PAH ther­a­py tanks in Phase III, oblit­er­at­ing Bellerophon's stock

Bellerophon, a small com­pa­ny in New Jer­sey, has lost its bet that it could treat high blood pres­sure in the lungs with its last-stage lead ther­a­py. The com­pa­ny’s stock is crash­ing on the news that its Phase III tri­al is be­ing scrapped mid­way.

Fabi­an Tenen­baum

Why is Bellerophon ditch­ing the ef­fort? Fu­til­i­ty. An in­ter­im analy­sis showed the first 75 pa­tients en­rolled in the pul­monary ar­te­r­i­al hy­per­ten­sion (PAH) tri­al were not re­spond­ing as hoped. The Da­ta Mon­i­tor­ing Com­mit­tee deemed the over­all change in 6 minute walk dis­tance — the pri­ma­ry end­point of the tri­al — “in­suf­fi­cient to sup­port the con­tin­u­a­tion of the study.”

The com­pa­ny was test­ing its tech­nol­o­gy called the IN­Opulse, which de­liv­ers in­haled ni­tric ox­ide to pa­tients’ res­pi­ra­to­ry sys­tem in puls­es. The com­pa­ny de­scribes the de­vice as an ex­ten­sion of the con­tin­u­ous flow in­haled ni­tric ox­ide sys­tem that hos­pi­tals use on new­borns. This tri­al was Bellerophon’s most ad­vanced pro­gram, and its re­main­ing pipeline is al­so de­pen­dent on this plat­form work­ing.

In­vestors are not hap­py. The com­pa­ny’s stock $BLPH nose­dived 72% when the mar­ket opened Tues­day morn­ing, de­liv­er­ing Bellerophon to pen­ny-stock ter­ri­to­ry. As of press time, shares were go­ing for $0.62.

The com­pa­ny’s CEO Fabi­an Tenen­baum had this to say in a state­ment:

While we are dis­ap­point­ed in the over­all ef­fi­ca­cy re­sults of this study, we are en­cour­aged by the pos­i­tive da­ta in he­mo­dy­nam­ics and pleased with the safe­ty and tol­er­a­bil­i­ty pro­file of IN­Opulse. Over the next few weeks, we in­tend to fur­ther an­a­lyze the full da­ta set avail­able to us from this in­ter­im analy­sis in or­der to de­ter­mine the next steps in our PAH pro­gram.

Bellerophon says it has enough re­sources to get through the first half of 2019.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 85,300+ biopharma pros reading Endpoints daily — and it's free.

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

An­ti­body leg­end Till­man Gern­gross is el­bow­ing his way in­to the Covid-19 R&D cru­sade: 'I don’t see this end­ing any­time soon'

One of the most influential — and outspoken — scientists at work in the field of antibody discovery is jumping into the frenzied race to create new therapeutics to treat and prevent Covid-19. And he’s operating with the conviction that the current outbreak now once again spreading like wildfire will create plenty of demand for what he has in mind.

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.

Endpoints Premium

Premium subscription required

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

Full Bril­in­ta study re­sults show the blood thin­ner re­duces rate of sec­ondary stroke

AstraZeneca once projected its Brilinta drug to peak at $3.5 billion in sales, and though the blood thinner never reached that lofty goal, it received the latest positive signs in a string of recent good news.

The pharma released full details from its THALES study Thursday morning, which measured the effects of Brilinta and aspirin against aspirin alone in treating patients who had an acute ischemic stroke or transient ischemic attack. When taken twice daily with once-a-day aspirin for 30 days, the Brilinta combo reduced the risk of stroke and death by 17 percent, meeting the primary endpoint of the study.

Norbert Bischofberger, Kronos CEO

Gilead­'s ex-R&D chief Bischof­berg­er heads back to the biotech gi­ant to pick up a pair of late-stage drugs that had been put aside

Norbert Bischofberger knows entospletinib well.

Back during his long, blockbuster run as head of R&D at Gilead, researchers had once held some high hopes for this drug. But to make it work, he and the team felt it would need a new companion diagnostic to identify patients. There was talk of a combo approach to give it more punch. But the market was small, making them wonder if it would be worth going through a lengthy development cycle to get it through a pivotal.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 85,300+ biopharma pros reading Endpoints daily — and it's free.

The $1B Mer­ck-Bay­er drug that di­vid­ed car­di­ol­o­gists in March gets pri­or­i­ty re­view

Three months after Merck published in the New England Journal of Medicine data that left doctors and investors divided over just how well its experimental heart drug worked, the FDA has handed that drug priority review. A decision is now due by January 20, 2021.

Merck first announced the drug, known as vericiguat, as a Phase III success last November. In 2016, Merck had paid $1 billion upfront for US rights to the Bayer-developed drug. Early projections foresaw a few hundred million a year in sales, but the unspecified late-stage success raised the possibility for far more. After all, Novartis’s flagship heart drug, Entresto, was earning $1.7 billion per year and was expected to reach up to $4 billion in annual sales.

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.

Source: Shutterstock

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.

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

→Merck KGaA has taken an early option on an immuno-oncology program developed at F-star Therapeutics. This is their second option in the collaboration. And they added a pair of preclinical discovery programs to the alliance as well.

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.