AveX­is wins the in­side track at the FDA with gene ther­a­py ‘break­through’

Chica­go-based AveX­is has lined up the lat­est break­through ther­a­py des­ig­na­tion from the FDA, gain­ing an in­side track at the agency for its ear­ly-stage gene ther­a­py for lethal cas­es of spinal mus­cu­lar at­ro­phy in chil­dren. The biotech says it won that mark of dis­tinc­tion af­ter reg­u­la­tors got a pre­lim­i­nary look at the re­sults from their Phase I study.

AveX­is was able to score a $95 mil­lion IPO last Feb­ru­ary as it joined the first wave of biotechs to hit the pub­lic mar­ket this year, a lit­tle af­ter the once red-hot gene ther­a­py field had be­gun to cool some­what. The of­fer­ing fol­lowed a $65 mil­lion crossover round last fall which was joined by Roche.

Like a num­ber of oth­er gene ther­a­py com­pa­nies, AveX­is in-li­censed vec­tor tech from Re­genX, which banked IP de­vel­oped by Penn’s James Wil­son, a pi­o­neer in gene ther­a­py work. Its study is be­ing done in col­lab­o­ra­tion with The Re­search In­sti­tute at Na­tion­wide Chil­dren’s Hos­pi­tal — which has a num­ber of gene ther­a­py projects un­der­way — and Ohio State Uni­ver­si­ty.

The next step for AveX­is: A Type B meet­ing with reg­u­la­tors to go over their de­vel­op­ment plans. The FDA set up the BTD ef­fort so it could help ac­cel­er­ate promis­ing drugs through the clin­ic. And now AveX­is should get VIP treat­ment and an open door when need­ed at the agency.

Roger Perlmutter, Merck R&D chief (YouTube)

UP­DAT­ED: Mer­ck makes a triple play on Covid-19: buy­ing out a vac­cine biotech, part­ner­ing on an­oth­er pro­gram and adding an an­tivi­ral to the mix

Merck is making a triple play in a sudden leap into the R&D campaign against Covid-19.

Tuesday morning the pharma giant simultaneously announced plans to buy an Austrian biotech that has been working on a preclinical vaccine candidate, added a collaboration on another vaccine with the nonprofit IAVI and inked a deal with Ridgeback Biotherapeutics on an early-stage antiviral.

The deal with IAVI covers recombinant vesicular stomatitis virus (rVSV) technology that is the basis for Merck’s successful Ebola Zaire virus vaccine. That’s going into the clinic later this year.

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The Advance Clinical leadership team: CEO Yvonne Lungershausen, Sandrien Louwaars - Director Business Development Operations, Gabriel Kremmidiotis - Chief Scientific Officer, Ben Edwards - Chief Strategy Officer

How Aus­tralia De­liv­ers Rapid Start-up and 43.5% Re­bate for Ear­ly Phase On­col­o­gy Tri­als

About Avance Clinical

Avance Clinical is an Australian owned Contract Research Organisation that has been providing high-quality clinical research services to the local and international drug development industry for 20 years. They specialise in working with biotech companies to execute Phase 1 and Phase 2 clinical trials to deliver high-quality outcomes fit for global regulatory standards.

As oncology sponsors look internationally to speed-up trials after unprecedented COVID-19 suspensions and delays, Australia, which has led the world in minimizing the pandemic’s impact, stands out as an attractive destination for early phase trials. This in combination with the streamlined regulatory system and the financial benefits including a very favourable exchange rate and the R & D cash rebate makes Australia the perfect location for accelerating biotech clinical programs.

As biotech IPOs siz­zle on vir­tu­al Wall Street, 3 new play­ers roll the dice on meg­a­money gam­bles top­ping $325M

Back in early January when I interviewed Generation Bio CEO Geoff McDonough on his $110 million mega-raise, he was thinking in terms of taking another 12 to 18 months to get into the clinic and then filing an IPO. They were, after all, still preclinical after 4 years in the lab.

