Ver­sant de­buts Ridge­line's start­up #4, armed with $30M and al­ter­na­tive TCR cell ther­a­pies for sol­id tu­mors

For all the it­er­a­tions and ad­vances in TCR ther­a­pies for can­cer, any ex­per­i­men­tal treat­ments in­volv­ing T cell re­cep­tors share one trait: By de­f­i­n­i­tion, they on­ly rec­og­nize anti­gens pre­sent­ed as pep­tides on the ma­jor his­to­com­pat­i­bil­i­ty com­plex (MHC) on cells.

Gen­naro De Libero

Ver­sant reck­ons it’s time to ex­pand the ar­se­nal. With $30 mil­lion in ini­tial fund­ing, its Ridge­line Dis­cov­ery En­gine in Switzer­land has been work­ing on a non-pep­tidic ap­proach that it says has tu­mor-ag­nos­tic po­ten­tial, es­pe­cial­ly in sol­id tu­mors. They’ve named it Mat­ter­horn, af­ter a Swiss moun­tain as they did with the three oth­er com­pa­nies that have emerged from the Basel-based in­cu­ba­tor.

The key dis­cov­er­ies by found­ing sci­en­tists Gen­naro De Libero and Lu­cia Mori at the Uni­ver­si­ty of Basel have to do with MR1, or MHC class I-re­lat­ed mol­e­cule 1.

When they be­gan prob­ing the pro­tein more than 10 years ago, MR1 was most­ly known for bind­ing to mol­e­cules of bac­te­r­i­al ori­gin — and thus its role in in­fec­tions. But De Libero and Mori pre­dict­ed that en­doge­nous mol­e­cules could be pre­sent­ed as well. And they were right. Not on­ly did they find metabo­lites tied to tu­mor cell pro­lif­er­a­tion that are not found on healthy cells, they al­so stum­bled up­on a class of T cells that could specif­i­cal­ly tar­get MR1.

“We pub­lished that the fre­quen­cy of these cells in the blood is sim­i­lar to the fre­quen­cy of pep­tide-spe­cif­ic T cells,” De Libero told End­points News. “And they were over­looked.”

Be­cause MR1 is iden­ti­cal across hu­mans act­ing as a sur­veil­lance sys­tem for aber­rant me­tab­o­lism, it lends it­self to an off-the-shelf ther­a­py, with no HLA match­ing nec­es­sary.

“The idea then is to gen­er­ate a li­brary of com­pounds that are metabo­lites, that ac­cu­mu­late in the tu­mor, to­geth­er with a li­brary of pre­de­fined T cell re­cep­tors,” he said. “So that if your tu­mor ex­press­es a com­pound A, you will uti­lize re­cep­tor A that you know is spe­cif­ic to that com­bi­na­tion.”

Lu­cia Mori

Al­though Mat­ter­horn’s tar­gets and re­cep­tors are new, the method of get­ting them in­to T cells isn’t. Much like CAR-T and oth­er TCR ther­a­pies, sci­en­tists will knock out en­doge­nous TCR genes and trans­duce T cells with their own re­cep­tors.

Based on the pre­clin­i­cal da­ta he and Mori, the CSO of Mat­ter­horn, along­side 10 staffers have gen­er­at­ed to date, De Libero be­lieves that their li­brary would ul­ti­mate­ly con­sist of a rel­a­tive­ly small num­ber of metabo­lites and cor­re­spond­ing T cell re­cep­tors — “much less than 100” in to­tal.

Alex May­weg

The re­search so far al­so in­di­cates that while cer­tain tu­mors car­ry rare metabo­lites, there are oth­er metabo­lites that are present in a whole bas­ket of dif­fer­ent tu­mors and tis­sues. ‘We don’t want to say that we have the sil­ver bul­let against every­thing here, but it has a breadth that no oth­er T cell ther­a­py right now re­al­ly has,” Alex May­weg, man­ag­ing di­rec­tor at Ver­sant and a board mem­ber at Mat­ter­horn, said.

The plan now is to go through the col­lec­tion of MR1 T cells and re­cep­tors they’ve as­sem­bled and nom­i­nate a lead clin­i­cal can­di­date lat­er this year, aim­ing to be ready for the clin­ic in ear­ly 2022. Mean­while, the Ridge­line team will fade out as Mat­ter­horn grows its in­ter­nal pay­roll to 15 to 20 by the end of this year.

Biotech in­vestors and CEOs see two paths to growth, but are they equal­ly vi­able?

The dynamic in the biotech market has been highly volatile in the last few years, from the high peaks immediately after the COVID vaccine in 2021, to the lowest downturns of the last 20 years in 2022. This uncertainty makes calling the exact timing of the market’s turn something of a fool’s errand, according to Dr. Chen Yu, Founder and Managing Partner of TCG Crossover (TCG X). He speaks with RBC’s Noël Brown, Head of US Biotechnology Investment Banking, about the market’s road ahead and two possible paths for growth.

Dave Marek, Myovant CEO

My­ovant board balks as ma­jor­i­ty own­er Sum­it­o­mo swoops in with a $2.5B deal to buy them out

Three years after Sumitomo scooped up Roivant’s 46% stake in the publicly traded Myovant $MYOV as part of a 5-company, $3 billion deal, they’re coming back for the whole thing.

