J&J snags rights to a gene ther­a­py port­fo­lio from lit­tle MeiraGTx, a spin­off from Sam Wak­sal’s Kad­mon

Af­ter get­ting a quick les­son on lit­tle MeiraGTx’s gene ther­a­py ef­forts — in­clud­ing their home made on/off switch — J&J is go­ing all in. And like oth­er deals it’s made re­cent­ly, J&J’s re­search or­ga­ni­za­tion un­der Math­ai Mam­men is demon­strat­ing its en­thu­si­asm for small biotechs that are short on his­to­ry and long on promise.

The phar­ma gi­ant fol­lowed up on the ini­tial re­search al­liance they signed last fall with the biotech and snapped up com­mer­cial rights to a slate of clin­i­cal-stage, once-and-done de­vel­op­ment pro­grams aimed at eye dis­eases. J&J will pay $100 mil­lion in a cash up­front for the deal and is putting an­oth­er $340 mil­lion on the ta­ble in mile­stones.

Alexan­dria Forbes

J&J is al­so on the hook now for de­vel­op­ment costs for the port­fo­lio, which in­cludes can­di­dates for in­her­it­ed reti­nal dis­eases: achro­matop­sia caused by mu­ta­tions in ei­ther CNGB3 or CN­GA3 and X-linked re­tini­tis pig­men­tosa. Tied in is a pact on col­lab­o­rat­ing on AAV man­u­fac­tur­ing tech­nol­o­gy, which they will work on to­geth­er.

J&J is ty­ing it­self up to a New York-based com­pa­ny that spun out of Kad­mon, Sam Wak­sal’s come­back biotech af­ter his prison sen­tence for in­sid­er trad­ing — a case that al­so took celebri­ty home­mak­er Martha Stew­art down with him. Wak­sal bowed out just as Kad­mon went pub­lic, as he was barred from be­ing an of­fi­cer in a pub­lic com­pa­ny again.

J&J cre­at­ed an ex­ten­sive net­work of deal­mak­ing of­fices around the world sev­er­al years ago, and it’s been pay­ing some top prices to land new clin­i­cal-stage drugs to bring in­to the pipeline. That strat­e­gy was on dis­play when J&J snagged the BC­MA CAR-T at Shang­hai’s Leg­end, with plans to go up against blue­bird for the first-in-class ti­tle.

“Our gene reg­u­la­tion plat­form, based on RNA struc­ture, was in­vent­ed and de­vel­oped en­tire­ly in-house at MeiraGTx and is a re­flec­tion of our com­mit­ment to ground-break­ing sci­ence,” said MeiraGTx CEO Alexan­dria Forbes, a sci­en­tist who took a lat­er spin at a hedge fund be­fore helm­ing her first biotech.

Gilead CEO Dan O'­Day of­fers a de­tailed ex­pla­na­tion on remde­sivir ac­cess — re­as­sur­ing an­a­lysts that Covid-19 da­ta are com­ing fast

After coming under heavy fire from consumer groups ready to pummel them for grabbing the FDA’s orphan status for remdesivir — reserved to encourage the development of rare disease therapies — Gilead CEO Daniel O’Day had some explaining to do about the company’s approach to providing access to this drug to patients suffering from Covid-19. And he set aside time over the weekend to patiently explain how they are making their potential pandemic drug available in a new program — one he feels can better be used to address a growing pack of infected patients desperately seeking remdesivir under compassionate use provisions.

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Ven­ture Cap­i­tal as a Strate­gic Part­ner: Fu­el­ing In­no­va­tion be­yond Fi­nance

The average level of investment required for a biotech start-up to succeed is increasing every year, elevating the pressure even further on venture capital to make smart financial investments. Financial investment alone, however, does not always guarantee that exciting innovations can be transformed into real businesses that make a meaningful difference to patients.

Beyond just capital

At Astellas Venture Management (AVM) – a wholly-owned venture capital organization within Astellas, headquartered in the San Francisco Bay Area – capital is just one of the ingredients we offer to add value to our biotechnology investments and partnerships. We generally take a strategic investor approach for companies in our invested portfolio, providing access to expertise, technology and/or resources in addition to the injection of finance. An equity investment from AVM can include access to Astellas’ research and development (R&D) capabilities and expertise, and a global network of partner academic institutions and biotechnology companies, to help advance and accelerate the start-up’s innovation.

