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.

Tal Zaks, Moderna CMO (Moderna via YouTube)

UP­DAT­ED: NI­AID and Mod­er­na spell out a 'ro­bust' im­mune re­sponse in PhI coro­n­avirus vac­cine test — but big ques­tions re­main to be an­swered

The NIAID and Moderna have spelled out positive Phase I safety and efficacy data for their Covid-19 vaccine mRNA-1273 — highlighting the first full, clear sketch of evidence that back-to-back jabs at the dose selected for Phase III routinely produced a swarm of antibodies to the virus that exceeded levels seen in convalescent patients — typically in multiples indicating a protective response.

Moderna execs say plainly that this first stage of research produced exactly the kind of efficacy they hoped to see in humans, with a manageable safety profile.

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Trans­port Sim­u­la­tion Test­ing for Your Ther­a­py is the Best Way to As­sure FDA Ex­pe­dit­ed Pro­gram Ap­proval

Modality Solutions is an ISO:9001-registered biopharmaceutical cold chain engineering firm with unique transport simulation capabilities that support accelerated regulatory approval for biologics and advanced therapeutic medicinal products (ATMP). Our expertise combines traditional validation engineering approaches with regulatory knowledge into a methodology tailored for the life sciences industry. We provide insight and execution for the challenges faced in your cold chain logistics network.

Tillman Gerngross, Adagio Therapeutics CEO

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Dartmouth professor and Adimab CEO Tillman Gerngross tells me he’s raised $50 million from a group of close VCs to spin out a new company — Adagio Therapeutics — with a full C-suite team assembled to hire up a staff and keep rolling toward the clinic.

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Who are the women blaz­ing trails in bio­phar­ma R&D and lead­ing the fight against Covid-19? Nom­i­nate them for End­points' spe­cial re­port

One of the many inequalities the pandemic has laid bare is the gender imbalance in biomedical research. A paper examining Covid-19 research authorship wondered out loud: Where are the women?

It’s a question that echoes beyond our current times. In the biopharma world, not only are women under-represented in R&D roles (particularly at higher levels), their achievements and talents could also be undermined by stereotypes and norms of leadership styles. The problem is even more dire for women of color.

GSK’s Shin­grix leader Guil­laume Pfe­fer has jumped on board Flag­ship to helm a biotech hy­brid as Afeyan’s lat­est CEO-part­ner

After spending 4 years in a senior post with GlaxoSmithKline’s star team positioning Shingrix for a blockbuster approval, Guillaume Pfefer is headed back to the biotech world — in style.

Pfefer has signed on to join Noubar Afeyan’s busy group of partners at Flagship, and he’s taking the helm of an upstart — which today is being merged with another Flagship startup — with some grand plans of its own. The announcement this morning notes that Pfefer will run Kintai Therapeutics, one of the grads of the Flagship labs.

Donald Trump and Anthony Fauci (AP Images)

Covid-19 roundup: Rus­sia hack­ers tar­get US, UK vac­cine and drug re­searchers; Fau­ci fires back at White House cam­paign to un­der­mine him

Russia has tried to steal a Covid-19 vaccine and therapeutics researcher from pharmaceutical and academic institutions in the US, UK and Canada, Britain’s National Cyber Security Centre said Thursday.

The NCSC said that hacking attempts came from a group known as APT129, also known as “Cozy Bear,” that “almost certainly operate as part of Russian intelligence services.” The Canadian Communication Security Establishment, US Department for Homeland Security, the Cybersecurity Infrastructure Security Agency, and the National Security Agency shared the assessment, the NCSC said.

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John Furey, Imvax CEO

A neu­ro­sur­geon spent the past 30 years de­vel­op­ing a neoanti­gen tu­mor vac­cine. Now he has $112M for a piv­otal test

As a neurosurgeon, David Andrews knew there wasn’t much he could do for his glioma patients after resecting — rarely fully — their tumor. Even with the best treatment and care available, median overall survival is just somewhere between 14 and 16 months.

Then in the 1990s, his mentor at Thomas Jefferson University introduced him to Renato Baserga, a pathologist who had been studying the effect of using antisense oligonucleotide to knock out the insulin-like growth factor type 1 receptor in cancers. As IGF-R1 drives tumor growth and metastasis, the preclinical reasoning went, implanting a molecule targeting the receptor together with the tumor material near lymph nodes can slow down the spread of the cancer.

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Jeff Albers, Blueprint CEO

Di­ag­nos­tic champ Roche buys its way in­to the RET ti­tle fight with Eli Lil­ly, pay­ing $775M in cash to Blue­print

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Roche gains rights to co-develop and co-commercialize the drug, with sole marketing responsibility for places outside the US and China (where CStone has staked its claim).

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Mer­ck KGaA takes its I/O op­tion on F-star Ther­a­peu­tics; Nephron spends $215M, eye­ing spot in Covid-19 vac­cine chain

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Any biotech going public these days wouldn’t feel right if they didn’t upsize the offering. And that’s just what Phase I biotech Pandion Therapeutics did. The autoimmune company is now selling 7 million shares, a 1.5 million share bump, for $16 to $18 a share.