The PC­SK9 of NASH? Re­gen­eron and Al­ny­lam join forces to tack­le a promis­ing tar­get for se­vere liv­er dis­eases

The Re­gen­eron Ge­net­ics Cen­ter $REGN rou­tine­ly sifts through a moun­tain of se­quenc­ing da­ta. And every now and then, the re­searchers there will pan out a glit­ter­ing find­ing that looks like it could rank right up there with PC­SK9.

Here’s one.

John Maraganore

This evening, in a study pub­lished in the New Eng­land Jour­nal of Med­i­cine, they are point­ing to one such dis­cov­ery that they be­lieve has some big im­pli­ca­tions for se­vere liv­er dis­eases, in­clud­ing the Holy Grail of NASH. And with­out a log­i­cal an­ti­body strat­e­gy to put in­to play — giv­en an in­tra­cel­lu­lar tar­get in the spot­light — they’ve en­list­ed an ex­pe­ri­enced crew at Al­ny­lam to bring their RNAi gene si­lenc­ing plat­form in­to the game to find the most ef­fi­cient way to pro­vide the key to coun­ter­ing a raft of ail­ments.

“We ob­vi­ous­ly saw the da­ta and we were blown away,” says Al­ny­lam $AL­NY CEO John Maraganore, who’s now in wait-and-see mode as their lead drug patisir­an rolls up to an Au­gust PDU­FA date. “This tar­get  — HSD17B13 — from a hu­man ge­net­ics per­spec­tive it re­al­ly is the  PC­SK9 of NASH.”

Aris Baras

Re­gen­eron prides it­self on mov­ing ag­gres­sive­ly when they find some­thing promis­ing for clin­i­cal de­vel­op­ment. And in Al­ny­lam, they feel they are work­ing with close kin in that re­spect.

“These guys at Al­ny­lam have a sim­i­lar ap­proach and track record,” says Aris Baras, head of the RGC. And he feels this tar­get de­serves speed and care, which in­spired a 50/50 deal on re­search costs and any com­mer­cial po­ten­tial that comes out of the pre­clin­i­cal align­ment.

How fast can Al­ny­lam move here? “We’re not giv­ing guid­ance,” says Maraganore, but he says you shouldn’t be sur­prised if there’s a pro­gram in the clin­ic next year. (Yes, that is fast.)

The deal, he adds, calls for shared eco­nom­ics in de­vel­op­ment, with Al­ny­lam do­ing the crit­i­cal ear­ly de­vel­op­ment and Re­gen­eron pick­ing up the late-stage work. That will be their first RNAi study of their own, says Maraganore, but there’s no re­al mys­tery in how that works. And they’ll stay close­ly in­volved through­out.

The study of hu­man ge­net­ics in these cen­ters is be­gin­ning to have a re­al im­pact on R&D ef­fi­cien­cy, says Baras. And this project in par­tic­u­lar, he be­lieves, high­lights the promise of the field in quick­ly ze­ro­ing in on a vi­able drug pro­gram.

By set­ting up a large pile of ge­net­ic da­ta on thou­sands of pa­tients with liv­er dis­ease and com­par­ing it with se­quenc­ing da­ta on a group of tens of thou­sands of healthy peo­ple, Baras tells me, the re­searchers at Re­gen­eron’s ge­net­ics arm nar­rowed down pos­si­ble ge­net­ic trig­gers for dis­ease to a loss-of-func­tion ge­net­ic vari­a­tion in HSD17B13.

Ful­ly shut down by the vari­a­tion, the in­ves­ti­ga­tors found that eclips­ing the gene re­duced en­zy­mat­ic ac­tiv­i­ty:

— Cut­ting the risk of al­co­holic cir­rho­sis by 73%.

— Low­er­ing the risk of non­al­co­holic cir­rho­sis by 49%.

— And cut­ting the risk of al­co­holic liv­er dis­ease by 53%, and non­al­co­holic liv­er dis­ease by 30%, with an as­so­ci­at­ed re­duc­tion in risk of NASH.

Re­gen­eron is al­so go­ing af­ter some small mol­e­cule pro­grams in the process.

“We think this is big bi­ol­o­gy and there are dif­fer­ent shots on goal with dif­fer­ent modal­i­ties,” says Baras.

We’ll stay fo­cused here at End­points on this one to see whether it lives up to the pre­clin­i­cal promise. Tra­di­tion sug­gests that any pro­gram that goes in­to the clin­ic will have at best around a 1 in 10 shot at an ap­proval. Ge­net­ics aims to change those odds for the bet­ter.

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.

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: 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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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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A Sin­ga­pore VC rais­es $200M for a new round, but will Covid-19 pre­vent it from rais­ing the rest?

A top Singaporean biotech venture fund is nearly halfway toward its largest ever fund, but in a sign of what could be in store for VCs amid a global economic freeze, said they could face headwinds raising the other half.

Vickers Venture Partners has secured $200 million out of a targeted $500 million for its 6th fund, first announced in early 2018. They’ve given themselves 13 months to complete the financing, Vickers founder Finian Tan told Deal Street Asia, but the financial frost settling amid the Covid-19 pandemic could slow efforts.

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Strug­gling Unum ex­ecs are ready to con­sid­er a sale, merg­er or any deal that comes its way

Unum $UMRX is working its way through a survival plan of sorts.

After getting hit with a trio of FDA holds in its brief public history and triggering its second pivot to a new lead drug program while laying off 60% of the staff, the troubled penny stock biotech Unum Therapeutics has hatched new plans to secure financial backing while lining up a go-forward strategy for the company.

First, Lincoln Park Capital Fund has agreed to buy up to $25 million of the long-suffering stock, as Unum directs. And the executive team — led by CEO Chuck Wilson — has put everything on the table for consideration: a sale, acquisition, merger, licensing deal, you name it. The ACTR707 program, meanwhile, is being formally wrapped up — their second failed lead program.