Three biotechs set terms in a hunt for $475M-plus; Leap Ther­a­peu­tics earns a tri­al win across DKK1 high ex­press­ing tu­mors

We have a slate of biotech IPO terms to re­port this morn­ing.

De­sign Ther­a­peu­tics is ap­ply­ing a high val­u­a­tion to the com­pa­ny. The pre­clin­i­cal biotech wants to sell 12 mil­lion shares at $18 to $20 a pop, rais­ing more than $200 mil­lion with a hoped-for de­but val­u­a­tion of $1.1 bil­lion.

The gene­TAC com­pa­ny chose {DS­GN} for its tick­er.

The pre­clin­i­cal on­col­o­gy biotech Ike­na {IK­NA} says it will be sell­ing 7.8 mil­lion shares at $15 to $17 each for around $125 mil­lion. If suc­cess­ful, the com­pa­ny could de­but with a mar­ket cap worth more than $600 mil­lion.

Ike­na’s lead drug is an in­hibitor of the TEAD tran­scrip­tion fac­tor in the Hip­po sig­nal­ing path­way.

Edge­wise Ther­a­peu­tics {EWTX} is ac­tu­al­ly in the clin­ic, with a Phase I tri­al un­der­way for EDG-5506. The drug has am­bi­tions in dy­s­trophinopathies, tar­get­ing Duchenne mus­cu­lar dy­s­tro­phy, Beck­er mus­cu­lar dy­s­tro­phy, and limb-gir­dle mus­cu­lar dy­s­tro­phy.

The biotech is out to sell 10 mil­lion shares at $14 to $16 a share look­ing to raise about $150 mil­lion. — John Car­roll

Leap Ther­a­peu­tics takes home win in DKK1 ‘bas­ket’ study

Leap Ther­a­peu­tics’ DKK1 tar­get­ing mon­o­clon­al an­ti­body DKN-01 scored a Phase II win in a “bas­ket” study try­ing the drug as a monother­a­py or a com­bi­na­tion with pa­cli­tax­el in pa­tients with ei­ther ep­ithe­lial en­dome­tri­al can­cer, ep­ithe­lial ovar­i­an can­cer, or car­ci­nosar­co­ma.

Across the 111-pa­tient study’s six sub­groups, Leap found that DKN-01 per­formed best in a buck­et of pa­tients tagged as DKK1 “high ex­press­ing,” the biotech said. In­ves­ti­ga­tors found pa­tients with EEC and those with a high fre­quen­cy of Wnt sig­nal­ing path­way mu­ta­tions ex­pressed the DKK1 pro­tein at ab­nor­mal­ly high lev­els, po­ten­tial­ly of­fer­ing up two sub­groups for fu­ture stud­ies.

DKK1 has been im­pli­cat­ed in the tu­mor growth, pro­lif­er­a­tion and im­muno­sup­pres­sion path­ways, en­abling tu­mors to evade the im­mune sys­tem. DKN-01 looks to reg­u­late DKK1 ex­pres­sion, giv­ing the in­nate im­mune sys­tem a shot at bet­ter tar­get­ing tu­mors.

In an odd foot­note, Leap had sev­en pa­tients it bun­dled in­to the DKK1 “high ex­press­ing” cat­e­go­ry even though they didn’t have the tu­mor ma­te­r­i­al avail­able to de­ter­mine whether that bio­mark­er ac­tu­al­ly showed up in tests. On a call with an­a­lysts Mon­day, the Leap team ex­plained that it opt­ed to in­clude those pa­tients with­out work­able tu­mor biop­sies in­to the high ex­press­ing group af­ter con­sid­er­ing sec­ondary bio­mark­ers and DKN-01’s ef­fect on over­all tu­mor re­sponse rate and PFS. — Kyle Blanken­ship

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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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).