CEO Jeremy Bender (Day One)

With 'rapid' progress of pe­di­atric brain can­cer treat­ment, Day One sees broad ex­cite­ment in new crossover round

Rough­ly nine months af­ter emerg­ing from stealth, Day One Bio­phar­ma­ceu­ti­cals re­turned to the ven­ture cap­i­tal well and came away with a nine-fig­ure prize. And with the hefty crossover round, it rais­es the big ques­tion of whether they’re prep­ping an S-1 to en­ter a hot biotech IPO mar­ket.

Day One raised $130 mil­lion in a Se­ries B, the biotech an­nounced Wednes­day, with the fund­ing led by RA Cap­i­tal. The funds will help sup­port a va­ri­ety of pipeline projects, in­clud­ing the de­vel­op­ment of their lead com­pound DAY101 in prepa­ra­tion for a po­ten­tial com­mer­cial launch in 2023.

The rapid progress of that pro­gram ul­ti­mate­ly led to the ex­cite­ment for the round, CEO Je­re­my Ben­der told End­points News, and Day One has now raised a to­tal of $190 mil­lion since late 2019.

“As a re­sult of both some pub­li­ca­tion of da­ta that un­der­pinned the de­vel­op­ment plan, as well as build­ing out the full team in­clud­ing my­self, we start­ed hav­ing dis­cus­sions with in­vestors in Q4,” Ben­der said. “Those ac­cel­er­at­ed over time.”

Day One’s mis­sion is cen­tered around im­prov­ing the land­scape for pe­di­atric can­cers, an area that the com­pa­ny says phar­ma has left be­hind over the last sev­er­al years. Chil­dren rep­re­sent a much small­er pa­tient pool than adults, giv­ing the in­dus­try less mar­ket in­cen­tive, and for years ex­perts have said the bi­ol­o­gy has not been prop­er­ly un­der­stood.

With ad­di­tion­al con­cerns over safe­ty, many pe­di­atric treat­ments end up be­ing re­for­mu­la­tions of adult ther­a­pies like ra­di­a­tion and chemo. But that can come with heavy long-term side ef­fects. Day One aims to fill that gap, bring­ing ef­fec­tive and safe treat­ment specif­i­cal­ly with chil­dren in mind, Ben­der said.

They got start­ed with an old Take­da pro­gram in DAY101, for­mer­ly named TAK-580. The com­pound it­self is a pan-RAF in­hibitor that can cross the blood-brain bar­ri­er, block­ing mu­ta­tions that dri­ve can­cer in both child­hood and adult gliomas.

Day One has made sig­nif­i­cant progress with this pro­gram since it came out of stealth mode last May, Ben­der said, re­cent­ly launch­ing a piv­otal Phase II study in the most com­mon brain tu­mor in chil­dren — pe­di­atric low-grade glioma. Cur­rent­ly, pe­di­atric pa­tients with pLGG don’t have much in the way of ef­fec­tive treat­ment, uti­liz­ing typ­i­cal plat­inum-based chemother­a­py in the front­line set­ting with no clear fa­vorite be­yond that.

About a third of pa­tients see their tu­mors ef­fec­tive­ly cured through biop­sies and sur­gi­cal re­moval, Ben­der said, but the “vast ma­jor­i­ty” of the rest go on to re­ceive these sys­temic chemo treat­ments. “It’s hard on pa­tients and par­ents, but the on­ly clear stan­dard of care,” he said.

The com­pa­ny is look­ing to en­roll 60 pa­tients in a sin­gle-arm, open-la­bel tri­al, which would form the ba­sis for an ap­proval pack­age once the da­ta read out topline re­sults in the sec­ond half of 2022. The ex­per­i­men­tal drug al­so re­ceived break­through ther­a­py des­ig­na­tion from the FDA in the fall, and it was around that time when Se­ries B in­ter­est from VCs start­ed ramp­ing up.

With Wednes­day’s fund­ing, Day One has enough run­way to get through the end of 2022 and through the Phase II read­out “at min­i­mum,” Ben­der said. Should every­thing go well with the study, a com­mer­cial launch of DAY101 could be in the cards as ear­ly as 2023.

