An­oth­er hic­cup for GW Phar­ma's seizure drug ri­val, as Zo­genix dis­clos­es FDA re­view de­lay

Zo­genix has had a trou­bling 2020 so far. Ear­li­er this month, its ex­per­i­men­tal seizure drug met the main goal in a piv­otal study in pa­tients with Lennox-Gas­taut Syn­drome, but the com­pa­ny saw its shares plum­met af­ter the mag­ni­tude of the ther­a­py’s ef­fect fell short of Wall Street ex­pec­ta­tions. On Thurs­day, the drug de­vel­op­er said that the FDA had ex­tend­ed the re­view of the drug in pa­tients with Dravet syn­drome by three months.

Last April, Zo­genix said the FDA had re­buffed re­view­ing the drug’s mar­ket­ing ap­pli­ca­tion for Dravet pa­tients in a refuse-to-file let­ter, cit­ing the lack of cer­tain non-clin­i­cal stud­ies key for the as­sess­ment of chron­ic ad­min­is­tra­tion of the drug as well as an in­cor­rect ver­sion of a clin­i­cal dataset. In No­vem­ber, the FDA ac­cept­ed the com­pa­ny’s re­sub­mit­ted ap­pli­ca­tion, and the agency was ex­pect­ed to make its fi­nal de­ci­sion by March 25.

How­ev­er, af­ter Zo­genix re­cent­ly sub­mit­ted ad­di­tion­al ef­fi­ca­cy da­ta on the drug — to be brand­ed Fin­tepla — at the be­hest of the US reg­u­la­tor. That in­for­ma­tion was con­sti­tut­ed as a ma­jor amend­ment, which pushed the FDA’s de­ci­sion date to June 25, the com­pa­ny said.

When Stifel’s Paul Mat­teis spoke to Zo­genix man­age­ment, ex­ec­u­tives told him that the in­for­ma­tion in ques­tion was around an ef­fi­ca­cy analy­sis, and not fo­cused on the drug’s safe­ty pro­file or tox­i­c­i­ty stud­ies. The com­pa­ny al­so sug­gest­ed that they had “ze­ro in­di­ca­tion” that the FDA was plan­ning an ad­vi­so­ry com­mit­tee meet­ing.

“We asked, just to make sure, whether or not these ef­fi­ca­cy analy­ses were re­lat­ed to an in­ter­pre­ta­tion of the PhI­II tri­als as suc­cess­ful or not: They af­firmed this is not the case, the ask is not about da­ta in­tegri­ty, and thus the ad­di­tion­al analy­ses that were sub­mit­ted to the agency sound to us like they’re prob­a­bly less about whether or not the drug works, and there­fore prob­a­bly less about ac­tu­al ap­prov­abil­i­ty,” Mat­teis wrote in a note.

If ap­proved, Fin­tepla, which is one-half of the no­to­ri­ous axed pre­scrip­tion weight-loss cock­tail fen-phen, will com­pete with GW Phar­ma­ceu­ti­cals’ pi­o­neer­ing cannabis-de­rived Epid­i­olex — which is ap­proved for both Dravet and LGS.

“To be fair this isn’t the first hic­cup with the sub­mis­sion, giv­en that there was an RTF last Spring. Some in­vestors might look at a pat­tern here and get wor­ried that there’s some­thing else go­ing on be­yond the sur­face. We ob­vi­ous­ly can’t be 100% cer­tain. But when we look at the clin­i­cal da­ta, the ef­fi­ca­cy of Fin­tepla in Dravet looks high­ly dif­fer­en­ti­at­ed, and the safe­ty pro­file of the drug — as as­sessed un­der FDA guide­lines — looks bet­ter than we had al­ways ex­pect­ed as it re­lates to CV risk,” Mat­teis added.

“(W)e as­sume a 80% prob­a­bil­i­ty-of-ap­proval, and in the mid-$20s, we think shares are dis­count­ing the rev­enue po­ten­tial of Fin­tepla in Dravet and are as­crib­ing lit­tle-to-no cred­it in LGS, where, even de­spite ef­fi­ca­cy not liv­ing up to ex­pec­ta­tions, still is a re­al mar­ket for this prod­uct.”

Echo­ing Stifel’s com­men­tary, SVB Leerink an­a­lyst Marc Good­man said he was still pos­i­tive about the ap­prov­abil­i­ty of the drug, de­spite the de­lay.

“(T)he FDA de­lays that are ma­jor amend­ments are usu­al­ly be­cause the FDA wants to ap­prove the prod­uct, which is good prece­dent,” he wrote in a note, not­ing that most in­vestors are con­cerned about safe­ty on­ly be­cause of the his­to­ry of fen-phen. 

But Zo­genix in­vestors ap­peared dis­mayed by the an­nounce­ment. The Cal­i­for­nia-based com­pa­ny’s stock $ZGNX tum­bled about 19% to $21.20 in morn­ing trad­ing.

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.