Recursion's leadership team has decided that conducting a dose optimization study in a sheep efficacy model of Tay-Sachs disease is prudent and in the best interest of patients before enrolling infants in a Phase 2 trial of REC-3599 in the ultra-rare indication infantile GM2 gangliosidosis. This decision will delay the GM2 Phase 2 trial start by approximately 2 years.
Recursion's Phase 2 trial of REC-994 in Cerebral Cavernous Malformation is on track and we expect to enroll the first patient in the coming weeks.
The Phase 2 portion of Recursion's adaptive Phase 2/3 trial of REC-2282 in Neurofibromatosis Type 2 is on track to enroll in Q2.
Recursion's Phase 2 trial of REC-4881 in Familial Adenomatous Polyposis is expected to enroll the first patient in either Q2 or potentially Q3, 2022 due to challenges with site opening presented by the Covid-19 Omicron variant.
Preparations for Recursion's Phase 1 study of REC-3964 in recurrent C. difficile colitis are on track, and we expect to begin enrollment in the second half of 2022.
Partial response (PR) observed in ATRC-101 monotherapy cohort as well as a confirmed complete response (CR) observed in pembrolizumab combination cohort in Phase 1b clinical trial
Clinical activity observed in multiple tumor types and significantly associated with ATRC-101 target expression; target diagnostic validated and planning for participant selection based on target expression
ATRC-101 has been well-tolerated with no dose-limiting toxicities observed
Conference call and webcast with accompanying slides scheduled for today at 4:30 p.m. EST
Conference Call and Webcast Details
Atreca will host a live conference call and webcast today at 4:30 p.m. EST. To access the conference call by telephone, please dial (800) 373-6606 (Domestic) or 409-937-8918 (International). The conference ID number is 5089907.
The live audio webcast and accompanying slide presentation can be accessed via the Events section of the Company’s investor relations website at https://ir.atreca.com/news-and-events/event-calendar. An archived replay of the webcast will be available on the Company's website for 90 days following the live event.
Eisai Co., Ltd. (Headquarters:Tokyo, CEO:Haruo Naito, "Eisai") and Biogen Inc. (Nasdaq: BIIB, Corporate headquarters:Cambridge, Massachusetts, CEO:Michel Vounatsos, "Biogen") announced today that Eisai has initiated a submission to the Pharmaceuticals and Medical Devices Agency (PMDA) of application data under the prior assessment consultation system inJapanfor the investigational anti-amyloid beta (Aβ) protofibril antibody lecanemab (BAN2401). The lecanemab Clarity AD Phase 3 clinical study for mild cognitive impairment (MCI) due to Alzheimer's disease (AD) and mild AD (collectively known as early AD) is ongoing.
The PMDA's process, known as "prior assessment consultation," is conducted at the development stage before the new drug application submission, which is based on available quality, non-clinical and clinical data. By identifying and resolving any potential issues prior to submission, the aim is to shorten application review time.
Based on discussions with the Ministry of Health, Labour and Welfare (MHLW) and PMDA, Eisai applied to PMDA for permission to utilize the "prior assessment consultation" process for lecanemab with the aim of shortening the review period. The agency approved Eisai's request and Eisai has submitted the non-clinical lecanemab data to PMDA. The additional data of the application package will be submitted hereafter. Eisai plans to obtain the primary endpoint data from the Clarity AD study in the fall of 2022, and based on the results of the study, aims to file for the manufacturing and marketing approval in Japan during Eisai's fiscal year 2022.
In September 2021, Eisai initiated a rolling submission to the U.S. Food and Drug Administration (FDA) of a Biologics License Application (BLA) for lecanemab, an investigational agent under the Accelerated Approval pathway for the treatment of early AD with confirmed amyloid pathology, and expects to complete this rolling submission in the beginning of Eisai's fiscal year 2022. Based on the results of the Clarity AD study as the confirmatory study, Eisai plans to submit for full approval of lecanemab to the U.S. FDA during fiscal year 2022. Eisai and Biogen are committed to providing innovative treatments to persons living with early AD, their families and healthcare professionals who are waiting for new treatment options, as early as possible.
Ocugen Inc said on Friday U.S. regulators have declined to issue an emergency use authorization (EUA) for Covaxin, the COVID-19 vaccine developed by its Indian partner Bharat Biotech, for use in individuals aged 2 to 18 years.
Shares of Ocugen slumped 30% premarket on the news.
Ocugen said it intends to continue working with the U.S. Food and Drug Administration to evaluate the process for getting an EUA for pediatric use of Covaxin.
