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.

The DCT-OS: A Tech­nol­o­gy-first Op­er­at­ing Sys­tem - En­abling Clin­i­cal Tri­als

As technology-enabled clinical research becomes the new normal, an integrated decentralized clinical trial operating system can ensure quality, deliver consistency and improve the patient experience.

The increasing availability of COVID-19 vaccines has many of us looking forward to a time when everyday things return to a state of normal. Schools and teachers are returning to classrooms, offices and small businesses are reopening, and there’s a palpable sense of optimism that the often-awkward adjustments we’ve all made personally and professionally in the last year are behind us, never to return. In the world of clinical research, however, some pandemic-necessitated adjustments are proving to be more than emergency stopgap measures to ensure trial continuity — and numerous decentralized clinical trial (DCT) tools and methodologies employed within the last year are likely here to stay as part of biopharma’s new normal.

As­traZeneca caps PD-L1/CT­LA-4/chemo com­bo come­back with OS win. Is treme­li­mum­ab fi­nal­ly ready for ap­proval?

AstraZeneca’s closely-watched POSEIDON study continues to be the rare bright spot in its push for an in-house PD-L1/CTLA-4 combo.

Combining Imfinzi and tremelimumab with physicians’ choice of chemotherapy helped patients with stage IV non-small cell lung cancer live longer, the company reported — marking the first time the still-experimental tremelimumab has demonstrated an OS benefit.

For AstraZeneca and CEO Pascal Soriot, the positive readout — which is devoid of numbers — offers much-needed validation for the big bet they made on Imfinzi plus tremelimumab, after the PD-L1/CTLA-4 regimen failed multiple trials in head and neck cancer as well as lung cancer.

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Onno van de Stolpe, Galapagos CEO (Thierry Roge/Belga Mag/AFP via Getty Images)

Gala­pa­gos chops in­to their pipeline, drop­ping core fields and re­or­ga­niz­ing R&D as the BD team hunts for some­thing 'trans­for­ma­tive'

Just 5 months after Gilead gutted its rich partnership with Galapagos following a bitter setback at the FDA, the Belgian biotech is hunkering down and chopping the pipeline in an effort to conserve cash while their BD team pursues a mission to find a “transformative” deal for the company.

The filgotinib disaster didn’t warrant a mention as Galapagos laid out its Darwinian restructuring plans. Forced to make choices, the company is ditching its IPF molecule ’1205, while moving ahead with a Phase II IPF study for its chitinase inhibitor ’4617.

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Ron DePinho (file photo)

A 'fly­over' biotech launch­es in Texas with four Ron De­Pin­ho-found­ed com­pa­nies un­der its belt

In his 13 years at Genzyme, Michael Wyzga noticed something about East Coast drugmakers. Execs would often jet from Boston or New York to San Francisco to find more assets, and completely miss the work being done in flyover states, like Texas or Wisconsin.

“If it doesn’t come out of MGH or MIT or Harvard, probably not that interesting,” he said of the mindset.

Now, he and some well-known industry players are looking to change that, and they’ve reeled in just over $38 million to do it.

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Stéphane Bancel, Getty

Mod­er­na CEO brush­es off US sup­port for IP waiv­er, eyes more than $19B in Covid-19 vac­cine sales in 2021

Moderna is definitively more concerned with keeping pace with Pfizer in the race to vaccinate the world against Covid-19 than it is with Wednesday’s decision from the Biden administration to back an intellectual property waiver that aims to increase vaccine supplies worldwide.

In its first quarter earnings call on Thursday, Moderna CEO Stéphane Bancel shrugged off any suggestion that the newly US-backed intellectual property waiver would impact his company’s vaccine or bottom line. Still, the company’s stock price fell by about 9% in early morning trading.

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Ad­comm splits slight­ly in fa­vor of FDA ap­prov­ing Chemo­Cen­tryx’s rare dis­ease drug

The FDA’s Arthritis Advisory Committee on Thursday voted 10 for and 8 against the approval of ChemoCentryx’s $CCXI investigational drug avacopan as a treatment for adults with a rare and serious disease known as anti-neutrophil cytoplasmic autoantibody (ANCA)-vasculitis.

The vote on whether the FDA should approve the drug was preceded by a split vote of 9 to 9 on whether the efficacy data support approval, and 10 to 8 that the safety profile of avacopan is adequate enough to support approval.

Paul Hastings, Nkarta CEO

With no up­front pay­ment or mile­stones on the line, Nkar­ta and CRISPR join forces on CAR-NK search

Most deals in biotech come with hefty upfront payments attached, and the promise of big biobucks if a program works out. Not this one.

Nkarta has struck what CEO Paul Hastings calls a “real collaboration” with CRISPR Therapeutics to co-develop and commercialize two CAR-NK therapies, in addition to an NK+T program. The duo will split all R&D costs — and any worldwide profits — 50/50, Hastings said.

Brent Saunders (Richard Drew, AP Images)

OcuWho? Star deal­mak­er turned aes­thet­ics czar Brent Saun­ders flips back in­to biotech. But who’s he team­ing up with now?

Brent Saunders went on a tear of headline-blazing deals building Allergan, merging and rearranging a variety of big companies into one before an M&A pact with Pfizer blew up and sent him on a bout of biotech drug deals. That didn’t work so well, so under pressure, he got his buyout at AbbVie — which needed a big franchise like Botox. And it was no big surprise to see him riding the SPAC wave into a recent $1 billion-plus deal that left him in the executive chairman’s seat at an aesthetics outfit — now redubbed The Beauty Health Company — holding a big chunk of the equity.

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Drug pric­ing watch­dog joins the cho­rus of crit­ics on Bio­gen's ad­u­canum­ab: What about charg­ing $2,560 per year?

As if Biogen’s aducanumab isn’t controversial enough, the researchers at drug pricing watchdog ICER have drawn up the contours of a new debate: If the therapy does get approved for Alzheimer’s by June, what price should it command?

Their answer: At most $8,290 per year — and perhaps as little as $2,560.

Even at the top of the range, the proposed price is a fraction of the $50,000 that Wall Street has reportedly come to expect (although RBC analyst Brian Abrahams puts the consensus figure at $11.5K). With critics, including experts on the FDA’s advisory committee, making their fierce opposition to aducanumab’s approval loud and clear, the pricing pressure adds one extra wrinkle Biogen CEO Michel Vounatsos doesn’t need as he orders full-steam preparation for a launch.