Ve­rastem wins break­through nod for com­bo ther­a­py in ovar­i­an can­cer; EMA picks up speedy re­view of Bio­Mar­in's gene ther­a­py

Ve­rastem has earned the FDA’s break­through ther­a­py des­ig­na­tion for its nov­el RAF/MEK in­hibitor VS-6766 as a com­bo ther­a­py with in-house FAK in­hibitor de­fac­tinib to treat sec­ond-line, re­cur­rent low-grade serous ovar­i­an can­cer re­gard­less of KRAS mu­tant sta­tus, the com­pa­ny said Mon­day.

The com­bi­na­tion is be­ing test­ed in the in­ves­ti­ga­tor-ini­ti­at­ed Phase I/II FRAME tri­al, where ear­ly re­sults showed an ORR of 52% with KRAS mu­tant ORR at 70%, KRAS wild-type ORR at 44%, and KRAS sta­tus un­de­ter­mined ORR at 0%. The most com­mon side ef­fects seen in the study were rash, cre­a­tine ki­nase el­e­va­tion, nau­sea, hy­per­biliru­bine­mia and di­ar­rhea.

Ve­rastem’s drug takes a nov­el dual-tar­get ap­proach to the RAS path­way, which is im­pli­cat­ed in can­cer re­cur­rence with about 30% of all can­cer tu­mors ex­press­ing mu­tant RAS.

Ve­rastem is al­so test­ing the VS-6766 alone and com­bi­na­tion with de­fac­tinib as part of the phase II reg­is­tra­tion RAMP 201 study. — Kyle Blanken­ship

EMA grants Bio­Marin’s re­quest for a speedy re­view of gene ther­a­py

The Eu­ro­pean Med­i­cines Agency will speed­i­ly re­view Bio­Marin’s val­oc­toco­gene rox­a­parvovec, a gene ther­a­py to treat se­vere he­mo­phil­ia A, with an opin­ion ex­pect­ed in the first half of next year, the com­pa­ny said.

With the EMA agree­ing to give the ther­a­py a clos­er look, Bio­Marin said it plans to sub­mit an as­so­ci­at­ed mar­ket­ing ap­pli­ca­tion by June, based on piv­otal Phase III da­ta that in­cludes one-year fol­low-up re­sults as well as four and three years of fol­low-up from an on­go­ing Phase I/II dose es­ca­la­tion study.

Mean­while, Bio­Marin al­so ex­pects to sub­mit two-year fol­low-up safe­ty and ef­fi­ca­cy da­ta from that Phase III test to the FDA, with a po­ten­tial re­sub­mis­sion of its ap­pli­ca­tion sched­uled for Q2 2022.

The FDA shot down Bio­Marin’s ap­pli­ca­tion for the ther­a­py back in Au­gust, cit­ing the need to see that two-year fol­low-up da­ta. — Kyle Blanken­ship

Francesco De Ru­ber­tis’ lega­cy play Centes­sa sets terms for $285M IPO

Ahead of an ex­pect­ed IPO lat­er this week, Centes­sa is set­ting terms for what it hopes is a mas­sive raise.

The com­pa­ny ex­pects to see $285 mil­lion come in with the raise, of­fer­ing 15 mil­lion shares at a price range of $18 to $20. At the mid­point of that range, Centes­sa would have a mar­ket val­ue of about $1.8 bil­lion, ac­cord­ing to Re­nais­sance Cap­i­tal.

Centes­sa comes from the mind of Francesco De Ru­ber­tis, mak­ing a lega­cy play for a 10-in-1 hold­ing com­pa­ny. His vi­sion re­volves around tak­ing the as­set-cen­tric mind­set that he’s been preach­ing at Medicxi over the years and rolling up a bunch of biotech up­starts in­to one out­fit.

They’ll be go­ing for­ward with­out Mon­cef Slaoui, the for­mer GSK ex­ec and Op­er­a­tion Warp Speed chief fol­low­ing a “sub­stan­ti­at­ed” sex­u­al ha­rass­ment case for which he sub­se­quent­ly apol­o­gized. — Max Gel­man

Scoop: Boehringer qui­et­ly shut­ters a PhII for one of its top drugs — now un­der re­view

Boehringer Ingelheim has quietly shut down a small Phase II study for one of its lead drugs.

The private pharma player confirmed to Endpoints News that it had shuttered a study testing spesolimab as a therapy for Crohn’s patients suffering from bowel obstructions.

A spokesperson for the company tells Endpoints:

Taking into consideration the current therapeutic landscape and ongoing clinical development programs, Boehringer Ingelheim decided to discontinue our program in Crohn’s disease. It is important to note that this decision is not based on any safety findings in the clinical trials.

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Vas Narasimhan (Photographer: Jason Alden/Bloomberg via Getty Images)

No­var­tis de­tails plans to axe 8,000 staffers as Narasimhan be­gins sec­ond phase of a glob­al re­org

We now know the number of jobs coming under the axe at Novartis, and it isn’t small.

