In pre­emp­tive strike, Sanders, Cum­mings de­mand PTC chief Peltz spell out his price for con­tro­ver­sial Duchenne MD treat­ment

It’s time for Stu­art Peltz to take the Con­gres­sion­al hot seat on drug pric­ing.

PTC CEO Stu­art Peltz

The CEO of PTC Ther­a­peu­tics ac­quired Marathon’s steroid de­flaza­cort for $140 mil­lion up front a few days ago with an eye to sell it to a small pop­u­la­tion of Duchenne mus­cu­lar dy­s­tro­phy pa­tients. And that comes with as much of a glar­ing spot­light as Sen­a­tor Bernie Sanders and Rep. Eli­jah Cum­mings can bring to it.

In a new let­ter di­rect­ed to Peltz, the two ac­tivist law­mak­ers not­ed in a pre­emp­tive strike that the CEO has been in touch with the Par­ent Pro­ject for Mus­cu­lar Dy­s­tro­phy to talk price. And they have a re­tail num­ber in mind that PTC $PTCT won’t like­ly cot­ton to: The UK net price of $1,000 to $1,200 a month, which is what many par­ents are pay­ing to­day.

Rep. Eli­jah Cum­mings

“We urge you to keep the price of this rel­a­tive­ly com­mon steroid at its cur­rent im­por­ta­tion cost,” the two law­mak­ers note. They al­so are ask­ing what Peltz’s plans are in pur­su­ing an­oth­er or­phan ap­proval for ju­ve­nile arthri­tis.

Peltz wasn’t ready to tell an­a­lysts what he thought PTC’s price would be when he an­nounced the deal to ac­quire the con­tro­ver­sial steroid. That was a sub­ject that he said would be re­vis­it­ed in light of the con­tro­ver­sy over the $89,000 price. It’s un­like­ly, though, that he was think­ing of a range stretch­ing from $12,000 to $14,400 a year.

Peltz is quite fa­mil­iar with con­tro­ver­sy, though. He man­aged to per­suade the Eu­ro­peans to ap­prove ataluren for Duchenne mus­cu­lar dy­s­tro­phy, even though that ther­a­py has now failed three straight stud­ies, in­clud­ing two for DMD. He was barred at the door by the FDA, but used agency’s reg­u­la­tions to force a re­view and PDU­FA date for the drug.

Marathon and its drug be­came vir­tu­al­ly ra­dioac­tive af­ter it priced de­flaza­cort at $89,000 af­ter gain­ing a nar­row FDA ap­proval as an or­phan ther­a­py, spawn­ing a fresh wave of out­rage over price goug­ing that left PhRMA re­view­ing its mem­ber­ship rules and whether Marathon CEO’s Jeff Aronin should be oust­ed from the board. Sanders and Cum­mings al­so fol­lowed up re­cent­ly by ask­ing the FDA why it han­dled a cheap, old steroid as an or­phan drug, in a pro­gram that will now be re­viewed by the GAO.

Aronin no­to­ri­ous­ly pledged to Duchenne fam­i­lies that his com­pa­ny had done the “heavy lift­ing” on de­flaza­cort with a de­vel­op­ment pro­gram that re­quired 17 tri­als. But it turned out the com­pa­ny bought the ef­fi­ca­cy da­ta it need­ed for on­ly a low six fig­ures, ac­cord­ing to the Wall Street Jour­nal, and much of its “heavy lift­ing” in­clud­ed small pre­clin­i­cal and clin­i­cal stud­ies that like­ly were done on the cheap.

Now Peltz can try to find out if you can take a cheap old steroid that’s been avail­able for years from over­seas sources at a mar­gin­al cost and reprice it for a US mar­ket with­out com­ing un­der a full as­sault from out­raged law­mak­ers. That’s no easy task.

On the oth­er hand, Mar­tin Shkre­li prac­ti­cal­ly in­vent­ed the sim­mer­ing con­tro­ver­sy over price goug­ing with his de­ci­sion to up the price of an old gener­ic more than 5000%. Shkre­li re­signed from his post at Tur­ing — and is now fac­ing un­re­lat­ed fraud charges — but the biotech nev­er dis­count­ed the price, de­spite Con­gres­sion­al hear­ings, an­gry law­mak­ers and an on­line mob that shrieked for ret­ri­bu­tion. There are no laws re­strict­ing drug prices, as Peltz is well aware of.

To be con­tin­ued.

UP­DAT­ED: Mer­ck pulls Keytru­da in SCLC af­ter ac­cel­er­at­ed nod. Is the FDA get­ting tough on drug­mak­ers that don't hit their marks?

In what could be an early shot in the battle against drugmakers that whiff on confirmatory studies to support accelerated approvals, the FDA ordered Bristol Myers Squibb late last year to give up Opdivo’s approval in SCLC. Now, Merck is next on the firing line — are we seeing the FDA buckling down on post-marketing offenders?

Merck has withdrawn its marketing approval for PD-(L)1 inhibitor Keytruda in metastatic small cell lung cancer as part of what it describes as an “industry-wide evaluation” by the FDA of drugs that do not meet the post-marketing checkpoints on which their accelerated nods were based, the company said Monday.

