FDA ex­e­cutes a 180 on Ther­a­peu­tic­sMD ther­a­py, hand­ing it an OK — and stok­ing con­cerns about po­lit­i­cal in­flu­ence

A year af­ter the FDA hand­ed Ther­a­peu­tic­sMD $TXMD a re­jec­tion let­ter for its ther­a­py pre­vent­ing vagi­nal pain dur­ing sex, the agency has done a com­plete 180 — ap­prov­ing the treat­ment af­ter wav­ing off the safe­ty con­cerns that had once war­rant­ed a CRL.

The de­ci­sion to drop the CRL and al­low the ap­pli­ca­tion to pro­ceed with­out meet­ing spec­i­fied hur­dles is one of three the FDA ex­e­cut­ed in the weeks af­ter Scott Got­tlieb jumped in­to the top job at the FDA, rais­ing con­cerns that po­lit­i­cal in­flu­ence was chang­ing the for­tunes of some of the com­pa­nies with busi­ness be­fore the agency. In this case, Got­tlieb — hand­ed a man­date to speed ap­provals — won the nom­i­na­tion the day af­ter the re­jec­tion was an­nounced by Ther­a­peu­tic­sMD.

Scott Got­tlieb

Two oth­er com­pa­nies al­so earned a re­prieve un­der Got­tlieb: Am­i­cus, where CEO John Crow­ley had lob­bied of­fi­cials claim­ing that their re­jec­tion of its ex­per­i­men­tal Fab­ry drug mi­gala­s­tat would de­lay it by 5 to 7 years, far more than the 2 years he told share­hold­ers; and Eli Lil­ly, which re­cent­ly won over an ex­pert pan­el on a ques­tion­able low dose of baric­i­tinib. 

In Am­i­cus’ case, the FDA put the drug on the agency’s in­side track, of­fer­ing a fast pri­or­i­ty re­view ahead of an ex­pect­ed ap­proval.

In this case, Ther­a­peu­tic­sMD — which is chaired by ex-HHS sec­re­tary Tom­my Thomp­son, who ran a group of promi­nent Bush ad­min­is­tra­tion Re­pub­li­cans back­ing Don­ald Trump for pres­i­dent — told in­vestors last year that the agency had flagged con­cerns about the ab­sence of safe­ty da­ta be­yond the 12-week stretch test­ed in the clin­ic. It al­so didn’t help ini­tial­ly that there are oth­er prod­ucts for the con­di­tion, less­en­ing the ur­gency of an ap­proval.

The agency doesn’t pub­lish CRLs and nev­er of­fered an ex­pla­na­tion for its re­ver­sal, an agency stan­dard on in­di­vid­ual com­pa­nies. 

Their estra­di­ol vagi­nal in­serts will be sold as Imvexxy.

Tar­get­ing a Po­ten­tial Vul­ner­a­bil­i­ty of Cer­tain Can­cers with DNA Dam­age Re­sponse

Every individual’s DNA is unique, and because of this, every patient responds differently to disease and treatment. It is astonishing how four tiny building blocks of our DNA – A, T, C, G – dictate our health, disease, and how we age.

The tricky thing about DNA is that it is constantly exposed to damage by sources such as ultraviolet light, certain chemicals, toxins, and even natural biochemical processes inside our cells.¹ If ignored, DNA damage will accumulate in replicating cells, giving rise to mutations that can lead to premature aging, cancer, and other diseases.

Roivant par­lays a $450M chunk of eq­ui­ty in biotech buy­out, grab­bing a com­pu­ta­tion­al group to dri­ve dis­cov­ery work

New Roivant CEO Matt Gline has crafted an all-equity upfront deal to buy out a Boston-based biotech that has been toiling for several years now at building a supercomputing-based computational platform to design new drugs. And he’s adding it to the Erector set of science operations that are being built up to support their network of biotech subsidiaries with an eye to growing the pipeline in a play to create a new kind of pharma company.

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

UP­DAT­ED: Mer­ck takes a swing at the IL-2 puz­zle­box with a $1.85B play for buzzy Pan­dion and its au­toim­mune hope­fuls

When Roger Perlmutter bid farewell to Merck late last year, the drugmaker perhaps best known now for sales giant Keytruda signaled its intent to take a swing at early-stage novelty with the appointment of discovery head Dean Li. Now, Merck is signing a decent-sized check to bring an IL-2 moonshot into the fold.

