News brief­ing: Four biotechs an­nounce IPO terms, set­ting the pace to round out a busy year; FDA sets PDU­FA date for Kad­mon's graft-ver­sus-host drug

Four more biotechs set the terms for their IPOs, lin­ing up yet an­oth­er busy week on Wall Street.

Sil­ver­back Ther­a­peu­tics, which ini­tial­ly filed for a $100 mil­lion raise, is now shoot­ing for $125 mil­lion from 7 mil­lion shares at a range of $17 to $19. About $70 mil­lion is tagged for the com­pa­ny’s lead Phase I/Ib an­ti­body-drug con­ju­gate, SBT6050, for ad­vanced or metasta­t­ic HER2-ex­press­ing sol­id tu­mors. In­ter­im da­ta from the Phase I dose-es­ca­la­tion co­horts are ex­pect­ed in the sec­ond half of 2021. An­oth­er $55 mil­lion is set aside for Sil­ver­back’s two oth­er can­di­dates, which have yet to reach the clin­ic.

Kin­nate Bio­phar­ma is look­ing for a $170 mil­lion raise, and set a $16 to $18 range for its 10 mil­lion share of­fer­ing. About $105 is ear­marked for its RAF in­hibitors, in­clud­ing its lead pre­clin­i­cal can­di­date KIN002787 for pa­tients with lung can­cer, melanoma and oth­er sol­id tu­mors. An IND is com­ing in the first half of 2021, the com­pa­ny says. Back in Au­gust, it hooked a $98 mil­lion Se­ries C.

Seer al­so set its shares at a $16 to $18 range. The com­pa­ny, which is work­ing on next-gen pro­teome analy­sis tests, is of­fer­ing 8.8 mil­lion shares, go­ing for a $150 raise. About $65.0 mil­lion would go to its Pro­teo­graph Prod­uct Suite, which al­ready has one col­lab­o­ra­tor and could have a sec­ond by the end of the year.

Sig­ilon Ther­a­peu­tics is seek­ing $101 mil­lion from 5.6 mil­lion shares at a $17 to $19 range. Be­tween $30 to $35 mil­lion will be set aside for its lead can­di­date, SIG-001, which is in a Phase I/II tri­al to pre­vent bleed­ing episodes in pa­tients with he­mo­phil­ia A. An­oth­er $30 to $35 mil­lion would fund a scale-up of the com­pa­ny’s GMP man­u­fac­tur­ing process­es SIG-001 and SIG-005, its pre­clin­i­cal can­di­date for pa­tients with mu­copolysac­cha­ri­do­sis type 1, or MPS-1.

More than 72 biotech and bio­phar­ma com­pa­nies and count­ing have hit Nas­daq so far this year, and head of health­care list­ings Jor­dan Saxe pre­dict­ed round­ing out the year with just un­der $14 bil­lion in pro­ceeds.

FDA sets PDU­FA date for Kad­mon’s graft-ver­sus-host drug

Kad­mon’s chron­ic graft-ver­sus-host drug belu­mo­sudil is in the FDA’s hands.

The agency ac­cept­ed the biotech’s NDA un­der its re­al-time on­col­o­gy re­view (RTOR) pi­lot pro­gram, and set the PDU­FA date for May 30, 2021. The sub­mis­sion was based on pos­i­tive re­sults from a piv­otal, open-la­bel tri­al dubbed ROCK­star, which en­rolled 132 pa­tients who had re­ceived at least two pri­or lines of ther­a­py.

Belu­mo­sudil is de­signed to tamp down the in­flam­ma­to­ry re­sponse seen af­ter hematopoi­et­ic stem cell trans­plant by block­ing Rho-as­so­ci­at­ed coiled-coil ki­nase 2 (ROCK2). In­stead of a com­para­tor arm, Kad­mon set the bar at a 30% over­all re­sponse rate, based on con­ver­sa­tions with the FDA. Pa­tients were giv­en 200 mg of the drug ei­ther once or twice dai­ly. At six months af­ter the com­ple­tion of en­roll­ment, Kad­mon saw an ORR of 73% and 75% in the re­spec­tive arms.

That’s it for da­ta, un­til the full re­port is read out at ASH in De­cem­ber. The com­pa­ny’s stock $KDMN was up 6.68% on Mon­day, at $4.39 per share. Kad­mon was found­ed by Sam Wak­sal, the biotech ex­ec who was sen­tenced to prison for his in­sid­er trad­ing con­vic­tion in­volv­ing Martha Stew­art.

