News brief­ing: Zio­pharm dra­ma con­tin­ues with new let­ter from ac­tivist group; Al­ny­lam, Cen­to­gene join forces on AT­TRv screen­ing pro­gram

As Fri­day’s dead­line to sub­mit writ­ten con­sent ap­proach­es, Wa­ter­Mill As­set Man­age­ment Corp is con­tin­u­ing to put the pres­sure on Zio­pharm On­col­o­gy in its ac­tivist at­tack.

The 3.3% mi­nor­i­ty stake­hold­er sent a new let­ter to in­vestors Tues­day morn­ing, em­pha­siz­ing the need for board­room change ahead of the self-im­posed due date. Wa­ter­Mill again pushed for the full slate of its three board nom­i­nees — Jaime Vieser, Hol­ger Weis and Wa­ter­Mill founder Robert Post­ma — to be se­lect­ed, and de­tailed nu­mer­ous ac­counts of what it says are at­tempts by Zio­pharm to “dis­tort” the truth.

Wa­ter­Mill’s let­ter pro­vid­ed new de­tails of al­leged events that it says jus­ti­fy the need for new board mem­bers. This in­cludes an in­ci­dent ear­li­er this year when Zio­pharm CEO Lau­rence Coop­er pur­port­ed­ly hung up on a group of in­vestors rep­re­sent­ing about 25% of the com­pa­ny’s share­hold­ers on a con­fer­ence call.

The Wa­ter­Mill group al­so stat­ed that Zio­pharm had reached out to in­ter­view two of the mi­nor­i­ty in­vestors’ board nom­i­nees on Oct 30, but is­sued a press re­lease on Nov 2 say­ing the nom­i­nees were “un­qual­i­fied” be­fore the group could re­spond. Wa­ter­Mill launched its ac­tivist at­tack on Nov 2 with an 8:45 am press re­lease, and Zio­pharm’s re­lease came out that day at 9 am.

Tues­day’s let­ter comes about two weeks af­ter proxy ad­vis­er In­sti­tu­tion­al Share­hold­er Ser­vices rec­om­mend­ed the as­cen­sion of Vieser and Weis to the Zio­pharm board while ex­clud­ing Post­ma. Both sides claimed vic­to­ry af­ter that re­port, with Zio­pharm say­ing ISS had re­ject­ed Wa­ter­Mill’s “full slate” of nom­i­nees and Wa­ter­Mill claim­ing ISS had en­dorsed their pro­pos­al for “board­room change.”

Zio­pharm ob­ject­ed, how­ev­er, to ISS’s ap­par­ent ap­proval of Weis as a nom­i­nee, say­ing Weis had played a key role in the 2018 bank­rupt­cy of the biotech De­meRx. Wa­ter­Mill on Tues­day again said the com­pa­ny was en­gag­ing in “base­less smears” as two for­mer De­meRx ex­ec­u­tives have vouched for Weis.

Over the last six months, three Zio­pharm board mem­bers have re­signed or been re­placed with the lat­est in­di­vid­ual re­sign­ing last week. The board has shrunk from eight to sev­en, and Zio­pharm has not nom­i­nat­ed any of Wa­ter­Mill’s pro­posed can­di­dates. — Max Gel­man

Al­ny­lam, Cen­to­gene join forces on AT­TRv screen­ing pro­gram

Cen­to­gene and Al­ny­lam have launched a new joint clin­i­cal screen­ing pro­gram to ge­net­i­cal­ly screen for those at risk of hered­i­tary transthyretin-re­lat­ed amy­loi­do­sis.

Al­so known as AT­TRv, the dis­or­der is caused by mis­fold­ed pro­teins which pro­gres­sive­ly build up and in­ter­fere with or­gan func­tion. The screen­ing pro­gram will fol­low up on a 2017 ef­fort to test 5,000 pa­tients across Ger­many, Aus­tria and Switzer­land with polyneu­ropa­thy and/or car­diomy­opa­thy — with no known ori­gin. Over 1% of those pa­tients were even­tu­al­ly di­ag­nosed with AT­TRv and were both clin­i­cal­ly and re­gion­al­ly mapped.

Since then, Cen­to­gene has dis­cov­ered new AT­TRv bio­mark­ers, which will be used to mon­i­tor pa­tients in the new screen­ing pro­gram over a two-year process. Both symp­to­matic and asymp­to­matic transthyretin amy­loi­do­sis pa­tients will take part in screen­ings, which Cen­to­gene will use to val­i­date the bio­mark­ers and bet­ter un­der­stand treat­ment op­tions. — Con­ner Mitchell

Melanoma im­munother­a­pies the fo­cus of new Check­mate-Bris­tol My­ers Squibb col­lab­o­ra­tion

Late-stage melanoma and the im­mune sys­tem’s abil­i­ty to fight the can­cer will be at the fore­front of a new clin­i­cal col­lab­o­ra­tion be­tween Check­mate Phar­ma­ceu­ti­cals and Bris­tol My­ers Squibb.

