Here's your in­vi­ta­tion to this year's End­points 11 awards. Let's cel­e­brate the best of biotech to­geth­er

Over in the pub­lic mar­ket, we’re watch­ing the wheat be­ing re­morse­less­ly sep­a­rat­ed from the chaff as the biotech sec­tor went from every­one’s dar­ling to the or­phan child of Wall Street. That was in­evitable. But while there’s been a knock-on ef­fect in the ven­ture in­vest­ing side, we’re see­ing clear ev­i­dence that big play­ers are stick­ing with their game plan: Ma­jor tech­no­log­i­cal break­throughs are open­ing doors to biotech star­tups that didn’t ex­ist five years ago — and the so­phis­ti­cat­ed in­vestors don’t want to miss out.

It’s against that dra­mat­ic back­drop that I picked this year’s End­points 11 — eleven up-and-com­ing pri­vate biotechs that are promis­ing to make some rad­i­cal al­ter­ations in the ways in which dis­eases are treat­ed, or some­times pre­vent­ed.

They’re all mak­ing some big promis­es, and we all know that biotechs can’t keep all the promis­es they make. But that’s part of what makes this busi­ness so ex­cit­ing.

Over the past 10 years, we watched the de-risk­ing strat­e­gy get dis­card­ed in drug de­vel­op­ment as the move to swing for the fences be­came pro­gres­sive­ly more ir­re­sistible. And along the way we’ve added Mod­er­na and BioN­Tech to the list of biotechs that have gone from doubt­ed mR­NA rev­o­lu­tion­ar­ies to con­firmed mar­ket dis­rup­tors with tens of bil­lions in new rev­enue to show for it.

I don’t know which of these biotechs on the 2022 list will make it in the long run. Some­times the tim­ing is all wrong. Some­times it all just doesn’t work as billed. Sev­er­al of these play­ers are bet­ting big on drugs that were tried and dis­card­ed. They are go­ing to be right or they are go­ing to be wrong.

But I do know one thing: They all rep­re­sent what is best about the biotech in­dus­try. They are run by some of the top peo­ple in the busi­ness, with a track record for suc­cess. They all have big dreams. And we’ll be cel­e­brat­ing that pi­o­neer­ing spir­it in a so­cial gath­er­ing at The 105 in Sea­port the evening of Sep­tem­ber 22.

You are in­vit­ed to join us for an evening of net­work­ing and cel­e­bra­tion as we hand out this year’s awards, with a spe­cial fire­side chat with No­bel win­ner Phil Sharp — one of the sci­en­tif­ic greats who co-found­ed the biotech in­dus­try. I hope to see you there, tick­ets per­mit­ting. You can get your tick­et here.

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.

Casey McPherson shows his daughters Rose (left) and Weston around Everlum Bio, a lab that he co-founded to spark a treatment for Rose and others with ultra-rare conditions. (Ilana Panich-Linsman)

Fa­ther starts lab af­ter in­tel­lec­tu­al prop­er­ty is­sues stymie rare dis­ease drug de­vel­op­ment

Under bright lab lights, Casey McPherson holds his 6-year-old daughter, Rose. His free hand directs Rose’s gaze toward a computer screen with potential clues in treating her one-of-a kind genetic condition.

Gray specks on the screen show her cells that scientists reprogrammed with the goal of zeroing in on a custom medicine. McPherson co-founded the lab, Everlum Bio, to spark a treatment for Rose — and others like her. A regarded singer-songwriter, McPherson never imagined going into drug development.

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Benjamine Liu, TrialSpark CEO

Paul Hud­son and Tri­alSpark's mu­tu­al de­sire to speed up de­vel­op­ment con­verges in three-year, six-drug goal

A unicorn startup that originally set out to hasten clinical studies for biopharma partners dug further into its revised path of internal drug development by linking arms with Sanofi in a pact that the biotech’s CEO said originated from the top.

TrialSpark and the Big Pharma on Tuesday committed to in-licensing and/or acquiring six Phase II/Phase III drugs within the next three years.

“I’ve known Paul Hudson for a while and we were discussing the opportunity to really re-imagine a lot of different parts of pharma,” TrialSpark CEO Benjamine Liu told Endpoints News, “and one of the things that we discussed was this opportunity to accelerate the development of new medicines in mutual areas of interest.”

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New Chroma Medicine board member Jeff Marrazzo

Jeff Mar­raz­zo has found a buzzy new biotech cause to cham­pi­on. And once again, he's all in

Jeff Marrazzo is one of those biotech execs who has always been focused on the next big goal. He has a track record for meeting objectives, relentlessly staying on message, and breaking new ground.

The fact that he stayed around for a couple of years after Roche’s $4.3 billion Spark buyout, making sure the organization he founded weathered Covid-19, is one example. And that came after he carefully guided the company to the first-ever US approval of a gene therapy — no easy task.

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Take­da to pull key hy­poparathy­roidism drug from the mar­ket en­tire­ly by end of 2024 af­ter years of man­u­fac­tur­ing woes

Takeda on Tuesday morning made an announcement that almost 3,000 people with the rare disease known as hypoparathyroidism were fearing.

Due to unresolved supply issues and manufacturing woes, Takeda said it will cut its losses and discontinue its hypoparathyroidism drug, known as Natpara (parathyroid hormone), halting all manufacturing of the drug by the end of 2024.

The decision to not re-commercialize Natpara will be a blow to not only the 2,400 people who were awaiting supplies of their reliable injection since 2019, but also the additional nearly 400 people who were accessing the drugs via the company’s Special Use Program as Takeda sought to resolve these manufacturing issues over the past five years.

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.

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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.

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Astel­las, Pan­th­er­na add or­gan to mR­NA tie-up; Rock­et launch­es sale of six fig­ures worth of stock

Astellas and Pantherna have expanded their November 2021 pact surrounding the latter’s mRNA platform to include a new target organ, the duo announced Tuesday morning, though they did not specify what that target is.

German biotech Pantherna is home to two platform technologies — one that designs mRNAs for non-vaccine therapies and another that designs LNPs. Astellas and Pantherna’s deal appears to mainly revolve around the first platform, which Astellas said it is using to research direct reprogramming, or turning cells from one kind into another without an intermediate stem cell phase.

Andrew Crockett, KalVista CEO

KalVista ends a PhII study ear­ly af­ter pa­tients suf­fer se­vere and life-threat­en­ing side ef­fects

KalVista took a beating Tuesday after announcing it would scrap a Phase II trial for one of its experimental drugs.

The biotech said in an early morning press release that it is terminating the study for KVD824 after multiple patients in every treatment group saw unsafe, elevated levels of certain liver enzymes. By ending the trial now, KalVista hopes to save some money and funnel it toward another study for its lead program, CEO Andrew Crockett said in a statement.