Ve­rastem's low-stakes come­back gam­ble on an Ab­b­Vie/In­fin­i­ty castoff pays off with pos­i­tive CLL da­ta

Ve­rastem CEO $VSTM Robert For­rester was able to pick up the late-stage can­cer drug du­velis­ib from a bad­ly wound­ed In­fin­i­ty Phar­ma­ceu­ti­cals late last year for ex­act­ly noth­ing up front. And now he’s set­ting out to prove that the drug is a steal, of­fer­ing up one in­com­plete but pos­i­tive snap­shot of top-line Phase III da­ta to prove that he has a shot at a near-term ap­proval that could be worth up to $300 mil­lion a year in the US mar­ket alone.

Robert For­rester

Ve­rastem’s new­ly ac­quired PI3K-delta/gam­ma in­hibitor hit the pri­ma­ry in the 4-year study, achiev­ing a pro­gres­sion-free sur­vival rate of 13.3 months among re­lapsed or re­frac­to­ry chron­ic lym­pho­cyt­ic leukemia /small lym­pho­cyt­ic lym­phoma pa­tients com­pared to 9.9 months for No­var­tis’ Arz­er­ra (ofa­tu­mum­ab). Break it down to me­di­an PFS in the sub­set of hard-to-treat pa­tients with 17p dele­tion ran­dom­ized to du­velis­ib, and you al­so get a sig­nif­i­cant­ly high­er 12.7-month rate for their drug com­pared to 9.0 months for the com­para­tor, along with a shot at a clear­ly de­fined niche.

For Ve­rastem, that hit on the pri­ma­ry end­point is good enough to start dis­cus­sions with the FDA on fil­ing for an ap­proval. In For­rester’s words, “it’s every­thing we hoped for and more.”

In­vestors loved what they saw, with Ve­rastem shares spik­ing 44% on the news.

“This fits nice­ly in the CLL mar­ket­place,” For­rester adds, which is tran­si­tion­ing from chemo be­ing the tra­di­tion­al route of treat­ment to a new set of oral drugs like du­velis­ib that can help an old­er group of pa­tients treat them­selves at home, main­tain­ing their qual­i­ty of life as well as life ex­pectan­cy.

The PFS rate, though, is one of sev­er­al key mea­sures the tri­al stud­ied. Re­searchers al­so ex­plored over­all re­sponse rates as well as over­all sur­vival for the sec­on­daries. Pressed, For­rester con­ced­ed that he has the ORR da­ta in hand, but won’t re­lease it un­til a lat­er sci­en­tif­ic con­fer­ence some­time in the near fu­ture.

What he does say, though, is that the da­ta back Ve­rastem’s plan to roll this drug out them­selves as a sec­ond or third-line CLL drug in the US, look­ing to grab 15% to 20% of the mar­ket in that niche, which For­rester es­ti­mates is worth $200 mil­lion to $300 mil­lion a year. Part­ners can be found for the rest of the world.

Ve­rastem is al­so adding a pro­gram for pe­riph­er­al T-cell lym­phoma for du­velis­ib as it looks to ex­pand in­di­ca­tions fol­low­ing the first OK, pro­vid­ed that comes through.

Du­velis­ib was once a block­buster prospect, up un­til In­fin­i­ty out­lined pos­i­tive but dis­ap­point­ing Phase II da­ta, with a 46% ORR rate for in­do­lent non-Hodgkin lym­phoma. Ab­b­Vie, which had inked an $805 mil­lion deal with In­fin­i­ty to get this in their pipeline, prompt­ly turned their backs on the drug and walked — sat­is­fied that the pos­i­tive da­ta did not trans­late in­to a sol­id com­mer­cial op­por­tu­ni­ty.

In­fin­i­ty, which had seen two pre­vi­ous lead drugs crushed by bad da­ta, was hit hard. Its stock price was evis­cer­at­ed, the biotech was forced to re­struc­ture and lay off staffers, and CEO Ade­lene Perkins was charged with un­load­ing the drug AS­AP.

That’s when For­rester called. He wound up get­ting the drug in a deal that in­cludes just $28 mil­lion in mile­stones: $6 mil­lion for a pos­i­tive Phase III and $22 mil­lion on ap­proval.

Ve­rastem has al­so known what it’s like to be stung by fail­ure. Close to two years ago now, af­ter ini­tial­ly de­fend­ing the da­ta, For­rester ad­mit­ted that their lead drug de­fac­tinib (VS-6063) had failed its most ad­vanced study for mesothe­lioma, a dif­fi­cult-to-treat type of lung can­cer as­so­ci­at­ed with re­peat­ed con­tact with as­bestos. This came af­ter the drug showed poor ef­fi­ca­cy and se­ri­ous ad­verse events in a study for non-small cell lung can­cer. Its stock was crushed, and that biotech al­so re­struc­tured.

The FDA has been known to ap­prove drugs based on sim­i­lar da­ta, or worse. But there are still some im­por­tant ques­tions on du­velis­ib that will need to be asked and an­swered. But Ve­rastem is one step clos­er to a ma­jor ob­jec­tive.

Covid-19 roundup: Eu­rope pur­chas­es 80M dos­es of Mod­er­na's vac­cine; CO­V­AXX se­cures $2.8B in emerg­ing mar­ket pre-or­ders

With the announcement of its vaccine efficacy data last week, Moderna is starting to line up customers for its Covid-19 mRNA jabs.

The Massachusetts-based biotech announced Wednesday it has agreed to sell an initial round of 80 million doses to the European Commission, with the option to double the amount to 160 million. Once the member states rubber stamp the approval, the deal will be finalized.