Check­mate, the clin­i­cal stage bio­phar­ma­ceu­ti­cal com­pa­ny, will put CMP-001 — its fore­most can­cer im­munother­a­py treat­ment — to the test through two dif­fer­ent tri­als with Bris­tol My­ers Squibb and Op­di­vo, the com­pa­ny’s PD-1 block­ing an­ti­body.

The first tri­al will in­volve a sin­gle arm Phase II study us­ing a com­bi­na­tion of CMP-001 and Op­di­vo in sub­jects who have un­re­sectable or metasta­t­ic melanoma that re­fracts PD-1 block­ing both as a monother­a­py op­tion or in con­junc­tion with oth­er ther­a­pies.

The sec­ond tri­al will in­volve a ran­dom­ized Phase II study of CMP-001 as a first-line ther­a­py com­bined with Op­di­vo com­pared to us­ing on­ly Op­di­vo as a monother­a­py in sub­jects with un­re­sectable or metasta­t­ic melanoma.

Check­mate will spon­sor the col­lab­o­ra­tion while Bris­tol My­ers Squibb sup­plies its drug, ac­cord­ing to the terms of the agree­ment. — Con­ner Mitchell

Athi­ra Phar­ma award­ed $15M NIA grant for clin­i­cal tri­al of Alzheimer’s ther­a­peu­tic

The Phase II clin­i­cal tri­al for a po­ten­tial Alzheimer’s dis­ease ther­a­putic re­ceived a boost Tues­day.

Seat­tle-based Athi­ra Phar­ma an­nounced that it re­ceived a $15 mil­lion grant from the Na­tion­al In­sti­tute on Ag­ing to sup­port the clin­i­cal tri­al for its small mol­e­cule ther­a­peu­tic, ATH-1017. A po­ten­tial can­di­date for treat­ing those with mild-to-mod­er­ate cas­es of Alzheimer’s, ATH-1017 will be stud­ied over the next three years thanks in part to the grant fund­ing, Athi­ra Phar­ma said.

The study will as­sess the im­pact of ATH-1017 on cer­tain brain wave sig­nals re­lat­ed to learn­ing, mem­o­ry for­ma­tion and high­er cog­ni­tive func­tions, the com­pa­ny said. — Con­ner Mitchell

Non-small cell lung can­cer drug re­ceives break­through ther­a­py des­ig­na­tion

A lead­ing drug can­di­date for treat­ing pa­tients with non-small cell lung can­cer has been giv­en a break­through ther­a­py des­ig­na­tion by the FDA.

Re­potrec­tinib, a cre­ation of San Diego, Cal­i­for­nia-based Turn­ing Point Ther­a­peu­tics, showed pos­i­tive re­sults dur­ing ini­tial da­ta from Phase 1 and Phase 2 por­tions of clin­i­cal stud­ies — enough to ex­pe­dite the drug’s fur­ther de­vel­op­ment through the FDA des­ig­na­tion, the com­pa­ny said Tues­day.

Re­potrec­tinib has al­ready re­ceived three fast track des­ig­na­tions from the FDA for us­es tan­gen­tial­ly re­lat­ed to non-small cell lung can­cer pa­tients af­ter the drug was found to pro­mote an­ti­tu­mor ac­tiv­i­ty and durable re­spons­es among ki­nase in­hibitor treat­ment-naïve and pre-treat­ed pa­tients, Turn­ing Point Ther­a­peu­tics said.

The com­pa­ny will present new Phase 2 study da­ta at a mi­ni-oral pre­sen­ta­tion at the World Con­fer­ence on Lung Can­cer on Jan 31, 2021.—Con­ner Mitchell

No­vo Nordisk Foun­da­tion ini­tia­tive gains sta­tus as own foun­da­tion to sup­port, fund re­search and start-ups

BioIn­no­va­tion In­sti­tute, a 2017 ini­tia­tive of the No­vo Nordisk Foun­da­tion, is now an in­de­pen­dent foun­da­tion.

Based in Copen­hagen, BII was es­tab­lished as an ex­ten­sion of the NNF to ad­vance re­search and en­tre­pre­neur­ship with­in the biotech­nol­o­gy sphere. In just two years, BII fund­ed some 85 re­search projects and star­tups thanks to $77.6 mil­lion from NNF.

