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.

A New Fron­tier: The In­ner Ear

What happens when a successful biotech venture capitalist is unexpectedly diagnosed with a chronic, life-disrupting vertigo disorder? Innovation in neurotology.

That venture capitalist was Jay Lichter, Ph.D., and after learning there was no FDA-approved drug treatment for his condition, Ménière’s disease, he decided to create a company to bring drug development to neurotology. Otonomy was founded in 2008 and is dedicated to finding new drug treatments for the hugely underserved community living with balance and hearing disorders. Helping patients like Jay has been the driving force behind Otonomy, a company heading into a transformative 2020 with three clinical trial readouts: Phase 3 in Ménière’s disease, Phase 2 in tinnitus, and Phase 1/2 in hearing loss. These catalysts, together with others in the field, highlight the emerging opportunity in neurotology.
Otonomy is leading the way in neurotology
Neurotology, or the treatment of inner ear neurological disorders, is a large and untapped market for drug developers: one in eight individuals in the U.S. have moderate-to-severe hearing loss, tinnitus or vertigo disorders such as Ménière’s disease.1 With no FDA-approved drug treatments available for these conditions, the burden on patients—including social anxiety, lower quality of life, reduced work productivity, and higher rates of depression—can be significant.2, 3, 4

Af­ter 4 years of furor, the FTC and New York state ac­cuse Mar­tin Shkre­li of run­ning a drug mo­nop­oly. And this time they plan to squash it

Pharma bro Martin Shkreli was jailed, publicly pilloried and forced to confront some lawmakers in Washington riled by his move to take an old generic and move the price from $17.50 per pill to $750. But through 4 years of controversy and public revulsion, his company never backed away from the price — left uncontrolled by a laissez faire federal policy on a drug’s cost.

Now the FTC and the state of New York plan to pry his fingers off the drug once and for all and open it up to some cheap competition.

J&J's Spra­va­to gets no love from NICE, jeop­ar­diz­ing its prospects in the UK

UK’s cost-effectiveness watchdog NICE is taking the same track laid out by ICER — J&J’s pharmaceutical version of the hallucinogenic anesthetic ketamine, Spravato, is low value for money. On Tuesday, the agency refused to endorse the therapy for inclusion as a reimbursable drug on the the UK’s National Health System.

Cognizant of the myriad of approved antidepressants that often don’t work, EU regulators endorsed J&J’s low-dose, nasal-spray formulation of ketamine last month for treatment-resistant depression.

Patrik Jonsson, the president of Lilly Bio-Medicines

Who knew? Der­mi­ra’s board kept watch as its stock price tracked Eli Lil­ly’s se­cret bid­ding on a $1.1B buy­out

In just 8 days, from December 6 to December 14, the stock jumped from $7.88 to $12.70 — just under the initial $13 bid. There was no hard news about the company that would explain a rise like that tracking closely to the bid offer, raising the obvious question of whether insider info has leaked out to traders.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

Short at­tack­er Sahm Ad­ran­gi draws crosshairs over a fa­vorite of Sanofi’s new CEO — with PhII da­ta loom­ing

Sahm Adrang Kerrisdale

Kerrisdale chief Sahm Adrangi took a lengthy break from his series of biotech short attacks after his chief analyst in the field pulled up stakes and went solo. But he’s making a return to drug development this morning, drawing crosshairs over a company that’s one of new Sanofi CEO Paul Hudson’s favorite collaborators.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 70,700+ biopharma pros reading Endpoints daily — and it's free.

UP­DAT­ED: Ac­celeron of­fers thumbs up on a PhII suc­cess for would-be block­buster drug — and shares rock­et up

There’s no public data yet, but Acceleron $XLRN says that its first major trial readout of 2020 is a success.

In a Phase II study of 106 patients with pulmonary arterial hypertension (PAH), Acceleron’s experimental drug sotatercept hit its primary endpoint: a significant reduction in pulmonary vascular resistance. The drug also met three different secondary endpoints, including the 6-minute walking test.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 70,700+ biopharma pros reading Endpoints daily — and it's free.

Civi­ca and Blue Cross Blue Shield launch new ven­ture to low­er gener­ic prices

Five years after Martin Shkreli put a smug face to the volatile prices companies can charge even for generic drugs, payers and governments are coming up with outside-the-box solutions.

The latest fix is a new venture from the Blue Cross Blue Shield Association, 18 of its members and Civica, the generics company founded in 2018 by hospitals fed up with high prices for drugs that had long-since lost patent protection. While Civica focused on drugs that hospitals purchased, the new company will aim to lower prices on drugs that, like Shkreli’s Daraprim, are purchased by individuals.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 70,700+ biopharma pros reading Endpoints daily — and it's free.

Merck Invests in State-Of-The-Art Biotech Development Facility in Switzerland

Mer­ck KGaA match­es lofty R&D goals with €250M in­vest­ment in­to a new clin­i­cal man­u­fac­tur­ing site in Switzer­land

As Merck KGaA strives to prove itself as a capable biopharma R&D player, it has begun construction on a €250 million facility dedicated to developing and manufacturing drugs for use in clinical trials.

The German drugmaker chose a location at Corsier-sur-Vevey, Switzerland, where it already has a commercial manufacturing site, in order to “bridge together research and manufacturing.”

“This investment in the Merck Biotech Development Center reflects our commitment to speed up the availability of new medicines for patients in need, and confirms the importance of Switzerland as our prime hub for the manufacturing of biotech medicines,” CEO Stefan Oschmann said at the groundbreaking ceremony, according to a statement.

Breast can­cer ap­proval in tow, As­traZeneca, Dai­ichi armed an­ti­body scores in key gas­tric can­cer study

AstraZeneca kicked off Monday with a flurry of good news. Apart from unveiling positive results on its stroke trial testing its clot-fighter Brilinta, and welcoming its experimental IL-23 inhibitor brazikumab back from Allergan — the British drugmaker also disclosed some upbeat gastric cancer data on its HER2-positive oncology therapy it is collaborating on with Daiichi Sankyo.

Buoyed by the performance of its oncology drugs, last March AstraZeneca chief Pascal Soriot bet big to partner with Daiichi on the cancer drug, with $1.35 billion upfront in a deal worth up to roughly $7 billion. Roughly 8 months later, as 2019 drew to a close, the FDA swiftly approved the drug — trastuzumab deruxtecan — for use in breast cancer, months ahead of the expected decision date.