CEO Jeremy Bender (Day One)

It’s a new $DAWN for Day One as start­up piv­ots to an IPO for more fund­ing to back its work in pe­di­atric can­cer

Pe­di­atric can­cer biotech Day One Phar­ma­ceu­ti­cals is head­ed for Nas­daq, in a move that comes less than three months af­ter their work on a brain can­cer treat­ment led to a nine-fig­ure crossover round backed by some blue-chip in­vestors.

The South San Fran­cis­co-based com­pa­ny is pen­cil­ing in $100 mil­lion as their ini­tial IPO tar­get, though the ul­ti­mate raise will like­ly be high­er. Tues­day’s fil­ing comes af­ter RA Cap­i­tal led a $130 mil­lion B round for the com­pa­ny in Feb­ru­ary, fol­low­ing what CEO Je­re­my Ben­der said at the time was “rapid” progress of their lead pro­gram.

When Day One prices in a few weeks, it will trade un­der the tick­er $DAWN.

As the phar­ma in­dus­try con­tin­ues de­vel­op­ing and push­ing for­ward can­cer treat­ments, Day One ex­ecs ar­gue that the main­stream play­ers left pe­di­atric can­cer be­hind. Adult pa­tients make up a sig­nif­i­cant­ly big­ger por­tion of the mar­ket, the com­pa­ny said, and there have been lags in un­der­stand­ing pe­di­atric bi­ol­o­gy prop­er­ly.

Day One’s mis­sion, then, is to try to fill that gap by de­vel­op­ing tar­get­ed ther­a­pies aimed specif­i­cal­ly at chil­dren. Their pro­posed treat­ments are en­tire­ly new ef­forts rather than re­for­mu­lat­ing adult treat­ments that come with heavy side ef­fects, like ra­di­a­tion ther­a­py and chemo.

Their lead pro­gram is a for­mer Take­da pro­gram called DAY101, an oral pan-RAF in­hibitor that can cross the blood-brain bar­ri­er and block mu­ta­tions in gliomas. As such, Day One is aim­ing for an in­di­ca­tion to treat pe­di­atric low-grade gliomas, the most com­mon form of brain tu­mors in chil­dren. The cur­rent stan­dard of care is plat­inum-based chemo, with no clear fa­vorite be­yond that, Ben­der told End­points News in Feb­ru­ary.

The biotech re­cent­ly launched a Phase II study for the ex­per­i­men­tal drug, with the goal of read­ing out top-line re­sults in the first half of 2022. Day One plans to ul­ti­mate­ly en­roll 60 pa­tients in the sin­gle-arm, open-la­bel study and hopes it can form the ba­sis of an ap­proval pack­age.

With­in its S-1, Day One plans to fun­nel its cash not on­ly to­ward this Phase II tri­al, but al­so launch a new Phase III study for the front­line treat­ment of pe­di­atric low grade gliomas next year. Its goals al­so in­clude a Phase II tri­al for RAF-al­tered sol­id tu­mors in pa­tients old­er than 12, as well as a Phase Ib/II study com­bin­ing DAY101 with Mer­ck Kg­GA’s MEK in­hibitor pi­masert­ib to treat adult MAPK-al­tered sol­id tu­mors.

The S-1 al­so pro­vid­ed some de­tails re­gard­ing that deal with Take­da and the com­pa­ny’s eq­ui­ty. Day One on­ly paid $1 mil­lion for the DAY101 can­di­date and at the time of the agree­ment, De­cem­ber 2019, of­fered a Take­da sub­sidiary about a 12% stake in the com­pa­ny. The sub­sidiary, Mil­len­ni­um Phar­ma­ceu­ti­cals, will con­tin­ue to own that stake when Day One goes pub­lic.

Ben­der for his part, owns a 3% stake, while CMO and founder Sam Black­man has a 4.6% stake. The biggest stake­hold­er is Canaan Part­ners at 21.9%, fol­lowed up by the At­las Ven­ture Fund at 16.9%.

Day One al­so not­ed that Derek DiRoc­co, the RA Cap­i­tal part­ner who joined the board with Feb­ru­ary’s raise, will be leav­ing the com­pa­ny af­ter a very short stint once the biotech be­gins trad­ing pub­licly.

Biotech IPOs con­tin­ue to boom as 2021 march­es for­ward, with more than 50 com­pa­nies hav­ing ei­ther filed or priced their IPOs so far this year. For the year, the com­bined biotech raise is ap­proach­ing $6.5 bil­lion, and is ex­pect­ed to reach near­ly $7 bil­lion lat­er this week when four more com­pa­nies make their Nas­daq de­buts.

Pi­o­neer­ing Click Chem­istry in Hu­mans

Reimagining cancer treatments

Cancer is a leading cause of death worldwide, accounting for nearly 10 million deaths in 2020, which is nearly one in six deaths. Recently, we have seen incredible advances in novel cancer therapies such as immune checkpoint inhibitors, cell therapies, and antibody-drug conjugates that have revamped cancer care and improved survival rates for patients.

Despite this significant progress in therapeutic targeting, why are we still seeing such a high mortality rate? The reason is that promising therapies are often limited by their therapeutic index, which is a measure of the effective dose of a drug, relative to its safety. If we could broaden the therapeutic indices of currently available medicines, it would revolutionize cancer treatments. We are still on the quest to find the ultimate cancer medicine – highly effective in several cancer types, safe, and precisely targeted to the tumor site.

