‘There was a grow­ing weari­ness’: Rush­ing against a pan­dem­ic clock, As­pen Neu­ro­sciences se­cures $70M Se­ries A

Howard Federoff

Just be­fore Christ­mas­time, Howard Federoff got a tip from Wash­ing­ton: There was a new virus in Chi­na. And this one could be bad.

News re­port of the virus had not yet ap­peared. Federoff, a neu­ro­sci­en­tist, was briefed be­cause years be­fore, he was vet­ted as part of a group — he didn’t give a name for the group — to con­sult for the US gov­ern­ment on emerg­ing sci­en­tif­ic is­sues. His day job, though, was CEO of As­pen Neu­ro­sciences, a Parkin­son’s cell ther­a­py start­up that days be­fore had come out of stealth mode and gave word to in­vestors they were hop­ing to raise $70 mil­lion. That, Federoff re­al­ized, would be dif­fi­cult if a pan­dem­ic shut down the glob­al econ­o­my.

“I start­ed think­ing rather ear­ly on…There might be some­thing on the hori­zon that we don’t ful­ly un­der­stand,” Federoff told End­points News. “We knew that if some­thing did change, it could change rather quick­ly.”

Op­er­at­ing with in­sight and knowl­edge oth­er biotechs lacked ac­cess to, Federoff went in­to over­drive try­ing to close be­fore Covid-19 hit the US, and he emerged to­day with $70 mil­lion in Se­ries A fund­ing led by Or­biMed. The oth­er in­vestors in­clud­ed Fra­zier Health Part­ners, Sam Alt­man and ARCH Ven­ture Part­ners, the VC whose leader Robert Nelsen be­came one of the ear­li­est and most promi­nent voic­es call­ing for change.

”We’ve had long con­ver­sa­tions,” Federoff said of him and Nelsen.

With the Se­ries A, Federoff has con­vinced A-list in­vestors to back one ver­sion of a long-sought so­lu­tion to Parkin­son’s. As­pen will use stem cells grown from Parkin­son’s pa­tients’ own skin tis­sue to grow dopamine neu­rons that can be im­plant­ed in­to the brain and hope­ful­ly re­place the de­gen­er­at­ing neu­rons. The idea has been around for decades, with the first trans­plant oc­cur­ring in the 80s, but it was nev­er scaleable. The tech­nol­o­gy to pro­duce stem cells on de­mand didn’t ex­ist.

The com­pa­ny has a ri­val in Blue­Rock, which us­es donor stem cells and which Bay­er ac­quired in Au­gust at a val­u­a­tion of $1 bil­lion.

Over the win­ter, though, the in­vestor hunt be­came less about pitch­ing the sci­ence — which Federoff says every­one agreed was promis­ing — than about beat­ing the clock and in­vestors’ ris­ing wor­ries about the econ­o­my. He pre­pared to work fast, turn­ing an ear­ly meet­ing with Fra­zier at the JP Mor­gan Health­care Con­fer­ence in­to a piv­otal one. As the months passed, he phoned in­vestors mul­ti­ple times a day to keep fund­ing on track.

“They were al­ready in from the stand­point of the sci­ence,” Federoff said. “I could tell that there was a grow­ing weari­ness about whether all that they had pre­vi­ous­ly con­sid­ered as part of their own re­spec­tive port­fo­lios out­side of As­pen would all be pos­si­ble.”

The mon­ey he se­cured will help fund their Phase I tri­al on Parkin­son’s and a sec­ond pro­gram that us­es a form of gene ther­a­py to im­plant stem cells that have a ge­net­ic mark­er for Parkin­son’s edit­ed out. The plan had been to start a tri­al in 2021, but Federoff knows there are no more guar­an­tees.

“At this time it’s not clear what Covid-19 will do to pro­jec­tions,” he said.

The Avance 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.

As­traZeneca trum­pets the good da­ta they found for Tagris­so in an ad­ju­vant set­ting for NSCLC — but many of the ex­perts aren’t cheer­ing along

AstraZeneca is rolling out the big guns this evening to provide a salute to their ADAURA data on Tagrisso at ASCO.

Cancer R&D chief José Baselga calls the disease-free survival data for their drug in an adjuvant setting of early stage, epidermal growth factor receptor-mutated NSCLC patients following surgery “momentous.” Roy Herbst, the principal investigator out of Yale, calls it “transformative.”

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Pablo Legorreta, founder and CEO of Royalty Pharma AG, speaks at the annual Milken Institute Global Conference in Beverly Hills, California (Patrick T. Fallon/Bloomberg via Getty Images)

Cap­i­tal­iz­ing Pablo: The world’s biggest drug roy­al­ty buy­er is go­ing pub­lic. And the low-key CEO di­vulges a few se­crets along the way

Pablo Legorreta is one of the most influential players in biopharma you likely never heard of.

