Was PTC Ther­a­peu­tics play­ing games when it tried to defuse the de­flaza­cort con­tro­ver­sy with a $35,000 an­nu­al price?

PTC Ther­a­peu­tics CEO Stu­art Peltz

When PTC Ther­a­peu­tics $PTCT CEO Stu­art Peltz rolled out the biotech’s Q1 re­port this morn­ing, he not­ed that com­pa­ny ex­ecs came up with what they be­lieve is a “sus­tain­able” price for the cheap, old steroid ac­quired from the con­tro­ver­sial crew at Marathon Phar­ma­ceu­ti­cals for $140 mil­lion up front and tar­get­ed at the Duchenne com­mu­ni­ty in the US.

The net price, he said, would av­er­age $35,000 a year for a pa­tient that weighed 25 kilo­grams, or 55 pounds.

“We be­lieve this rep­re­sents sus­tain­able pric­ing which bal­ances pro­vid­ing ac­cess to all el­i­gi­ble pa­tients in the US in an ul­tra or­phan pop­u­la­tion while main­tain­ing suf­fi­cient in­fra­struc­ture and pro­grams, in­clud­ing con­tin­ued in­vest­ment in Duchenne,” Peltz said.

That $35,000 net — the price pay­ers could ex­pect af­ter dis­counts — sound­ed like quite a drop from the $89,000 an­nu­al price point that trig­gered a storm of protest for Marathon, un­til you start to do the math on what these pa­tients ac­tu­al­ly weigh.

Matthew Eck­ler, RBC

RBC’s Matthew Eck­ler did the math, and came up with a much high­er num­ber. If you con­sid­er that most of the boys are in the 35- to 40-ki­lo range, he says, you’re re­al­ly talk­ing about an av­er­age range more like $45,000 to $50,000 a year.

That’s 45 to 50 times more than the $1,000 av­er­age price Duchenne par­ents have been pay­ing to bring in de­flaza­cort, a gener­ic out­side the US which has been avail­able for decades at a low price. But now that it’s ap­proved and avail­able in the US, those for­eign sup­pli­ers are be­ing closed to Amer­i­can fam­i­lies. And PTC’s “net” price may ac­tu­al­ly wind up high­er than the $54,000 price Marathon field­ed, but failed to sell.

Ac­cord­ing to com­mu­ni­ty ac­tivist Chris­tine Mc­Sh­er­ry of the non­prof­it Jett Foun­da­tion, many of the old­er pa­tients like her son now weigh 60 to 100 ki­los, and at that range the price — pro­vid­ed PTC sticks to the weight/cost ra­tio — par­ents would ac­tu­al­ly be forced to buy the steroid with a net price high­er than Marathon’s $89,000 tag.

So, I asked Eck­ler, for boys weigh­ing 75 ki­los this would mean a price of $105,000?

Eck­ler replied:

Based on what they have told us yes, but that as­sumes the cost per mg is the same for all size boys.

It’s pos­si­ble that they could work out agree­ments with pay­ors where the cost per mg is not sta­t­ic based on the weight of boys. At this point (we) will have to wait and see. Re­gard­less, there will be boys who are much heav­ier than the 25mg guide­line and thus (their) cost is go­ing to be more.

PTC did not ex­plain what it’s whole­sale ac­qui­si­tion cost, or WAC, was or how the weight/price for­mu­la would work. It’s ex­treme­ly un­usu­al for com­pa­nies to avoid the high WAC price tag en­tire­ly. Most be­gin with that and then ne­go­ti­ate pri­vate­ly with pay­ers.

Even at $35,000, an­a­lysts like Eck­ler ex­pect the head­lines to con­tin­ue to run against PTC, now that the com­pa­ny has picked up the price goug­ing ti­tle that Marathon has been try­ing so hard to shed. And that could play hav­oc with PTC’s plans to start work­ing with the US Duchenne com­mu­ni­ty as it tries to force the FDA to re­verse its po­si­tion on ataluren — a drug that failed its last three stud­ies in a row — and ap­prove it for US mar­ket­ing. The drug is avail­able in Eu­rope.

Notes Eck­ler:

Drug pric­ing has re­mained in the head­lines as a non-par­ti­san po­lit­i­cal ral­ly­ing cry, and we don’t an­tic­i­pate this will change in the near term. Ul­ti­mate­ly, we see this as­set as neg­a­tive on an NPV ba­sis giv­en: 1) the sig­nif­i­cant up­front in­vest­ment ($140M vs. 2017 sales guid­ance of $5-$10M) for a drug which we view as hav­ing ~120M in peak sales po­ten­tial; 2) the avail­abil­i­ty of oth­er cor­ti­cos­teroids for DMD pa­tients; and 3) the like­li­hood of con­tin­ued pric­ing con­tro­ver­sy and pay­or push­back re­sult­ing in a tem­pered com­mer­cial launch.