Over the next 10 years, NNF can award $569.9 mil­lion to BII for sup­port of ear­ly-stage life sci­ence projects and start­up op­por­tu­ni­ties. How­ev­er, des­ig­nat­ing BII as its own foun­da­tion will al­low oth­er or­ga­ni­za­tions and foun­da­tions to take part in fi­nanc­ing its out­puts.

BII said Tues­day it ex­pects to em­ploy rough­ly 500 re­searchers, in­no­va­tors and en­tre­pre­neurs over the com­ing years. — Con­ner Mitchell

Sutro Bio­phar­ma pro­pos­es pub­lic of­fer­ing of 5 mil­lion shares of com­mon stock two years af­ter $85M IPO

A Cal­i­for­nia-based bio­phar­ma­ceu­ti­cal com­pa­ny on Mon­day pro­posed a pub­lic of­fer­ing of 5 mil­lion shares of com­mon stock — two years af­ter fil­ing for an $85 mil­lion IPO.

Sutro Bio­phar­ma, which fo­cus­es on pro­tein en­gi­neer­ing to cre­ate can­cer and au­toim­mune ther­a­peu­tics, an­nounced the pro­posed un­der­writ­ten pub­lic of­fer­ing of the 5 mil­lion com­mon stock shares and said it would al­so of­fer un­der­writ­ers a 30-day op­tion to pur­chase up to an ad­di­tion­al 750,000 shares.

Cowen, Piper San­dler and Wells Far­go Se­cu­ri­ties are act­ing as joint book-run­ning man­agers in the of­fer­ing. Wed­bush Pac­Grow and JMP Se­cu­ri­ties are act­ing as co-man­agers for the of­fer­ing.

Pro­ceeds from the pro­posed of­fer­ing will al­low Sutro Bio­phar­ma to con­tin­ue its clin­i­cal de­vel­op­ment of STRO-001 and STRO-002, two an­ti­body-drug con­ju­gates fo­cused on pa­tients with non-Hodgkin lym­phoma and ovar­i­an can­cers, re­spec­tive­ly.

In Sutro Bio­phar­ma’s 2018 IPO, it of­fered 5.67 mil­lion com­mon stock shares priced at $15 per share. The com­pa­ny’s stock is cur­rent­ly priced at $21.13. — Con­ner Mitchell

Flori­da blank check com­pa­ny to file $75M IPO, fo­cus on in­vest­ing in nov­el life sci­ence com­pa­nies

A blank check com­pa­ny based in Mi­a­mi, Flori­da is wad­ing in­to life sci­ence in­vest­ment op­por­tu­ni­ties through a $75 mil­lion ini­tial pub­lic of­fer­ing.

Big Cy­press Ac­qui­si­tion, found­ed just this year, ex­pects to call on a mar­ket val­ue of $95 mil­lion. The com­pa­ny will of­fer 7.5 mil­lion units of com­mon stock — priced at $10 — and each unit will al­so hold one-half of a war­rant, which can be ex­er­cised at $11.50.

Laden­burg Thal­mann is the on­ly bookrun­ner on the of­fer­ing, BCA said. The com­pa­ny is led by Samuel Re­ich, who co-found­ed Bis­cayne Neu­rother­a­peu­tics in 2011 (it was lat­er ac­quired by Su­per­nus). Re­ich is joined at BCA by chair­man Jef­frey Spra­gens, who co-found­ed SafeStitch Med­ical in 2005 and was its CEO un­til 2013.

BCA says it plans on tar­get­ing growth-ori­ent­ed life sci­ence com­pa­nies that of­fer prod­ucts with the po­ten­tial to ad­dress un­met needs in the med­ical field. — Con­ner Mitchell

How Pa­tients with Epilep­sy Ben­e­fit from Re­al-World Da­ta

Amanda Shields, Principal Data Scientist, Scientific Data Steward

Keith Wenzel, Senior Business Operations Director

Andy Wilson, Scientific Lead

Real-world data (RWD) has the potential to transform the drug development industry’s efforts to predict and treat seizures for patients with epilepsy. Anticipating or controlling an impending seizure can significantly increase quality of life for patients with epilepsy. However, because RWD is secondary data originally collected for other purposes, the challenge is selecting, harmonizing, and analyzing the data from multiple sources in a way that helps support patients.

$DNA is once again on NYSE; FDA clears Soliris chal­lenger for the mar­ket; Flag­ship’s think­ing big again with eR­NA; and more

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I still remember the uncertainty in the air last year when nobody was sure whether ASCO would cancel their in-person meeting. But it’s now back again for the second virtual conference, and Endpoints News is here for it. Check out our 2-day event reviewing the landscape of cancer R&D and send news our way.

