Crinetics Pharmaceuticals to Participate in the SVB Securities Global Biopharma Conference

On February 10, 2023 Crinetics Pharmaceuticals, Inc. (Nasdaq: CRNX), reported that company management will participate in a fireside chat at the annual SVB Securities Global Biopharma Conference, which is being held in a virtual format February 14-16, 2023 (Press release, Crinetics Pharmaceuticals, FEB 10, 2023, View Source [SID1234627058]).

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Details of the presentation are as follows:

Date: Tuesday, February 14, 2023
Time: 3:40 p.m. Eastern Time
The live and archived webcast will be accessible on the Events & Presentations page in the Investors section on the Crinetics’ website at www.crinetics.com/events.

Cerus Corporation to Participate in Upcoming Investor Conferences

On February 10, 2023 Cerus Corporation (Nasdaq: CERS) reported that the Company will participate in two upcoming investor conferences (Press release, Cerus, FEB 10, 2023, View Source [SID1234627057]).

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William ‘Obi’ Greenman, Cerus’ president and chief executive officer, is scheduled to be in attendance at BTIG’s MedTech, Digital Health, Life Science & Diagnostic Tools Conference on Tuesday, February 14th and Wednesday, February 15th.
William ‘Obi’ Greenman and Kevin Green, Cerus’ vice president and chief financial officer, are scheduled to participate in a fireside chat at Cowen’s 43rd Annual Health Care Conference on Wednesday, March 8th at 1:30 PM EST. A live webcast of the fireside chat will be available at https://wsw.com/webcast/cowen132/cers/2009322. A replay will be available after the event.

Abecma (idecabtagene vicleucel) Reduced the Risk of Disease Progression or Death by 51% Versus Standard Regimens in Earlier Lines of Therapy for Relapsed and Refractory Multiple Myeloma Based on Results from Phase 3 KarMMa-3 Study

On February 10, 2023 Bristol Myers Squibb (NYSE: BMY) and 2seventy bio, Inc. (Nasdaq: TSVT) reported the first publication and presentation of positive results from KarMMa-3, a pivotal Phase 3, open-label, global, randomized, controlled study evaluating Abecma (idecabtagene vicleucel) compared with standard combination regimens in adults with relapsed and refractory multiple myeloma after two to four prior lines of therapy, including an immunomodulatory agent, a proteasome inhibitor, and an anti-CD38 monoclonal antibody, and who were refractory to their last regimen (Press release, Bristol-Myers Squibb, FEB 10, 2023, View Source [SID1234627055]). Data from KarMMa-3 are being published in The New England Journal of Medicine and simultaneously presented at the EBMT and the European Hematology Association (EHA) (Free EHA Whitepaper)’s (EHA) (Free EHA Whitepaper) 5th European CAR T-cell Meeting on Friday, February 10 in an oral presentation during the Best Abstract Session.

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At a median follow up of 18.6 months, treatment with Abecma (n=254)demonstrated a clinically meaningful and statistically significant improvement in the primary endpoint of progression-free survival (PFS) compared with standard regimens (n=132), with a median PFS of 13.3 months (95% CI: 11.8-16.1) vs. 4.4 months (95% CI: 3.4-5.9), respectively (HR:0.49; p<0.0001). This represents a 51% reduction in risk of disease progression or death with Abecma. Based on results from KarMMa-3, Abecma is the first and only chimeric antigen receptor (CAR) T cell therapy to demonstrate superiority over standard regimens in triple-class exposed relapsed and refractory multiple myeloma in a randomized, controlled Phase 3 trial.

"In earlier lines of treatment for multiple myeloma, regimens consisting of immunomodulatory agents, proteasome inhibitors, and anti-CD38 monoclonal antibodies are often used to help manage the disease. This shift in the treatment paradigm leaves many patients who are triple-class exposed with relapsed and refractory disease and in need of new treatment options sooner," said Paula Rodriguez-Otero, M.D., Ph.D., Department of Hematology, Clinica Universidad de Navarra, Pamplona, Spain. "Results from the KarMMa-3 study with Abecma clearly demonstrate the benefit of earlier use of a CAR T cell therapy in providing the longest progression-free survival for patients with relapsed and refractory multiple myeloma compared to current standard regimens for these patients."

