Pro­teosta­sis stock soars on cys­tic fi­bro­sis break­through sta­tus

Shares are sky­rock­et­ing at Pro­teosta­sis $PTI, a small biotech in Cam­bridge, MA, fol­low­ing news that reg­u­la­tors hand­ed out break­through sta­tus to the com­pa­ny’s cys­tic fi­bro­sis ther­a­py.

The good news fol­lows pos­i­tive da­ta an­nounced last year for the com­pa­ny’s first-in-class CFTR am­pli­fi­er, and a $50 mil­lion IPO just be­fore that.

Meenu Chhabra

The drug, called PTI-428, is meant to boost lev­els of the CFTR pro­tein, which is lack­ing in cys­tic fi­bro­sis pa­tients. The ther­a­py is an add-on treat­ment for pa­tients with the F508del mu­ta­tion in the CFTR gene who are al­ready tak­ing an ap­proved CFTR mod­u­la­tor or as part of Pro­teosta­sis’ triple com­bo reg­i­men that in­cludes PTI-808, a po­ten­tia­tor, and PTI-801, a cor­rec­tor.

This new des­ig­na­tion was giv­en out based on da­ta from a re­cent Phase II study in­clud­ing 24 CF pa­tients with the F508del mu­ta­tion tak­ing Ver­tex’s drug Orkam­bi.

“We be­lieve the break­through ther­a­py des­ig­na­tion for PTI-428 re­flects the strength of the re­cent Phase II study re­sults for our am­pli­fi­er, a nov­el and pro­pri­etary class of CFTR mod­u­la­tors,” said Meenu Chhabra, pres­i­dent and CEO of Pro­teosta­sis, in a state­ment.  “PTI-428 can po­ten­tial­ly be added to cur­rent and fu­ture stan­dards of care, of­fer­ing the po­ten­tial for im­prove­ment in pul­monary func­tion for pa­tients with cys­tic fi­bro­sis.”

An­a­lysts at Baird Eq­ui­ty Re­search were pos­i­tive on the news, but ac­knowl­edged in­vestors’ skep­ti­cism that a break­through sta­tus could be mean­ing­ful to stock per­for­mance.

Bri­an Sko­r­ney

“We ac­knowl­edge that break­through des­ig­na­tion has turned out to be much less of a val­i­da­tion of a drug’s ben­e­fit than many hoped for when it start­ed in 2012,” wrote an­a­lyst Bri­an Sko­r­ney in a re­search note this morn­ing. “How­ev­er, for a ~$100 mil­lion mar­ket cap com­pa­ny where many in­vestors just don’t be­lieve the da­ta, we think it like­ly pro­vides a ma­te­ri­al­ly high­er de­gree of con­fi­dence in the re­sults of the Phase II study, with the un­der­stand­ing that the FDA doesn’t put a ton of dili­gence in­to ver­i­fy­ing the strength of re­sults that are the ba­sis of break­through ther­a­py des­ig­na­tion. While not an ex­haus­tive list, a skim in­di­cates that most of the drugs grant­ed break­through des­ig­na­tion do seem to have mean­ing­ful clin­i­cal im­pact.”

Pro­teosta­sis’ stock is up 52% in pre-mar­ket trad­ing, go­ing for $6.29 per share at time of pub­lish­ing.

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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Bob Nelsen (Photo by Michael Kovac/Getty Images)

With stars aligned and cash in re­serve, Bob Nelsen's Re­silience plans a makeover at 2 new fa­cil­i­ty ad­di­tions to its drug man­u­fac­tur­ing up­start

Bob Nelsen’s new, state-of-the-art drug manufacturing initiative is taking shape.

Just 3 months after gathering $800 million of launch money, a dream team board and a plan to shake up a field where he found too many bottlenecks and inefficiencies for the era of Covid-19, Resilience has snapped up a pair of facilities now in line for a retooling.

The company has acquired a 310,000-square-foot plant in Boston from Sanofi along with a 136,000-square-foot plant in Ontario to add to a network which CEO Rahul Singhvi says is just getting started on building his company’s operations up. The Sanofi deal comes with a contract to continue manufacturing one of its drugs.

