Pro­teosta­sis shares zoom north as their cys­tic fi­bro­sis dou­blet caus­es a stir in small study -- trig­ger­ing an on­line brawl

Some­times it’s the small­est stud­ies that gain the biggest re­ac­tions from the in­vest­ment crowd that fol­lows biotech.

Case in point: Lit­tle Pro­teosta­sis Ther­a­peu­tics hit big-time pay dirt on Thurs­day, watch­ing its stock $PTI swoop up 448% on some in­trigu­ing ear­ly da­ta on a hand­ful of pa­tients suf­fer­ing from cys­tic fi­bro­sis.

Meenu Chhabra, CEO

Break­ing 21 pa­tients ho­mozy­gous for the F508del mu­ta­tion in­to var­i­ous dose groups for their CF dou­blet ther­a­py, re­searchers teased out some sig­nif­i­cant re­sults for the few peo­ple in the high dose co­hort that was evalu­able at the time of the Phase I cut­off for their read­out.

For the 300 mg ppFEV1 co­hort there was a sta­tis­ti­cal­ly sig­nif­i­cant in­crease from base­line to day 7 of 6.3%, which reg­is­tered as a 5.9% in­crease on day 14. Com­pared to a place­bo, the same PTI-801 300 mg/PTI-808 300 mg co­hort hit +8.3% and +6.6% at 7 and 14 days. They added that they al­so tracked a sta­tis­ti­cal­ly sig­nif­i­cant de­crease in sweat chlo­ride con­cen­tra­tion from base­line.

There’s an­oth­er 400 mg co­hort that’s be­ing tracked now, with a triplet to fol­low.

The kick­er is here:

“The cur­rent im­prove­ment in ppFEV1 for stan­dard of care dou­blets is on av­er­age 3%-4% in the F508del ho­mozy­gous pa­tient pop­u­la­tion,” said Dami­an Downey, a lec­tur­er in res­pi­ra­to­ry med­i­cine at Queen’s Uni­ver­si­ty Belfast.

The num­bers got a close re­view by an­a­lysts in the field. Leerink’s Joseph Schwartz not­ed Pro­teosta­sis’ mixed re­sults as an add-on for Orkam­bi, from Ver­tex. But, he added, “PTI’s own pro­pri­etary com­bo bodes well in our view for pre­lim­i­nary triplet da­ta ex­pect­ed this quar­ter.”

Baird’s Bri­an Sko­r­ney al­so gave the da­ta a thumbs up as an in­di­ca­tor of the po­ten­tial of the triple:

The com­bo of PTI-808 and PTI-801 showed ini­tial FEV1 re­sults that are com­pa­ra­ble to Ver­tex’s Orkam­bi and Symdeko in cys­tic fi­bro­sis pa­tients. Al­though we be­lieve this com­bo would have a dif­fi­cult de­vel­op­ment path­way, giv­en the avail­abil­i­ty of the Ver­tex dou­blets, we do be­lieve it cre­ates an op­por­tu­ni­ty for it to de­vel­op its am­pli­fi­er, PTI-428, in a pro­pri­etary triple com­bi­na­tion. We will see ini­tial triple com­bo da­ta this quar­ter that we be­lieve will be a sig­nif­i­cant pos­i­tive cat­a­lyst for the stock.

Ver­tex stock dropped 3.5%, which trig­gered a de­fen­sive scrum on Twit­ter as some of the com­pa­ny’s de­fend­ers turned ug­ly on the pres­sure. So far, Ver­tex has stared down re­peat­ed chal­lenges to its fron­trun­ner sta­tus. But it didn’t get off so light­ly on this run.

Pro­teosta­sis’ KOLs, mean­while, will con­tin­ue to talk up their new-found sta­tus in CF.

”Dou­blet com­bi­na­tions, the cur­rent stan­dard of care for many CF pa­tients, are the bench­mark for cur­rent treat­ment in cys­tic fi­bro­sis.  These da­ta are the first re­sults seen us­ing an en­tire­ly new CF dou­blet, com­pare fa­vor­ably to stan­dard of care, and demon­strate the po­ten­tial of next-gen­er­a­tion CFTR mod­u­la­tors to fur­ther im­prove out­comes in this dis­ease,” said Carsten Schwarz, head of the Adult Cys­tic Fi­bro­sis Cen­tre, Lung-Trans­plan­ta­tion Pro­gram, and En­doscopy Unit, De­part­ment of Pe­di­atric Pneu­mol­o­gy and Im­munol­o­gy, Char­ité, Berlin Uni­ver­si­ty Med­ical Cen­ter. “I look for­ward to re­sults from the fourth dos­ing co­hort and to un­der­stand­ing the po­ten­tial of an­oth­er nov­el com­bi­na­tion, PTI-801, PTI-808 and PTI-428.”

Re­searchers will be adding more da­ta this quar­ter and in ear­ly 2019.

The fight­ing has just be­gun.