Merck will shell out roughly $1.85 billion for Pandion Pharmaceuticals, a biotech hoping to gin up regulatory T cells (Tregs) to treat a range of autoimmune disorders, the drugmaker said Thursday.

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Fol­low biotechs go­ing pub­lic with the End­points News IPO Track­er

The Endpoints News team is continuing to track IPO filings for 2021, and we’ve designed a new tracker page for the effort.

Check it out here: Biopharma IPOs 2021 from Endpoints News

You’ll be able to find all the biotechs that have filed and priced so far this year, sortable by quarter and listed by newest first. As of the time of publishing on Feb. 25, there have already been 16 biotechs debuting on Nasdaq so far this year, with an additional four having filed their S-1 paperwork.

Doug Ingram (file photo)

Why not? Sarep­ta’s third Duchenne MD drug sails to ac­cel­er­at­ed ap­proval

Sarepta may be running into some trouble with its next-gen gene therapy approach to Duchenne muscular dystrophy. But when it comes to antisense oligonucleotides, the well-trodden regulatory path is still leading straight to an accelerated approval for casimersen, now christened Amondys 45.

We just have to wait until 2024 to find out if it works.

Amondys 45’s approval was unceremonious, compared to its two older siblings. There was no controversy within the FDA over approving a drug based on a biomarker rather than clinical benefit, setting up a powerful precedent that still haunts acting FDA commissioner Janet Woodcock as biotech insiders weighed her potential permanent appointment; no drama like the FDA issuing a stunning rejection only to reverse its decision and hand out an OK four months later, which got more complicated after the scathing complete response letter was published; no anxious tea leaf reading or heated arguments from drug developers and patient advocates who were tired of having corticosteroids as their loved ones’ only (sometimes expensive) option.

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With dust set­tled on ac­tivist at­tack, Lau­rence Coop­er leaves Zio­pharm to a new board

Laurence Cooper has done his part.

In the five years since he left a tenured position at Houston’s MD Anderson Cancer Center to become CEO of Boston-based Ziopharm, he’s steered the small-cap immunotherapy player through patient deaths in trials, clinical holds, short attacks and, most recently, an activist attack on the board.

So when the company has “fantastic news” like an IND clearance for a TCR T cell therapy program, he’s ready to pass on the baton.

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S&P ex­pects steady ero­sion in Big Phar­ma's cred­it pro­file in 2021 as new M&A deals roll in — but don't un­der­es­ti­mate their un­der­ly­ing strength

S&P Global has taken a look at the dominant forces shaping the pharma market and come to the conclusion that there will be more downgrades than upgrades in 2021 — the 8th straight year of steady decline.

But it’s not all bad news. Some things are looking up, and there’s still plenty of money to be made in an industry that enjoys a 30% to 40% profit margin, once you factor in steep R&D expenses.

Steve Cutler, Icon CEO (Icon)

In the biggest CRO takeover in years, Icon doles out $12B for PRA Health Sci­ences to fo­cus on de­cen­tral­ized clin­i­cal work

Contract research M&A had a healthy run in recent years before recently petering out. But with the market ripe for a big buyout and the Covid-19 pandemic emphasizing the importance of decentralized trials, Wednesday saw a tectonic shift in the CRO world.

Icon, the Dublin-based CRO, will acquire PRA Health Sciences for $12 billion in a move that will shake up the highest rungs of a fragmented market. The merger would combine the 5th- and 6th-largest CROs by 2020 revenue, according to Icon, and the merger will set the newco up to be the second-largest global CRO behind only IQVIA.

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J&J ad­comm live blog: J&J work­ing on vac­cine for vari­ants, tri­al sched­uled for sum­mer

This week, Moderna announced it would begin testing a modified version of their mRNA vaccine to tackle the vaccine-resistant B1.351 variant that popped up in South Africa. Pfizer said it would test giving people an extra boost of its original vaccine to accomplish the same.

J&J revealed at the adcomm that they, too, have been working on a modified vaccine to tackle emerging variants.

The company didn’t reveal much detail, including how they modified the vaccine or if they were targeting the same B 1.351 variant, but Johan van Hoof said they would begin testing a new construct in the summer. The FDA has said they would allow modified vaccines for variants to be authorized after quick immunological studies that track whether the vaccine elicits antibodies against the new variant.