Ocugen had entered into a deal with vaccine maker Bharat Biotech in late 2020, under which it would develop, supply, and commercialize Covaxin for the U.S. market. (https://reut.rs/35nVA1P)
Covaxin, which is not cleared for any age group in the United States, is one of the two most widely used COVID vaccines in India and also has an emergency use listing from the World Health Organization
When Russia began to stack troops along Ukraine's border—up to 190,000 of them—Karuna Therapeutics' partners began to prepare clinical trial participants for the company's schizophrenia treatment in psychiatric hospitals across the country.
That, combined with additional contingency plans, is why CEO Steven Paul, M.D., is confident there won't be much, if any, delay to the initial phase 3 readout for KarXT, despite earlier concerns that the timing would be pushed out.
Karuna’s partners in Ukraine were enrolling “quite recently,” but the trial was paused when the invasion began, according to Paul. Ultimately, a “substantial number of patients” were enrolled prior to the invasion, and data was collected on many of them prior to the start of the conflict.
“That data is all collected, it’s all protected, we have it and we will use it in the trial,” he said.
Paul’s efforts to assuage concerns come after the company disclosed last week in a Securities and Exchange Commission filing that the projected timeline for the data was being withdrawn. The disclosure also noted that the company were unsure of when data would in fact be available. Karuna hadoriginally reported during fourth-quarter earnings that the ETA for top-line data from the EMERGENT-3 phase 3 trial was the second half of this year.
Even though 10 of the company's 19 clinical trial sites for the late-stage study are in Ukraine, Paul said the disclosure was "a tempest in the teapot."
“I think we spooked people,” he said. “There are many, many companies, both drug companies and other companies, that are doing business in Ukraine that will be disrupted to a much, much, greater extent than we will because we were far along.”
Karuna CEO Steven Paul (Karuna Therapeutics)
Contributing to Paul’s bullish projection is that the company’s concurrent phase 3 trial, EMERGENT-2, is recruiting only in the U.S. Karuna has estimated that data from that trial will be available in the middle of this year. Paul also said Karuna could shift recruiting for EMERGENT-3 to be mainly in the U.S.
“We’ve always had the possibility of just using those sites to enroll the remainder of the patients,” he said.
As for the company’s EMERGENT-4 trial, which is looking at the long-term effects of KarXT among patients enrolled in either EMERGENT-2 or EMERGENT-3, Paul is likewise confident the war won’t hinder data, given the company’s ability to rely on U.S. recruitment. Right now, five out of 32 sites listed for that trial are in Ukraine.
The impact of the war on clinical trials has been felt across the pharmaceutical and biotech industry, one of the many economic implications occurring in the backdrop of a conflict that’s now cost thousands of lives.
Ascendis Pharma A/S reported Wednesday that the war could impact the readout for a phase 3 trial of an adult growth hormone deficiency treatment. Tricidia on Thursday delayed a readout for a late-stage study in chronic kidney disease from the third quarter to the fourth quarter. The company said that 15% of enrolled randomized patients were based in Ukraine.
Russia’s decision to invade has brought condemnation from across the biotech industry. Business leaders issued a letter last week calling for “complete economic disengagement” with the country and called the war a “criminal act deliberately committed by Russia.”
The tumult comes as Karuna is trying to keep its head above water in a market that's pummeled biotechs in the last year. Karuna's stock has jumped more than $10 per share since disclosing the potential delay, from $99.19 on Feb. 24 to $109.25 on March 1. Paul said that even amid the choppy financial waters facing the industry, the company has fared well.
"Now, of course, like many biotech companies we've settled back, but we're still doing quite well, relatively speaking, [in] this biotech market which as you know has been a tough one of late," he said.
The company’s hopes are primarily pinned to the schizophrenia treatment, KarXT, which was shown to be both tolerable and, on average, improved patients' symptoms of the mental disorder in a phase 2 trial. The treatment acts as a control panel for muscarinic acetylcholine receptors in the brain, turning them on in the central nervous system as treatment and turning them off in peripheral tissues to avoid side effects.
Paul said he discovered the original compounds of the drug by accident while working at Eli Lilly looking into treatments for dementia-related psychosis. He retired from the company in 2010 after a 17-year stint, leaving as executive vice president of science and technology and as president of Lilly Research Laboratories.
The ongoing phase 3 trials for Karuna were initiated after the FDA advised the company in a end-of-phase 2 meeting in 2020 that one more trial was needed to demonstrate efficacy. Although Paul set out to clarify the timeline for top-line data, he wouldn’t divulge when the company is likely to apply for a new drug application with federal regulators, saying the company will apply when ready.
