Long jour­ney for DBV peanut patch ends in re­jec­tion by FDA

DBV Tech­nolo­gies’ long and wind­ing saga with the FDA has end­ed — for now, at least — with a CRL for their peanut al­ler­gy skin patch.

The re­jec­tion comes 21 months af­ter the com­pa­ny first filed for ap­proval. At the time, the French biotech was in a two-way race with Cal­i­for­nia’s Aim­mune to pro­duce the first FDA-ap­proved treat­ment for peanut al­ler­gy. But since then Aim­mune cleared that hur­dle, while DBV, af­ter a with­draw­al and re­sub­mis­sion, in­di­cat­ed that com­mu­ni­ca­tion be­tween them and reg­u­la­tors had bro­ken down.  In June, the com­pa­ny be­gan cut­ting pro­grams and em­ploy­ees.

As de­tailed in DBV’s press re­lease, the con­cerns raised in the CRL may take more than a few months to rem­e­dy. On top of CMC is­sues, DBV said that the agency al­so had ques­tions about how the ad­he­sive­ness, or lack there­of, of their skin patch could af­fect ef­fi­ca­cy.  They want­ed DBV to mod­i­fy the patch and then con­duct a new hu­man fac­tor study and pro­vide new clin­i­cal da­ta.

Daniel Tassé

CEO Daniel Tassé struck a con­cil­ia­to­ry tone, adding they plan to meet with the FDA to dis­cuss re­sub­mis­sion be­fore up­dat­ing in­vestors. The com­pa­ny had €225.9 mil­lion – $266.4 mil­lion – in cash as of June 30. They did not in­di­cate how much run­way that gave them, but the re­struc­tur­ing plan ini­ti­at­ed in June was in­tend­ed to keep them sol­vent through the first quar­ter of 2021.

“We plan to ful­ly col­lab­o­rate with the FDA with re­gards to the out­stand­ing is­sues and be­lieve that the EPIT patch tech­nol­o­gy plat­form lends it­self well to po­ten­tial mod­i­fi­ca­tions to en­hance patch func­tion­al­i­ty,” he said in a state­ment, us­ing the brand­ed name for their patch tech­nol­o­gy.

The com­pa­ny’s stock {DB­VT}, hav­ing al­ready tum­bled from $19.83 to $6.70 this year, fell to $4.74 on the news.

Trou­ble for DBV be­gan in De­cem­ber of 2018, when the com­pa­ny with­drew their first BLA, say­ing reg­u­la­tors in­di­cat­ed their ini­tial ap­pli­ca­tion lacked “suf­fi­cient de­tail re­gard­ing da­ta on man­u­fac­tur­ing pro­ce­dures and qual­i­ty con­trols.” That set­back gave Aim­mune a win­dow to file for their peanut pow­der cap­sule, lat­er known as Pal­forzia, al­low­ing it to be­come the first FDA-ap­proved peanut al­ler­gy ther­a­py in Jan­u­ary.

Nei­ther ther­a­pies are cures. Rather, they try to get kids to build tol­er­ance to peanuts by re­peat­ed ex­po­sure — through es­ca­lat­ing dos­es of pow­der in Aim­mune’s case and through es­ca­lat­ing dos­es of their “Vi­askin” patch in DBV’s case. Ear­ly da­ta point­ed to Aim­mune hav­ing the more ef­fec­tive but per­haps less tol­er­a­ble ther­a­py, as DBV failed to reach sta­tis­ti­cal sig­nif­i­cance in late-stage tri­als be­fore head­ing to the FDA.

DBV re-filed last Au­gust and the FDA ac­cept­ed the sub­mis­sion in Oc­to­ber, set­ting up an ad­vi­so­ry com­mit­tee hear­ing in May and a PDU­FA date in Au­gust. In March, though, DBV dis­closed the FDA had con­cerns that kids could eas­i­ly scratch off their patch, in­ter­fer­ing with its over­all ef­fi­ca­cy, and that the agency had can­celed their ad­vi­so­ry com­mit­tee hear­ing.

The com­pa­ny sub­mit­ted new da­ta to as­suage those con­cerns. In June, though, they an­nounced that they had yet to hear back from the FDA and, fac­ing the pos­si­bil­i­ty of re­jec­tion in Au­gust, were lay­ing off staff and cut­ting pro­grams to stay above wa­ter long-term.

That an­nounce­ment di­vid­ed an­a­lysts. SVB Leerink’s Joseph Schwartz called it “a sticky sit­u­a­tion.” Baird’s Bri­an Sko­r­ney, who had been bull­ish on Aim­mune, pre­pared fu­ner­al rites. “At this point, we view DBV as ef­fec­tive­ly dead in the wa­ter,” he wrote in a note fol­low­ing the an­nounce­ment.

The re­jec­tion means Aim­mune will have a clear path to take the peanut al­ler­gy mar­ket, but the com­pa­ny is fac­ing its own is­sues. They had planned ex­ten­sive train­ing ses­sions with al­ler­gists to help launch Pal­forzia, but Covid-19 stunt­ed those ef­forts. The com­pa­ny said last week they re­port­ed ze­ro Pal­forzia sales in Q2. Their stock is down to $13.60 from a post-ap­proval high of over $36 in Jan­u­ary, al­though Sko­r­ney is still con­fi­dent they’re worth far more.

“We be­lieve in­ter­est in the prod­uct re­mains high, were en­cour­aged to hear man­age­ment’s com­ments sur­round­ing an uptick in new starts in Ju­ly,” he said in a note last week. “That be­ing said, we are re­duc­ing our price tar­get to $47 as we be­lieve near-term COVID head­winds could con­tin­ue to ham­per the launch, in­creas­ing the po­ten­tial for a di­lu­tive cap­i­tal raise.”

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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