I asked an­oth­er an­gry Duchenne par­ent what she planned to do now that PTC has post­ed a price.

“Still go­ing to Mex­i­co,” she replied, re­fer­ring to the low-cost phar­ma­cies that sell drugs cheap along the bor­der.

One big stum­bling block that PTC faces is gener­ic pred­nisone, which is shap­ing up as the pre­ferred steroid among pay­ers — for some ob­vi­ous rea­sons. I asked Ex­press Scripts about their ap­proach to de­flaza­cort (brand name Em­flaza), and they replied:

We are re­quir­ing a pri­or au­tho­riza­tion for this med­ica­tion. For the man­age­ment of DMD, our cri­te­ria re­quires pa­tients to try the low­er cost, equal­ly ef­fec­tive pred­nisone be­fore Em­flaza. Ex­cept for rare cas­es, the low­er cost pred­nisone should work for pa­tients. The price of pred­nisone is any­where from $0.08 per tablet to $1.50 per tablet.

The neg­a­tive sen­ti­ment isn’t sit­ting too well with the in­vest­ment com­mu­ni­ty, which has dri­ven PTC’s shares down by 10% so far to­day.

BY­OD Best Prac­tices: How Mo­bile De­vice Strat­e­gy Leads to More Pa­tient-Cen­tric Clin­i­cal Tri­als

Some of the most time- and cost-consuming components of clinical research center on gathering, analyzing, and reporting data. To improve efficiency, many clinical trial sponsors have shifted to electronic clinical outcome assessments (eCOA), including electronic patient-reported outcome (ePRO) tools.

In most cases, patients enter data using apps installed on provisioned devices. At a time when 81% of Americans own a smartphone, why not use the device they rely on every day?

Near­ly a year af­ter Au­den­tes' gene ther­a­py deaths, the tri­al con­tin­ues. What hap­pened re­mains a mys­tery

Natalie Holles was five months into her tenure as Audentes CEO and working to smooth out a $3 billion merger when the world crashed in.

Holles and her team received word on the morning of May 5 that, hours before, a patient died in a trial for their lead gene therapy. They went into triage mode, alerting the FDA, calling trial investigators to begin to understand what happened, and, the next day, writing a letter to alert the patient community so they would be the first to know. “We wanted to be as forthright and transparent as possible,” Holles told me late last month.

The brief letter noted two other patients also suffered severe reactions after receiving a high dose of the therapy and were undergoing treatment. One died a month and a half later, at which point news of the deaths became public, jolting an emergent gene therapy field and raising questions about the safety of the high doses Audentes and others were now using. The third patient died in August.

“It was deeply saddening,” Holles said. “But I was — we were — resolute and determined to understand what happened and learn from it and get back on track.”

Eleven months have now passed since the first death and the therapy, a potential cure for a rare and fatal muscle-wasting disease called X-linked myotubular myopathy, is back on track, the FDA having cleared the company to resume dosing at a lower level. Audentes itself is no more; last month, Japanese pharma giant Astellas announced it had completed working out the kinks of the $3 billion merger and had restructured and rebranded the subsidiary as Astellas Gene Therapies. Holles, having successfully steered both efforts, departed.

Still, questions about precisely what led to the deaths of the 3 boys still linger. Trial investigators released key details about the case last August and December, pointing to a biological landmine that Audentes could not have seen coming — a moment of profound medical misfortune. In an emerging field that’s promised cures for devastating diseases but also seen its share of safety setbacks, the cases provided a cautionary tale.

Audentes “contributed in a positive way by giving a painful but important example for others to look at and learn from,” Terry Flotte, dean of the UMass School of Medicine and editor of the journal Human Gene Therapy, told me. “I can’t see anything they did wrong.”

Yet some researchers say they’re still waiting on Astellas to release more data. The company has yet to publish a full paper detailing what happened, nor have they indicated that they will. In the meantime, it remains unclear what triggered the events and how to prevent them in the future.

“Since Audentes was the first one and we don’t have additional information, we’re kind of in a holding pattern, flying around, waiting to figure out how to land our vehicles,” said Jude Samulski, professor of pharmacology at UNC’s Gene Therapy Center and CSO of the gene therapy biotech AskBio, now a subsidiary of Bayer.

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Pascal Soriot (AstraZeneca via YouTube)

Af­ter be­ing goad­ed to sell the com­pa­ny, Alex­ion's CEO set some am­bi­tious new goals for in­vestors. Then Pas­cal So­ri­ot came call­ing

Back in the spring of 2020, Alexion $ALXN CEO Ludwig Hantson was under considerable pressure to perform and had been for months. Elliott Advisers had been applying some high public heat on the biotech’s numbers. And in reaching out to some major stockholders, one thread of advice came through loud and clear: Sell the company or do something dramatic to change the narrative.

