On November 3, 2022 CymaBay Therapeutics, Inc. (NASDAQ: CBAY), a clinical-stage biopharmaceutical company focused on developing therapies for liver and other chronic diseases with high unmet need, reported that management will participate in the Credit Suisse 31st Healthcare Conference taking place November 7-10, 2022 in Rancho Palos Verdes, CA and the 34th Annual Piper Sandler Healthcare Conference taking place November 29 to December 1, 2022 in New York, NY (Press release, CymaBay Therapeutics, NOV 3, 2022, https://ir.cymabay.com/news/detail/545/cymabay-therapeutics-to-present-at-upcoming-investment-conferences [SID1234622860]).

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Credit Suisse 31st Healthcare Conference

34th Annual Piper Sandler Healthcare Conference

On November 3, 2022 C4 Therapeutics, Inc. (C4T) (Nasdaq: CCCC), a clinical-stage biopharmaceutical company dedicated to advancing targeted protein degradation science to develop a new generation of small-molecule medicines and transform how disease is treated, reported financial results for the third quarter ended September 30, 2022, as well as recent business highlights (Press release, C4 Therapeutics, NOV 3, 2022, View Source [SID1234622859]).

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"In the third quarter we remained focused on clinical execution as we continued to enroll patients in the dose escalation portions of the Phase 1/2 clinical trials of CFT7455 and CFT8634. Additionally, we received FDA clearance of the investigational new drug application for our Phase 1/2 clinical trial of CFT1946, which is expected to initiate by year-end, marking our third program cleared to enter the clinic," said Andrew Hirsch, president and chief executive officer of C4 Therapeutics. "Our recent progress demonstrates the power of our TORPEDO platform to create MonoDAC and BiDAC degrader medicines that have the potential to transform patients’ lives. We are well-financed to advance our clinical pipeline through key inflection points and leverage our discovery platform to progress our next wave of research programs focused on novel and historically undruggable targets."

THIRD QUARTER 2022 AND RECENT HIGHLIGHTS

CFT7455: CFT7455 is a novel degrader of IKZF1/3 for the treatment of multiple myeloma (MM) and non-Hodgkin’s lymphomas (NHL), including peripheral T-cell lymphoma and mantle cell lymphoma.

Advanced Enrollment in Phase 1/2 Clinical Trial: C4T progressed the Phase 1/2 clinical trial of CFT7455. Cohort B1 is exploring CFT7455 as a monotherapy for relapsed or refractory MM, and Cohort C is exploring CFT7455 as a monotherapy for NHL.
CFT8634: CFT8634 is a degrader of BRD9 for the treatment of synovial sarcoma and SMARCB1-null solid tumors.

Advanced Enrollment in Phase 1/2 Clinical Trial: C4T progressed the Phase 1/2 clinical trial of CFT8634. Cohort A is exploring CFT8634 as a monotherapy for synovial sarcoma and SMARCB1-null solid tumors.

Accepted to Present at the 2022 Connective Tissue Oncology Society (CTOS) Annual Meeting: Brian A. Van Tine, MD, PhD, Professor of Medicine, Division of Oncology, Section of Medical Oncology, the Washington University School of Medicine, Siteman Cancer Center, will present a trial-in-progress poster titled "A Phase 1/2 Study of CFT8634, A Novel Bifunctional Degradation Activating Compound (BiDAC) Degrader of BRD9, in Synovial Sarcoma and SMARCB1-Null Tumors" at the 2022 CTOS Annual Meeting on November 17, 2022 at 5 pm PT.
CFT1946: CFT1946 is a degrader targeting BRAF-V600 mutations for the treatment of solid tumors including non-small cell lung cancer (NSCLC), colorectal cancer and melanoma.

Investigational New Drug (IND) Application Clearance Achieved: In September 2022, C4T received a Study May Proceed Letter from the United States Food and Drug Administration (FDA) to begin a Phase 1/2 trial of CFT1946 for the treatment of BRAF-V600 mutant solid tumors.
Research Activities

Presented at Discovery on Target and the 5th Annual Targeted Protein Degradation Summit. In October 2022, C4T delivered presentations that highlighted how C4T’s platform has been built to enable rational and efficient discovery of MonoDAC degraders through the design and evolution of a diverse chemical library combined with various screening approaches.
UPCOMING 2022 MILESTONES

CFT1946: Initiate a Phase 1/2 clinical trial of CFT1946 for the treatment of BRAF V600-mutant solid tumors by year-end 2022.
CFT8919: Complete IND-enabling activities for CFT8919, a potent and selective degrader of EGFR L858R, for the treatment of non-small cell lung cancer, by year-end 2022.
UPCOMING 2022 EVENTS

