Mesoblast gets a $110M life­line from Surg­Cen­ter De­vel­op­ment; uniQure still un­sure if gene ther­a­py spurred can­cer event

Mesoblast faced rough wa­ters in 2020, but on Mon­day were thrown a fi­nan­cial life­line.

The Aus­tralian stem cell ther­a­py play­er has raised $110 mil­lion in a pri­vate place­ment, the com­pa­ny an­nounced, of­fer­ing 60 mil­lion shares to the US in­vestor group Surg­Cen­ter De­vel­op­ment. Surg­Cen­ter re­ceived the shares at a 6.5% dis­count from Mesoblast’s clos­ing price on Feb. 25.

Mesoblast plans to use the funds to boost sup­ply of its lead can­di­date remestem­cel-L ahead of what they hope is a po­ten­tial ap­proval in pe­di­atric GvHD when they re­turn to the FDA, as well as ad­vanc­ing man­u­fac­tur­ing and de­vel­op­ment of their rexleme­stro­cel-L plat­form for chron­ic heart fail­ure and chron­ic low back pain.

US reg­u­la­tors turned away remestem­cel-L last Oc­to­ber af­ter ob­ject­ing to the com­pa­ny’s tri­al de­sign. The com­pa­ny sub­mit­ted its ap­pli­ca­tion for the ex­per­i­men­tal drug on the ba­sis of one sin­gle-arm, open-la­bel study rather than a ran­dom­ized tri­al, even though it demon­strat­ed a sta­tis­ti­cal­ly sig­nif­i­cant ben­e­fit in its pri­ma­ry end­point against the his­tor­i­cal con­trol rate.

Mesoblast al­so spun a rexleme­stro­cel-L miss as a win in heart fail­ure in De­cem­ber, not­ing that while the ex­per­i­men­tal cell ther­a­py did not sig­nif­i­cant­ly re­duce hos­pi­tal­iza­tions over place­bo in in­di­vid­u­als with re­cur­ring heart fail­ure, the pro­gram man­aged to hit sec­on­daries in mor­tal­i­ty-rate re­duc­tion for ear­li­er stages of the dis­ease.

uniQure still un­sure if its gene ther­a­py spurred liv­er can­cer case

Af­ter the FDA placed a hold on uniQure’s he­mo­phil­ia B gene ther­a­py back in De­cem­ber due to a case of liv­er can­cer, the com­pa­ny still isn’t sure whether or not the event is re­lat­ed to the treat­ment.

In a 10-K fil­ing sub­mit­ted to the SEC on Mon­day, uniQure dis­closed that while they don’t think the event will de­lay their re­sponse to reg­u­la­tors or their even­tu­al BLA, they’re still un­able to de­ter­mine a “pos­si­ble causal re­la­tion­ship.” UniQure cit­ed a lack of ad­e­quate da­ta.

Re­searchers col­lect­ed sam­ples from the liv­er le­sion in ear­ly 2021 af­ter de­tect­ing it in an ul­tra­sound, the com­pa­ny said, and in mid-Feb­ru­ary, they re­port­ed the analy­sis of the le­sion to the FDA.

No oth­er cas­es of liv­er can­cer were re­port­ed in the group of 67 pa­tients.

Fol­low­ing sev­er­al gene ther­a­py sna­fus, the FDA is tak­ing a cau­tious ap­proach to the med­i­cines. Sol­id Bio has had its Duchenne mus­cu­lar dy­s­tro­phy pro­gram in and out of a clin­i­cal hold, while Bio­Marin faced a ma­jor de­lay on he­mo­phil­ia A as reg­u­la­tors de­mand­ed more da­ta to demon­strate dura­bil­i­ty.

And last month, blue­bird bio sus­pend­ed its work on its ear­ly-stage study as well as the Phase III sick­le cell tri­al for Lenti­Glo­bin in or­der to in­ves­ti­gate two more se­ri­ous side ef­fects among pa­tients in the pro­gram.

Promi­nent French in­vestors launch new in­cu­ba­tor

The French gov­ern­ment and a promi­nent in­vest­ment firm in the coun­try are team­ing up to start a new in­cu­ba­tor.

Kur­ma Part­ners and the French na­tion­al bank Bpifrance are lead­ing the way for Ar­go­b­io, which will aim to launch at least five biotechs over the next five years. Ar­go­b­io is get­ting start­ed with €50 mil­lion, and al­so saw fund­ing from An­geli­ni Phar­ma, Evotec and the In­sti­tut Pas­teur.