"With Abecma, our first-in-class anti-BCMA CAR T cell therapy, we sought to deliver a personalized therapy that provides durable outcomes with a single infusion to advance the multiple myeloma treatment paradigm for patients," said Anne Kerber, senior vice president, Cell Therapy Development, Bristol Myers Squibb. "This represents the third New England Journal of Medicine publication for Abecma, showing the clear clinical benefit of using Abecma across lines of therapy for patients with triple-class exposed relapsed and refractory multiple myeloma to provide the best chance for lasting disease control."

Results for the key secondary endpoint of overall response rate also met statistical significance with the majority of patients (71%) treated with Abecma achieving a response, and 39% achieving a complete response or stringent complete response. In comparison, less than half of patients (41%) who received standard regimens achieved a response, with 5% experiencing a complete response or stringent complete response (p<0.0001). Responses with Abecma were durable with a median duration of 14.8 months (95% CI: 12.0-18.6) compared with 9.7 months (95% CI: 5.4-16.3) for standard regimens. Clinical benefit with Abecma was consistently observed across difficult-to-treat subgroups.

"For relapsing triple-class exposed multiple myeloma patients, median progression-free survival is just 4.6 months and there is no established standard treatment approach that provides durable responses," said Sergio Giralt, M.D., Division of Hematologic Malignancies, Memorial Sloan Kettering Cancer Center. "In this study, we are seeing efficacy among a population with historically difficult-to-treat disease, with a significant improvement in progression-free survival and deep and lasting responses. These results from KarMMa-3 introduce the potential for this anti-BCMA CAR T cell therapy to become a standard of care earlier in the treatment course for relapsed and refractory multiple myeloma."

Abecma exhibited a consistent and generally predictable safety profile, including no new safety signals, with mostly low-grade occurrences of cytokine release syndrome (CRS) and neurotoxicity. In patients treated with Abecma, 88% experienced any grade CRS, with Grade 3/4 events occurring in 4% of patients. Two patients (1%) experienced a Grade 5 CRS event. Median time to onset of CRS was 1 day (range: 1-14) and median duration of CRS was 3.5 days (range: 1-51). Any grade neurotoxicity occurred in 15% of patients, with Grade 3/4 neurotoxicity occurring in 3% of patients, and no Grade 5 events reported. Median time to onset of neurotoxicity was 3 days (range: 1-317) and median duration of neurotoxicity was 2 days (range: 1-37).

"The KarMMa-3 study is the first with a BCMA-directed CAR T therapy to demonstrate superiority versus standard regimens for patients with relapsed and refractory multiple myeloma, illustrating the potential of Abecma to change the standard of care of triple-class exposed multiple myeloma in early lines," said Steve Bernstein, M.D., chief medical officer, 2seventy bio. "We are pleased to present and have these data published to build on the compelling efficacy profile of Abecma demonstrating significant improvement in progression-free survival and we look forward to working with regulatory authorities to make Abecma available to more myeloma patients who could benefit from this important treatment option."

Bristol Myers Squibb and 2seventy bio intend to include these data in a planned supplemental Biologics License Application submission to the U.S. Food and Drug Administration (FDA) in 2023. Abecma is the first-in-class B-cell maturation antigen (BCMA)-directed CAR T cell immunotherapy approved by the FDA for the treatment of adult patients with relapsed or refractory multiple myeloma after four or more prior lines of therapy, including an immunomodulatory agent, a proteasome inhibitor, and an anti-CD38 monoclonal antibody. Please see the Important Safety Information section below, including Boxed WARNINGS for Abecma regarding CRS, neurologic toxicities, Hemophagocytic Lymphohistiocytosis/Macrophage Activation Syndrome and Prolonged Cytopenia. Abecma is also approved in the European Union, Switzerland, Japan, Canada, the United Kingdom and Israel for adult patients with triple-class exposed relapsed or refractory multiple myeloma after three to four or more prior lines of therapy.

