Up­start GeneTx launch­es, go­ing head to head with Ovid on rare An­gel­man syn­drome

An Illi­nois non­prof­it has launched a start­up de­vel­op­ing a drug to treat An­gel­man syn­drome – the ge­net­ic con­di­tion that’s al­so be­ing tack­led by Je­re­my Levin’s high­er pro­file up­start Ovid Ther­a­peu­tics $OVID.

Paula Evans

Levin, who you might know as the for­mer busi­ness de­vel­op­ment chief at Bris­tol-My­ers, was al­so Te­va’s CEO for a brief stint be­fore jump­ing in­to the biotech start­up busi­ness. Ovid, his lat­est ven­ture, just closed a $75 mil­lion IPO last year with a drug to take on An­gel­man syn­drome, a rare dis­ease that af­fects the ner­vous sys­tem.

Now a new con­tender in the field has emerged: GeneTx Bio­ther­a­peu­tics, the start­up just launched by the Foun­da­tion for An­gel­man Syn­drome Ther­a­peu­tics (FAST).

FAST chair­per­son Paula Evans will come on as CEO, while the non­prof­it’s CSO Allyson Berent joins as COO. Both are serv­ing on an un­paid ba­sis, the foun­da­tion said in a state­ment.

Je­re­my Levin, Ovid

GeneTx’s drug hope­ful is called GTX-101, an an­ti­sense ther­a­py that works to si­lence genes by bind­ing to the mes­sen­ger RNA pro­duced by that gene.

“For the past eight years, FAST has nar­row­ly fo­cused on in­vest­ing in lead­ing-edge tech­nolo­gies to treat the un­der­ly­ing cause and de­bil­i­tat­ing symp­toms of An­gel­man syn­drome,” Berent said in a state­ment. “Our ag­gres­sive re­search agen­da and col­lab­o­ra­tion with Texas A&M AgriL­ife Re­search has iden­ti­fied and char­ac­ter­ized a promis­ing ther­a­peu­tic to ad­dress this dev­as­tat­ing dis­or­der. An­gel­man syn­drome has such a sig­nif­i­cant un­met need, for which there are no ap­proved ther­a­peu­tics, and the land­scape for bring­ing mean­ing­ful treat­ments to the com­mu­ni­ty is sig­nif­i­cant­ly ex­pand­ing.”

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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