November 8, 2022: Management will participate in a fireside chat at the Credit Suisse 31st Annual Healthcare Conference.
November 17, 2022: An investigator will present C4T’s trial-in-progress poster for CFT8634 at the CTOS Annual Meeting.
December 1, 2022: Management will participate in a fireside chat at the Evercore ISI HealthCONx Conference.
THIRD QUARTER 2022 FINANCIAL RESULTS

Revenue: Total revenue for the third quarter of 2022 was $6.8 million, compared to $8.5 million for the third quarter of 2021. Total revenue reflects revenue recognized under collaboration agreements with Roche, Biogen, and Calico.

Research and Development (R&D) Expense: R&D expense for the third quarter of 2022 was $29.7 million, compared to $24.3 million for the third quarter of 2021. The increase in R&D expense was primarily attributable to increased personnel expenses, and clinical expenses from the ongoing CFT7455 and CFT8634 Phase 1/2 clinical trials.

General and Administrative (G&A) Expense: G&A expense for the third quarter of 2022 was $9.6 million, compared to $8.5 million for the third quarter of 2021. The increase in G&A expense was primarily attributable to increased personnel expenses.

Net Loss and Net Loss per Share: Net loss for the third quarter of 2022 was $32.0 million, compared to $24.7 million for the third quarter of 2021. Net loss per share for the third quarter of 2022 was $0.65, compared to $0.51 for the third quarter of 2021.

Cash Position and Financial Guidance: Cash, cash equivalents and marketable securities as of September 30, 2022, were $366.0 million, compared to $451.5 million as of December 31, 2021. The decrease in cash was primarily driven by expenditures to fund operations. C4T expects that its cash, cash equivalents and marketable securities as of September 30, 2022, together with future payments expected to be received under existing collaboration agreements, will be sufficient to fund planned operating expenses and capital expenditures to the end of 2024.

On November 3, 2022 Allogene Therapeutics, Inc. (Nasdaq: ALLO), a clinical-stage biotechnology company pioneering the development of allogeneic CAR T (AlloCAR T) products for cancer, reported that it will present updated data from its Phase 1 UNIVERSAL trial of ALLO-715, an anti-BCMA AlloCAR T product candidate for relapsed/refractory multiple myeloma, in a poster presentation at the 64th Annual Meeting of the American Society of Hematology (ASH) (Free ASH Whitepaper) taking place December 10-13, 2022 in New Orleans, LA (Press release, Allogene, NOV 3, 2022, View Source [SID1234622857]).

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"UNIVERSAL is the first trial to demonstrate significant responses can be achieved with an allogeneic CAR T product candidate. We look forward to presenting updated clinical data with ALLO-715 as we aim to alleviate the barriers patients with multiple myeloma often face when seeking to access cell therapy," said Rafael Amado, M.D., Executive Vice President of Research & Development of Allogene.

The ASH (Free ASH Whitepaper) abstract includes longer-term follow up from UNIVERSAL, including the updated safety experience and efficacy results from two expansion cohorts using lymphodepletion of FC (fludarabine, cyclophosphamide) and ALLO-647 at 39mg or 60mg and a single dose of ALLO-715 at 320M CAR positive cells.

The Company will host an in-person and virtual Research & Development Showcase on Tuesday, November 29, 2022 in New York City. This event will include a discussion of updated data from the UNIVERSAL trial with single dose ALLO-715 and next steps for the program.

The ASH (Free ASH Whitepaper) abstracts are now available at www.hematology.org. Details on the ASH (Free ASH Whitepaper) presentation are as follows:

Allogene Poster Presentation

Session: 704. Cellular Immunotherapies: Early Phase and Investigational Therapies: Poster I
Abstract #1972
Title: Universal Updated Phase 1 Data Highlights Role of Allogeneic Anti-BCMA ALLO-715 Therapy for Relapsed/Refractory Multiple Myeloma
Presenter: Sham Mailankody, MBBS, M.D.
Session Date & Time: Saturday, December 10, 2022; 5:30 PM – 7:30 PM Central Time

