Pick­ing up the pieces left from an Alzheimer’s im­plo­sion, Ax­o­vant is start­ing over by div­ing in­to gene ther­a­py

Af­ter get­ting blast­ed by the fail­ure of its lead drug for Alzheimer’s, Vivek Ra­maswamy’s Ax­o­vant $AX­ON is go­ing to try and blaze a new path for­ward by de­vel­op­ing a gene ther­a­py for Parkin­son’s.

Pa­van Cheru­vu

The biotech has un­veiled a deal in which it will hand over $30 mil­lion in cash to Ox­ford Bio­Med­ica for what will now be called AXO-Lenti-PD, a gene ther­a­py de­signed to spur dopamine pro­duc­tion in the brain. And to help fund the next phase, par­ent com­pa­ny Roivant is buy­ing $25 mil­lion worth of shares.

There’s a hefty set of mile­stones on the ta­ble for Ox­ford Bio­Med­ica, adding up to $812 mil­lion for their suc­cess. And Ax­o­vant ex­ecs say they’ll be ready to take this gene ther­a­py in­to the clin­ic be­fore the end of the year.

Ax­o­vant has a long way to go be­fore it can win back the trust of the in­vestors who lost big on its high-wire act on Alzheimer’s. But they are tak­ing the first steps in the jour­ney. And the in­vestors cheered on the move this morn­ing, with Ax­o­vant’s shares rock­et­ing up 160%. Ox­ford Bio­med­ica $OXB,mean­while, jumped 17% on the news.

“I re­al­ly see this as the start of pipeline ex­pan­sion,” Ax­o­vant CEO Pa­van Cheru­vu tells me.

“This” is a troi­ka of genes de­signed to turn cells in­to “dopamine fac­to­ries,” says the CEO. And it makes them one of the few biotechs in gene ther­a­py — along­side Voy­ager — to go af­ter a neu­ro­sciences tar­get.

They’ll be start­ing on this new drug sev­er­al years af­ter Ox­ford Bio­Med­ica pub­lished pos­i­tive re­sults from a small study of the gene ther­a­py, which they say demon­strat­ed its abil­i­ty to pro­vide a durable as­sis­tance to pa­tients.

Ax­o­vant im­plod­ed with spec­tac­u­lar ef­fect fol­low­ing the re­cent com­plete fail­ure of its lead ef­fort on Alzheimers, which has tak­en down one play­er af­ter the next that has tried to tack­le the dis­ease. CEO David Hung sub­se­quent­ly left, tak­ing his crew with him and wip­ing his hands of the en­tire com­pa­ny, which went through a painful re­struc­tur­ing. Now Cheru­vu is bring­ing in a new crew of his own to help lead the com­pa­ny in a new di­rec­tion.

Fras­er Wright

The CEO has re­cruit­ed Fras­er Wright — who had a se­nior role at gene ther­a­py pi­o­neer Spark Ther­a­peu­tics — as chief tech­nol­o­gy of­fi­cer. A for­mer Penn in­ves­ti­ga­tor, Wright is al­so a vet­er­an of the gene ther­a­py pro­gram at The Chil­dren’s Hos­pi­tal of Philadel­phia, which pro­vid­ed Spark with its ini­tial pipeline in gene ther­a­py.

He’ll be work­ing with oth­er new re­cruits, in­clud­ing ex-Te­va R&D chief Michael Hay­den, who is com­ing on board as a se­nior sci­en­tif­ic ad­vis­er at Roivant as well as head of Ax­o­vant’s sci­en­tif­ic ad­vi­so­ry board. Al­ler­gan $AGN CMO Gavin Cor­co­ran, mean­while, is jump­ing ship to be­come the new head of R&D at Ax­o­vant.

 

Biogen CEO Michel Vounatsos (via Getty Images)

With ad­u­canum­ab caught on a cliff, Bio­gen’s Michel Vounatsos bets bil­lions on an­oth­er high-risk neu­ro play

With its FDA pitch on the Alzheimer’s drug aducanumab hanging perilously close to disaster, Biogen is rolling the dice on a $3.1 billion deal that brings in commercial rights to one of the other spotlight neuro drugs in late-stage development — after it already failed its first Phase III.

The big biotech has turned to Sage Therapeutics for its latest deal, close to a year after the crushing failure of Sage-217, now dubbed zuranolone, in the MOUNTAIN study.

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Pascal Soriot (AP Images)

As­traZeneca, Ox­ford on the de­fen­sive as skep­tics dis­miss 70% av­er­age ef­fi­ca­cy for Covid-19 vac­cine

On the third straight Monday that the world wakes up to positive vaccine news, AstraZeneca and Oxford are declaring a new Phase III milestone in the fight against the pandemic. Not everyone is convinced they will play a big part, though.