Covid-19 roundup: In­ter­cept, blue­bird and a grow­ing list of biotechs feel the pain as pan­dem­ic man­gles FDA, R&D sched­ules

Around 100 staffers at Boston area hospitals have now tested positive for Covid-19, spotlighting the growing risk that the pandemic will sideline many of the most essential workers in healthcare as caseloads peak in the US and around the globe. With more than 3,400 deaths, Spain has become the latest country to surpass the official death count attributed to the new coronavirus in China, where the outbreak originated. As of Thursday morning, confirmed global cases had crossed 470,000 and the death count eclipsed 21,000.

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Af­ter crit­ics lam­bast­ed Gilead for grab­bing the FDA's spe­cial rare drug sta­tus on remde­sivir, they're giv­ing it back

Two days after Gilead won orphan drug status for remdesivir as a potential treatment for Covid-19, they’re handing it back.

The company was slammed from several sides after Gilead reported that the FDA had come through with the special status, which comes with 7 years of market exclusivity, the waiver of FDA fees and some tax credits as well. Typically, everyone who can get orphan status lands it without much of a fuss, but Democratic presidential candidate Bernie Sanders, Public Citizen and other consumer groups were outraged.

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Mod­er­na CEO Stéphane Ban­cel out­lines a short path for emer­gency use of a coro­n­avirus vac­cine

NIAID director Anthony Fauci has left no doubts that it takes 12 to 18 months to get a new vaccine tested and in commercial use, in the best of circumstances. But in times of a global emergency — like these — maybe there’s another, faster route to follow.

In an SEC filing on Tuesday, Moderna $MRNA staked out a record-setting pathway to getting their mRNA vaccine into the frontline of the healthcare response as early as this fall. The SEC filing notes that CEO Stéphane Bancel told Goldman Sachs that an emergency use approval could allow the vaccine to go to healthcare workers and certain individuals in a matter of months — presumably provided the NIH sees the safety and efficacy data they would need from the Phase I.

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Caught in a Covid-19 mael­strom, Eli Lil­ly locks down clin­i­cal tri­als as multi­bil­lion-dol­lar R&D ops de­rail

The Covid-19 pandemic has derailed Eli Lilly’s $6 billion R&D operations.

The pharma giant reported Monday morning that it has decided to hit the brakes on most new study starts and pause enrollment for most ongoing studies. Lilly adds that it is continuing dosing for ongoing studies, “but with study-by-study consideration.”

The pandemic has severely disrupted healthcare systems around the globe, says Lilly, making it difficult or impossible to conduct studies at many research sites. And there’s no timeline for when it expects to get back on track.

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UP­DAT­ED: Ver­tex joins Mer­ck, Pfiz­er — re­vamp­ing multi­bil­lion-dol­lar tri­al strat­e­gy as biotech R&D crum­bles

You can add Pfizer, Merck and — as we found out Friday morning — Vertex to the growing list of pharma giants hitting the pause button on a range of clinical trials. But not everyone in R&D is getting a red light.

Vertex says that it’s doing its best to keep working its pipeline strategy, coming up with a plan “to enable virtual clinic visits and home delivery of study drug to ensure study continuity and medical monitoring, and to facilitate study procedures.”

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Covid-19 roundup: Bris­tol My­ers sus­pends clin­i­cal tri­als, grounds field team; Vir ush­ers an­ti­body can­di­dates to hu­man test­ing

The global nature of the Covid-19 pandemic is manifesting more profoundly every day. With Spain’s death toll now surpassing China’s and India on full lockdown, the number of confirmed cases around the world has exceeded 436,000 while recoveries edged close to 112,000.

While the outbreak derails R&D at another pharma giant, several drugmakers have some encouraging updates on both experimental and repurposed molecules. Philanthropic campaigns in anticipation of the economic fallout continue. An Australian biotech is taking extreme measures to hunker down. There’s also an alternative epidemiology model emerging out of the UK, stirring up more discussion regarding the true extent of the infections in the country.

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As share buy­backs come un­der scruti­ny, what's in store for the bio­phar­ma in­dus­try?

Stock buybacks are not to be permitted for companies that will be bailed out in the coronavirus stimulus package, Congressional leaders have signaled. To what degree the biopharma industry has relied on buybacks for earnings growth in recent years, and if the trend continues, are the big questions as scrutiny into the practice heightens and balance sheets weaken with the coronavirus pandemic wreaking havoc on global economies.

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