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Gene ther­a­py from Bio­gen's $800M buy­out flops in mid-stage study, deal­ing blow to new am­bi­tions

The #2 candidate from Biogen’s $800 million ocular gene therapy buyout has failed in a mid-stage trial, dealing an early blow to the big biotech’s plans to revitalize its pipeline with new technologies.

Biogen announced that the candidate, an experimental treatment for a rare and progressive form of blindness called X-linked retinitis pigmentosa (XLRP), failed to sufficiently improve vision in patients’ treated eye — patients only received an injection in one eye — after a year, on a standard scale, compared to their untreated eye. The company said they saw “positive trends” on several secondary endpoints, including visual acuity, but declined to say whether the trial actually hit any of those endpoints.

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Vas Narasimhan (Photographer: Simon Dawson/Bloomberg via Getty Images)

No­var­tis whiffs on En­tresto study af­ter heart at­tacks — but that does­n't mean it's go­ing down qui­et­ly

If Novartis learned one thing from its interaction with the FDA over its latest heart failure approval for Entresto, it was that missing a primary endpoint may not be the nail in the coffin. Now, Entresto has missed again on a late-stage study in high-risk heart patients, and it’s already sowing the seeds for a path forward regardless.

Novartis’ Entresto couldn’t best standard-of-care ramipril in staving off a composite of deaths and heart failure events in patients with left ventricular systolic dysfunction and/or pulmonary congestion who have had a prior heart attack, according to topline data from the Phase III PARADISE-MI study revealed Saturday at the virtual American College of Cardiology meeting.

Michael Dell (Richard Drew, AP Images)

'Dude, you're get­ting a Del­l' — as a new deep-pock­et biotech in­vestor

What happens when you marry longtime insiders in the global biotech VC game with the family fund of tech billionaire Michael Dell, a synthetic biology legend out of MIT and Harvard and the former director of the NCI?

Today, the answer is a newly financed, $200 million biotech SPAC now cruising the industry for a top player interested in finding a short cut to Nasdaq.

Orion Biotech Opportunities priced their blank check company today, raising $200 million with Dell’s multibillion-dollar MSD group’s commitment on investing another $20 million in a forward-purchase agreement.

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BAR­DA slows its $9B en­gine for new Covid-19 ther­a­peu­tics

The Biomedical Advanced Research and Development Authority is cooling its jets in looking for new, potential Covid-19 treatments, at least in the near term.

An HHS spokesperson told Endpoints News via email, “to date, BARDA has obligated more than $9 billion for the development and/or purchase of 13 therapeutics, beginning in February 2020 with support to develop Regeneron’s monoclonal antibody therapeutic. Therapeutics are an important element of the COVID-19 response, and we are focused on the programs currently underway and/or in negotiation using the funds available to us.”

Scynex­is takes out $60M loan ahead of ex­pect­ed ap­proval; Com­pass Ther­a­peu­tics ac­quires new bis­pe­cif­ic an­ti­body in buy­out

As the PDUFA date for their vaginal yeast infections quickly approaches, Scynexis is taking out a loan to ensure it can hit the ground running for a potential commercial launch.

Scynexis closed terms on a $60 million loan with Hercules Capital and Silicon Valley Bank, the New Jersey biotech announced Friday, with its ibrexafungerp candidate set for a June 1 PDUFA. Approval of the program is anticipated, the company said, after which the oral antifungal will be branded as Brexafemme.

Bris­tol My­ers backs up its case for heart drug mava­camten as FDA weighs app in car­diomy­opa­thy

When Bristol Myers Squibb signed off on its $13 billion acquisition of MyoKardia back in October, it was making a big bet that lead drug mavacamten could prove a game changer in cardiac myopathy. Now, with the drug up for FDA review, Bristol Myers is backing up its case with new quality of life data.

Patients dosed with myosin inhibitor mavacamten posted a clinically significant increase in scores on the Kansas City Cardiomyopathy Questionnaire, a catch-all summary of symptoms and quality of life markers, over placebo at 30 weeks, according to data from the Phase III EXPLORER-HCM study presented Saturday at the virtual American College of Cardiology meeting.

UP­DAT­ED: Apel­lis bags FDA nod for Soliris chal­lenger with a dif­fer­ent path­way to PNH — but can it slay the gi­ant?

With a blockbuster rare disease giant in its sights in Alexion’s Soliris, small biotech Apellis has reason to think its competitor is worthy of the spotlight. Now, with the FDA on its side, Apellis will get its chance to be the David to Alexion’s Goliath.

The FDA on Friday approved Empaveli (pegcetacoplan), a C3 complement inhibitor the biotech thinks can prove a worthy challenger to Alexion’s C5 inhibitors Soliris and follow-up drug Ultomiris in rare disease paroxysmal nocturnal hemoglobinuria (PNH).