PhI­II de­tails boost Acor­da as ex­ecs rush to FDA with the clock tick­ing on a time bomb

Ron Co­hen, Acor­da

Shares of Acor­da got a boost af­ter the mar­ket closed on Mon­day as in­vestors got a chance to look over the de­tailed Phase III da­ta for its in­haled lev­odopa drug CVT-301. And while we al­ready knew that the top-line da­ta on the pri­ma­ry end­point for Parkin­son’s dis­ease were pos­i­tive, it seemed to help that this late-stage ther­a­py ap­pears on track to ar­rive at the FDA lat­er this month as com­pa­ny ex­ecs look to re­place its big mon­ey­mak­er, now poised to lose patent pro­tec­tion.

Fail­ure, or even the hint of a set­back here, would be cat­a­stroph­ic. So the p=0.009 val­ue for a sta­tis­ti­cal­ly sig­nif­i­cant im­prove­ment in mo­tor func­tion among pa­tients ex­pe­ri­enc­ing OFF times was re­as­sur­ing. The switch from OFF to ON— mark­ing pe­ri­ods of the day when the drug is work­ing— and stay­ing ON at 60 min­utes, a key sec­ondary end­point, was al­so pos­i­tive. But the third step in the sec­ondary re­view ran in­to trou­ble, with no sig­nif­i­cant change to the Uni­fied Parkin­son’s Dis­ease Rat­ing Scale — III at 20 min­utes.

An­a­lysts al­so flagged some wor­ries about the lack of sig­nif­i­cant im­prove­ment in to­tal OFF time, which could crimp its roll­out, pro­vid­ed reg­u­la­tors of­fer a green light in the first place.

Acor­da’s shares jumped 10% af­ter in post-mar­ket trad­ing.

Paul Mat­teis at Leerink of­fered a quick thumbs up:

We be­lieve the full re­sult – con­veyed at the Move­ment Dis­or­ders So­ci­ety An­nu­al Meet­ing – sup­ports our fair­ly high, 90% odds of FDA ap­proval. With­in the full da­ta we are en­cour­aged with what looks to be an ac­cept­able safe­ty pro­file as well as a low, place­bo-like dropout rate. Sec­ondary end­points al­so most­ly trend or are clear­ly in fa­vor of drug – in­clud­ing the pa­tient glob­al im­pres­sion of change – though the lack of dif­fer­ence (p-val­ue of 0.98) on to­tal OFF time may emerge as a sub­ject of dis­cus­sion as in­vestors de­bate the size of the com­mer­cial op­por­tu­ni­ty. We mod­el ~$278MM in CVT-301 sales in 2022E, and we ex­pect the prod­uct to be ap­proved in the mid­dle of next year.

Acor­da shift­ed to sur­vival mode in ear­ly April af­ter a US dis­trict court tossed four key patents on its flag­ship drug Ampyra, leav­ing it with one to stand on in­to next year. The com­pa­ny chopped 20% of its staff as it scram­bles to re­struc­ture while gam­bling that it can field new drugs in short or­der.

But Acor­da CEO Ron Co­hen left his mar­ket­ing team in place, gam­bling that an on-time ap­proval would al­low for a quick hand­off of CVT-301 as they scram­ble to pre­vent a plunge in rev­enue. Ampyra pro­vides the bulk of the com­pa­ny’s mon­ey.

Even with high odds of an ap­proval, though, biotech is known for any num­ber of sna­fus that can af­flict the progress of a drug. And this time there’s no room for er­ror as they stay fo­cused on the high wire act.

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 Covid-19 vac­cine -- shares rock­et up

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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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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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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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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Phase III read­outs spell dis­as­ter for Genen­tech’s lead IBD drug

Roche had big plans for etrolizumab. Eyeing a hyper-competitive IBD and Crohn’s market where they have not historically been a player, the company rolled out 8 different Phase III trials, testing the antibody for two different uses across a range of different patient groups.

On Monday, Roche released results for 4 of those studies, and they mark a decided setback for both the Swiss pharma and their biotech sub Genentech, potentially spelling an end to a drug they put over half-a-decade and millions of dollars behind.

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).