Pro­teosta­sis shares shat­tered as weak triplet da­ta in­spires a scorch­ing back­lash

You al­most have to pity Pro­teosta­sis $PTI to­day. The mi­cro-cap biotech sal­lied out in­to the stock mar­ket ear­ly in the day try­ing to float some mediocre ef­fi­ca­cy re­sults for their triple for cys­tic fi­bro­sis and got gunned down on every side. 

Al­ready quick to en­tice skep­ti­cism af­ter ear­li­er shots on goal aroused a short at­tack from Ker­ris­dale Cap­i­tal, some promi­nent an­a­lysts made short work of Pro­teosta­sis’ num­bers this morn­ing. Its share price is down 65% to an all-time low and the mar­ket cap has shriv­eled to a pal­try $76 mil­lion — less than cur­rent as­sets on hand.

That’s a far cry from the big spike of last Oc­to­ber, when some pos­i­tive dou­blet da­ta whipped up a bit of con­tro­ver­sial sup­port for the biotech — al­ways dan­ger­ous when you fac­tor in the Sturm und Drang re­ac­tions from the brigade of Ver­tex sup­port­ers out there anx­ious to mug any­thing that could com­pare with one of their cham­pi­on’s new triplets.

Ge­of­frey Porges

Even by ex­clud­ing some pa­tients from their per pro­to­col eval­u­a­tion, though, these weren’t com­pet­i­tive num­bers, and Pro­teosta­sis shares were quick­ly led to the slaugh­ter. 

Here’s Ge­of­frey Porges from SVB Leerink:

Bot­tom Line: Pro­teosta­sis’ (PTI, OP) pro­pri­etary triple ther­a­py phase I re­sults for ho­mozy­gous F508del cys­tic fi­bro­sis (CF) did not match the ef­fi­ca­cy of Ver­tex’s CF med­i­cines, and will not com­pete for our es­ti­mat­ed $10bn peak CF sales to Ver­tex. Pro­teosta­sis’ triple in­creased lung func­tion mea­sured by per­cent pre­dict­ed forced ex­pi­ra­to­ry vol­ume (FEV1) from base­line by +5%, com­pared to the +13-14% hur­dle es­tab­lished by Ver­tex.

Leerink’s Joseph Schwartz has been a bull­ish ad­vo­cate of the triples and re­mains mod­est­ly hope­ful about bet­ter num­bers with longer ther­a­py. But even Schwartz can’t swal­low da­ta points in­volv­ing tiny pa­tient num­bers.

Al­though the com­pa­ny re­port­ed an FEV ben­e­fit of 5.2% when ‘428 was added on­to Orkam­bi, some in­vestors ques­tion the FEV ben­e­fit of ‘428 be­cause the da­ta was high­ly vari­able and large­ly dri­ven by two pa­tients on place­bo who wors­ened more than one would ex­pect. Un­for­tu­nate­ly, pa­tients did not gain a sta­tis­ti­cal­ly sig­nif­i­cant im­prove­ment in FEV for both stud­ies — 10 mg or 30 mg ‘428 added to Symdeko (28 days) and 400 mg ‘801 added to Symdeko (14 days) com­pared to place­bo. How­ev­er, the pa­tients in the ‘801 + Symdeko co­hort ex­pe­ri­enced an av­er­age sweat chlo­ride im­prove­ment of -20 mM (p<0.05) com­pared to base­line. Based on to­day’s up­date, PTI will de­pri­or­i­tize their add-on study and fo­cus on their pro­pri­etary triplet and dou­blet com­bi­na­tion ther­a­py.

Will in­vestors come back for more? Many turned the chan­nel this morn­ing and don’t seem pre­pared to give this lat­est ri­val to Ver­tex’s throne an­oth­er chance.

Not on board to in­spect the da­ta was Baird’s Bri­an Sko­r­ney, who had called the Oc­to­ber re­sults vi­able. Sko­r­ney dropped cov­er­age soon af­ter the Oc­to­ber da­ta were re­leased and Pro­teosta­sis raised cash with its in­flat­ed share price.

Martin Shkreli [via Getty]

Pris­on­er #87850-053 does not get to add drug de­vel­op­er to his list of cred­its

Just days after Retrophin shed its last ties to founder Martin Shkreli, the biotech is reporting that the lead drug he co-invented flopped in a pivotal trial. Fosmetpantotenate flunked both the primary and key secondary endpoints in a placebo-controlled trial for a rare disease called pantothenate kinase-associated neurodegeneration, or PKAN.

Endpoints News

Basic subscription required

Unlock this story instantly and join 58,000+ biopharma pros reading Endpoints daily — and it's free.

We­bi­nar: Re­al World End­points — the brave new world com­ing in build­ing fran­chise ther­a­pies

Several biopharma companies have been working on expanding drug labels through the use of real world endpoints, combing through the data to find evidence of a drug’s efficacy for particular indications. But we’ve just begun. Real World Evidence is becoming an important part of every clinical development plan, in the soup-through-nuts approach used in building franchises.

I’ve recruited a panel of 3 top experts in the field — the first in a series of premium webinars — to look at the practical realities governing what can be done today, and where this is headed over the next few years, at the prodding of the FDA.

ZHEN SU — Merck Serono’s Senior Vice President and Global Head of Oncology
ELLIOTT LEVY — Amgen’s Senior Vice President of Global Development
CHRIS BOSHOFF — Pfizer Oncology’s Chief Development Officer

A premium subscription to Endpoints News is required to attend this webinar. Please upgrade to either an Insider or Enterprise plan for access. Already have Endpoints Premium? Please sign-in below. You can contact our Subscriptions team at with any issues.