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Pascal Soriot, AstraZeneca CEO (AP Images)

Pas­cal So­ri­ot cash­es in As­traZeneca’s chips on Mod­er­na for $1.2B cash in­jec­tion

While still working to prove its own Covid-19 vaccine, AstraZeneca has reportedly capitalized on the success of another.

The company has sold off its 7.7% stake in Moderna and turned it into $1.2 billion in cash, according to the Times, beefing up the reserves just as Pascal Soriot is wrapping up his $39 billion acquisition of Alexion and its rare disease pipeline.

AstraZeneca’s stock sale follows a similar move by Merck in December. But like its pharma brethren, the British giant is keeping its R&D collaborations with Moderna.

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Af­ter bail­ing on Covid-19 vac­cines, Mer­ck will team up with J&J to pro­duce its shot as part of un­usu­al Big Phar­ma pact

Merck took a big gamble when it opted to jump into the Covid-19 vaccine race late, and made an equally momentous decision to back out in late January. Now, looking to chip in on the effort, Merck reportedly agreed to team up with one of the companies that has already crossed the finish line.

President Joe Biden on Tuesday is expected to announce a partnership between drugmakers Merck and Johnson & Johnson to jointly produce J&J’s recombinant protein Covid-19 vaccine that received the FDA’s emergency use authorization Saturday, the Washington Post reported.

Ab­b­Vie tees up a biotech buy­out af­ter siz­ing up their Parkin­son's drug spun out of Ke­van Shokat's lab

AbbVie has teed up a small but intriguing biotech buyout after looking over the preclinical work it’s been doing in Parkinson’s disease.

The company is called Mitokinin, a Bay Area biotech spun out of the lab of UCSF’s Kevan Shokat, whose scientific explorations have formed the academic basis of a slew of startups in the biotech hub. One of Shokat’s PhD students in the lab, Nicholas Hertz, co-founded Mitokinin using their lab work on PINK1 suggesting that amping up its activity could play an important role in regulating the mitochondrial dysfunction contributing to Parkinson’s disease pathogenesis and progression.

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Paul Sekhri

The next big biotech su­per­star? Paul Sekhri has some thoughts on that

It occasionally occurs to Paul Sekhri that if they pull this off, his company will be on the front page of the New York Times and a lead story in just about every major news outlet on the planet. He tries not to dwell on it, though.

“I just want to be laser-focused on getting to that point,” Sekhri says, before acknowledging, “Yes, it absolutely crossed my mind.”

Sekhri, a longtime biopharma executive with tenures at Sanofi and Novartis, is now entering year three as CEO of eGenesis, the biotech that George Church protégé Luhan Yang founded to genetically alter pigs so that they can be used for organ transplants. He led them through one megaround and has just closed another, raising $125 million from 17 different investors to push the first-ever (humanized) pig to human transplants into the clinic.

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Fi­bro­Gen shares skid low­er as a sur­prise ad­comm rais­es risks on roxa OK

FibroGen will likely have to delay its US rollout for roxadustat once again.

In an unexpected move, the FDA is convening its Cardiovascular and Renal Drugs Advisory Committee to review the NDA in an advisory committee meeting. The date is yet to be confirmed.

Just a few weeks ago, SVB Leerink analyst Geoffrey Porges predicted that the roxa approval could come ahead of the PDUFA date on March 20 — effusive despite already being let down once by the FDA’s extension of its review back in December. AstraZeneca, which is partnered with FibroGen on the chronic kidney disease-related anemia drug, disclosed regulators had requested further clarifying analyses of clinical data.

In­tro­duc­ing End­pointsF­DA+, 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.

UP­DAT­ED: Feds clear the road for J&J to start de­liv­er­ing mil­lions of dos­es of their Covid-19 vac­cine — but frets linger about run­ner-up sta­tus

All the pieces needed to trigger a third wave of Covid-19 vaccine supply to start washing over the US fell neatly into place over the weekend.

After providing for a brief mime of regulatory judiciousness, the FDA stamped their emergency approval on J&J’s Covid-19 vaccine Saturday, adding to the Biden administration’s plan aimed at ending the pandemic in the near term — at least in the US. The CDC came through on Sunday with its stamp of approval and J&J is reportedly expected to start delivering vaccine sometime in the next few days.

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