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.

In a stun­ning set­back, Amarin los­es big patent fight over Vas­cepa IP. And its high-fly­ing stock crash­es to earth

Amarin’s shares $AMRN were blitzed Monday evening, losing billions in value as reports spread that the company had lost its high-profile effort to keep its Vascepa patents protected from generic drugmakers.

Amarin had been fighting to keep key patents under lock and key — and away from generic rivals — for another 10 years, but District Court Judge Miranda Du in Las Vegas ruled against the biotech. She ruled that:
(A)ll the Asserted Claims are invalid as obvious under 35 U.S.C.§ 103. Thus, the Court finds in favor of Defendants on Plaintiff’s remaining infringementclaim, and in their favor on their counterclaims asserting the invalidity of the AssertedClaims under 35 U.S.C. § 103.

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UP­DAT­ED: Have a new drug that promis­es to fight Covid-19? The FDA promis­es fast ac­tion but some de­vel­op­ers aren't hap­py

After providing an emergency approval to use malaria drugs against coronavirus with little actual evidence of their efficacy or safety in that setting, the FDA has already proven that it has set aside the gold standard when it comes to the pandemic. And now regulators have spelled out a new approach to speeding development that promises immediate responses in no uncertain terms — promising a program offering the ultimate high-speed pathway to Covid-19 drug approvals.

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The race to de­vel­op Covid-19 drugs and vac­cines is on — here’s what’s hap­pen­ing in the UK

Weeks away from the results of ongoing US and China trials testing its experimental antiviral remdesivir, Gilead is going to trial the failed Ebola drug in a small group of coronavirus patients in England and Scotland. The United Kingdom is also home to a range of other therapeutic efforts, as the pandemic rages on across the globe.

On Tuesday, Southampton, UK-based startup Synairgen kicked off a mid-stage placebo-controlled study testing its experimental drug, SNG001 — an inhaled formulation of interferon-beta-1a — that has previously shown to be safe and effective in improving lung function in asthma patients with a respiratory viral infection in a pair of Phase II trials.

‘There was a grow­ing weari­ness’: Rush­ing against a pan­dem­ic clock, As­pen Neu­ro­sciences se­cures $70M Se­ries A

Just before Christmastime, Howard Federoff got a tip from Washington: There was a new virus in China. And this one could be bad.

News report of the virus had not yet appeared. Federoff, a neuroscientist, was briefed because years before, he was vetted as part of a group — he didn’t give a name for the group — to consult for the US government on emerging scientific issues. His day job, though, was CEO of Aspen Neurosciences, a Parkinson’s cell therapy startup that days before had come out of stealth mode and gave word to investors they were hoping to raise $70 million. That, Federoff realized, would be difficult if a pandemic shut down the global economy.

FDA puts pe­di­atric aGVHD drug on pri­or­i­ty re­view lane — will they go vir­tu­al with the ad­comm?

Despite worries about regulatory delays due to new work arrangements under Covid-19, the FDA appears intent to go full speed ahead with its everyday work, not only granting priority review to a stem cell therapy for acute graft versus host disease but also plotting an advisory committee meeting for it.

With a PDUFA date of September 30, the journey of the drug — remestemcel-L, or Ryoncil — could shed light on the agency’s capacity to facilitate drug development unrelated to Covid-19.

Once fu­ri­ous over No­var­tis’ da­ta ma­nip­u­la­tion scan­dal, the FDA now says it’s noth­ing they need to take ac­tion on

Back in the BP era — Before Pandemic — the FDA ripped Novartis for its decision to keep the agency in the dark about manipulated data used in its application for Zolgensma while its marketing application for the gene therapy was under review.

Civil and criminal sanctions were being discussed, the agency noted in a rare broadside at one of the world’s largest pharma companies. Notable lawmakers cheered the angry regulators on, urging the FDA to make an example of Novartis, which fielded Zolgensma at $2.1 million — the current record for a one-off therapy.

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Covid-19 roundup: GSK, Am­gen tai­lor R&D work to fit the coro­n­avirus age; Doud­na's ge­nomics crew launch­es di­ag­nos­tic lab

You can add Amgen and GSK to the list of deep-pocket drug R&D players who are tailoring their pipeline work to fit a new age of coronavirus.

Following in the footsteps of a lineup of big players like Eli Lilly — which has suspended patient recruitment for drug studies — Amgen and GSK have opted to take a more tailored approach. Amgen is intent on circling the wagons around key studies that are already fully enrolled, and GSK has the red light on new studies while the pandemic plays out.

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Covid-19 roundup: Trump push­es his new fa­vorite, untest­ed drug; CRISPR out­lines crip­pling im­pact of Covid-19

President Trump has a new favorite Covid-19 drug.

After a conversation with Japanese Prime Minister Shinzo Abe, Politico reports, the president is pressuring the FDA to issue emergency use authorization for favipiravir, a flu drug that showed glimpses of success in China but remains unproven and carries a list of worrying side effects. The push comes after a week-plus in which the White House touted a potentially effective but unproven malaria medication despite the concerns of scientific advisors such as NIAID director Anthony Fauci. And Trump ally Rudy Giuliani has been talking up unproven cell therapy efforts on Twitter.

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ITeos nabs $125M as they prep Keytru­da com­bi­na­tion tri­al — if Covid-19 will let them

For iTeos, it turned out, $75 million could only last so long.

Two years after announcing their eye-catching Series B raise, the Belgian biotech is back with an even larger Series B-2: $125 million.

The now $175 million financing – $25 million of the first B round is considered part of the second – illustrates the vast capital available for those with promising new immuno-oncology compounds, particularly those that might be used in combination with existing therapies. In December, iTeos announced a collaboration with Merck to test its lead compound with Keytruda this year. The proceeds will push forward that trial and help fund the ongoing Phase I/II trials for that compound, EOS-850, and a second one, EOS-448.

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