Re­searchers’ fo­cus at Ar­go­b­io will span rare dis­eases, neu­ro­log­i­cal dis­or­ders, on­col­o­gy, and im­munol­o­gy, while al­so look­ing to de­vel­op plat­form tech­nolo­gies they feel could be promis­ing.

“Ar­go­b­io rep­re­sents the re­al­iza­tion of a long­stand­ing idea at Kur­ma,” Kur­ma part­ner Pe­ter Neubeck said in a state­ment.

BY­OD Best Prac­tices: How Mo­bile De­vice Strat­e­gy Leads to More Pa­tient-Cen­tric Clin­i­cal Tri­als

Some of the most time- and cost-consuming components of clinical research center on gathering, analyzing, and reporting data. To improve efficiency, many clinical trial sponsors have shifted to electronic clinical outcome assessments (eCOA), including electronic patient-reported outcome (ePRO) tools.

In most cases, patients enter data using apps installed on provisioned devices. At a time when 81% of Americans own a smartphone, why not use the device they rely on every day?

Voting in the 2020 election (AP Images)

The right to vote is fun­da­men­tal — a let­ter from biotech­nol­o­gy in­dus­try lead­ers

Biotech Voices is a collection of exclusive opinion editorials from some of the leading voices in biopharma on the biggest industry questions today. Think you have a voice that should be heard? Reach out to senior editors Kyle Blankenship and Amber Tong.

We oppose all attempts to introduce laws that reduce the rights of US citizens to vote or that restrict them from exercising that right. The right to vote is fundamental to democracy. States that have enacted, or are proposing to enact, legislation to restrict voting are undermining our democracy and posing a threat to our nation. As leaders of the life sciences industry, we stand for what we believe is right for our country, our enterprises, our employees and those who benefit from our work. We join the first groups of business leaders who have challenged these laws and will continue to make our collective voices heard on this matter.

Pascal Soriot (AstraZeneca via YouTube)

Af­ter be­ing goad­ed to sell the com­pa­ny, Alex­ion's CEO set some am­bi­tious new goals for in­vestors. Then Pas­cal So­ri­ot came call­ing

Back in the spring of 2020, Alexion $ALXN CEO Ludwig Hantson was under considerable pressure to perform and had been for months. Elliott Advisers had been applying some high public heat on the biotech’s numbers. And in reaching out to some major stockholders, one thread of advice came through loud and clear: Sell the company or do something dramatic to change the narrative.

In the words of the rather dry SEC filing that offers a detailed backgrounder on the buyout deal, Alexion stated: ‘During the summer and fall of 2020, Alexion also continued to engage with its stockholders, and in these interactions, several stockholders encouraged the company to explore strategic alternatives.’

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Near­ly a year af­ter Au­den­tes' gene ther­a­py deaths, the tri­al con­tin­ues. What hap­pened re­mains a mys­tery

Natalie Holles was five months into her tenure as Audentes CEO and working to smooth out a $3 billion merger when the world crashed in.

Holles and her team received word on the morning of May 5 that, hours before, a patient died in a trial for their lead gene therapy. They went into triage mode, alerting the FDA, calling trial investigators to begin to understand what happened, and, the next day, writing a letter to alert the patient community so they would be the first to know. “We wanted to be as forthright and transparent as possible,” Holles told me late last month.

The brief letter noted two other patients also suffered severe reactions after receiving a high dose of the therapy and were undergoing treatment. One died a month and a half later, at which point news of the deaths became public, jolting an emergent gene therapy field and raising questions about the safety of the high doses Audentes and others were now using. The third patient died in August.

“It was deeply saddening,” Holles said. “But I was — we were — resolute and determined to understand what happened and learn from it and get back on track.”

Eleven months have now passed since the first death and the therapy, a potential cure for a rare and fatal muscle-wasting disease called X-linked myotubular myopathy, is back on track, the FDA having cleared the company to resume dosing at a lower level. Audentes itself is no more; last month, Japanese pharma giant Astellas announced it had completed working out the kinks of the $3 billion merger and had restructured and rebranded the subsidiary as Astellas Gene Therapies. Holles, having successfully steered both efforts, departed.

Still, questions about precisely what led to the deaths of the 3 boys still linger. Trial investigators released key details about the case last August and December, pointing to a biological landmine that Audentes could not have seen coming — a moment of profound medical misfortune. In an emerging field that’s promised cures for devastating diseases but also seen its share of safety setbacks, the cases provided a cautionary tale.