Hookipa Phar­ma reads out in­ter­im re­sults from Phase II CMV vac­cine tri­al

Hookipa Phar­ma said no one from a small group of Phase II par­tic­i­pants treat­ed with its pro­phy­lac­tic cy­tomegalovirus vac­cine came down with the dis­ease, ac­cord­ing to in­ter­im re­sults.

The analy­sis was based on 41 par­tic­i­pants — 8 of whom re­ceived 3 dos­es, 19 of whom re­ceived 2 dos­es, and 14 of whom re­ceived a place­bo. Com­pared to the place­bo, re­searchers saw a 48% re­duc­tion in CMV viremia, and a 42% re­duc­tion in the use of an­tivi­ral ther­a­py. There were no cas­es of CMV dis­ease in the treat­ment arm, com­pared to 2 in the place­bo group.

“While these in­ter­im da­ta are from a small group of pa­tients, they of­fer ear­ly in­sight in­to the po­ten­tial of a three-dose sched­ule of HB-101 to help pro­tect kid­ney trans­plant re­cip­i­ents against CMV dis­ease,” CEO Jo­ern Aldag said in a state­ment.

The can­di­date is go­ing head-to-head with Mod­er­na’s mR­NA-1647, which pro­duced pos­i­tive re­sults from a proof-of-con­cept study back in Jan­u­ary. Re­searchers said they saw an in­creas­ing lev­el of neu­tral­iz­ing an­ti­body titers in those who had re­ceived a third vac­ci­na­tion.

“This could re­al­ly be a com­pa­ny builder,” CEO Stéphane Ban­cel said at the time, adding that the can­di­date could turn in­to a $2 bil­lion to $5 bil­lion an­nu­al fran­chise.

ZS Per­spec­tive: 3 Pre­dic­tions on the Fu­ture of Cell & Gene Ther­a­pies

The field of cell and gene therapies (C&GTs) has seen a renaissance, with first generation commercial therapies such as Kymriah, Yescarta, and Luxturna laying the groundwork for an incoming wave of potentially transformative C&GTs that aim to address diverse disease areas. With this renaissance comes several potential opportunities, of which we discuss three predictions below.

Allogenic Natural Killer (NK) Cells have the potential to displace current Cell Therapies in oncology if proven durable.

Despite being early in development, Allogenic NKs are proving to be an attractive new treatment paradigm in oncology. The question of durability of response with allogenic therapies is still an unknown. Fate Therapeutics’ recent phase 1 data for FT516 showed relatively quicker relapses vs already approved autologous CAR-Ts. However, other manufacturers, like Allogene for their allogenic CAR-T therapy ALLO-501A, are exploring novel lymphodepletion approaches to improve persistence of allogenic cells. Nevertheless, allogenic NKs demonstrate a strong value proposition relative to their T cell counterparts due to comparable response rates (so far) combined with the added advantage of a significantly safer AE profile. Specifically, little to no risk of graft versus host disease (GvHD), cytotoxic release syndrome (CRS), and neurotoxicity (NT) have been seen so far with allogenic NK cells (Fig. 1). In addition, being able to harness an allogenic cell source gives way to operational advantages as “off-the-shelf” products provide improved turnaround time (TAT), scalability, and potentially reduced cost. NKs are currently in development for a variety of overlapping hematological indications with chimeric antigen receptor T cells (CAR-Ts) today, and the question remains to what extent they will disrupt the current cell therapy landscape. Click for more details.

What lured Hal Bar­ron away?; Top FDA minds on ac­cel­er­at­ed ap­proval re­forms; ‘Dead wrong’ Aduhelm ad blitz; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

Nothing can really compete with Hal Barron’s departure from GlaxoSmithKline as the news of the week, but we do have plenty of original reporting and analysis from the Endpoints team in this edition. Enjoy and have a nice weekend.

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Mer­ck wins le­gal bat­tle over in­sur­ance cov­er­age af­ter ran­somware at­tack

Merck has emerged victorious from a years-long legal battle with insurers over the coverage of more than a billion dollars in losses from the malware NotPetya, with a New Jersey Superior Court judge concluding that the responsibility is on insurers to clarify their policies around cyber attacks.

The pharma giant was one of several victims of a global cyber attack back in 2017 that also hit Danish shipping company Maersk, American food company Mondelēz, French construction giant Saint-Gobain and even the systems monitoring the Chernobyl nuclear power stations, Bloomberg reported back in 2019.