But these other investors at Myovant want more than what the Japanese pharma company is currently offering to pay at this stage.

Sumitomo is bidding $22.75 a share for the outstanding stock, which now represents 48% of the company after Sumitomo bumped its ownership since the original deal with Roivant. Myovant, however, created a special committee on the board, and they’re shaking their heads over the offer.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 150,300+ biopharma pros reading Endpoints daily — and it's free.

Justin Klee (L) and Joshua Cohen, Amylyx co-CEOs (Cody O'Loughlin/The New York Times; courtesy Amylyx)

Ad­vo­cates, ex­perts cry foul over Amy­lyx's new ALS drug, cit­ing is­sues with price, PhI­II com­mit­ment

Not 24 hours after earning the first ALS drug approval in five years, Amylyx Pharmaceuticals’ Relyvrio is already drawing scrutiny. And it’s coming from multiple fronts.

In an investor call Friday morning, Amylyx revealed that it would charge about $158,000 per year, a price point that immediately drew backlash from ALS advocates and some outside observers. The cost reveal had been highly anticipated in the immediate hours after Thursday evening’s approval, though Amylyx only teased Relyvrio would cost less than previously approved drugs.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 150,300+ biopharma pros reading Endpoints daily — and it's free.

Albert Bourla, Pfizer CEO (Gian Ehrenzeller/Keystone via AP)

Can a smart­phone app de­tect Covid? Pfiz­er throws down $116M to find out

What can a cough say about a patient’s illness? Quite a bit, according to ResApp Health — and Pfizer’s listening.

The pharma giant is shelling out about $116 million ($179 million AUD) to scoop up the University of Queensland spinout and its smartphone technology that promises to diagnose Covid and other respiratory illnesses based on cough and breathing sounds, the university announced last week.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 150,300+ biopharma pros reading Endpoints daily — and it's free.

Big Phar­ma heavy­weights seek tweaks to FDA's clin­i­cal out­come as­sess­ment guid­ance

Pfizer, GSK, Janssen, Regeneron, Boehringer Ingelheim and at least a half dozen other companies are calling on the FDA to provide significantly more clarity in its draft guidance from this summer on clinical outcome assessments, which are a type of patient experience.

The draft is the third in a series of four patient-focused drug development guidance documents that the FDA had to create as part of the 21st Century Cures Act, and they describe how stakeholders (patients, caregivers, researchers, medical product developers and others) can collect and submit patient experience data and other relevant information for medical product development and regulatory decision-making.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 150,300+ biopharma pros reading Endpoints daily — and it's free.

Rob Etherington, Clene CEO

Star­tup's gold nanocrys­tal ALS drug flops a PhII tri­al, a re­minder of the dis­ease's ob­sta­cles de­spite Amy­lyx OK

Despite the FDA approving an ALS drug for the first time in five years last week, the disease continues to fluster researchers, and another biotech is feeling the pain of a mid-stage failure.

Clene Nanomedicine reported early Monday that its ALS program, which uses gold nanocrystals to try to catalyze intracellular reactions, did not achieve its Phase II primary or secondary endpoints. And in a press release, the company noted for the first time that it’s speaking with “potential strategic partners” about the program — language that typically indicates a biotech is preparing to sell off an asset.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 150,300+ biopharma pros reading Endpoints daily — and it's free.

Marc Dunoyer, Alexion CEO (AstraZeneca via YouTube)

Up­dat­ed: As­traZeneca nabs a small rare dis­ease gene ther­a­py play­er for 667% pre­mi­um

AstraZeneca is kicking off the fourth quarter with a little M&A Monday for a gene editing player recently overcoming a second clinical hold to its only program in human studies.

The Big Pharma and its subsidiary Alexion are buying out little LogicBio for $2.07 per share. That’s good for a massive 667% premium over its Friday closing price, when it headed into the weekend at 27 cents and just weeks after Nasdaq said LogicBio would have to delist, which has been put on hold as the biotech requests a hearing. It’s one of two biotech deals to commence October, alongside the news of Incyte buying a vitiligo-focused biotech.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 150,300+ biopharma pros reading Endpoints daily — and it's free.

Land­mark Amy­lyx OK spurs de­bate; Some... pos­i­tive? Alzheimer's da­ta; Can­cer tri­al bot­tle­neck; Sanofi's CRISPR bet; 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.

After brief stops in Paris and Boston, John Carroll and the Endpoints crew are staying on the road in October with their return for a live/streaming EUBIO22 in London. The hybrid event fireside chats and panels on mRNA, oncology and the crazy public market. We hope you can join him there.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 150,300+ biopharma pros reading Endpoints daily — and it's free.

An­oth­er warn­ing let­ter for Lupin as FDA iden­ti­fies de­fi­cien­cies at In­dia-based site

With few new details of what needs fixing, Lupin disclosed last week that the FDA recently sent a warning letter to its Tarapur, India-based site.

After an inspection from March 22 to April 4, Lupin disclosed in an April stock filing that it received a Form 483 with four observations, but it didn’t offer any details on the observations.

Similar to comments made in April, the company said last week it does not believe the FDA slap will disrupt its drug supplies or the existing revenues from operations of this facility.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 150,300+ biopharma pros reading Endpoints daily — and it's free.