLC patients had a response after taking a combination of an in-house TKI dubbed lazertinib and amivantamab, a bispecific antibody targeting both EGFR and cMET engineered on partner Genmab’s platform, J&J reported at ESMO. All were treatment-naïve, and none has seen their cancer progress at a median follow-up of seven months.

Anthony Fauci (AP Images)

A press of­fi­cer at An­tho­ny Fau­ci’s NI­AID was un­masked as a hard-right Covid troll. He just re­tired to­day

William B Crews had been a public affairs specialist at the NIH’s National Institute of Allergy and Infectious Diseases.

That ended today when he informed the agency of his decision to retire, after he was identified as the managing editor at RedState, a prominent Trump loyalist website.

Crews’ RedState duties are performed under the alias streiff. While enjoying the benefits of pseudonymity, he disparaged and worked against NIAID with an incendiary level of rhetoric in the midst of a pandemic.