Renee Wegrzyn (AIChE)

Biden turns to biotech for his in­au­gur­al ARPA-H di­rec­tor

Pres­i­dent Joe Biden ear­ly Mon­day un­veiled his in­tent to ap­point Re­nee We­grzyn, VP of busi­ness de­vel­op­ment at Gink­go Bioworks, as the first di­rec­tor of the Ad­vanced Re­search Pro­jects Agency for Health (ARPA-H), a new NIH-housed agency that looks to fill R&D gaps and go a step fur­ther than the NIH to at­tack riski­er, po­ten­tial­ly more re­ward­ing bio­med­ical in­no­va­tion.

We­grzyn is a prac­ti­cal choice as she’s spent time in in­dus­try and at both of the in­sti­tu­tions that in­spired the cre­ation of ARPA-H – the De­fense Ad­vanced Re­search Pro­jects Agency (DARPA) and In­tel­li­gence Ad­vanced Re­search Pro­jects Ac­tiv­i­ty. At DARPA, she re­ceived a ser­vice medal for her work, and pri­or to that, We­grzyn spent sev­en years at Booz Allen Hamil­ton as a tech­ni­cal and strat­e­gy ad­vi­sor to DARPA, and al­most five years as an ad­vi­sor to the Nu­clear Threat Ini­tia­tive.

With ex­pe­ri­ence lead­ing teams de­vel­op­ing gene ther­a­pies, and work­ing on emerg­ing in­fec­tious dis­eases, neu­ro­mod­u­la­tion, and syn­thet­ic bi­ol­o­gy, We­grzyn will join ARPA-H’s in­au­gur­al em­ploy­ee, Adam Rus­sell, who’s serv­ing as act­ing deputy di­rec­tor.

De­tails of what ex­act­ly ARPA-H will fo­cus on ini­tial­ly have been few and far be­tween, but ac­cord­ing to the Con­gres­sion­al Re­search Ser­vice, the agency will work on tech­nolo­gies “that could have ap­pli­ca­tions across a wide range of dis­eases, rather than fo­cus on spe­cif­ic dis­eases,” and avoid ar­eas that are al­ready well-fund­ed by NIH or the pri­vate sec­tor.

But ARPA-H is go­ing to have to fight to grow its bud­get. Biden ini­tial­ly sought $6.5 bil­lion for the new life sci­ence in­cu­ba­tor over three years, but Con­gress has on­ly al­lot­ted $1 bil­lion so far, and while the House has sought $2.75 bil­lion more in next year’s bud­get, avail­able un­til Sept. 30, 2025, the Sen­ate has on­ly sought an­oth­er $1 bil­lion.

A cloud of dis­agree­ment al­so has emerged be­tween those who want it to be housed in but in­de­pen­dent from NIH (the cur­rent ad­min­is­tra­tion), and those that want it to be en­tire­ly in­de­pen­dent (Rep. An­na Es­hoo’s House bill passed this sum­mer and made clear that ARPA-H should be in­de­pen­dent).

A month ear­li­er, Sec­re­tary Xavier Be­cer­ra for­mal­ly an­nounced the es­tab­lish­ment of ARPA-H as an in­de­pen­dent en­ti­ty with­in the NIH, as HHS had pre­vi­ous­ly stip­u­lat­ed that “NIH may not sub­ject ARPA-H to NIH poli­cies.”

Lat­er to­day, on the 6oth an­niver­sary of Pres­i­dent John F. Kennedy’s Moon­shot speech, We­grzyn will join Biden at the John F. Kennedy Pres­i­den­tial Li­brary in Boston as he dis­cuss­es his vi­sion for “end­ing can­cer as we know it.”

The ad­di­tion of We­grzyn is al­so an­oth­er top fe­male sci­en­tist ap­point­ment by Biden, as he’s re­cent­ly nom­i­nat­ed Arati Prab­hakar to serve as the next di­rec­tor of the Of­fice of Sci­ence and Tech­nol­o­gy Pol­i­cy (OSTP), which is a Sen­ate-con­firmed po­si­tion, to re­place the Broad In­sti­tute’s Er­ic Lan­der, who left OSTP af­ter de­mean­ing his col­leagues. Biden al­so named Alon­dra Nel­son as OSTP’s Deputy Di­rec­tor for Sci­ence and So­ci­ety in Feb­ru­ary.

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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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.

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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.