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The 2021 top 100 bio­phar­ma in­vestors: As the pan­dem­ic hit and IPOs boomed, VCs swung in­to ac­tion like nev­er be­fore

The global pandemic may have roiled economies, killed hundreds of thousands and throttled entire industries, but the only effect it had on biopharma venture investing was to help turbocharge the field to giddy new heights.

Below you’ll find the new top 100 venture investors in the industry, ranked by the number of deals they were publicly involved in, as tracked by DealForma chief Chris Dokomajilar. The numbers master then calculated the estimated amount of money they put into each deal — divvying up the cash by the number of players — to indicate how they managed their syndicates.

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Hal Barron, Endpoints UKBIO19

GSK, Vir's hopes for a Covid-19 an­ti­body fall flat in NIH 'mas­ter pro­to­col' with no ben­e­fit in hos­pi­tal­ized pa­tients

GlaxoSmithKline and Vir Biotechnology were hopeful that one of their partnered antibodies would carve out a win after getting the invite to a major NIH study in hospitalized Covid-19 patients. But just like Eli Lilly, the pair’s drug couldn’t hit the mark, and now they’ll be left to take a hard look at the game plan.

The NIH has shut down enrollment for GSK and Vir’s antibody VIR-7831 in its late-stage ACTIV-3 trial after the drug showed negligible effect in achieving sustained recovery in hospitalized Covid-19 patients, the partners said Wednesday.

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As Brain­Storm con­tin­ues to tout ‘clear sig­nal’ on ALS drug, the FDA of­fers a rare pub­lic slap­down on the da­ta

A little more than a week after BrainStorm acknowledged that regulators at the FDA had informed them that the biotech needed more data before it could expect to gain an approval for its ALS treatment NurOwn — while still touting a “clear signal” of efficacy and not ruling out an application — the agency has decided to clarify the record in a most unusual statement.

The FDA statement amounts to a straight slap own, offering a different set of efficacy numbers from the company’s public presentation last November and ruling out any chance of statistical significance.

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In­tro­duc­ing End­points FDA+, our new pre­mi­um week­ly reg­u­la­to­ry news re­port led by Zachary Bren­nan

CRLs. 483s. CBER, CDER and RWE. For biopharma professionals, these acronyms command attention because of the fundamental role FDA plays in drug development. Now Endpoints is doubling down on regulatory coverage, and launching a weekly report focusing on developments out of White Oak, with analysis and insight into what it all means.

Coverage will be led by our new senior editor, Zachary Brennan. He joins Endpoints from POLITICO, where he covered pharma. Prior to that he was the managing editor for Regulatory Focus, a news publication from the Regulatory Affairs Professionals Society.

Antoine Papiernik, Sofinnova managing director (Business Wire)

Sofinno­va Part­ners stays fo­cused on late-stage deals with a new, $540M crossover fund

One of Europe’s most high-profile biopharma investors is getting $540 million to invest in new crossover deals for late-stage companies.

The Paris-based VC says the fresh Sofinnova Crossover Fund raise positions them as the “largest crossover investor in Europe dedicated to late-stage biopharma and medtech investments.”

They got a leg up in France after winning a special “Tibi” designation from the French government, giving them access to a pool of €6 billion that helped them gain an edge with institutional investors. Since they were founded close to 50 years ago, the venture group has backed more than 500 companies and currently has more than €2 billion under management.

Eli Lil­ly claims suc­cess in a new JAK in­di­ca­tion: hair loss

Over the last decade, drugmakers have proven JAK inhibitors can treat a smattering of immune-related diseases ranging from rheumatoid arthritis to Covid-19. Now Eli Lilly has pulled out a new one.

Lilly and its biotech partner Incyte announced Wednesday that their JAK inhibitor baricitinib effectively regrew patients’ hair in a Phase III trial for alopecia areata, an autoimmune condition that can cause sudden, severe and patchy hair loss. Lilly didn’t break down the results from the 546-patient trial, but the primary endpoint was improvement on a standard score for alopecia symptoms.

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Thank you, next: Take­da hands Ovid $196M cash to rein back in Phase III-ready seizure drug, re­viv­ing bat­tered stock

Soticlestat made it.

Takeda is bringing the drug back into its fold more than four years after first entrusting the team at Ovid with the mid-stage clinical work. For all that — generating what they saw as positive Phase II data in Dravet syndrome and Lennox-Gastaut syndrome — the biotech has been rewarded with $196 million in upfront cash, with another $660 million reserved for regulatory and commercial milestones.

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Presage teams with Mer­ck on its Phase 0 test­ing; Kem­Pharm AD­HD drug wins ap­proval in chil­dren aged 6 and up

Seattle-based Presage Biosciences, which approaches drug development through its microdosing platform, has some new partnerships and cash to come with them.

Presage closed a $13 million financing round Tuesday, aiming to expand its network of clinical trial sites and advance development of its microdosing injection devices. They also closed partnership deals with Merck and Maverick Therapeutics.

The financing included $7 million from new investors, including the LabCorp Venture Fund, Bristol Myers Squibb, and InHarv Partners. An additional $6 million convertible note from Takeda Ventures will convert to equity.