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.

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.

The End­points 11: They've got mad mon­ey and huge am­bi­tions. It's time to go big or go home

These days, selecting a group of private biotechs for the Endpoints 11 spotlight begins with a sprint to get ahead of IPOs and the M&A teams at Big Pharma. I’ve had a couple of faceplants earlier this year, watching some of the biotechs on my short list choose a quick leap onto Nasdaq or into the arms of a buyer.

Vividion, you would have been a great pick for the Endpoints 11. I’m sorry I missed you.

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Dave Lennon, former president of Novartis Gene Therapies

So what hap­pened with No­var­tis Gene Ther­a­pies? 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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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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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.

FDA+ roundup: Bs­U­FA III ready for show­time, court tells FDA to re-work com­pound­ing plan, new guid­ance up­dates and more

The FDA has now spelled out what exactly will be included in the third iteration of Biosimilar User Fee Act (BsUFA) from 2023 through 2027, which similarly to the prescription drug deal, sets fees that industry has to pay for submitting applications, in exchange for firm timelines that the agency must meet.

This latest deal includes several sweeteners for the biosimilar industry, which has yet to make great strides in the US market, with shorter review timelines for safety labeling updates and updates to add or remove an indication that does not contain efficacy data.

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Joshua Liang, Clover Biopharmaceuticals CEO

With world still in sore need of dos­es, Clover says its Covid-19 vac­cine is 67% ef­fec­tive in Phase III

With concerns about the Delta variant rising and much of the world still in desperate need of vaccine doses, a Chinese biotech announced Wednesday that a new shot has shown positive results in a large trial against Covid-19, including new variants.

Clover Biopharmaceuticals announced Wednesday that its vaccine candidate showed 79% efficacy against the Delta variant in a Phase II/III trial dubbed Spectra, and 67% effective against Covid-19 overall.

Jean Bennett (Brent N. Clarke/Invision/AP Images)

Lux­tur­na in­ven­tor Jean Ben­nett starts a new gene ther­a­py com­pa­ny to tack­le rare dis­eases left be­hind by phar­ma, VCs

A few years ago Jean Bennett found herself in a surprising place for a woman who invented the first gene therapy ever approved in the United States: No one, it seemed, wanted her work.

Bennett, who designed and co-developed Luxturna, approved in 2018 for a rare form of blindness, had kept building new gene therapies for eye diseases at her University of Pennsylvania lab. But although the results in animals looked promising, pharma companies and investors kept turning down the pedigreed ophthalmology professor.

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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.