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.

MedTech clinical trials require a unique regulatory and study design approach and so engaging a highly experienced CRO to ensure compliance and accurate data across all stages is critical to development milestones.

In­no­v­a­tive MedTech De­mands Spe­cial­ist Clin­i­cal Tri­al Reg­u­la­to­ry Af­fairs and De­sign

Avance Clinical is the Australian CRO for international biotechs providing world-class clinical research services with FDA-accepted data across all phases. With Avance Clinical, biotech companies can leverage Australia’s supportive clinical trials environment which includes no IND requirement plus a 43.5% Government incentive rebate on clinical spend. The CRO has been delivering clinical drug development services for international biotechs for FDA and EMA regulatory approval for the past 24 years. The company has been recognized for the past two consecutive years with the prestigious Frost & Sullivan CRO Best Practices Award and a finalist in Informa Pharma’s Best CRO award for 2022.

Who are the women blaz­ing trails in bio­phar­ma R&D? Nom­i­nate them for End­points' 2022 spe­cial re­port

Over the past three years, Endpoints News has spotlighted 60 women who have blazed trails and supercharged R&D across the biopharma world. And judging from the response we’ve received, to both our special reports and live events, telling their stories — including any obstacles they may have had to overcome — has inspired our readers in many different ways.

But change takes time, and the fact remains that women are still underrepresented at the upper ranks of the drug-making world.

Up­dat­ed: Amid mas­sive re­struc­tur­ing, Bio­gen looks to re­duce phys­i­cal pres­ence in Boston

Biogen is putting a sizable chunk of office and research space in Kendall Square and Weston, MA up for sublease, marking another big change as the biotech grapples with the aftershock of a disastrous and controversial rollout for its Alzheimer’s drug.

The subbleases are “part of Biogen’s overall implementation of the ‘Future of Work,’ which is allowing us to optimize our footprint and reduce the amount of space we occupy, taking into consideration new elements such as the hybrid work model,” Biogen spokesperson Ashleigh Koss wrote in a statement to Endpoints News, adding that the company has had subleases across several buildings for years.

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Hervé Affagard, MaaT Pharma CEO

One year in­to clin­i­cal hold, FDA has more ques­tions about 'pooled' mi­cro­bio­me ther­a­py

The FDA is still wary about a trial testing a microbiome therapy in patients with steroid-resistant acute graft-versus-host disease (aGVHD).

A year after MaaT Pharma’s IND application in the US was first met with a clinical hold, the French biotech said the agency is maintaining the hold. The crux of the matter, MaaT suggested, has to do with the way it puts together its drug candidate, which is administered as an enema (i.e. an injection of fluid into the bowel).

Pfiz­er launch­es re­bate pro­gram for rare dis­ease pa­tients who have to stop tak­ing Panzy­ga

Pfizer is launching its second-ever rebate program, this time for Panzyga, its treatment for a rare neurological disease of the peripheral nerves.

The program began last month, according to STAT which first reported the news, and offers a refund of out-of-pocket costs for patients who must discontinue their course before the fifth treatment for “clinical reasons.”

Panzyga was approved back in 2018 to treat primary immunodeficiency (PI) in patients two years and older and chronic immune thrombocytopenia (cITP) in adults. It has since picked up an indication in chronic inflammatory demyelinating polyneuropathy (CIDP), a condition that’s characterized by weakness of the arms or legs, tingling or numbness, and a loss of deep tendon reflexes, according to the NIH.

Horizon's back-to-school campaign for children with cystinosis includes an all about me poster as part of a care package box.

Hori­zon read­ies kids and fam­i­lies for back to school with week­long ac­tiv­i­ties around rare dis­ease cysti­nosis

Going back to school is usually a bumpy readjustment from summer freedom for all kids, but especially for kids with chronic health conditions. Horizon Therapeutics is hoping to help smooth the way for some who have the rare disease cystinosis. Cystinosis is a genetic disease that causes the amino acid cystine to build up in different tissues and organs.

The “Gear Up” for school campaign is running all week with different online and at-home events and activities for families and children with cystinosis. Each family who signed up receives a care package mailed to their home including an activity coloring book “Michael’s Show-and-Tell.” The book tells Michael’s story about living with cystinosis while offering kids matching, coloring and finding object games along with information.

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Alessandro Maselli, Catalent CEO

Catal­ent ac­quires North Car­oli­na CD­MO for $475M, boost­ing oral solids work

As Catalent has been expanding its reach in the US this year, as well as recently completing a C-suite shuffle, the company announced last night that it has acquired the CDMO Metrics Contract Services for $475 million from Mayne Pharma Group.

The acquisition will increase Catalent’s capabilities in oral solid formulation development, manufacturing and packaging as well as expand its capacity to handle more highly potent compounds.

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Phar­mas spend mil­lions on di­a­betes ad­ver­tis­ing, but few pa­tients can re­call brand names — sur­vey

While many Big Pharma diabetes brands spend millions of dollars on TV ads every year, most people with type 2 diabetes don’t recognize specific drug brand names, according to a new study.

No brand garnered more than 30% recognition in Phreesia Life Science’s latest in-office patient survey of more than 4,000 adults with type 2 diabetes. Eli Lilly’s Trulicity topped the list as the most recognized brand with 29% of those surveyed recalling it, followed by Boehringer Ingelheim and Lilly’s Jardiance at 27% and Merck’s Januvia and Novo Nordisk tying for the third spot with 24%. Meanwhile, 76% of the patients surveyed were familiar with the generic treatment metformin.

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Faced with thou­sands of opi­oid law­suits, En­do says it will like­ly file for bank­rupt­cy 'im­mi­nent­ly'

Endo International will likely be the next pharma company to file for bankruptcy under a mountain of opioid lawsuits.

The Dublin, Ireland-based company revealed in its Q2 results on Tuesday that it’s in talks with first lien creditors, and that “these negotiations will likely result in a pre-arranged filing under Chapter 11 of the U.S. Bankruptcy Code by Endo International plc and substantially all of its subsidiaries, which could occur imminently.”