Acor­da’s aw­ful, no good, just plain bad week gets worse as FDA hands ex­ecs an­oth­er de­lay on In­bri­ja — bat­tered shares tum­ble again

Acor­da’s sna­fu-plagued at­tempt to get a fast FDA mar­ket­ing de­ci­sion on a crit­i­cal­ly im­por­tant new drug has just run in­to a fresh set­back — and it’s painful to watch.

Two days af­ter the biotech’s shares $ACOR cratered on the loss of an ap­peal aimed at pro­tect­ing its patents on Ampyra — its on­ly re­al as­set on the mar­ket — the biotech is back with news that the FDA wants more time to con­sid­er new in­fo it asked for on CMC re­lat­ed to In­bri­ja (lev­odopa in­hala­tion pow­der), adding a three-month de­lay to the process and ex­tend­ing the PDU­FA to Jan­u­ary 5 of next year.

Ron Co­hen

Usu­al­ly a de­lay of this na­ture would be seen as a speed bump to a bio­phar­ma com­pa­ny. But Acor­da’s first at­tempt to file their ap­pli­ca­tion was met with a rare refuse-to-file no­tice, say­ing the pack­age was in­com­plete. That forced the com­pa­ny to re­set the clock on In­bri­ja, a Parkin­son’s drug which it needs to start mar­ket­ing last year, as they look to re­place Ampyra with the new drug.

Ev­i­dent­ly, though, the re-filed ap­pli­ca­tion was still light on re­quired in­for­ma­tion. And at this stage that qual­i­fies as an­oth­er self-in­flict­ed wound Acor­da can’t af­ford. Now’s the time to ex­e­cute, not ex­plain.

Paul Mat­teis at Stifel was quick to weigh in Thurs­day morn­ing.

We ex­pect the stock to be un­der pres­sure on the news; one of the over­ar­ch­ing fears (or bear cas­es) has been that as a drug-de­vice com­bo, In­bri­ja, de­spite hav­ing rel­a­tive­ly clean clin­i­cal da­ta, is at greater than av­er­age risk of re­ceiv­ing a com­plete re­sponse let­ter. In turn, the ex­ten­sion, ac­cord­ing to Acor­da, is re­lat­ed to re­cent sub­mis­sions made by the com­pa­ny re­gard­ing “CMC” (man­u­fac­tur­ing). FDA de­ter­mined these sub­mis­sions con­sti­tute a ma­jor amend­ment – such de­ter­mi­na­tion is a stan­dard ra­tio­nale for ex­tend­ing a PDU­FA date by 3 months (from Oc­to­ber to now Jan­u­ary). Our the­sis on the stock is that In­bri­ja is like­ly to at­tain ap­proval and even­tu­al­ly grow in­to an im­por­tant drug for Parkin­son’s OFFs, but that the ini­tial roll­out may be slow.

Ampyra, mean­while, faces im­mi­nent gener­ic com­pe­ti­tion for the fran­chise that will carve it to a frac­tion of its for­mer size as the com­pa­ny waits im­pa­tient­ly to get on the field with their new drug — pro­vid­ed they get an OK, which is not guar­an­teed.

Ear­li­er in the week the com­pa­ny’s stock dropped 25%. This morn­ing it’s down 17% in pre-mar­ket trad­ing.

“We look for­ward to con­tin­u­ing our con­struc­tive di­a­logue with FDA,” said Acor­da CEO Ron Co­hen in a state­ment. 

Inside FDA HQ (File photo)

The FDA just ap­proved the third Duchenne MD drug. And reg­u­la­tors still don’t know if any of them work

Last year Sarepta hit center stage with the FDA’s controversial reversal of its CRL for the company’s second Duchenne muscular dystrophy drug — after the biotech was ambushed by agency insiders ready to reject a second pitch based on the same disease biomarker used for the first approval for eteplirsen, without actual data on the efficacy of the drug.

On Wednesday the FDA approved the third Duchenne MD drug, based on the same biomarker. And regulators were ready to act yet again despite the lack of efficacy data.

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Cell and Gene Con­tract Man­u­fac­tur­ers Must Em­brace Dig­i­ti­za­tion

The Cell and Gene Industry is growing at a staggering 30% CAGR and is estimated to reach $14B by 20251. A number of cell, gene and stem cell therapy sponsors currently have novel drug substances and products and many rely on Contract Development Manufacturing Organizations (CDMO) to produce them with adherence to stringent regulatory cGMP conditions. Cell and gene manufacturing for both autologous (one to one) and allogenic (one to many) treatments face difficult issues such as: a complex supply chain, variability on patient and cellular level, cell expansion count and a tight scheduling of lot disposition process. This complexity affects quality, compliance and accountability in the entire vein-to-vein process for critically ill patients.

