Jeremy Bender, Day One CEO

Day One to file NDA for pe­di­atric brain can­cer drug by sum­mer

Day One Bio­phar­ma­ceu­ti­cals will ask reg­u­la­tors to ap­prove its pe­di­atric brain can­cer drug tovo­rafenib be­fore the sec­ond half of 2023, the biotech said Sun­day.

The fil­ing will in­clude top-line da­ta from a piv­otal Phase II study that Day One re­port­ed over the week­end, show­ing that 64% of 69 evalu­able pa­tients re­spond­ed to the monother­a­py. Pa­tients re­mained on treat­ment for a me­di­an of 8.4 months, with 77% of them still re­ceiv­ing the drug as of the Sept. 28, 2022 da­ta cut­off.

Pa­tients 6 months to 25 years of age took part in the FIRE­FLY-1 study and re­ceived tovo­rafenib once a week. The pa­tients have re­cur­rent or pro­gres­sive pe­di­atric low-grade glioma, which is the most com­mon form of brain tu­mor found in chil­dren, Day One said.

That 64% ORR is the same fig­ure as was re­port­ed in a small­er dataset of 25 pa­tients last sum­mer, which led to a stock spike and an ap­prox­i­mate­ly $150 mil­lion of­fer­ing. The ex-Take­da drug has break­through and rare pe­di­atric dis­ease des­ig­na­tions from the FDA.

Three pa­tients ful­ly re­spond­ed to the treat­ment while 41 ben­e­fit­ed par­tial­ly, 10 of which were un­con­firmed, Day One said this week­end. The Cal­i­for­nia biotech said the drug was “gen­er­al­ly well-tol­er­at­ed,” with change in hair col­or be­ing the most com­mon side ef­fect as seen in 75% of 77 treat­ed pa­tients. Oth­er side ef­fects in­clud­ed an in­crease in cre­a­tine phos­pho­k­i­nase, ane­mia, fa­tigue and mac­u­lopapu­lar rash.

Day One will sub­mit more da­ta to present at a med­ical meet­ing next quar­ter. Up­com­ing da­ta from all pa­tients in the tri­al will be in­clud­ed in the NDA fil­ing, CEO Je­re­my Ben­der said in a state­ment.

“We look for­ward to con­tin­u­ing our dis­cus­sions with reg­u­la­to­ry au­thor­i­ties with the hope of bring­ing this ther­a­py to chil­dren in need of new op­tions as soon as pos­si­ble,” Ben­der said.

The biotech said there are no cur­rent treat­ments ap­proved for pLGG. Day One said “most chil­dren with pLGG sur­vive their can­cer,” but if they don’t go in­to re­mis­sion af­ter surgery, they might have to un­der­go ag­gres­sive ther­a­pies for years.

Day One went pub­lic in 2021 on the nar­ra­tive that many drug de­vel­op­ers de-em­pha­sized pe­di­atric can­cer pa­tients in their clin­i­cal de­vel­op­ment. The biotech is fo­cused on new treat­ments, rather than re­pur­pos­ing adult can­cer drugs with heavy side ef­fects.

Sen. Ron Wyden (D-OR) (Francis Chung/E&E News/Politico via AP Images)

In­fla­tion re­bates in­com­ing: Wyden calls on CMS to move quick­ly as No­var­tis CEO pledges re­ver­sal

Senate Finance Chair Ron Wyden (D-OR) this week sent a letter to the head of the Centers for Medicare & Medicaid Services seeking an update on how and when new inflation-linked rebates will take effect for drugs that see major price spikes.

The newly signed Inflation Reduction Act requires manufacturers to pay a rebate to Medicare when they increase drug prices faster than the rate of inflation.

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Ma­gen­ta halts stem cell work and may sell it­self fol­low­ing pa­tient death, clin­i­cal hold

Magenta Therapeutics said it is halting work on its stem cell transplant drug pipeline and may sell itself, a week after the company reported the death of a patient in an early stage trial of its antibody-drug conjugate.

The Cambridge, MA-based company said it will conduct a “review of strategic alternatives,” and that could include an “acquisition, merger, business combination, or other transaction.”

