Mak­e­na's ac­cel­er­at­ed ap­proval with­draw­al hear­ing set for Oc­to­ber as Co­vis wants to do more re­search

The long-await­ed FDA hear­ing to re­view Co­vis Phar­ma’s ac­cel­er­at­ed ap­proval for its con­tro­ver­sial preterm birth in­jec­tion, which failed its con­fir­ma­to­ry tri­al, will take place from Oct. 17-19.

Celia Wit­ten

The FDA’s Ob­stet­rics, Re­pro­duc­tive and Uro­log­ic Drugs Ad­vi­so­ry Com­mit­tee will con­duct the hear­ing about the fu­ture of Mak­e­na (hy­drox­yprog­es­terone caproate in­jec­tion), the com­pa­ny said in a state­ment yes­ter­day.

In a show of fair­ness, the hear­ing will al­low for both Co­vis and FDA’s Cen­ter for Drug Eval­u­a­tion and Re­search to each make two-hour pre­sen­ta­tions, and both CDER and Co­vis will be giv­en a full hour to ask ques­tions af­ter the op­pos­ing side makes its pre­sen­ta­tion. An ad­di­tion­al four hours will be set aside for pub­lic com­ments, which will al­low for the gener­ic man­u­fac­tur­ers of Mak­e­na to al­so speak.

The FDA’s Celia Wit­ten, who’s pre­sid­ing over the hear­ing, pre­vi­ous­ly told Co­vis that the de­sign of any fu­ture con­fir­ma­to­ry tri­al(s) is not an is­sue to be de­cid­ed as part of this hear­ing process.

Even still, Co­vis said in a state­ment that it has urged FDA to al­low the com­pa­ny to do ad­di­tion­al re­search to ful­ly ex­plore the ef­fi­ca­cy of Mak­e­na for in­di­cat­ed pa­tients, “and looks for­ward to pre­sent­ing pro­posed study op­tions at the Oc­to­ber hear­ing.”

The com­pa­ny said it’s de­vel­op­ing:

Op­tions that im­prove up­on the pre­vi­ous spon­sor’s pro­pos­al for ad­di­tion­al stud­ies. Specif­i­cal­ly, Co­vis is ex­plor­ing two al­ter­na­tive op­tions: (1) con­duct­ing a prospec­tive his­tor­i­cal con­trol study, con­sis­tent with FDA guid­ance, and/or (2) con­duct­ing a ran­dom­ized clin­i­cal tri­al (RCT) re­ly­ing on an analy­sis of ret­ro­spec­tive datasets for the pur­pose of iden­ti­fy­ing rel­e­vant sub­groups for study.

Adam Ura­to

But some physi­cians are ques­tion­ing the need to keep Mak­e­na on the mar­ket as these ad­di­tion­al stud­ies are on­go­ing.

“If the drug stays on the mar­ket, preg­nant women across the US will con­tin­ue to be in­ject­ed with a syn­thet­ic hor­mone that has no proven ben­e­fit and pos­si­ble short and long-term harms,” Adam Ura­to, a ma­ter­nal-fe­tal med­i­cine spe­cial­ist in Fram­ing­ham, MA, tells End­points News.

Plans for this hear­ing have dragged on af­ter FDA first pro­posed to with­draw Mak­e­na’s ap­proval fol­low­ing an­oth­er 2019 ad­comm that vot­ed against keep­ing the ac­cel­er­at­ed ap­proval (9-7) af­ter a re­view of tri­al re­sults that did not con­firm the ben­e­fit of Mak­e­na as a treat­ment to re­duce the risk of preterm birth.

Co­vis, mean­while, in­sists the da­ta are mixed.

“We are com­mit­ted to fur­ther study to clar­i­fy the risk-ben­e­fit of this drug, giv­en the in­con­sis­tent ef­fi­ca­cy out­comes be­tween the land­mark Meis study and PRO­LONG, and the im­por­tant role Mak­e­na plays in this high-risk and un­der­served pa­tient pop­u­la­tion,” said Co­vis CEO Michael Porter in a state­ment.

Biotech in­vestors and CEOs see two paths to growth, but are they equal­ly vi­able?

The dynamic in the biotech market has been highly volatile in the last few years, from the high peaks immediately after the COVID vaccine in 2021, to the lowest downturns of the last 20 years in 2022. This uncertainty makes calling the exact timing of the market’s turn something of a fool’s errand, according to Dr. Chen Yu, Founder and Managing Partner of TCG Crossover (TCG X). He speaks with RBC’s Noël Brown, Head of US Biotechnology Investment Banking, about the market’s road ahead and two possible paths for growth.

Casey McPherson shows his daughters Rose (left) and Weston around Everlum Bio, a lab that he co-founded to spark a treatment for Rose and others with ultra-rare conditions. (Ilana Panich-Linsman)

Fa­ther starts lab af­ter in­tel­lec­tu­al prop­er­ty is­sues stymie rare dis­ease drug de­vel­op­ment

Under bright lab lights, Casey McPherson holds his 6-year-old daughter, Rose. His free hand directs Rose’s gaze toward a computer screen with potential clues in treating her one-of-a kind genetic condition.