Franz-Werner Haas, CureVac CEO

UP­DAT­ED: On the heels of a snap $1B raise, Cure­Vac out­lines plans to seek emer­gency OK for their Covid-19 vac­cine in a mat­ter of months

CureVac is going from being one of the quietest players in the race to develop a new vaccine to fight the worst public health crisis in a century to a challenger for the multibillion-dollar market that awaits the first vaccines to make it over the finish line. Typically low-key at a time of brash comments and incredibly ambitious development timelines from the leaders, CureVac now is jumping straight into the spotlight.

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US gov­ern­ment re­port­ed­ly be­gins prepar­ing for Covid-19 chal­lenge tri­als. Are they eth­i­cal?

Controversial human challenge trials for potential Covid-19 vaccines reportedly have a new booster — the US government.

Scientists working for the government have begun manufacturing a strain of the novel coronavirus that could be used in such studies, Reuters reported Friday morning. The trials would enroll healthy volunteers to be vaccinated and then intentionally infected with a weakened coronavirus.

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Trevor Martin (Mammoth)

Eye­ing in-vi­vo edit­ing, Mam­moth li­cens­es Jen­nifer Doud­na’s new CRISPR en­zyme

Last month, Jennifer Doudna revealed in Science a new, “hyper-compact” CRISPR enzyme that was half the size of traditional CRISPR enzymes and could, she suspected, offer a new, more versatile tool for gene editing.

Now, the University of California-Berkeley has licensed that enzyme, known as Casφ, exclusively to a biotech startup she and two former students set up three years ago: Mammoth Biosciences. It’s the second new CRISPR protein Mammoth has licensed from Doudna’s lab, after they licensed Cas14 in 2019.

Sanofi vet Kather­ine Bowdish named CEO of PIC Ther­a­peu­tics; As the world Terns: Liv­er dis­ease biotech makes ex­ec­u­tive changes

PIC Therapeutics hasn’t raised much money, yet. But the fledgling biotech has attracted a high-profile player to the helm.

The Boston-based biotech has handed the reins to Katherine Bowdish as its president and CEO. Bowdish will also join the board of directors of PIC. Bowdish joins from Sanofi where she served as VP and head of R&D strategy, as well as helping launch and lead Sanofi Sunrise, a venture investment and partnering vehicle at Sanofi. Before that, Bowdish held several exec roles at Permeon Biologics, Anaphore, Alexion Pharmaceuticals and Prolifaron (acquired by Alexion).

Clockwise from left: Canaccord Genuity principal Michelle Gilson, Canaccord Genuity CSO Brian Mueller and BioMarin CSO Hank Fuchs (Canaccord Genuity webcast)

Bio­Marin CSO diss­es ri­vals for the he­mo­phil­ia A gene ther­a­py crown: Way be­hind, fac­ing big re­cruit­ment chal­lenges and at best a .6 on the gen-one scale

The leader in the race to a hemophilia A gene therapy does not like to be compared unfavorably to the competition. And when their top execs do the comparing, don’t look for any modesty — BioMarin, they say, owns the lead.

As Factor VIII expression wanes over time, quite a few analysts have raised questions about the kind of future BioMarin’s gene therapy — a supposed once-and-done treatment — faces if it stops working. But just 7 days away from their PDUFA date, with high odds of success, the top execs clearly feel that they are way out front, while promising their rivals will discover there’s a tough slog ahead trying to pursue trials where large numbers of patients are ineligible for new therapies.

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Cal­lid­i­tas bets up to $102M on a biotech buy­out, snag­ging a once-failed PBC drug

After spending years developing its oral formulation of the corticosteroid budesonide, Sweden’s Calliditas now has its sights set on the primary biliary cholangitis field.

The company will buy out France-based Genkyotex, and it’s willing to bet up to €87 million ($102 million) that Genkyotex’s failed Phase II drug, GKT831, will do better in late-stage trials.

Under the current agreement, Calliditas $CALT will initially pay €20.3 million in cash for 62.7% of Genkyotex (or €2.80 a piece for 7,236,515 shares) in early October, then circle back for the rest of Genkyotex’s shares under the same terms. If nothing changes, the whole buyout will cost Calliditas €32.3 million, plus up to  €55 million in contingent rights.

James Wilson, WuXi Global Forum at JPM20

FDA puts up a red light for Pas­sage Bio’s first gene ther­a­py pro­gram, de­lay­ing a pro­gram from James Wilson's group at Penn

Gene therapy pioneer James Wilson spearheaded animal studies demonstrating the potential of new treatments injected directly into the brain, looking to jumpstart a once-and-done fix for an extraordinarily rare disease called GM1 gangliosidosis in infants. His team at the University of Pennsylvania published their work on monkeys and handed it over to Passage Bio, a Wilson-inspired startup building a pipeline of gene therapies — with an IND for PBGM01 to lead the way.

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