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.

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David Liu, Liu Group

David Liu un­veils newest ad­vance­ment in CRISPR tech: Prime Edit­ing

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Bhaskar Chaudhuri. Frazier Healthcare Partners

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HBM Healthcare Investments, Vivo Capital, BlackRock, Omega Funds, Pivotal BioVentures, and Goldman Sachs jumped on board, joining Bain Capital Life Sciences, OrbiMed and RA Capital Management in backing Arcutis’ lead topical cream for plaque psoriasis.

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The remedy for the most common side effect for one of the most common multiple sclerosis drugs is simple: aspirin.

Taking aspirin with Biogen’s Tecfidera will reduce the flush, a sometimes painful form of red skin irritation, many patients experiences. The problem is that the aspirin has to be taken at least 30 minutes before Tecfidera, turning a simple twice-a-day, one-dose oral drug into a staggered two-drug regimen.

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Vas Narasimhan. Getty Images

Failed PhI­II fe­vip­iprant tri­als pour more cold wa­ter on No­var­tis' block­buster R&D en­gine — and spread the chill to a high-pro­file biotech

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CD47 play­er Tril­li­um chops dis­cov­ery ef­forts and 40% of staff; Brii Bio inks deal to bring an­tibi­otics to Chi­na

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Take­da tees up $420M deal for celi­ac an­ti­dote, con­tin­u­ing R&D re­fo­cus

Sometime in the 1st century AD, a patient presented to Arataeus looking like a varicose ghost. He was “emaciated and atrophied, pale, feeble and incapable of performing any of his accustomed works,” the Greek physician wrote, with hollow temples and huge veins running all over his body.

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