Audentes “contributed in a positive way by giving a painful but important example for others to look at and learn from,” Terry Flotte, dean of the UMass School of Medicine and editor of the journal Human Gene Therapy, told me. “I can’t see anything they did wrong.”

Yet some researchers say they’re still waiting on Astellas to release more data. The company has yet to publish a full paper detailing what happened, nor have they indicated that they will. In the meantime, it remains unclear what triggered the events and how to prevent them in the future.

“Since Audentes was the first one and we don’t have additional information, we’re kind of in a holding pattern, flying around, waiting to figure out how to land our vehicles,” said Jude Samulski, professor of pharmacology at UNC’s Gene Therapy Center and CSO of the gene therapy biotech AskBio, now a subsidiary of Bayer.

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Launched by MIT grads, a small start­up gets $20M to back a ro­bot­ics rev­o­lu­tion in cell ther­a­py man­u­fac­tur­ing

As co-director of an experimental cellular therapy process development and manufacturing group at UCSF specializing in T cell therapies for autoimmune conditions, Jonathan Esensten has learned a lot about the challenges involved when his group hand-fashions a cell therapy. Esensten — who was a postdoc in Wendell Lim’s lab and counts the legendary Jeffrey Bluestone as a mentor — gives them all high marks at being great at what they do, but time and again there are variations in the treatments they construct.

Anand Shah (FDA)

For­mer head of FDA’s med­ical and sci­en­tif­ic af­fairs on Covid: ‘FDA has nev­er been test­ed like this’

Anand Shah has served the American public in a unique way, crisscrossing over the last two administrations between serving as an attending radiation oncologist focused on prostate cancer at NIH, serving as CMO at the Center for Medicare and Medicaid Innovation, and most recently, leading the FDA’s operations on medical and scientific affairs from within the commissioner’s office.

Shah, who stepped down from the FDA in January, caught up with Endpoints News in a phone interview on Tuesday afternoon, offering his thoughts on the agency’s latest decision to pause the J&J vaccinations in the US, and reflecting on his time at an agency during this once-in-a-lifetime pandemic.

UP­DAT­ED: J&J paus­es vac­cine roll­out as feds probe rare cas­es of blood clots

The FDA and CDC have jointly decided to stop administering J&J’s Covid-19 vaccine after reviewing data involving six reported US cases of a rare and severe type of blood clot in individuals after receiving the vaccine.

CDC will convene a meeting of its Advisory Committee on Immunization Practices on Wednesday to further review these cases and assess their potential significance. “FDA will review that analysis as it also investigates these cases. Until that process is complete, we are recommending a pause in the use of this vaccine out of an abundance of caution,” Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research and Anne Schuchat, Principal Deputy Director of the CDC, said in a joint statement Tuesday morning.

Patrizia Cavazzoni, new CDER director

Pa­trizia Cavaz­zoni named per­ma­nent di­rec­tor of CDER, adding to ques­tions around where Wood­cock will end up

Patrizia Cavazzoni on Monday became the permanent director of the FDA’s Center for Drug Evaluation and Research, which puts to rest the idea that Janet Woodcock, Cavazzoni’s predecessor, might return to lead CDER if she isn’t made permanent commissioner.

Woodcock, who’s currently serving as acting commissioner and principal medical advisor to the commissioner, a position she was detailed to last year, may not make the move to permanent commissioner because of lingering questions from Senate Democrats. She previously served as director of CDER since 1994. Cavazzoni took over as acting director of CDER when Woodcock moved over to Operation Warp Speed to run the therapeutics side of the Trump-era program.

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Barbara Weber, Tango Therapeutics CEO (Tango)

It takes two to Tan­go: The biotech us­ing CRISPR to dis­cov­er new can­cer gene tar­gets rides a $353M SPAC deal to Nas­daq

Editor’s note: Interested in following biopharma’s fast-paced IPO market? You can bookmark our IPO Tracker here.

The latest biotech-SPAC deal has arrived, and it’s dancing its way to Nasdaq to the tune of several hundred million dollars.

Tango Therapeutics and its CRISPR-focused search for new cancer genes is reverse merging with Boxer Capital’s blank-check company, the biotech announced Wednesday morning. With a spotlight on three lead programs, Tango expects total proceeds to equal about $353 million in the deal, which includes the roughly $167 million held in the SPAC and an additional $186 million in PIPE financing.