Memorial Sloan Kettering Cancer Center disclosures: Dr. Giralt and Memorial Sloan Kettering Cancer Center have financial interests associated with the research described in this release.

About KarMMa-3

KarMMa-3 (NCT03651128) is a pivotal, Phase 3, open-label, global, randomized, controlled trial evaluating Abecma compared to standard regimens in patients with relapsed and refractory multiple myeloma who have received two to four prior lines of treatment, including an immunomodulatory agent, a proteasome inhibitor, and an anti-CD38 monoclonal antibody, and were refractory to the last treatment regimen. Patients were randomized to receive Abecma or standard regimens that consisted of combinations that included daratumumab, pomalidomide, and dexamethasone (DPd), daratumumab, bortezomib, and dexamethasone (DVd), ixazomib, lenalidomide, and dexamethasone (IRd), carfilzomib and dexamethasone (Kd) or elotuzumab, pomalidomide and dexamethasone (EPd) chosen based on their most recent treatment regimen and investigator discretion. The primary endpoint evaluated in this study is progression-free survival, defined as time from randomization to the first documentation of progressive disease or death due to any cause, whichever occurs first. Key secondary endpoints include overall response rate and overall survival.

Blueprint Medicines Announces Partial Clinical Hold for Phase 1/2 VELA Trial of BLU-222

On February 10, 2023 Blueprint Medicines Corporation (NASDAQ: BPMC) reported that the U.S. Food and Drug Administration (FDA) verbally informed the company on February 8, 2023 that it has placed a partial clinical hold on the Phase 1/2 VELA trial of BLU-222 due to visual adverse events (AEs) observed in a limited number of patients (Press release, Blueprint Medicines, FEB 10, 2023, View Source [SID1234627054]). Patients currently enrolled in the trial are continuing on study drug at this time, and additional patients will not be enrolled until the partial clinical hold is resolved.

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BLU-222 is currently being evaluated in the Phase 1 dose escalation portion of the VELA trial. Patients have been treated with BLU-222 at doses ranging from 50 mg BID to 800 mg BID to date, with evidence of clinical benefit observed and no discontinuations due to AEs.

The reported visual AEs consisted of transient, reversible episodes of light sensitivity and blurred vision. All events were Grade 1, except one Grade 3 event involving light sensitivity and blurred vision in a patient treated at 600 mg BID. All events resolved with dose interruption or reduction. No treatment-emergent abnormal findings, including uveitis, have been observed in patients who have received detailed ophthalmologic examinations.

"Patient safety is our first priority, and we are working closely with the FDA to investigate the reported visual adverse events as well as amend the VELA trial protocol to provide specific guidance to investigators on how to monitor for and manage these events should they occur," said Becker Hewes, M.D., Chief Medical Officer at Blueprint Medicines. "We have confidence in the benefit-risk profile of BLU-222 based on the activity and safety data we have seen to date in the dose escalation study. In addition, we recognize the urgency to treat patients with CDK2-vulnerable cancers, many of whom have seen their disease progress after exhausting all other options, and we aim to resume enrollment as expeditiously and responsibly as possible."

Consistent with prior guidance, Blueprint Medicines plans to present initial dose escalation data from the VELA trial of BLU-222 in the first half of 2023.

Ayala Pharmaceuticals Reports Financial Results For the Fiscal Year Ended October 31, 2022

On February 10, 2023 Ayala Pharmaceuticals, Inc. (f/k/a Advaxis Inc.) (the "Company," "New Ayala," "we," "us" or "our") (OTCQX: ADXS), a clinical-stage oncology company, reported financial results for the fiscal year ended October 31, 2022 (Press release, Ayala Pharmaceuticals, FEB 10, 2023, View Source [SID1234627053]).