Gray specks on the screen show her cells that scientists reprogrammed with the goal of zeroing in on a custom medicine. McPherson co-founded the lab, Everlum Bio, to spark a treatment for Rose — and others like her. A regarded singer-songwriter, McPherson never imagined going into drug development.

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Benjamine Liu, TrialSpark CEO

Paul Hud­son and Tri­alSpark's mu­tu­al de­sire to speed up de­vel­op­ment con­verges in three-year, six-drug goal

A unicorn startup that originally set out to hasten clinical studies for biopharma partners dug further into its revised path of internal drug development by linking arms with Sanofi in a pact that the biotech’s CEO said originated from the top.

TrialSpark and the Big Pharma on Tuesday committed to in-licensing and/or acquiring six Phase II/Phase III drugs within the next three years.

“I’ve known Paul Hudson for a while and we were discussing the opportunity to really re-imagine a lot of different parts of pharma,” TrialSpark CEO Benjamine Liu told Endpoints News, “and one of the things that we discussed was this opportunity to accelerate the development of new medicines in mutual areas of interest.”

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Take­da to pull key hy­poparathy­roidism drug from the mar­ket en­tire­ly by end of 2024 af­ter years of man­u­fac­tur­ing woes

Takeda on Tuesday morning made an announcement that almost 3,000 people with the rare disease known as hypoparathyroidism were fearing.

Due to unresolved supply issues and manufacturing woes, Takeda said it will cut its losses and discontinue its hypoparathyroidism drug, known as Natpara (parathyroid hormone), halting all manufacturing of the drug by the end of 2024.

The decision to not re-commercialize Natpara will be a blow to not only the 2,400 people who were awaiting supplies of their reliable injection since 2019, but also the additional nearly 400 people who were accessing the drugs via the company’s Special Use Program as Takeda sought to resolve these manufacturing issues over the past five years.

Big Phar­ma heavy­weights seek tweaks to FDA's clin­i­cal out­come as­sess­ment guid­ance

Pfizer, GSK, Janssen, Regeneron, Boehringer Ingelheim and at least a half dozen other companies are calling on the FDA to provide significantly more clarity in its draft guidance from this summer on clinical outcome assessments, which are a type of patient experience.

The draft is the third in a series of four patient-focused drug development guidance documents that the FDA had to create as part of the 21st Century Cures Act, and they describe how stakeholders (patients, caregivers, researchers, medical product developers and others) can collect and submit patient experience data and other relevant information for medical product development and regulatory decision-making.

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Astel­las, Pan­th­er­na add or­gan to mR­NA tie-up; Rock­et launch­es sale of six fig­ures worth of stock

Astellas and Pantherna have expanded their November 2021 pact surrounding the latter’s mRNA platform to include a new target organ, the duo announced Tuesday morning, though they did not specify what that target is.

German biotech Pantherna is home to two platform technologies — one that designs mRNAs for non-vaccine therapies and another that designs LNPs. Astellas and Pantherna’s deal appears to mainly revolve around the first platform, which Astellas said it is using to research direct reprogramming, or turning cells from one kind into another without an intermediate stem cell phase.

Marc Dunoyer, Alexion CEO (AstraZeneca via YouTube)

Up­dat­ed: As­traZeneca nabs a small rare dis­ease gene ther­a­py play­er for 667% pre­mi­um

AstraZeneca is kicking off the fourth quarter with a little M&A Monday for a gene editing player recently overcoming a second clinical hold to its only program in human studies.

The Big Pharma and its subsidiary Alexion are buying out little LogicBio for $2.07 per share. That’s good for a massive 667% premium over its Friday closing price, when it headed into the weekend at 27 cents and just weeks after Nasdaq said LogicBio would have to delist, which has been put on hold as the biotech requests a hearing. It’s one of two biotech deals to commence October, alongside the news of Incyte buying a vitiligo-focused biotech.

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Dave Marek, Myovant CEO

My­ovant board balks as ma­jor­i­ty own­er Sum­it­o­mo swoops in with a $2.5B deal to buy them out

Three years after Sumitomo scooped up Roivant’s 46% stake in the publicly traded Myovant $MYOV as part of a 5-company, $3 billion deal, they’re coming back for the whole thing.

But these other investors at Myovant want more than what the Japanese pharma company is currently offering to pay at this stage.

Sumitomo is bidding $22.75 a share for the outstanding stock, which now represents 48% of the company after Sumitomo bumped its ownership since the original deal with Roivant. Myovant, however, created a special committee on the board, and they’re shaking their heads over the offer.

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Albert Bourla, Pfizer CEO (Gian Ehrenzeller/Keystone via AP)

Can a smart­phone app de­tect Covid? Pfiz­er throws down $116M to find out

What can a cough say about a patient’s illness? Quite a bit, according to ResApp Health — and Pfizer’s listening.

The pharma giant is shelling out about $116 million ($179 million AUD) to scoop up the University of Queensland spinout and its smartphone technology that promises to diagnose Covid and other respiratory illnesses based on cough and breathing sounds, the university announced last week.

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