On November 3, 2022 Abeona Therapeutics Inc. (Nasdaq: ABEO) reported that it has entered into a securities purchase agreement to sell 7,065,946 shares of its common stock, and, in lieu of shares of common stock, pre-funded warrants exercisable for 543,933 shares of common stock, and accompanying warrants to purchase 7,609,879 shares of its common stock to a group of new and existing institutional investors in a private placement (Press release, Abeona Therapeutics, NOV 3, 2022, View Source [SID1234622855]). The offering price for each share of common stock and accompanying warrant was $4.60, and the offering price for each pre-funded warrant and accompanying warrant was $4.59, which equals the offering price per share of the common stock and accompanying warrant, less the $0.01 per share exercise price of each pre-funded warrant. Each accompanying warrant will represent the right to purchase one share of the Company’s common stock at an exercise price of $4.75 per share of common stock. The pre-funded warrants and the accompanying warrants will be exercisable immediately, and will expire five years from the date of issuance. Gross proceeds of the private placement are expected to be approximately $35,000,000, before deducting placement agent fees and other expenses. The private placement is expected to close on November 7, 2022, subject to the satisfaction of customary closing conditions.

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The private placement included participation from new and existing institutional investors including Adage Capital Management LP, Armistice Capital, Deerfield Management Company, L.P., EcoR1 Capital and Nantahala Capital as well as other specialist biotech investors.

Abeona intends to use the net proceeds from the proposed private placement for development, working capital and general corporate purposes. Abeona estimates that the net proceeds from the proposed private placement plus its existing financial resources are sufficient to fund operations into the third quarter of 2024.

Cantor Fitzgerald & Co. acted as sole placement agent on the transaction.

The securities being issued and sold in the private placement, including the shares of common stock underlying the warrants, have not been registered under the Securities Act of 1933, as amended (the "Securities Act"), or any states’ securities laws and may not be offered or sold in the United States, except pursuant to an effective registration statement or an applicable exemption from the registration requirements of the Securities Act. Abeona has agreed to file a registration statement with the Securities and Exchange Commission (the "SEC") registering the resale of the shares of common stock and the shares of common stock issuable upon exercise of the warrants issued in this private placement. Any offering of securities under a resale registration statement will only be made by means of a prospectus.

This press release shall not constitute an offer to sell or the solicitation of an offer to buy, nor shall there be any sale of securities of Abeona in any state or other jurisdiction in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of any such state or jurisdiction.

On November 2, 2022 Pacylex, a clinical-stage NMT inhibitor company developing first-in-class therapies for leukemia, lymphoma, and solid tumor cancers, reported that the U.S. Food and Drug Administration (FDA) has cleared the Investigational New Drug (IND) application for PCLX-001 for the treatment of acute myeloid leukemia (AML) (Press release, Pacylex Pharmaceuticals, NOV 2, 2022, View Source [SID1234645059]). The open IND enables a Phase 1/2 clinical study to start in the coming months at The University of Texas MD Anderson Cancer Center, which was recently awarded a US Department of Defense (DOD) grant for this purpose.

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Last month the U.S. FDA also granted PCLX-001 Orphan Drug Designation for "treatment of patients with acute myeloid leukemia." PCLX-001 is a first-in-class N-myristoylation inhibitor in clinical development by Pacylex. PCLX-001 is currently being studied in non-Hodgkin lymphoma (NHL) and solid tumor cancer patients at 4 sites in Canada. Pacylex has the first and only NMT inhibitor in clinical studies.

"With this IND clearance, Pacylex opens up a second clinical indication for the investigation of PCLX-001 in patients," said Michael Weickert, PhD, CEO of Pacylex. In the ongoing studies of PCLX-001, seventeen patients have been accrued through 5 dose levels of oral, once-per-day PCLX-001 with no dose limiting toxicities observed.

"Non clinical data suggests that AML may be the cancer type most sensitive to NMT inhibitors, so we are excited to move PCLX-001 into clinical studies in AML patients in the near future," said John Mackey, MD, CMO of Pacylex. Luc Berthiaume, PhD, CSO of Pacylex commented, "It is gratifying to see the pre-clinical work pioneered in my lab, translating to clinical investigations and the potential to help patients in the near future." Data on the scientific rationale for PCLX-001 in AML patients, will be presented at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition from 10-13 December 2022.

The ongoing clinical PCLX-001 trial in NHL and solid tumor patients is registered at ClinicalTrials.gov Identifier: NCT04836195.

PCLX-001

PCLX-001 (aka DDD86481) is a first-in-class, small molecule NMT inhibitor originally developed by the University of Dundee Drug Discovery Unit as part of a program to treat African sleeping sickness, funded by Welcome Trust. Pacylex is developing PCLX-001 in the form of a once-a-day pill initially to treat leukemia and lymphoma. PCLX-001 has also been shown to inhibit the growth of lung and breast cancer tumors in animal models. In leukemia, lymphoma and breast cancer patients, the levels of NMT2 are correlated with survival, suggesting an important biological role in these cancers.