Zaf­gen's sec­ond try at Prad­er-Willi syn­drome trig­gers an­oth­er safe­ty alarm in pre­clin­i­cal tox study

Zaf­gen’s $ZFGN lat­est shot at cre­at­ing the first ther­a­py to treat pa­tients with rare cas­es of obe­si­ty brought on by Prad­er-Willi syn­drome has hit a safe­ty alarm be­fore the drug even man­aged to make it in­to the clin­ic.

The suc­ces­sor to be­lo­ranib — which blew up with spec­tac­u­lar ef­fect sev­er­al years ago fol­low­ing a clin­i­cal hold by the FDA — ZGN-1258 was sup­posed to be the biotech’s come­back drug in the field. In­stead, the biotech re­port­ed af­ter the mar­ket closed on Mon­day that it is sus­pend­ing plans for an IND af­ter re­searchers tracked mus­cle de­te­ri­o­ra­tion in a ro­dent mod­el of the dis­ease.

Zaf­gen’s stock shriv­eled at the men­tion of a new safe­ty alert. The shares plum­met­ed 35% af­ter the bell.

From their state­ment to­day:

Non­clin­i­cal da­ta showed de­gen­er­a­tion and oth­er anom­alies in rat mus­cle tis­sue to dif­fer­ent de­grees in both ve­hi­cle and dose arms of the stud­ies. The ef­fects were ab­sent from oth­er an­i­mal species in long term mod­els, and im­por­tant­ly, this find­ing has not been ob­served in any of the Com­pa­ny’s oth­er MetAP2 in­hibitors or clin­i­cal tri­als and ap­pears to be spe­cif­ic to ZGN-1258. Zaf­gen will pro­vide an up­date on plans for ZGN-1258 at a lat­er time, if war­rant­ed, fol­low­ing fur­ther eval­u­a­tion.

The move to shelve the IND came af­ter Zaf­gen re­port­ed in an SEC fil­ing late last year that every­thing had been lined up to ini­ti­ate clin­i­cal tri­als ex­cept for 1 last pre­clin­i­cal tri­al, which had to be re­done by a new CRO af­ter the first CRO had con­duct­ed it “im­prop­er­ly.”

Any fresh hint of a safe­ty warn­ing is tox­ic to Zaf­gen, which had to re­or­ga­nize and ul­ti­mate­ly re­place the se­nior ex­ecs who went on to oth­er en­deav­ors af­ter the first big crash. The biotech re­cent­ly re­port­ed what it called en­cour­ag­ing ear­ly da­ta about their lead drug, ZGN-1061 for obe­si­ty re­lat­ed to di­a­betes. Re­searchers re­port­ed pos­i­tive im­prove­ments in weight loss and blood sug­ar lev­els for di­a­bet­ics.

The lead drug, though, has al­so been un­der a cloud since the FDA dropped a clin­i­cal hold on the pro­gram last No­vem­ber, cit­ing CV safe­ty con­cerns. Leerink’s Joseph Schwartz has re­mained fo­cused on a key bio­mark­er for CV risk, even as the com­pa­ny pur­sued a risky ex-US tri­al.

Di­a­betes is a field with a huge pa­tient pop­u­la­tion, and even a hint of a safe­ty is­sue could prove lethal for any drug in de­vel­op­ment for this group.

And obe­si­ty drugs in gen­er­al are al­ways un­der a mi­cro­scope at the FDA, even af­ter the lat­est gen­er­a­tion hit the mar­ket, though they proved dra­mat­i­cal­ly un­suc­cess­ful as a com­mer­cial prod­uct.

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.