Bio­gen bags an AAV gene ther­a­py pro­gram from Mass­a­chu­setts Eye and Ear; Biotechs raised $1B-plus in lat­est round of fol­low-ons

→ Bio­gen has picked up a new, pre­clin­i­cal gene ther­a­py pro­gram from Mass­a­chu­setts Eye and Ear for in­her­it­ed reti­nal de­gen­er­a­tion due to mu­ta­tions in the PRPF31 gene, among the most com­mon caus­es for au­to­so­mal dom­i­nant re­tini­tis pig­men­tosa. They’re build­ing on the work of Har­vard’s Er­ic Pierce. “The treat­ment of IRDs with high­ly ef­fec­tive AAV-based gene ther­a­pies is core to Bio­gen’s oph­thal­mol­o­gy strat­e­gy,” said Chris Hen­der­son, the re­search head at Bio­gen. “This agree­ment un­der­scores our com­mit­ment to that strat­e­gy and builds off of our ac­qui­si­tion of Night­star Ther­a­peu­tics in 2019 and our ac­tive clin­i­cal tri­als of gene ther­a­pies for dif­fer­ent ge­net­ic forms of IRD.”

→ Sarep­ta has inked a col­lab­o­ra­tion with Hansa to de­vel­op their ex­per­i­men­tal drug im­li­fi­dase as a pre-treat­ment for their gene ther­a­pies. The drug is in­tend­ed for use in pa­tients who have neu­tral­iz­ing an­ti­bod­ies that would pre­vent gene ther­a­pies for Duchenne mus­cu­lar dy­s­tro­phy and Limb-gir­dle mus­cu­lar dy­s­tro­phy from work­ing. Hansa gets a $10 mil­lion up­front and up to $397.5 mil­lion in mile­stones.

It’s been rain­ing mon­ey on Wall Street — at least when it comes to drug de­vel­op­ers. CRISPR Ther­a­peu­tics $CR­SP and Ac­celeron each raised a whop­ping $450 mil­lion this week af­ter pric­ing fol­low-on of­fer­ings. CRISPR priced 6,428,572 com­mon shares at a pub­lic of­fer­ing price of $70.00 per share, while Ac­celeron $XL­RN auc­tioned off 4,864,864 shares of com­mon stock at a price to the pub­lic of $92.50 per share.

The transat­lantic biotech play­er Immat­ics has com­plet­ed its flip on­to Nas­daq through the Arya Sci­ences Ac­qui­si­tion Corp. The can­cer drug biotech will trade as $IMTX af­ter it raised $253 mil­lion in the process. The SPAC was set up by Per­cep­tive Ad­vi­sors.

Seat­tle-based Ne­oleukin Ther­a­peu­tics, mean­while, raised $76.2 mil­lion $NLTX for its work on pro­tein ther­a­peu­tics.

The DCT-OS: A Tech­nol­o­gy-first Op­er­at­ing Sys­tem - En­abling Clin­i­cal Tri­als

As technology-enabled clinical research becomes the new normal, an integrated decentralized clinical trial operating system can ensure quality, deliver consistency and improve the patient experience.

The increasing availability of COVID-19 vaccines has many of us looking forward to a time when everyday things return to a state of normal. Schools and teachers are returning to classrooms, offices and small businesses are reopening, and there’s a palpable sense of optimism that the often-awkward adjustments we’ve all made personally and professionally in the last year are behind us, never to return. In the world of clinical research, however, some pandemic-necessitated adjustments are proving to be more than emergency stopgap measures to ensure trial continuity — and numerous decentralized clinical trial (DCT) tools and methodologies employed within the last year are likely here to stay as part of biopharma’s new normal.

As­traZeneca caps PD-L1/CT­LA-4/chemo com­bo come­back with OS win. Is treme­li­mum­ab fi­nal­ly ready for ap­proval?

AstraZeneca’s closely-watched POSEIDON study continues to be the rare bright spot in its push for an in-house PD-L1/CTLA-4 combo.

Combining Imfinzi and tremelimumab with physicians’ choice of chemotherapy helped patients with stage IV non-small cell lung cancer live longer, the company reported — marking the first time the still-experimental tremelimumab has demonstrated an OS benefit.

For AstraZeneca and CEO Pascal Soriot, the positive readout — which is devoid of numbers — offers much-needed validation for the big bet they made on Imfinzi plus tremelimumab, after the PD-L1/CTLA-4 regimen failed multiple trials in head and neck cancer as well as lung cancer.

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Ron DePinho (file photo)

A 'fly­over' biotech launch­es in Texas with four Ron De­Pin­ho-found­ed com­pa­nies un­der its belt

In his 13 years at Genzyme, Michael Wyzga noticed something about East Coast drugmakers. Execs would often jet from Boston or New York to San Francisco to find more assets, and completely miss the work being done in flyover states, like Texas or Wisconsin.