But with investors still clearly focused on biotech during the pandemic, a lot of things are going faster now. Including IPOing, which is sizzling for the right companies.

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Ax­o­vant spin­out Arvelle ups Se­ries A haul, de­ploy­ing $200M-plus to com­mer­cial­ize epilep­sy drug in Eu­rope

One of the characteristic features of the sprawling biotech group Vivek Ramaswamy has built at Roivant is its ability to whip up big-money financing deals for its subsidiaries. Axovant was a prime example, raising $315 million in an IPO — a monster by 2015 and even today’s standards — with an Alzheimer’s pitch before a spectacular flop forced the company to replot its course directly and pivot to gene therapy.

Piv­otal myas­the­nia gravis da­ta from ar­genx au­gur well for FcRn in­hibitors in de­vel­op­ment

Leading the pack of biotechs vying for a piece of the generalized myasthenia gravis (gMG) market with an FcRn inhibitor, argenx on Tuesday unveiled keenly anticipated positive late-stage data on its lead asset, bringing it one step closer to regulatory approval.

Despite steroids, immunosuppressants, acetylcholinesterase inhibitors, and Alexion’s Soliris, patients with the rare, chronic neuromuscular disorder (more than 100,000 in the United States and Europe) don’t necessarily benefit from these existing options, leaving room for the crop of FcRn inhibitors in development.

Af­ter de­cou­pling from Re­gen­eron, Sanofi says it’s time to sell the $13B stake picked up in the mar­riage

With Regeneron shares going for a peak price — after doubling from last fall — Sanofi is putting a $13 billion stake in their longtime partner on the auction block. And Regeneron is taking $5 billion of that action for themselves.

Sanofi — which has been decoupling from Regeneron for more than a year now — bought in big in early 2013, back when Regeneron’s stock was going for around $165 a share. Small investors flocked to the deal, buzzing about an imminent takeover. The buyout chatter wound down long ago.

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Covid-19 roundup: Janet Wood­cock steps aside — for now — as FDA drug czar; WHO hits the brakes on hy­droxy study af­ter lat­est safe­ty alarm

The biopharma industry will soon get a look at what the FDA will look like once CDER’s powerful chief Janet Woodcock retires from her post.

Long considered one of the most influential regulators in the agency, if not its single most powerful official when it counts, Woodcock is being detached to devote herself full-time to the White House’s special project to fast-forward new drugs and vaccines for the pandemic. The move comes a week after some quick reshuffling as Woodcock and CBER chief Peter Marks joined Operation Warp Speed. Initially they opted to recuse themselves from any FDA decisions on pandemic treatments and vaccines, after consumer advocates criticized the move as a clear conflict of interest in how the agency exercises oversight on new approvals.

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Andrew Hopkins, Exscientia founder and CEO (Exscientia)

Af­ter years of part­ner­ships, AI biotech Ex­sci­en­tia lands first ma­jor fi­nanc­ing round at $60M

After years racking up partnerships with biotechs and Big Pharma, the AI drug developer Exscientia has landed its first large financing round.

The UK-based company raised $60 million in a Series C round led by Novo Holdings — more than double the $26 million it garnered in a Series B 18 months ago. The round will help further the company’s expansion into the US and further what it calls, borrowing a term from the software world, its “full-stack capabilities,” i.e. its ability to develop drugs from the earliest stage to the market.

Janet Woodcock, director of the Center for Drug Evaluation and Research (AP Images)

Covid-19 roundup: Hit with new con­flict ac­cu­sa­tions, Janet Wood­cock steps out of the agen­cy's Covid-19 chain of com­mand

Two weeks ago, FDA drug chieftain Janet Woodcock was assuring a top Wall Street analyst that any vaccine approved for combating Covid-19 would have to meet high agency standards on safety and efficacy before it’s approved. But over the weekend, after she and Peter Marks took top positions with the public-private operation meant to speed a new vaccine to lightning-fast approvals — they both recused themselves from the review process after an advocacy group argued their roles close to the White House could pose a conflict of interest.

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