Day One is al­so look­ing at test­ing this pro­gram in adult sol­id tu­mors with RAF-al­tered mu­ta­tions, and ex­pects to launch a Phase II study with the fund­ing. The can­di­date had pre­vi­ous­ly been test­ed in melanoma, but not yet in the adult brain can­cer set­ting.

But the main mis­sion re­mains fo­cused on chil­dren, and there is clear en­thu­si­asm sur­round­ing DAY101 as ev­i­denced by Wednes­day’s raise, Ben­der said.

In ad­di­tion to RA Cap­i­tal, oth­er new in­vestors in­clud­ed Box­er Cap­i­tal, BVF Part­ners, Franklin Tem­ple­ton, Janus Hen­der­son In­vestors, Per­cep­tive Ad­vi­sors, T. Rowe Price and As­so­ci­ates and Viking Glob­al In­vestors. Ex­ist­ing in­vestors in Canaan, Ac­cess Biotech­nol­o­gy and At­las Ven­ture al­so par­tic­i­pat­ed in the round.

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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.

Uğur Şahin, BioNTech CEO (Kay Nietfeld/picture-alliance/dpa/AP Images)

De­spite falling Covid-19 sales, BioN­Tech main­tains '22 sales guid­ance

While Pfizer raked in almost $28 billion last quarter, its Covid-19 vaccine partner BioNTech reported a rise in total dose orders but a drop in sales.

The German biotech reported over $3.2 billion in revenue in Q2 on Monday, down from more than $6.7 billion in Q1, in part due to falling Covid sales. While management said last quarter that they anticipated a Covid sales drop — CEO Uğur Şahin said at the time that “the pandemic situation is still very much uncertain” — Q2 sales still missed consensus by 14%.

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Ted Love, Global Blood Therapeutics CEO

Up­dat­ed: Pfiz­er scoops up Glob­al Blood Ther­a­peu­tics and its sick­le cell ther­a­pies for $5.4B

Pfizer is dropping $5.4 billion to acquire Global Blood Therapeutics.

Just ahead of the weekend, word got out that Pfizer was close to clinching a $5 billion buyout — albeit with other potential buyers still at the table. The pharma giant, flush with cash from Covid-19 vaccine sales, apparently got out on top.

The deal immediately swells Pfizer’s previously tiny sickle cell disease portfolio from just a Phase I program to one with an approved drug, Oxbryta, plus a whole pipeline that, if all approved, the company believes could make for a $3 billion franchise at peak.

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FDA commissioner Rob Califf (Tom Williams/CQ Roll Call via AP Images)

With drug pric­ing al­most done, Con­gress looks to wrap up FDA user fee leg­is­la­tion

The Senate won’t return from its summer recess until Sept. 6, but when it does, it officially has 18 business days to finalize the reauthorization of the FDA user fee programs for the next 5 years, or else thousands of drug and biologics reviewers will be laid off and PDUFA dates will vanish in the interim.

FDA commissioner Rob Califf recently sent agency staff a memo explaining how, “Our latest estimates are that we have carryover for PDUFA [Prescription Drug User Fee Act], the user fee funding program that will run out of funding first, to cover only about 5 weeks into the next fiscal year.”

Pascal Soriot, AstraZeneca CEO (David Zorrakino/Europa Press via AP Images)

As­traZeneca and Dai­ichi Sankyo sprint to mar­ket af­ter FDA clears En­her­tu in just two weeks

Regulators didn’t keep AstraZeneca and Daiichi Sankyo waiting long at all for their latest Enhertu approval.

The partners pulled a win on Friday in HER2-low breast cancer patients who’ve already failed on chemotherapy, just two weeks after submitting a supplemental BLA. While this isn’t the FDA’s fastest approval — Bristol Myers Squibb won an OK for its blockbuster checkpoint inhibitor Opdivo in just five days back in March — it comes well ahead of Enhertu’s original Q4 PDUFA date.