“If it doesn’t come out of MGH or MIT or Harvard, probably not that interesting,” he said of the mindset.

Now, he and some well-known industry players are looking to change that, and they’ve reeled in just over $38 million to do it.

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Onno van de Stolpe, Galapagos CEO (Thierry Roge/Belga Mag/AFP via Getty Images)

Gala­pa­gos chops in­to their pipeline, drop­ping core fields and re­or­ga­niz­ing R&D as the BD team hunts for some­thing 'trans­for­ma­tive'

Just 5 months after Gilead gutted its rich partnership with Galapagos following a bitter setback at the FDA, the Belgian biotech is hunkering down and chopping the pipeline in an effort to conserve cash while their BD team pursues a mission to find a “transformative” deal for the company.

The filgotinib disaster didn’t warrant a mention as Galapagos laid out its Darwinian restructuring plans. Forced to make choices, the company is ditching its IPF molecule ’1205, while moving ahead with a Phase II IPF study for its chitinase inhibitor ’4617.

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Mod­er­na CEO brush­es off US sup­port for IP waiv­er, eyes more than $19B in Covid-19 vac­cine sales in 2021

Moderna is definitively more concerned with keeping pace with Pfizer in the race to vaccinate the world against Covid-19 than it is with Wednesday’s decision from the Biden administration to back an intellectual property waiver that aims to increase vaccine supplies worldwide.

In its first quarter earnings call on Thursday, Moderna CEO Stéphane Bancel shrugged off any suggestion that the newly US-backed intellectual property waiver would impact his company’s vaccine or bottom line. Still, the company’s stock price fell by about 9% in early morning trading.

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Ad­comm splits slight­ly in fa­vor of FDA ap­prov­ing Chemo­Cen­tryx’s rare dis­ease drug

The FDA’s Arthritis Advisory Committee on Thursday voted 10 for and 8 against the approval of ChemoCentryx’s $CCXI investigational drug avacopan as a treatment for adults with a rare and serious disease known as anti-neutrophil cytoplasmic autoantibody (ANCA)-vasculitis.

The vote on whether the FDA should approve the drug was preceded by a split vote of 9 to 9 on whether the efficacy data support approval, and 10 to 8 that the safety profile of avacopan is adequate enough to support approval.

Keytru­da gets ac­cel­er­at­ed OK in HER2-pos­i­tive gas­tric can­cer; Twin­Strand gets $50M to hunt for 'nee­dles in a haystack'

King Keytruda keeps racking up approvals, earning a new accelerated OK from the FDA on Wednesday.

Regulators handed down the approval for HER2-positive gastric cancer in the first-line setting, in combination with trastuzumab, fluoropyrimidine- and platinum-based chemo. It’s yet another thumbs-up for one of the best-selling drugs in the world.

Wednesday’s OK came on the basis of a 264-patient study with individuals who had not previously received treatment for their disease. Patients were randomized 1-to-1 to receive Keytruda or placebo every three weeks in addition to the chemotherapy regimen.

Brent Saunders (Richard Drew, AP Images)

OcuWho? Star deal­mak­er turned aes­thet­ics czar Brent Saun­ders flips back in­to biotech. But who’s he team­ing up with now?

Brent Saunders went on a tear of headline-blazing deals building Allergan, merging and rearranging a variety of big companies into one before an M&A pact with Pfizer blew up and sent him on a bout of biotech drug deals. That didn’t work so well, so under pressure, he got his buyout at AbbVie — which needed a big franchise like Botox. And it was no big surprise to see him riding the SPAC wave into a recent $1 billion-plus deal that left him in the executive chairman’s seat at an aesthetics outfit — now redubbed The Beauty Health Company — holding a big chunk of the equity.

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Drug pric­ing watch­dog joins the cho­rus of crit­ics on Bio­gen's ad­u­canum­ab: What about charg­ing $2,560 per year?

As if Biogen’s aducanumab isn’t controversial enough, the researchers at drug pricing watchdog ICER have drawn up the contours of a new debate: If the therapy does get approved for Alzheimer’s by June, what price should it command?

Their answer: At most $8,290 per year — and perhaps as little as $2,560.

Even at the top of the range, the proposed price is a fraction of the $50,000 that Wall Street has reportedly come to expect (although RBC analyst Brian Abrahams puts the consensus figure at $11.5K). With critics, including experts on the FDA’s advisory committee, making their fierce opposition to aducanumab’s approval loud and clear, the pricing pressure adds one extra wrinkle Biogen CEO Michel Vounatsos doesn’t need as he orders full-steam preparation for a launch.