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Crit­ics push back on Alzheimer’s As­so­ci­a­tion ad blitz to get Medicare to change its Aduhelm rul­ing: 'Dead wrong'

The latest Alzheimer’s Association advertising campaign encourages people to fight.

Not against the disease or for more research or treatments, but against the Centers for Medicare and Medicaid Services. More specifically, CMS’ recent reimbursement decision to only pay for Biogen and Eisai’s controversial Alzheimer’s drug Aduhelm for patients in clinical trials.

With CMS’ preliminary decision now in a 30-day comment period, patient advocates’ goal is to convince CMS to reverse its decision with a marketing blitz and public pressure.

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Dan O'Day, Gilead CEO (Jim Watson/AFP via Getty Images)

Fail­ing to con­firm clin­i­cal ben­e­fit, Gilead pulls 2 ac­cel­er­at­ed ap­proval in­di­ca­tions for can­cer drug

Gilead recently decided to pull two indications for its cancer drug Zydelig — in relapsed follicular B-cell non-Hodgkin lymphoma (FL) and relapsed small lymphocytic leukemia (SLL) — after failing to complete the confirmatory trials required as part of the accelerated approvals from 2014.

“As the treatment landscape for FL and SLL has evolved, enrollment into the confirmatory study has been an ongoing challenge,” Gilead said in a statement, noting it formally notified the FDA of its decision to voluntarily withdraw these indications.

Yingli Phar­ma brings small mol­e­cule re­search to the US in new pact with MD An­der­son; UCB's pso­ri­at­ic arthri­tis drug shows pos­i­tive PhI­II re­sults

Shanghai biotech Yingli Pharma wants to bring its small molecule drug research global — and a new pact with MD Anderson will help it get there.

Yingli and MD Anderson have inked a 5-year collaboration deal that will put its cancer candidates — some of which have already generated data in China — into trials in the US. The lead program is linperlisib, a PI3Kδ inhibitor that’s in a Phase III trial in follicular lymphoma, according to Yingli’s website. In the US, MD Anderson will work with Yingli to put the candidate in a Phase II trial for peripheral T cell lymphoma (PTCL), an uncommon and aggressive type of non-Hodgkin’s lymphoma.

Richard Pazdur (via AACR)

Time lim­its on ac­cel­er­at­ed ap­provals? FDA's on­col­o­gy chief Rick Paz­dur eyes po­ten­tial re­forms via in­ter­na­tion­al ap­proach­es

The spotlight on the accelerated approval pathway continues to shine bright, with the FDA’s top oncology official writing in an opinion that the pathway may be strengthened with bits and pieces of what other regulators in Europe and elsewhere have done with their expedited approval pathways, such as adding expiration dates for these faster approvals to ensure they confirm clinical benefit in a timely manner.

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Hal Barron, Endpoints UKBIO20 (Jeff Rumans)

'Al­tos was re­al­ly a once-in-a-life­time op­por­tu­ni­ty': Hal Bar­ron re­flects on his big move

By all accounts, Hal Barron had one of the best jobs in Big Pharma R&D. He made more than $11 million in 2020, once again reaping more than his boss, Emma Walmsley, who always championed him at every opportunity. And he oversaw a global R&D effort that struck a variety of big-dollar deals for oncology, neurodegeneration and more.

Sure, the critics never let up about what they saw as a rather uninspiring late-stage pipeline, where the rubber hits the road in the Big Pharma world’s hunt for the next big near-term blockbuster, but the in-house reviews were stellar. And Barron was firmly focused on bringing up the success rate in clinical trials, holding out for the big rewards of moving the dial from an average 10% success rate to 20%.

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Executive Director of the EMA Emer Cooke (AP Photo/Geert Vanden Wijngaert)

Eu­ro­pean Par­lia­ment signs off on strength­en­ing drug reg­u­la­tor's abil­i­ty to tack­le short­ages

The European Parliament on Thursday endorsed a plan to increase the powers of the European Medicines Agency, which will be better equipped to monitor and mitigate shortages of drugs and medical devices.

By a vote of 655 to 31, parliament signed off on a provisional agreement reached with the European Council from last October, in which the EMA will create two shortage steering groups (one for drugs, the other for devices), a new European Shortages Monitoring Platform to facilitate data collection and increase transparency, and on funding for the work of the steering groups, task force, working parties and expert panels that are to be established.

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