A bat­tered Zaf­gen seeks strate­gic op­tions, proph­esy­ing on­go­ing study will un­like­ly lift FDA hold on di­a­betes drug

Zaf­gen, which back in 2015 crossed the $50 mark, is now firm­ly in pen­ny stock ter­ri­to­ry. Blight­ed by safe­ty con­cerns across a range of its ex­per­i­men­tal drugs, the Boston drug de­vel­op­er is now seek­ing strate­gic op­tions, in­clud­ing a pos­si­ble sale — pre­dict­ing da­ta from the on­go­ing in vi­vo study for its di­a­betes drug will not be enough to con­vince the FDA to lift its clin­i­cal hold on the pro­gram.

The com­pa­ny’s shares $ZFGN tum­bled more than 15% to 69 cents in ear­ly Thurs­day trad­ing.

Zaf­gen’s woes go back years. In 2016, Zaf­gen de­sert­ed its be­lo­ranib pro­gram af­ter the ex­per­i­men­tal obe­si­ty drug was linked to pa­tient deaths in a piv­otal study. The FDA, wor­ried about ex­pos­ing more pa­tients to harm, im­posed a clin­i­cal hold on Zaf­gen’s sec­ond-gen­er­a­tion (obe­si­ty-re­lat­ed) di­a­betes drug ZGN-1061 last No­vem­ber, wary that the car­dio threat posed by be­lo­ranib was still in play. This Ju­ly, fol­low­ing months of dis­cus­sion with the reg­u­la­tor, an in vi­vo an­i­mal study de­sign and pro­to­col for ZGN-1061 was agreed up­on — da­ta from this study are ex­pect­ed by the end of 2019.

“Based on pre­lim­i­nary re­sults from this study re­viewed this week, Zaf­gen does not present­ly ex­pect the da­ta to war­rant res­o­lu­tion of the clin­i­cal hold for ZGN-1061,” Zaf­gen said on Thurs­day. The com­pa­ny — which saw a string of man­age­r­i­al de­par­tures last year in­clud­ing CEO Tom Hugh­es, who had led the com­pa­ny for nine years, and board mem­ber Bruce Booth, who ex­it­ed af­ter a dozen years — is seek­ing al­ter­na­tives in­clud­ing an ac­qui­si­tion, merg­er, busi­ness com­bi­na­tion, in-li­cens­ing, or an­oth­er strate­gic trans­ac­tion in­volv­ing the com­pa­ny or its as­sets.

Ear­li­er this year, Zaf­gen was forced to con­tend with yet an­oth­er set­back. Its ex­per­i­men­tal drug, ZGN-1258 — un­der de­vel­op­ment for rare cas­es of obe­si­ty brought on by rare ge­net­ic con­di­tion Prad­er-Willi syn­drome, which is char­ac­ter­ized by con­stant hunger — was slapped with a clin­i­cal hold by the FDA in March, even be­fore it hit the clin­ic. The hold em­anat­ed from da­ta that in­di­cat­ed mus­cle de­te­ri­o­ra­tion in a ro­dent mod­el of the dis­ease, which prompt­ed the com­pa­ny to sus­pend plans for an ap­pli­ca­tion to test the drug in hu­mans.

As of June 30, 2019, the com­pa­ny had cash, cash equiv­a­lents, and mar­ketable se­cu­ri­ties to­tal­ing $91.7 mil­lion, Zaf­gen dis­closed in Au­gust. In the sec­ond quar­ter, the com­pa­ny re­port­ed a net loss of about $12 mil­lion.

Secretary of health and human services Alex Azar speaking in the Rose Garden at the White House (Photo: AFP)

Trump’s HHS claims ab­solute au­thor­i­ty over the FDA, clear­ing path to a vac­cine EUA

The top career staff at the FDA has vowed not to let politics overrule science when looking at vaccine data this fall. But Alex Azar, who happens to be their boss’s boss, apparently won’t even give them a chance to stand in the way.

In a new memorandum issued Tuesday last week, the HHS chief stripped the FDA and other health agencies under his purview of their rule making ability, asserting all such power “is reserved to the Secretary.” Sheila Kaplan of the New York Times first obtained and reported the details of the September 15 bulletin.

Two wild weeks for Grail end in $8B Il­lu­mi­na buy­out

Grail’s whirlwind two weeks have ended in the wealthy arms of its former founder and benefactors.

Illumina has shelled out $8 billion to reacquire the closely-watched liquid biopsy startup they spun out just 5 years ago and sold off much of its shares just 3 years ago. The deal comes nearly two weeks after the well-heeled startup filed for a potentially massive IPO — one that was disrupted just a week later when Bloomberg reported that Illumina was in talks to buy their former spinout for up to $8 billion.

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Roche vaults to the front of the NL­RP3 clin­i­cal race, pay­ing $448M up­front to bag In­fla­zome

Roche is going all in on NLRP3.