The pharma giant is confirming a report from Swiss newspaper Tages-Anzeiger that it is chopping 8,000 jobs out of its 108,000 global staffers. A large segment will hit right at company headquarters in Basel, as CEO Vas Narasimhan axes some 1,400 of a little more than 11,000  jobs in Switzerland.

The first phase of the work is almost done, the company says in a statement to Endpoints News. Now it’s on to phase two. In the statement, Novartis says:

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Lina Gugucheva, NewAmsterdam Pharma CBO

Phar­ma group bets up to $1B-plus on the PhI­II res­ur­rec­tion of a once dead-and-buried LDL drug

Close to 5 years after then-Amgen R&D chief Sean Harper tamped the last spade of dirt on the last broadly focused CETP cholesterol drug — burying their $300 million upfront and the few remaining hopes for the class with it — the therapy has been fully resurrected. And today, the NewAmsterdam Pharma crew that did the Lazarus treatment on obicetrapib is taking another big step on the comeback trail with a €1 billion-plus regional licensing deal, complete with close to $150 million in upfront cash.

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How pre­pared is bio­phar­ma for the cy­ber dooms­day?

One of the largest cyberattacks in history happened on a Friday, Eric Perakslis distinctly remembers.

Perakslis, who was head of Takeda’s R&D Data Sciences Institute and visiting faculty at Harvard Medical School at the time, had spent that morning completing a review on cybersecurity for the British Medical Journal. Moments after he turned it in, he heard back from the editor: “Have you heard what’s going on right now?”

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(AP Photo/Gemunu Amarasinghe)

Some phar­ma com­pa­nies promise to cov­er abor­tion-re­lat­ed trav­el costs — while oth­ers won't go that far yet

As the US Department of Health and Human Services promises to support the millions of women who would now need to cross state lines to receive a legal abortion, a handful of pharma companies have said they will pick up employees’ travel expenses.

GSK, Sanofi, Johnson & Johnson, BeiGene, Alnylam and Gilead have all committed to covering abortion-related travel expenses just four days after the Supreme Court overturned Roe v. Wade and revoked women’s constitutional right to an abortion.

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Aurobindo Pharma co-founders P. V. Ram Prasad Reddy (L) and K. Nityananda Reddy

Au­robindo Phar­ma re­ceives warn­ing let­ter from In­di­a's SEC fol­low­ing more FDA ques­tion marks

Indian-based generics manufacturer Aurobindo Pharma has been in the crosshairs of the FDA for several years now, but the company is also attracting attention from regulators within the subcontinent.

According to the Indian business news site Business Standard, a warning letter was sent to the company from the Securities Exchange Board of India, or SEBI.

The letter is related to disclosures made by the company on an ongoing FDA audit of the company’s Unit-1 API facility in Hyderabad, India as well as observations made by the US regulator between 2019 and 2022.

New Charles River Laboratories High Quality (HQ) Plasmid DNA Centre of Excellence at Bruntwood SciTech’s Alderley Park in Cheshire, United Kingdom. (Charles River)

Charles Riv­er Lab­o­ra­to­ries to start cell and gene ther­a­py man­u­fac­tur­ing at UK site in Sep­tem­ber

While Massachusetts-based Charles River Laboratories has been on an acquisition spree, they are not against planting their flag. The latest move by the company sees them crossing the pond to establish a manufacturing site in the UK.

The company on Tuesday opened its cell and gene therapy manufacturing center at Bruntwood SciTech’s Alderley Park in Cheshire, United Kingdom. The expansion follows Charles River’s acquisition of Cognate BioServices and Cobra Biologics in 2021 for $875 million. Cognate is a plasmid DNA, viral vector and cell therapy CDMO.

Bristol Myers Squibb (Alamy)

CVS re­sumes cov­er­age of block­buster blood thin­ner af­ter price drop fol­lows Jan­u­ary ex­clu­sion

Following some backlash from the American College of Cardiology and patients, Bristol Myers Squibb and Pfizer lowered the price of their blockbuster blood thinner Eliquis, thus ensuring that CVS Caremark would cover the drug after 6 months of it being off the major PBM’s formulary.

“Because we secured lower net costs for patients from negotiations with the drug manufacturer, Eliquis will be added back to our template formularies for the commercial segment effective July 1, 2022, and patient choices will be expanded,” CVS Health said in an emailed statement. “Anti-coagulant therapies are among the non-specialty products where we are seeing the fastest cost increases from drug manufacturers and we will continue to push back on unwarranted price increases.”

#Can­nes­Lions2022: Con­sumer health ex­ecs call on agen­cies to in­volve pa­tients in cre­ative process

CANNES — When Tamara Rogers joined GSK back in 2018, “science was king and R&D were the gods.” Now the global chief marketing officer of consumer healthcare wants to make room for another supreme being: the consumer.

As health and wellness becomes more relevant to consumers amid the pandemic, four health-focused executives called on marketers to involve patients in their creative process in a panel discussion at the Cannes Lions advertising creativity festival.

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