David Reese, Amgen R&D chief

UP­DAT­ED: In a fresh dis­ap­point­ment, Am­gen spot­lights a ma­jor safe­ty is­sue with KRAS com­bo

Amgen had hoped that its latest study matching its landmark KRAS G12C drug Lumakras with checkpoint inhibitors would open up its treatment horizons and expand its commercial potential. Instead, the combo spurred safety issues that blunted efficacy and forced the pharma giant to alter course on its treatment strategy, once again disappointing analysts who have been tracking the drug’s faltering sales and limited therapeutic reach.

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GSK and IQVIA launch plat­form of US vac­ci­na­tion da­ta, show­ing drop in adult rates

Throughout the Covid-19 pandemic, the issue of vaccine uptake has been a point of contention, but a new platform from GSK and IQVIA is hoping to shed more light on vaccine data, via new transparency and general awareness.

The two companies have launched Vaccine Track, a platform intended to be used by public health officials, medical professionals and others to strengthen data transparency and display vaccination trends. According to the companies, the platform is intended to aid in increasing vaccine rates and will provide data on trends to assist public health efforts.

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Ab­b­Vie sur­veys emo­tion­al im­pact of chron­ic leukemia con­di­tion, finds 'roller coast­er' of emo­tions

Rare diseases often have more than just physical effects on patients — especially when it comes to chronic conditions. In the case of the rare slow-growing blood cancer chronic lymphocytic leukemia (CLL), AbbVie wanted to try to assess the mental and emotional toll on patients.

So it surveyed more than 300 CLL patients, caregivers and physicians. While each group differed in how they felt — caregivers overwhelmingly (81%) felt positive about their role, for instance — patients described a “roller coaster” of emotions traversing diagnosis to treatment to remission and even relapse for some.

Bernhardt Zeiher, outgoing Astellas CMO (Astellas)

Q&A: Astel­las' re­tir­ing head of de­vel­op­ment re­flects on gene ther­a­py deaths

For anyone who’s been following discussions about the safety alarms surrounding the adeno-associated viruses (AAV) commonly used to deliver gene therapy, Astellas should be a familiar name.

The Japanese pharma — which bought out Audentes Therapeutics near the end of 2019 and later built a gene therapy unit around the acquisition — rocked the field when it reported three patient deaths in a trial testing AT132, the lead program from Audentes designed to treat a rare muscle disease called X-linked myotubular myopathy (XLMTM).

When the company restarted the trial, it adjusted the dose and instituted a battery of other measures to try to prevent the same thing from happening again. But tragically, the first patient to receive the new regimen died just weeks after administration. The therapy remains under clinical hold, and just weeks ago, Astellas flagged another safety-related hold for a separate gene therapy candidate. In the process of investigating the deaths, the company has also taken flak about the way it disclosed information.

Big questions remain — questions that can have big implications about the future of AAV gene therapies.

Bernhardt Zeiher did not imagine any of it when he first joined Astellas as the therapeutic area leader in inflammation, immunology and infectious diseases. But his ascent to chief medical officer and head of development coincided almost exactly with Astellas’ big move into gene therapy, putting him often in the driver’s seat to grapple with the setbacks.

As Zeiher prepares to retire next month after a 12-year tenure — leaving the unfinished tasks to his successor, a seasoned cancer drug developer — he chatted with Endpoints News, in part, to discuss the effort to understand what happened, lessons learned and the criticism along the way.

The transcript has been lightly edited for length and clarity.

Endpoints: I want to also ask you a bit about the gene therapy efforts you’ve been working on. Astellas has really been at the forefront of discovering the safety concerns associated with AAV gene therapy. What’s that been like for you?

Zeiher: Well, I have to admit, it’s been a bit of a roller coaster. We acquired Audentes. Huge amount of enthusiasm. What we saw with AT132 — that was the lead program in XLMTM — was just remarkable efficacy. I mean, kids who went from being on ventilators, not able to eat for themselves, sit up, do things like that, to off ventilators, walking, you know, really — one investigator called it this Lazarus-like effect. It was just really dramatic efficacy. And then to have the safety events that occurred. So they actually occurred within that first year of the acquisition. So we had the three patient deaths. Me and my organization became very, very much involved. In fact, Ed Conner, who had been the chief medical officer, he left after some of the deaths, but I stepped in as the kind of acting chief medical officer, we had another chief medical officer who was involved, and then we had a fourth death, and I became acting again for a period of time.

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