Ivan Cheung, Eisai US chairman and CEO

Bio­gen, Ei­sai re­fresh amy­loid hy­poth­e­sis with PhI­II show­ing Alzheimer's med slows cog­ni­tive de­cline

In the first look at Phase III data for lecanemab, Eisai and Biogen’s follow-up Alzheimer’s drug to the embattled Aduhelm launch, results show the drug passed with flying colors on a test looking at memory, problem solving and other dementia metrics.

One of the most-watched Alzheimer’s therapies in the clinic, lecanemab met the study’s primary goal on the CDR-SB — Clinical Dementia Rating-Sum of Boxes — giving the biotech the confidence to ask for full approval in the US, EU and Japan by next March 31. The experimental drug reduced clinical decline on the scale by 27% compared to placebo at 18 months, the companies said Tuesday night Eastern time and Wednesday morning in Japan.

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Nooman Haque, head of life sciences and healthcare at Silicon Valley Bank, and John Carroll

I’m head­ed to Lon­don soon for #EU­BIO22. Care to join me?

It was great getting back to a live ESMO conference/webinar in Paris followed by a live pop-up event for the Endpoints 11 in Boston. We’re staying on the road in October with our return for a live/streaming EUBIO22 in London.

Silicon Valley Bank’s Nooman Haque and I are once again jumping back into the thick of it with a slate of virtual and live events on October 12. I’ll get the ball rolling with a virtual fireside chat with Novo Nordisk R&D chief Marcus Schindler, covering their pipeline plans and BD work.

Gilead names 'k­ing­pin­s' in coun­ter­feit HIV med law­suit

Gilead is mounting its counterfeit drug lawsuit, naming two “kingpins” and a complex network of conspirators who allegedly sold imitation bottles of its HIV meds, some of which ended up in US pharmacies.

The pharma giant on Wednesday provided an update on what it called a “large-scale, sophisticated counterfeiting conspiracy,” accusing two new defendants of “leading and orchestrating” a scheme to sell hundreds of millions of dollars in illegitimate drugs posing as meds such as Biktarvy and Descovy.

Vlad Coric, Biohaven CEO (Photo Credit: Andrew Venditti)

As Amy­lyx de­ci­sion waits in the wings, Bio­haven’s ALS drug sinks (again) in plat­form tri­al

The FDA’s decision on Amylyx’s ALS drug is set to come out sometime Thursday. In a space with few drugs, any approval would be a major landmark.

But elsewhere in the ALS field, things are a bit more tepid.

Thursday morning, Biohaven announced that its drug verdiperstat failed its arm of an ALS platform trial led by Massachusetts General Hospital. According to a press release, the drug did not meet its primary endpoint — improvement on an ALS functional status test — or any key secondary endpoints at 24 weeks. The trial had enrolled 167 patients, giving them either verdiperstat or placebo twice a day.

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Tar­sus looks to raise aware­ness of eye­lid mite dis­ease in cam­paign aimed at eye­care spe­cial­ists

Eyelid mite disease may be “gross” but it’s also fairly common, affecting about 25 million people in the US.

Called demodex blepharitis, it’s a well-known condition among eyecare professionals, but they often don’t always realize how common it is. Tarsus Pharmaceuticals wants to change that with a new awareness campaign called “Look at the Lids.”

The campaign and website debut Thursday — just three weeks after Tarsus filed for FDA approval for a drug that treats the disease.

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Marcelo Bigal, Ventus Therapeutics CEO

No­vo Nordisk joins No­var­tis, Roche in NL­RP3 are­na, bet­ting $70M cash on NASH, car­diometa­bol­ic us­es

As a drug target, the NLRP3 inflammasome has drawn serious interest from Big Pharma, inspiring a series of M&A deals from Novartis and Roche on top of venture investments by others. Now Novo Nordisk is jumping on the bandwagon — and the Danish pharma giant is taking the target where it knows best.

Novo Nordisk is getting its NLRP3 inhibitors from Ventus Therapeutics, a Versant-backed startup that set out to make some of the best NLRP3 drugs out there by incorporating new insights into the structure of the target complex.

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Work taking place in the clean rooms at Vor (Credit: Vor)

Vor Bio opts to keep man­u­fac­tur­ing op­er­a­tions in-house for de­vel­op­ing stem cell, CAR-T ther­a­pies

While it is not uncommon for a biotech to go down the route of having the product manufactured by a contract organization, one small biotech is looking to keep its card close to its chest.

Vor Biopharma has started manufacturing operations at an in-house facility at its HQ in Cambridge, MA after beginning construction last summer.

According to the biotech, the facility aims to develop Vor’s hematopoietic stem cells (eHSCs) and CAR-T therapies for patients with blood cancers. The site will initially manufacture a clinical supply of its candidate VCAR33allo to support its IND, which is slated to be submitted in the first half of next year. It also plans to transfer the production of VOR33 to the facility. Vor is getting to work quickly as engineering runs for VCAR33allo has started this week.

Aim­ing for fourth nod, Sarep­ta files an­oth­er DMD gene ther­a­py to FDA; Ax­some head­ed to­ward mi­graine re­sub­mis­sion

Sarepta Therapeutics has filed the data needed for an FDA accelerated approval, which would be the biotech’s fourth if granted by the agency.

The biotech has yet to complete confirmatory trials for those first three conditional nods. The filing for its fourth Duchenne muscular dystrophy treatment, disclosed Thursday, is not a surprise. Sarepta said in late-July it would do so after releasing positive results for the Roche-partnered gene therapy.