Over the last 24 years, Legorreta’s Royalty Pharma group has become, by its own reckoning, the biggest buyer of drug royalties in the world. The CEO and founder has bought up a stake in a lengthy list of the world’s biggest drug franchises, spending $18 billion in the process — $2.2 billion last year alone. And he’s become one of the best-paid execs in the industry, reaping $28 million from the cash flow last year while reserving 20% of the cash flow, less expenses, for himself.

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Paul Hudson, Sanofi CEO (Getty Images)

Sanofi CEO Paul Hud­son has $23B burn­ing a hole in his pock­et. And here are some hints on how he plans to spend that

Sanofi has reaped $11.1 billion after selling off a big chunk of its Regeneron stock at $515 a share. And now everyone on the M&A side of the business is focused on how CEO Paul Hudson plans to spend it.

After getting stung in France for some awkward politicking — suggesting the US was in the front of the line for Sanofi’s vaccines given American financial support for their work, versus little help from European powers — Hudson now has the much more popular task of managing a major cash cache to pull off something in the order of a big bolt-on. Or two.

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Look­ing to move past a tri­al fi­as­co, Ipsen poach­es their new CEO from Sanofi

Ipsen has turned to another Paris-based biopharma company for its next CEO.

Sanofi Pasteur chief David Loew is making the journey to Ipsen, 5 months after David Meek jumped ship to run a startup in late-stage development.

Loew arrives as Ipsen works to get back on track with their rare bone disease drug palovarotene, picked up in the $1.3 billion Clementia buyout, which was slammed with a partial hold after researchers observed cases of “early growth plate closure” in patients under the age of 14. But they are pushing ahead with the over-14 crowd after writing down slightly more than half of its initial development.

Iron­wood kicks de­layed-re­lease Linzess for­mu­la­tion to the curb af­ter tri­al fail­ure

The delayed-release formulation of Ironwood and Allergan’s bowel drug Linzess will not see the light of day.

The experimental drug, MD-7246, failed to help patients with abdominal pain associated with irritable bowel syndrome with diarrhea (IBS-D) in a mid-stage study, prompting the partners to abandon the therapy.

First approved in 2012, Linzess (known chemically as linaclotide) enhances the activity of the intestinal enzyme guanylate cyclase-C to increase the secretion of intestinal fluid and then transit through the intestinal tract, as well as reduce visceral pain, to relieve pain and constipation associated with IBS.

Dan O'Day, Gilead CEO (Andrew Harnik, AP Images)

UP­DAT­ED: Gilead leas­es part­ner rights to TIG­IT, PD-1 in a $2B deal with Ar­cus. Now comes the hard part

Gilead CEO Dan O’Day has brokered his way to a PD-1 and lined up a front row seat in the TIGIT arena, inking a deal worth close to $2 billion to align the big biotech closely with Terry Rosen’s Arcus. And $375 million of that comes upfront, with cash for the buy-in plus equity, along with $400 million for R&D and $1.22 billion in reserve to cover opt-in payments and milestones..

Hotly rumored for weeks, the 2 players have formalized a 10-year alliance that starts with rights to the PD-1, zimberelimab. O’Day also has first dibs on TIGIT and 2 other leading programs, agreeing to an opt-in fee ranging from $200 million to $275 million on each. There’s $500 million in potential TIGIT milestones on US regulatory events — likely capped by an approval — if Gilead partners on it and the stars align on the data. And there’s another $150 million opt-in payments for the rest of the Arcus pipeline.

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No­var­tis jumps in­to Covid-19 vac­cine hunt, as Big Phar­ma and big biotech com­mit to bil­lions of dos­es

After spending most of the pandemic on the sidelines, Novartis is offering its aid in the race to develop a Covid-19 vaccine.

AveXis, the Swiss pharma’s gene therapy subsidiary, has agreed to manufacture the vaccine being developed by Massachusetts Eye and Ear and Massachusetts General Hospital. The biotech will begin manufacturing this month, while the vaccine undergoes further preclinical testing. They’ve agreed to provide the vaccine for free for clinical trials beginning in the second half of 2020, but have not disclosed financials for after.

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Roger Perlmutter, Merck R&D chief (YouTube)

UP­DAT­ED: Backed by BAR­DA, Mer­ck jumps in­to Covid-19: buy­ing out a vac­cine, part­ner­ing on an­oth­er and adding an­tivi­ral to the mix

Merck execs are making a triple play in a sudden leap into the R&D campaign against Covid-19. And they have more BARDA cash backing them up on the move.

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.

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