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Pascal Soriot (AP Images)

UP­DAT­ED: As­traZeneca, Ox­ford on the de­fen­sive as skep­tics dis­miss 70% av­er­age ef­fi­ca­cy for Covid-19 vac­cine

On the third straight Monday that the world wakes up to positive vaccine news, AstraZeneca and Oxford are declaring a new Phase III milestone in the fight against the pandemic. Not everyone is convinced they will play a big part, though.

With an average efficacy of 70%, the headline number struck analysts as less impressive than the 95% and 94.5% protection that Pfizer/BioNTech and Moderna have boasted in the past two weeks, respectively. But the British partners say they have several other bright spots going for their candidate. One of the two dosing regimens tested in Phase III showed a better profile, bringing efficacy up to 90%; the adenovirus vector-based vaccine requires minimal refrigeration, which may mean easier distribution; and AstraZeneca has pledged to sell it at a fraction of the price that the other two vaccine developers are charging.

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Jason Kelly, Ginkgo Bioworks CEO (Kyle Grillot/Bloomberg via Getty Images)

Af­ter Ko­dak de­ba­cle, US lends $1.1B to a syn­thet­ic bi­ol­o­gy com­pa­ny and their big Covid-19, mR­NA plans

In mid-August, as Kodak’s $765 million government-backed push into drug manufacturing slowly fell apart in national headlines, Ginkgo Bioworks CEO Jason Kelly got a message from his company’s government liaison: HHS wanted to know if they, too, might want a loan.

The government’s decision to lend Kodak three quarters of a billion dollars raised eyebrows because Kodak had never made drugs before. But Ginkgo, while not a manufacturing company, had spent the last decade refining new ways to produce materials inside cells and building automated facilities across Boston.

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Vivek Ramaswamy (Jeff Rumans/JPM 2020)

Urovan­t's lead drug dis­ap­points in mid-stage study as first big FDA de­ci­sion looms

Just as Urovant gets ready for its first big FDA decision on vibegron, the drug has flopped in what would’ve been a follow-on indication.

In a Phase IIa trial involving women with abdominal pain due to irritable bowel syndrome, vibegron failed to meet the bar on improving “average worst abdominal pain” over 12 weeks, compared to placebo, among IBS-D patients.

There were actually slightly more responders in the placebo group than in the drug arm, with only 40.9% of those randomized to vigebron achieving at least a 30% decrease in “worst abdominal pain” in the past 24 hours. The trial enrolled 222 women but only 189 completed the study.

Bahija Jallal (file photo)

TCR pi­o­neer Im­muno­core scores a first with a land­mark PhI­II snap­shot on over­all sur­vival for a rare melanoma

Bahija Jallal’s crew at TCR pioneer Immunocore says they have nailed down a promising set of pivotal data for their lead drug in a frontline setting for a solid tumor. And they are framing this early interim readout as the convincing snapshot they need to prove that their platform can deliver on a string of breakthrough therapies now in the clinic or planned for it.

In advance of the Monday announcement, Jallal and R&D chief David Berman took some time to walk me through the first round of Phase III data for their lead TCR designed to treat rare, frontline cases of metastatic uveal melanoma that come with a grim set of survival expectations.

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FDA hands Liq­uidia and Re­vance a CRL and de­fer­ral, re­spec­tive­ly, as Covid-19 cre­ates in­spec­tion chal­lenge

Two biotechs said they got turned away by the FDA on Wednesday, in part due to pandemic-related travel restrictions.

North Carolina-based Liquidia Technologies was handed a CRL for its lead pulmonary arterial hypertension drug, citing the need for more CMC data and on-site pre-approval inspections, which the FDA hasn’t been able to conduct due to travel restrictions. The agency also deferred its decision on Revance Therapeutics’ BLA for its frown line treatment, because it needs to inspect the company’s northern California manufacturing facility. The action, Revance emphasized, was not a CRL.

News brief­ing: FDA re­quests new tri­al for Reata's Friedre­ich's atax­ia pro­gram; J&J's Trem­fya picks up ex­pand­ed la­bel in Eu­rope

Three months after Reata Pharmaceuticals suggested its Friedreich’s ataxia program omaveloxolone could be delayed, the company revealed that is indeed going to be the case.

Reata $RETA shares took a nosedive Wednesday after the biotech revealed that the FDA said supplemental data for its pivotal trial did not strengthen the case for approval. As a result, the drug is likely to need another study before the FDA takes up the case.

Jef­frey Hat­field takes over from Diego Mi­ralles as CEO of Vi­vid­ion; Drag­on­fly scores a new ex­ec with COO Alex Lu­gov­skoy

→ San Diego protein degradation startup Vividion Therapeutics has made a change at the top with Jeffrey Hatfield taking the helm as CEO, replacing Diego Miralles six months after Roche forked over $135 million to collaborate with Vividion on their small molecule degraders. Hatfield is chairman of the board at miRagen Therapeutics and previously held the CEO job at Zafgen and Vitae Pharmaceuticals. He also had a series of leadership roles at Bristol Myers Squibb from 1996-2004, including SVP, immunology and virology divisions.

Chi­na opens the door for biotech in­vestors in Hong Kong to buy Shang­hai stocks, and vice ver­sa

When Shanghai’s STAR board began opening its doors to biotech, it was considered not just a rival to Nasdaq but also the stock exchange in Hong Kong. Those perceptions may take an amicable turn as China expands a mutual access program with the city.

The changes mean investors in mainland China will be able to own Hong Kong biotech chapter stocks, while those in Hong Kong — a much more internationally connected group — would have access to those listed on STAR. In effect, it turns the Shanghai market into a globally accessible exchange overnight while also broadening a key source of revenue for HKEX.