Rit­ter bombs fi­nal PhI­II for sole lac­tose in­tol­er­ance drug — shares plum­met

More than two years ago Rit­ter Phar­ma­ceu­ti­cals man­aged to find enough sil­ver lin­ing in its Phase IIb/III study — af­ter miss­ing the top-line mark — to pro­pel its lac­tose in­tol­er­ance to­ward a con­fir­ma­to­ry tri­al. But as it turned out, the en­thu­si­asm on­ly set the biotech and its in­vestors up to be sore­ly dis­ap­point­ed.

An­drew Rit­ter

This time around there’s lit­tle left to sal­vage. Not on­ly did RP-G28 fail to beat place­bo in re­duc­ing lac­tose in­tol­er­ance symp­toms, pa­tients in the treat­ment group ac­tu­al­ly av­er­aged a small­er im­prove­ment. On a com­pos­ite score mea­sur­ing symp­toms like ab­dom­i­nal pain, cramp­ing, bloat­ing and gas, pa­tients giv­en the drug had a mean re­duc­tion of 3.159 while the place­bo co­hort saw a 3.420 drop on av­er­age (one-sided p-val­ue = 0.0106).

The mea­sures were tak­en on day 61, well in­to the re­al world ev­i­dence phase of the study (in which pa­tients con­sumed their nor­mal di­ets plus di­ary prod­ucts) fol­low­ing a 30-day treat­ment pe­ri­od.

None of the sec­ondary end­points were met. On­ly 36.2% of the RP-G28 arm count­ed as re­spon­ders with a mean­ing­ful ben­e­fit com­pared to 34.1%, but the one-sided p-val­ue was an abysmal 0.284. The oth­er dif­fer­ences al­so missed sta­tis­ti­cal sig­nif­i­cance.

All of Rit­ter’s eggs were in this one bas­ket. Its al­ready ham­mered stock $RT­TR took a fur­ther 69% plunge to $0.32.

Founder and CEO An­drew Rit­ter, who has suf­fered from se­ri­ous lac­tose in­tol­er­ance since child­hood, was bet­ting that his com­pa­ny could bring the first treat­ment to the mar­ket. RP-G28 is a for­mu­la­tion of galac­to-oligosac­cha­ride, which is de­signed to stim­u­late growth lac­tose me­tab­o­liz­ing bac­te­ria in the gut, there­by al­le­vi­at­ing symp­toms.

The drug failed to elic­it a sta­tis­ti­cal­ly sig­nif­i­cant re­sponse rate com­pared to place­bo in its pre­vi­ous tri­al, but the com­pa­ny in­sist­ed that the pri­ma­ry end­point was ac­tu­al­ly met — once they elim­i­nat­ed a site with “sig­nif­i­cant ir­reg­u­lar­i­ties” and changed the pri­ma­ry end­point.

That study, pub­lished in 2017, had 368 pa­tients. The cur­rent Phase III en­rolled 557 in to­tal.

“We are deeply dis­ap­point­ed in the re­sults of the phase 3 clin­i­cal tri­al,” Rit­ter said in a state­ment. “We be­lieve the clin­i­cal tri­al was well-de­signed and ex­e­cut­ed. We are con­tin­u­ing to an­a­lyze the re­sults of the tri­al to bet­ter un­der­stand the da­ta and clin­i­cal out­comes to as­sess a path for­ward, which may in­clude al­ter­na­tive strate­gic op­tions for the Com­pa­ny.”

Any plans will have to come quick. As of June 30, Rit­ter had $4.4 mil­lion in cash and cash equiv­a­lents, with a work­ing cap­i­tal of on­ly $2 mil­lion.

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.

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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.

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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.

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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.

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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.