The pharma giant is putting down $448 million (€380 million) upfront to snatch Novartis-backed Inflazome, which makes it a clinical player in the space overnight.

Dublin and Cambridge, UK-based Inflazome is the second NLRP3-focused biotech Roche has acquired in less than two years, and although no numbers were disclosed in the Jecure buyout, this is almost certainly a much larger deal.

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Eli Lilly CSO Dan Skovronsky (file photo)

UP­DAT­ED: #ES­MO20: Eli Lil­ly shows off the da­ta for its Verzenio suc­cess. Was it worth $18 bil­lion?

The press release alone, devoid of any number except for the size of the trial, added nearly $20 billion to Eli Lilly’s market cap back in June. Now investors and oncologists will get to see if the data live up to the hype.

On Sunday at ESMO, Eli Lilly announced the full results for its Phase III MonarchE trial of Verzenio, showing that across over 5,000 women who had had HR+, HER2- breast cancer, the drug reduced the odds of recurrence by 25%. That meant 7.8% of the patients on the drug arm saw their cancers return within 2 years, compared with 11.3% on the placebo arm.

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As­traZeneca pub­lish­es Covid-19 vac­cine PhI­II pro­to­cols in lock­step with Mod­er­na and Pfiz­er. How are they dif­fer­ent?

Following in the steps of Moderna and Pfizer, the other two American drugmakers currently in Phase III trials for their Covid-19 vaccines, AstraZeneca posted its own study protocols over the weekend. The move is the latest in a series of rare peeks behind the curtain, as such blueprints are typically shared once such trials are completed.

“Given the unprecedented global impact of the Coronavirus pandemic and the need for public information, AstraZeneca has published the detailed protocol and design of our AZD1222 clinical trial. As with most clinical development, protocols are not typically shared publicly due to the importance of maintaining confidentiality and integrity of trials. AstraZeneca continues to work with industry peers to ensure a consistent approach to sharing timely clinical trial information,” the company said in a statement.

Sebastian Nijman (file photo)

Roche looks to ge­net­ic mod­i­fiers for new drug tar­gets, team­ing up with Dutch biotech in $375M deal

Roche is gambling on a new way of discovering drug targets and, ultimately, promising to infuse more than $375 million into a small biotech if all goes well.

A spinout of the Netherlands Cancer Institute and Oxford University, Scenic Biotech set out to pioneer a field that’s gaining some traction among top VCs in the US: to harness the natural protecting powers of genetic modifiers — specific genes that suppress a disease phenotype.

Greg Friberg (File photo)

#ES­MO20: Am­gen team nails down sol­id ear­ly ev­i­dence of AMG 510’s po­ten­tial for NSCLC, un­lock­ing the door to a wave of KRAS pro­grams

The first time I sat down with Amgen’s Greg Friberg to talk about the pharma giant’s KRAS G12C program for sotorasib (AMG 510) at ASCO a little more than a year ago, there was high excitement about the first glimpse of efficacy from their Phase I study, with 5 of 10 evaluable non-small cell lung cancer patients demonstrating a response to the drug.

After decades of failure targeting KRAS, sotorasib offered the first positive look at a new approach that promised to open a door to a whole new approach by targeting a particular mutation to a big target that had remained “undruggable” for decades.

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#ES­MO20: Out to beat Tagris­so, J&J touts 100% ORR for EGFR bis­pe­cif­ic/TKI com­bo — fu­el­ing a quick leap to PhI­II

J&J’s one-two punch on EGFR-mutant non-small cell lung cancer has turned up some promising — although decidedly early — results, fueling the idea that there’s yet room to one up on third-generation tyrosine kinase inhibitors.

Twenty out of 20 advanced NSCLC patients had a response after taking a combination of an in-house TKI dubbed lazertinib and amivantamab, a bispecific antibody targeting both EGFR and cMET engineered on partner Genmab’s platform, J&J reported at ESMO. All were treatment-naïve, and none has seen their cancer progress at a median follow-up of seven months.

Exelixis CEO Michael Morrissey (file photo)

#ES­MO20: Look out Mer­ck. Bris­tol My­ers and Ex­elix­is stake out their com­bo’s claim to best-in-class sta­tus for front­line kid­ney can­cer

Now that the PD-(L)1 checkpoints are deeply entrenched in the oncology market, it’s time to welcome a wave of combination therapies — beyond chemo — looking to extend their benefit to larger numbers of patients. Bristol Myers Squibb ($BMY} and Exelixis {EXEL} are close to the front of that line.

Today at ESMO the collaborators pulled the curtain back on some stellar data for their combination of Opdivo (the PD-1) and Cabometyx (the TKI), marking a significant advance for the blockbuster Bristol Myers franchise while offering a big leg up for the team at Exelixis.

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