With an average efficacy of 70%, the headline number struck analysts as less impressive than the 95% and 94.5% protection that Pfizer/BioNTech and Moderna have boasted in the past two weeks, respectively. But the British partners say they have several other bright spots going for their candidate. One of the two dosing regimens tested in Phase III showed a better profile, bringing efficacy up to 90%; the adenovirus vector-based vaccine requires minimal refrigeration, which may mean easier distribution; and AstraZeneca has pledged to sell it at a fraction of the price that the other two vaccine developers are charging.

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John Maraganore, Alnylam CEO (Scott Eisen/Bloomberg via Getty Images)

Al­ny­lam gets the green light from the FDA for drug #3 — and CEO John Maraganore is ready to roll

Score another early win at the FDA for Alnylam.

The FDA put out word today that the agency has approved its third drug, lumasiran, for primary hyperoxaluria type 1, better known as PH1. The news comes just 4 days after the European Commission took the lead in offering a green light.

An ultra rare genetic condition, Alnylam CEO John Maraganore says there are only some 1,000 to 1,700 patients in the US and Europe at any particular point. The patients, mostly kids, suffer from an overproduction of oxalate in the liver that spurs the development of kidney stones, right through to end stage kidney disease.

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Jason Kelly, Ginkgo Bioworks CEO (Kyle Grillot/Bloomberg via Getty Images)

Af­ter Ko­dak de­ba­cle, US lends $1.1B to a syn­thet­ic bi­ol­o­gy com­pa­ny and their big Covid-19, mR­NA plans

In mid-August, as Kodak’s $765 million government-backed push into drug manufacturing slowly fell apart in national headlines, Ginkgo Bioworks CEO Jason Kelly got a message from his company’s government liaison: HHS wanted to know if they, too, might want a loan.

The government’s decision to lend Kodak three quarters of a billion dollars raised eyebrows because Kodak had never made drugs before. But Ginkgo, while not a manufacturing company, had spent the last decade refining new ways to produce materials inside cells and building automated facilities across Boston.

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Bax­ter con­tin­ues on-shoring push with $50M In­di­ana ex­pan­sion

It’s been a banner year for the once humdrum business of manufacturing drugs, particularly vaccines. Billions have been spent ramping up facilities for Covid-19 jabs, while individual CDMOs have expanded their facilities, apparently anticipating demand or responding to a government-led push to onshore drug manufacturing.

Now Baxter Biopharma Solutions, the CDMO wing of the many-armed healthcare giant Baxter, is getting in on the game. On Tuesday, they announced plans to spend $50 million to expand their flagship, 600,000 square-foot facility in Bloomington, IN.

Eu­ro­pean Union aims to es­tab­lish patent workaround in case of emer­gen­cies while try­ing to strength­en its own IP

The European Union is looking at ways to bypass patent protections and make it easier to make generic drugs in cases of emergency such as the Covid-19 pandemic, a new document says.

Normally, under WTO regulations, the practice known as “compulsory licensing” is allowed in exceptional circumstances and could be applied as a waiver to bypass patent holders. Wednesday’s document was published as part of the EU’s plan to shore up the intellectual property rights of its member states.

Vivek Ramaswamy (Jeff Rumans/JPM 2020)

Urovan­t's lead drug dis­ap­points in mid-stage study as first big FDA de­ci­sion looms

Just as Urovant gets ready for its first big FDA decision on vibegron, the drug has flopped in what would’ve been a follow-on indication.

In a Phase IIa trial involving women with abdominal pain due to irritable bowel syndrome, vibegron failed to meet the bar on improving “average worst abdominal pain” over 12 weeks, compared to placebo, among IBS-D patients.

There were actually slightly more responders in the placebo group than in the drug arm, with only 40.9% of those randomized to vigebron achieving at least a 30% decrease in “worst abdominal pain” in the past 24 hours. The trial enrolled 222 women but only 189 completed the study.

Pur­due Phar­ma pleads guilty in fed­er­al Oxy­Con­tin probe, for­mal­ly rec­og­niz­ing it played a part in the opi­oid cri­sis

Purdue Pharma, the producer of the prescription painkiller OxyContin, admitted Tuesday that, yes, it did contribute to America’s opioid epidemic.

The drugmaker formally pleaded guilty to three criminal charges, the AP reported, including getting in the way of the DEA’s efforts to combat the crisis, failing to prevent the painkillers from ending up on the black market and encouraging doctors to write more painkiller prescriptions through two methods: paying them in a speakers program and directing a medical records company to send them certain patient information. Purdue’s plea deal calls for $8.3 billion in criminal fines and penalties, but the company is only liable for a fraction of that total — $225 million.

Gen­mab ax­es an ADC de­vel­op­ment pro­gram af­ter the da­ta fail to im­press

Genmab $GMAB has opted to ax one of its antibody-drug conjugates after watching it flop in the clinic.

The Danish biotech reported Tuesday that it decided to kill their program for enapotamab vedotin after the data gathered from expansion cohorts failed to measure up. According to the company:

While enapotamab vedotin has shown some evidence of clinical activity, this was not optimized by different dose schedules and/or predictive biomarkers. Accordingly, the data from the expansion cohorts did not meet Genmab’s stringent criteria for proof-of-concept.