In the words of the rather dry SEC filing that offers a detailed backgrounder on the buyout deal, Alexion stated: ‘During the summer and fall of 2020, Alexion also continued to engage with its stockholders, and in these interactions, several stockholders encouraged the company to explore strategic alternatives.’

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Anand Shah (FDA)

For­mer head of FDA’s med­ical and sci­en­tif­ic af­fairs on Covid: ‘FDA has nev­er been test­ed like this’

Anand Shah has served the American public in a unique way, crisscrossing over the last two administrations between serving as an attending radiation oncologist focused on prostate cancer at NIH, serving as CMO at the Center for Medicare and Medicaid Innovation, and most recently, leading the FDA’s operations on medical and scientific affairs from within the commissioner’s office.

Shah, who stepped down from the FDA in January, caught up with Endpoints News in a phone interview on Tuesday afternoon, offering his thoughts on the agency’s latest decision to pause the J&J vaccinations in the US, and reflecting on his time at an agency during this once-in-a-lifetime pandemic.

Launched by MIT grads, a small start­up gets $20M to back a ro­bot­ics rev­o­lu­tion in cell ther­a­py man­u­fac­tur­ing

As co-director of an experimental cellular therapy process development and manufacturing group at UCSF specializing in T cell therapies for autoimmune conditions, Jonathan Esensten has learned a lot about the challenges involved when his group hand-fashions a cell therapy. Esensten — who was a postdoc in Wendell Lim’s lab and counts the legendary Jeffrey Bluestone as a mentor — gives them all high marks at being great at what they do, but time and again there are variations in the treatments they construct.

Barbara Weber, Tango Therapeutics CEO (Tango)

It takes two to Tan­go: The biotech us­ing CRISPR to dis­cov­er new can­cer gene tar­gets rides a $353M SPAC deal to Nas­daq

Editor’s note: Interested in following biopharma’s fast-paced IPO market? You can bookmark our IPO Tracker here.

The latest biotech-SPAC deal has arrived, and it’s dancing its way to Nasdaq to the tune of several hundred million dollars.

Tango Therapeutics and its CRISPR-focused search for new cancer genes is reverse merging with Boxer Capital’s blank-check company, the biotech announced Wednesday morning. With a spotlight on three lead programs, Tango expects total proceeds to equal about $353 million in the deal, which includes the roughly $167 million held in the SPAC and an additional $186 million in PIPE financing.

Kristin Fortney, BioAge Labs CEO

An­ti-ag­ing biotech up­start plucks a drug from Am­gen's dis­card pile, piv­ot­ing from heart fail­ure to mus­cle con­di­tions

Back in April 2019, Amgen quietly shut down a Phase I trial for a drug named AMG 986. There was no safety concern; the molecule just didn’t hit the mark on helping the small band of heart failure patients who received it.

A small biotech, though, believes it would stand a chance in the burgeoning anti-aging field.

BioAge Labs has licensed AMG 986 — now renamed BGE-105 — with plans to parlay the existing IND into a quick Phase I trial teasing out the pharmacodynamic effects and set the stage for mid-stage tests focused on acute muscle indications.

UP­DAT­ED: J&J paus­es vac­cine roll­out as feds probe rare cas­es of blood clots

The FDA and CDC have jointly decided to stop administering J&J’s Covid-19 vaccine after reviewing data involving six reported US cases of a rare and severe type of blood clot in individuals after receiving the vaccine.

CDC will convene a meeting of its Advisory Committee on Immunization Practices on Wednesday to further review these cases and assess their potential significance. “FDA will review that analysis as it also investigates these cases. Until that process is complete, we are recommending a pause in the use of this vaccine out of an abundance of caution,” Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research and Anne Schuchat, Principal Deputy Director of the CDC, said in a joint statement Tuesday morning.

Patrizia Cavazzoni, new CDER director

Pa­trizia Cavaz­zoni named per­ma­nent di­rec­tor of CDER, adding to ques­tions around where Wood­cock will end up

Patrizia Cavazzoni on Monday became the permanent director of the FDA’s Center for Drug Evaluation and Research, which puts to rest the idea that Janet Woodcock, Cavazzoni’s predecessor, might return to lead CDER if she isn’t made permanent commissioner.

Woodcock, who’s currently serving as acting commissioner and principal medical advisor to the commissioner, a position she was detailed to last year, may not make the move to permanent commissioner because of lingering questions from Senate Democrats. She previously served as director of CDER since 1994. Cavazzoni took over as acting director of CDER when Woodcock moved over to Operation Warp Speed to run the therapeutics side of the Trump-era program.

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