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Management Commentary

"We were pleased to close our previously announced merger in January 2023," said Kenneth A. Berlin, President and Chief Executive Officer of the Company. "Our immediate priorities include executing on Part B of the ongoing registration-enabling RINGSIDE study evaluating AL102 in desmoid tumors. We believe that AL102 has best in class potential and, if approved, may have important clinical advantages including safety, convenient once daily dosing and lower pill burden. For AL101, we expect to gain clarity this year on the development path in recurrent/metastatic adenoid cystic carcinoma (R/M ACC). We also continue to make progress on the development of ADXS-504 for the treatment of early prostate cancer. The dose escalation part of the investigator-sponsored trial at Columbia University has been completed and we are now enrolling patients in an expansion cohort."

Fiscal Year 2022 and Recent Business Highlights:

In October 2022, the Company, which was then known as Advaxis, Inc., entered into a definitive agreement to merge with the entity now known as Old Ayala, Inc. (which prior to the merger was named Ayala Pharmaceuticals, Inc.) The merged company is focused primarily on the development and commercialization AL102 for the treatment of desmoid tumors. The merger closed on January 19, 2023, at which time the Company changed its name to Ayala Pharmaceuticals, Inc.
Announced positive interim data from Part A of the Phase 2/3 RINGSIDE study of AL102 in desmoid tumors: Data showed that AL102 had efficacy across all cohorts, with early responses that deepened over time. AL102 was well tolerated at all three dosing regimens with no dose-limiting toxicities and no Grade 4/5 adverse events. The data were featured in an oral presentation at ESMO (Free ESMO Whitepaper) 2022 and in a poster at the CTOS 2022 annual meeting.
Initiated Part B (Phase 3 segment) of the RINGSIDE study in desmoid tumors: Part B of RINGSIDE is a double-blind placebo-controlled study enrolling up to 156 patients with progressive desmoid disease, randomized between AL102 or placebo. The primary endpoint is progression free survival with secondary endpoints including objective response rates, duration of response, and patient-reported quality of life measures.
Fast Track designation granted for AL102: The U.S. FDA granted Fast Track designation for AL102 for the treatment of progressing desmoid tumors. The designation holds important advantages that may expedite the development and regulatory review of AL102.
Presented data on AL101 in adenoid cystic carcinoma (ACC) at 2022 ASCO (Free ASCO Whitepaper) annual meeting: In a poster at ASCO (Free ASCO Whitepaper), an update was provided on the ACCURACY study, the only prospective study conducted to date in ACC patients carrying Notch-activating mutations. An overall disease control rate of 66.7% was observed. The median PFS in each of the 4mg and 6mg dose AL101 cohorts was 3.7 months and 6.7 months, respectively, among the patients who had a partial response.
Continued progress with Phase 1 clinical trial of ADXS-504 for early prostate cancer: Dose escalation has been completed and enrollment at second dose level is being expanded at Columbia University. Four out of six patients treated are still on study and PSA values are being followed up. ADXS-504 has been well tolerated with no serious adverse events reported.
New Ayala’s Consolidated Financial Results for the Fiscal Year Ended October 31, 2022

Cash position On October 31, 2022, the consolidated cash and cash equivalents position was $25.2 million.

Revenues: Revenues for the fiscal year 2022 were $250,000, compared with $3.2 million for the fiscal year 2021.

R&D expenses: Research and development expenses for fiscal year 2022 were $7.6 million, compared with $10.6 million for fiscal year 2021.

G&A expenses: General and administrative expenses for fiscal year 2021 were $8.9 million, compared to $11.5 million for fiscal year 2021.

Net loss: The net loss for the fiscal year ended October 31, 2022 was approximately $14.4 million or ($8.46) per share based on approximately 1.8 million weighted average shares outstanding. This compares with a net loss for fiscal year 2021 of approximately $17.9 million or ($11.07) per share based on approximately 1.6 million weighted average shares outstanding.

The above financial results, as well as the financial tables included in this press release, reflect the financial performance of the Company, then known as Advaxis Inc., prior to completion of the merger and do not include any contribution from Old Ayala.

For further details on the Company’s financial results, refer to our Annual report on Form 10-K for the twelve months ended October 31, 2022, filed with the Securities and Exchange Commission. Old Ayala will report consolidated financial results for the full year ended December 31, 2022 (a period completed prior to the merger) on or about March 31, 2023.