Op­ti­miz­ing Cell and Gene Ther­a­py De­vel­op­ment and Pro­duc­tion: How Tech­nol­o­gy Providers Like Corn­ing Life Sci­ences are Spurring In­no­va­tion

Remarkable advances in cell and gene therapy over the last decade offer unprecedented therapeutic promise and bring new hope for many patients facing diseases once thought incurable. However, for cell and gene therapies to reach their full potential, researchers, manufacturers, life science companies, and academics will need to work together to solve the significant challenges facing the industry.

Amid mon­key­pox fears, biotechs spring to ac­tion; Mod­er­na’s CFO trou­ble; Cuts, cuts every­where; Craft­ing the right pro­teins; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

It’s always a bittersweet moment saying goodbye, but as Josh Sullivan goes off to new adventures we are grateful for the way he’s built up the Endpoints Manufacturing section — which the rest of the team will now carry forward. If you’re not already, this may be a good time to sign up for your weekly dose of drug manufacturing news. Thank you for reading and wish you a restful weekend.

Bay­er sounds re­treat from a $670 mil­lion CAR-T pact in the wake of a pa­tient death

Two months after Atara Biotherapeutics hit the hold button on its lead CAR-T 2.0 therapy following a patient death, putting the company under the watchful eye of the FDA, its Big Pharma partners at Bayer are bowing out of a $670 million global alliance. And the move is forcing a revamp of Atara’s pipeline plans, even as research execs vow to continue work on the two drugs allied with Bayer 18 months ago, which delivered a $60 million cash upfront.

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Try­ing to shake up the Parkin­son's par­a­digm, Ab­b­Vie sub­mits NDA for con­tin­u­ous, 24-hour in­fu­sion ther­a­py

AbbVie is approaching the FDA with a new therapy to potentially treat Parkinson’s disease, using prodrugs of two medications commonly used for the condition.

The Big Pharma submitted its NDA for ABBV-951, a solution of levodopa and carbidopa prodrugs being evaluated in advanced Parkinson’s patients who don’t respond well to oral therapy, AbbVie announced Friday morning. Researchers are hoping a positive Phase III study that reads out in late October will help move things along quickly at the agency.

Sanofi and Re­gen­eron clear the fin­ish line in an in­flam­ma­to­ry esoph­a­gus dis­ease, leav­ing Take­da in the dust

With atopic dermatitis rivals breathing down Dupixent’s neck, Sanofi and Regeneron on Friday secured a first win in new territory in what Sanofi’s head of immunology and inflammation Naimish Patel called the fastest approval he’s ever seen.

The FDA approved Dupixent on Friday to treat patients 12 years and older with eosinophilic esophagitis (EoE), an inflammatory condition that causes swelling and scarring of the esophagus. The approval came just a couple months after regulators granted Dupixent priority review, and months ahead of its PDUFA date on Aug. 3.

Fu­ji­film con­tin­ues its biotech build­ing spree with new fa­cil­i­ty in Chi­na

A Japanese conglomerate is making a big play in China with the opening of a new facility, as it continues to expand.

Fujifilm Irvine Scientific has opened its new Innovation and Collaboration Center in Suzhou New District, China, an area in Jiangsu province specifically designated for technological and industrial development.

According to Fujifilm, the 12,000-square-foot site will be responsible for the company’s cell culture media optimization, analysis and design services. Cell culture media itself often requires customization of formulas and protocols to achieve the desired quantity and quality of therapeutic desired. Fujifilm Irvine Scientific is offering these services from its headquarters in California and Japan to its customers globally, as well as in China now.

Emer Cooke, EMA director (AP Photo/Geert Vanden Wijngaert)

Ahead of FDA, EMA rec­om­mends au­tho­riz­ing new gene ther­a­py treat­ment for ul­tra-rare dis­ease

Aromatic amino acid decarboxylase (AADC) deficiency is an ultra-rare genetic disease that leaves patients unable to produce certain hormones in the brain, such as dopamine and serotonin, usually leading to developmental delays, weak muscle tone and inability to control the movement of the limbs. It can also lead to multiple organ failure.

To date, there have been no treatments approved for AADC deficiency, which has been identified in less than 150 patients.

Ather­sys tries to post-hoc-an­a­lyze its way out of an­oth­er tri­al fail for stroke stem cell ther­a­py

Athersys’ stem cell therapy has failed yet again.

In a 206-person trial conducted in Japan, Athersys’ stem cell therapy for stroke failed its primary endpoint of “excellent outcome,” a combined measure of three stroke recovery scores.

While a greater percentage of patients in the treatment group reached the primary endpoint compared to placebo, that difference was not statistically significant.

Siddhartha Mukherjee (Brian Ach/Getty Images for The New Yorker)

All Blue's $733M bid to ac­quire Zymeworks turns hos­tile as board bat­tles back — af­ter a biotech celebri­ty jumps in

Yesterday, the team at All Blue Capital — bent on the takeover of a badly battered Zymeworks — brought in celebrated oncologist, Pulitzer prize-winning writer and biotech exec Siddhartha Mukherjee to add some glitz to their proposed board. But they’re still not winning over any converts.

This morning, Zymeworks’ board officially turned this acquisition offer into a hostile showdown, rejecting the unsolicited offer and marshaling its forces to prevent a buyout at $10.50 per share.

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