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.

As­traZeneca trum­pets the good da­ta they found for Tagris­so in an ad­ju­vant set­ting for NSCLC — but many of the ex­perts aren’t cheer­ing along

AstraZeneca is rolling out the big guns this evening to provide a salute to their ADAURA data on Tagrisso at ASCO.

Cancer R&D chief José Baselga calls the disease-free survival data for their drug in an adjuvant setting of early stage, epidermal growth factor receptor-mutated NSCLC patients following surgery “momentous.” Roy Herbst, the principal investigator out of Yale, calls it “transformative.”

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Paul Hudson, Sanofi CEO (Getty Images)

Sanofi CEO Paul Hud­son has $23B burn­ing a hole in his pock­et. And here are some hints on how he plans to spend that

Sanofi has reaped $11.1 billion after selling off a big chunk of its Regeneron stock at $515 a share. And now everyone on the M&A side of the business is focused on how CEO Paul Hudson plans to spend it.

After getting stung in France for some awkward politicking — suggesting the US was in the front of the line for Sanofi’s vaccines given American financial support for their work, versus little help from European powers — Hudson now has the much more popular task of managing a major cash cache to pull off something in the order of a big bolt-on. Or two.

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The Advance Clinical leadership team: CEO Yvonne Lungershausen, Sandrien Louwaars - Director Business Development Operations, Gabriel Kremmidiotis - Chief Scientific Officer, Ben Edwards - Chief Strategy Officer

How Aus­tralia De­liv­ers Rapid Start-up and 43.5% Re­bate for Ear­ly Phase On­col­o­gy Tri­als

About Avance Clinical

Avance Clinical is an Australian owned Contract Research Organisation that has been providing high-quality clinical research services to the local and international drug development industry for 20 years. They specialise in working with biotech companies to execute Phase 1 and Phase 2 clinical trials to deliver high-quality outcomes fit for global regulatory standards.

As oncology sponsors look internationally to speed-up trials after unprecedented COVID-19 suspensions and delays, Australia, which has led the world in minimizing the pandemic’s impact, stands out as an attractive destination for early phase trials. This in combination with the streamlined regulatory system and the financial benefits including a very favourable exchange rate and the R & D cash rebate makes Australia the perfect location for accelerating biotech clinical programs.

Pablo Legorreta, founder and CEO of Royalty Pharma AG, speaks at the annual Milken Institute Global Conference in Beverly Hills, California (Patrick T. Fallon/Bloomberg via Getty Images)

Cap­i­tal­iz­ing Pablo: The world’s biggest drug roy­al­ty buy­er is go­ing pub­lic. And the low-key CEO di­vulges a few se­crets along the way

Pablo Legorreta is one of the most influential players in biopharma you likely never heard of.

Over the last 24 years, Legorreta’s Royalty Pharma group has become, by its own reckoning, the biggest buyer of drug royalties in the world. The CEO and founder has bought up a stake in a lengthy list of the world’s biggest drug franchises, spending $18 billion in the process — $2.2 billion last year alone. And he’s become one of the best-paid execs in the industry, reaping $28 million from the cash flow last year while reserving 20% of the cash flow, less expenses, for himself.

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Dan O'Day, Gilead CEO (Andrew Harnik, AP Images)

UP­DAT­ED: Gilead leas­es part­ner rights to TIG­IT, PD-1 in a $2B deal with Ar­cus. Now comes the hard part

Gilead CEO Dan O’Day has brokered his way to a PD-1 and lined up a front row seat in the TIGIT arena, inking a deal worth close to $2 billion to align the big biotech closely with Terry Rosen’s Arcus. And $375 million of that comes upfront, with cash for the buy-in plus equity, along with $400 million for R&D and $1.22 billion in reserve to cover opt-in payments and milestones..

Hotly rumored for weeks, the 2 players have formalized a 10-year alliance that starts with rights to the PD-1, zimberelimab. O’Day also has first dibs on TIGIT and 2 other leading programs, agreeing to an opt-in fee ranging from $200 million to $275 million on each. There’s $500 million in potential TIGIT milestones on US regulatory events — likely capped by an approval — if Gilead partners on it and the stars align on the data. And there’s another $150 million opt-in payments for the rest of the Arcus pipeline.

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Bryan Roberts, Venrock

Ven­rock sur­vey shows grow­ing recog­ni­tion of coro­n­avirus toll, wan­ing con­fi­dence in ar­rival of vac­cines and treat­ments

When Venrock partner Bryan Roberts went to check the results from their annual survey of healthcare leaders, what he found was an imprint of the pandemic’s slow arrival in America.

The venture firm had sent their form out to hundreds of insurance and health tech executives, investors, officials and academics on February 24 and gave them two weeks to fill it out. No Americans had died at that point but the coronavirus had become enough of a global crisis that they included two questions about the virus, including “Total U.S. deaths in 2020 from the novel coronavirus will be:”.

Roger Perlmutter, Merck R&D chief (YouTube)

UP­DAT­ED: Backed by BAR­DA, Mer­ck jumps in­to Covid-19: buy­ing out a vac­cine, part­ner­ing on an­oth­er and adding an­tivi­ral to the mix

Merck execs are making a triple play in a sudden leap into the R&D campaign against Covid-19. And they have more BARDA cash backing them up on the move.

Tuesday morning the pharma giant simultaneously announced plans to buy an Austrian biotech that has been working on a preclinical vaccine candidate, added a collaboration on another vaccine with the nonprofit IAVI and inked a deal with Ridgeback Biotherapeutics on an early-stage antiviral.

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David Hoey (Vaxxas)

In for the long vac­cine game, Mer­ck buys in­to patch de­liv­ery tech with pan­dem­ic po­ten­tial

When Merck dived into the R&D fray for a Covid-19 vaccine earlier this week, execs made it clear that they’re not necessarily looking to be first — with CEO Ken Frazier throwing cold water on the hotly-discussed 12- to 18-month timelines. But when it does emerge from behind, the pharma giant clearly expects to play a significant part.

Part of that will depend on next-generation delivery technology that reshapes the world’s imagination of a vaccine.

No­var­tis jumps in­to Covid-19 vac­cine hunt, as Big Phar­ma and big biotech com­mit to bil­lions of dos­es

After spending most of the pandemic on the sidelines, Novartis is offering its aid in the race to develop a Covid-19 vaccine.

AveXis, the Swiss pharma’s gene therapy subsidiary, has agreed to manufacture the vaccine being developed by Massachusetts Eye and Ear and Massachusetts General Hospital. The biotech will begin manufacturing this month, while the vaccine undergoes further preclinical testing. They’ve agreed to provide the vaccine for free for clinical trials beginning in the second half of 2020, but have not disclosed financials for after.

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