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

Un­lock­ing ESG strate­gies for growth with Gilead Sci­ences

RBC Capital Markets explores what is material in ESG for biopharma companies with the ESG leads at Gilead Sciences. Gilead has long focused on sustainability but recognized a more robust framework was needed. Based on a materiality assessment, Gilead’s ESG strategy today focuses first on drug access and pricing, while also addressing D&I and climate change. Find out why Gilead’s board is “acutely aware” of the contribution that ESG makes to firm’s overall success.

On the hunt for the next Mod­er­na, in­vestors have pumped 'plat­form plays' with cash. Can any­thing slow the run­away train?

It didn’t take an expert to see that mRNA platforms could be huge.

Julie Sunderland partnered with both Moderna and BioNTech about a decade ago while she was running program-related investments for the Bill & Melinda Gates Foundation — and even then the potential for their platforms was obvious despite some well-founded concerns about whether the next-gen tech would ever cross the finish line.

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Roche's Alzheimer's drug spurred bio­mark­er changes but no cog­ni­tive im­pact — pa­per; vTv out-li­cens­es for­mer lead pro­gram

More than a year ago, Roche and Eli Lilly were forced to contend with a Phase II/III failure of their respective Alzheimer’s drugs. But while Eli Lilly essentially threw in the towels, Roche wasn’t ready to give up yet.

The Swiss drugmaker now has some biomarker data to spotlight as investigators continue monitoring patients in an open-label extension study.

Dubbed DIAN-TU, the study had been designed to see whether Roche’s gantenerumab and Lilly’s solanezumab could spur a cognitive benefit for a group of patients who had a rare, inherited form of Alzheimer’s that’d tied to early-onset. In short, they didn’t: Both failed the primary endpoint.

Fred Upton and Diana DeGette

New DARPA-like NIH agency preps for re­al­i­ty as E&C un­veils bi­par­ti­san Cures 2.0 draft bill

House Energy & Commerce leaders Fred Upton (R-MI) and Diana DeGette (D-CO) on Tuesday released new draft legislation with wide-ranging implications for public health, the FDA, NIH, and that would create a new, $6.5 billion federal advanced research agency under NIH, with an aim to cure cancer, Alzheimer’s and other difficult diseases.

Similar to DARPA, the new NIH division to be known as ARPA-H, would be run by a small group of program managers with more latitude to pursue high-risk, high-reward projects that other government agencies would likely shy away from.

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Sen. Ron Wyden (D-OR) with reporters in the Senate Subway (Graeme Sloan/Sipa via AP Images)

Top Wyden pri­or­i­ty for drug price re­forms: Medicare ne­go­ti­a­tions

As the Biden administration tries to wrangle the details of its infrastructure bill, Senate Finance Committee Chair Ron Wyden (D-OR) took a concrete step forward on drug pricing reforms on Tuesday and unveiled five principles for such reforms, including providing Medicare with the ability to negotiate prices.

“Allowing the Secretary of HHS to negotiate the price Medicare will pay creates a much needed mechanism to achieve fairer prices when the market has failed to do so,” Wyden wrote.

End­points News is now 5 years old. Here's how you can sup­port us for the next phase of growth

Endpoints News turned five years old over the weekend. I wanted to mark the happy occasion by extending our deepest gratitude to Endpoints’ premium subscribers while outlining several other ways to support us as we go broader and get bigger this year and beyond.

Same as any business, we’ve got to create value and get paid for delivering it. So if you depend on Endpoints to stay abreast on biopharma developments, we depend on you too.

Emma Walmsley, GlaxoSmithKline CEO (Credit: Fang Zhe/Xinhua/Alamy Live News)

Ac­tivist in­vestor El­liott in talks with oth­er Glax­o­SmithK­line in­vestors about re­plac­ing Em­ma Walm­s­ley, spin­ning off vac­cine busi­ness — re­port

As Emma Walmsley reveals details this Wednesday about the upcoming split of GlaxoSmithKline’s pharma and consumer units, some tough questions may be coming her way.

Elliott Management, the activist investor that’s previously threatened an attack on GSK (but eventually backed off), is floating more radical changes like replacing the CEO, further breaking up the company and spinning out the vaccine unit, or reviewing the focus on cancer drugs, the Financial Times reported.

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Covid-19 roundup: White House re­veals vac­cine do­na­tion plan; EU opts in for an­oth­er 150 mil­lion dos­es from Mod­er­na

President Joe Biden’s administration on Monday revealed the distribution list for 55 million of the 80 million doses of Covid-19 vaccines America plans to donate to lower-income nations:

Roughly 14 million doses will head to Latin America and the Caribbean, for a list of countries that includes Brazil, Argentina, Dominican Republic, Panama and Costa Rica.
Another 16 million doses are headed to Asia to help the following countries: India, Nepal, Bangladesh, Pakistan, Sri Lanka, Afghanistan, Maldives, Bhutan, Philippines, Vietnam, Indonesia, Thailand, Malaysia, Laos, Papua New Guinea, Taiwan, Cambodia, and the Pacific Islands.

Ken Frazier, Merck CEO (Bess Adler/Bloomberg via Getty Images)

Mer­ck­'s Keytru­da blazes a path in first-line cer­vi­cal can­cer, mak­ing good on drug­mak­er's push for ear­li­er pa­tients

In the years since I/O wonder drug Keytruda’s initial approval, Merck has struck an aggressive clinical trial program, which is now firmly focused on earlier lines of therapy. The drugmaker has scored some success there so far, and now it’s earned one of its biggest wins yet.

Keytruda plus chemotherapy with or without background Avastin significantly extended patients’ lives over those dosed with a placebo control in first-line patients with persistent, recurrent or metastatic cervical cancer, according to top-line data from the Phase III KEYNOTE-826 study revealed Tuesday.

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