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How to use ex­ter­nal con­trols: FDA spells out think­ing in new draft guid­ance

The use of real-world evidence to inform the FDA’s decision-making continues apace, with the agency releasing new draft guidance yesterday on how sponsors can compare outcomes of trial participants receiving a test treatment with outcomes in a group of people external to the trial.

The practice of externally controlled trials is common, particularly in oncology or other difficult areas where it’s not ethical or feasible to use internal controls.

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The Big Phar­ma axe: Mer­ck cuts chikun­gun­ya vax, Bris­tol My­ers drops Cy­tomX-part­nered pro­gram, and more

As fourth quarter earnings come in, Big Pharmas are disclosing changes to their pipelines during their investor calls, and sometimes more quietly in presentation appendices.

Merck dropped its chikungunya vaccine candidate, which completed a Phase II study. Merck acquired the vaccine through its purchase of Themis Bioscience in 2020. In developing a vaccine for chikungunya, a mosquito-borne virus, Valneva is the frontrunner, as it submitted its vaccine to the FDA at the end of December.

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Goldfinch Bio CEO Tony Johnson (L) and Karuna Therapeutics CEO Bill Meury

Karuna li­cens­es Goldfinch as­sets to com­pete with Boehringer In­gel­heim in neu­ro­science

Karuna Therapeutics is looking to compete with Boehringer Ingelheim on depression and anxiety with a new license to Goldfinch Bio’s assets, starting with $15 million to the shuttered biotech.

Karuna steps into an arena already being tested by Boehringer in multiple Phase II studies — the two are targeting transient receptor potential canonical 4 and 5, or TRPC4/5, which is thought to have a role in neuroscience indications. Goldfinch’s asset went through a Phase II in kidney diseases, but Karuna’s sights are set on mood and anxiety disorders for now.

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Teresa Graham, incoming Roche Pharmaceuticals CEO

In­com­ing Roche CEO builds out his top team, tap­ping Genen­tech vet to lead phar­ma di­vi­sion

Roche announced another leadership shuffle Thursday morning – the head of global product strategy, Teresa Graham, will take over as CEO of Roche Pharmaceuticals in March while the company’s corporate executive committee will make a spot for Levi Garraway, CMO and executive VP of global product development.

Thomas Schinecker will take over the top spot as Roche group CEO in March, leaving his spot as head of diagnostics.

Roche's headquarters in Basel, Switzerland (Kyle LaHucik for Endpoints News)

Roche ditch­es fi­nal PhI­II for can­cer hope­ful, re­ports set­back for key drug in $1.4B buy­out

Over the past few years, Roche has released news about its AKT inhibitor ipatasertib in drips — most of them negative. The drug yielded mixed data in a key prostate cancer trial, Phase III flops in triple-negative breast cancer forced the pharma giant to pull the plug there, and in mid-2022 Roche trimmed two more early-stage indications in prostate cancer after completing the trials.

Now, the last piece of the program is gone.

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Trodelvy notch­es a win in most com­mon form of breast can­cer

Following a promise last year to go “big and fast in breast cancer,” Gilead has secured a win for Trodelvy in the most common form.

The drug was approved to treat HR-positive, HER2-negative breast cancer patients who’ve already received endocrine-based therapy and at least two other systemic therapies for metastatic cancer, Gilead announced on Friday.

Trodelvy won its first indication in metastatic triple-negative breast cancer back in 2020, and has since added urothelial cancer to the list. HR-positive HER2-negative breast cancer accounts for roughly 70% of new breast cancer cases worldwide per year, according to senior VP of oncology clinical development Bill Grossman, and many patients develop resistance to endocrine-based therapies or worsen on chemotherapy.

Sanofi scraps PhI­II tri­al for Prin­cip­ia drug af­ter re­view­ing com­pe­ti­tion

Months after the FDA placed Phase III trials of Sanofi’s BTK inhibitor on hold, the company is winding down one of the studies.

Sanofi reported in its Q4 earnings that the URSA study “was discontinued after careful evaluation of the emerging competitive treatment landscape in” myasthenia gravis, a rare disease that causes muscle weakness.

The Phase III, placebo-controlled trial was testing tolebrutinib in patients with the moderate-to-severe form of the disease. It started in late 2021, according to records on clinicaltrials.gov, and was originally designed to recruit 154 participants who were receiving the standard of care.

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