Endpoints Premium

Premium subscription required

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

Hal Barron. GSK

GSK's Hal Bar­ron her­alds their sec­ond pos­i­tive piv­otal for cru­cial an­ti-BC­MA ther­a­py, point­ing to a push for quick OKs in a crowd­ed field

Hal Barron has his second positive round of Phase III data in hand for his anti-BCMA antibody drug conjugate belantamab mafodotin (GSK2857916). And GSK’s research chief says the data paves the way for their drive in search of an FDA approval for treating multiple myeloma.

It’s hard to overestimate the importance of this drug for GSK, a cornerstone of Barron’s campaign to make a dramatic impact on the oncology market and provide some long-lost excitement for the pharma giant’s pipeline. They’re putting this BCMA program at the front of that charge — looking to lead a host of rivals all aimed at the same target.

We don’t know what the data are yet, but DREAMM-2 falls on the heels of a promising set of data delivered 5 months ago for DREAMM-1. There investigators noted that complete responses among treatment-resistant patients rose to 15% in the extra year’s worth of data to look over, with a median progression-free survival rate of 12 months, up from 7.9 months reported earlier. The median duration of response was 14.3 months.

Endpoints News

Basic subscription required

Unlock this story instantly and join 58,000+ biopharma pros reading Endpoints daily — and it's free.

Brian Kaspar. AveXis via Twitter

AveX­is sci­en­tif­ic founder fires back at No­var­tis CEO Vas Narasimhan, 'cat­e­gor­i­cal­ly de­nies any wrong­do­ing'

Brian Kaspar’s head was among the first to roll at Novartis after company execs became aware of the fact that manipulated data had been included in its application for Zolgensma, now the world’s most expensive therapy.

But in his first public response, the scientific founder at AveXis — acquired by Novartis for $8.7 billion — is firing back. And he says that not only was he not involved in any wrongdoing, he’s ready to defend his name as needed.

I reached out to Brian Kaspar after Novartis put out word that he and his brother Allen had been axed in mid-May, two months after the company became aware of the allegations related to manipulated data. His response came back through his attorneys.

Endpoints News

Basic subscription required

Unlock this story instantly and join 58,000+ biopharma pros reading Endpoints daily — and it's free.

Why would Am­gen want to buy Alex­ion? An­a­lysts call hot­ly ru­mored takeover un­like­ly, but seize the mo­ment

A rumor that Amgen is closing in on buyout deal for Alexion has sparked a guessing game on just what kind of M&A strategy Amgen is pursuing and how much Alexion is worth.

Mizuho analyst Salim Syed first lent credence to the report out of the Spanish news outlet Intereconomía, which said Amgen is bidding as much as $200 per share. While the source may be questionable, “the concept of this happening doesn’t sound too crazy to me,” he wrote.

FDA asks why No­var­tis took two months to launch for­mal in­ter­nal probe, af­ter AveX­is flagged da­ta ma­nip­u­la­tion

And the plot thickens. Novartis $NVS officials are reportedly now scrambling to explain to the FDA why it took them two months to open an internal investigation into data discrepancies for their $2.1 million gene-therapy for spinal muscular dystrophy — the world’s most expensive drug.

Endpoints News

Basic subscription required

Unlock this story instantly and join 58,000+ biopharma pros reading Endpoints daily — and it's free.

Build­ing on suc­cess­ful PD-1 pact, Eli Lil­ly li­cens­es di­a­betes drug to Chi­nese part­ners at In­novent

Eli Lilly is expanding its partnership with China’s Innovent in a deal involving a diabetes drug sitting in its Phase I reserves.

The two companies had jointly developed one of China’s first homegrown PD-1 agents, scoring an approval for Tyvyt (sintilimab) late last year for relapsed/refractory classical Hodgkin’s lymphoma. This time around, Lilly is out-licensing a piece of its diabetes pipeline, a leading franchise that has historically produced the top-selling Trulicity and Humalog.

UP­DAT­ED: An em­bold­ened As­traZeneca splurges $95M on a pri­or­i­ty re­view vouch­er. Where do they need the FDA to hus­tle up?

AstraZeneca is in a hurry.

We learned this morning that the pharma giant — not known as a big spender, until recently — forked over $95 million to get its hands on a priority review voucher from Sobi, otherwise known as Swedish Orphan Biovitrum.

That marks another step down on price for a PRV, which allows the holder to slash 4 months off of any FDA review time.

Endpoints News

Basic subscription required

Unlock this story instantly and join 58,000+ biopharma pros reading Endpoints daily — and it's free.

Bob Smith, Pfizer

Pfiz­er is mak­ing a $500M state­ment to­day: Here’s how you be­come a lead play­er in the boom­ing gene ther­a­py sec­tor

Three years ago, Pfizer anted up $150 million in cash to buy Bamboo Therapeutics in Chapel Hill, NC as it cautiously stuck a toe in the small gene therapy pool of research and development.

Company execs followed up a year later with a $100 million expansion of the manufacturing operations they picked up in that deal for the UNC spinout, which came with $495 million in milestones.

And now they’re really going for it.

Endpoints News

Basic subscription required

Unlock this story instantly and join 58,000+ biopharma pros reading Endpoints daily — and it's free.