On January 2, 2024 Bicara Therapeutics, a clinical-stage biotechnology company developing dual-action biologics to elicit a potent and durable immune response, reported that Ryan Cohlhepp, Pharm.D., president & chief operating officer, will present at the 42nd Annual J.P. Morgan Healthcare Conference on Tuesday, January 9, 2024 at 5:30 p.m. Pacific Time (Press release, Bicara Therapeutics, JAN 2, 2024, View Source [SID1234638854]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Additional information about Bicara Therapeutics can be found on the company’s website at www.bicara.com.

On January 2, 2024 Akari Therapeutics, Plc (Nasdaq: AKTX), a late-stage biotechnology company developing advanced therapies for autoimmune and inflammatory diseases, reported that it closed a private placement financing with existing investors, Akari Chairman Dr. Ray Prudo and Director Samir R. Patel, M.D., on December 29, 2023, resulting in gross proceeds of approximately $2 million (Press release, Akari Therapeutics, JAN 2, 2024, View Source [SID1234638853]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

In connection with the financing, Akari issued 947,868 unregistered American Depository Shares ("ADSs"), each representing 2,000 of the company’s ordinary shares, at a purchase price of $2.11 per ADS.

Paulson Investment Company, LLC acted as the exclusive placement agent for this financing.

The ADSs described above were offered and sold in a private placement under Section 4(a)(2) of the Securities Act of 1933, as amended (the "Act") and Regulation D promulgated thereunder and have not been registered under the Act or state securities laws and may not be offered or sold in the United States absent registration with the Securities and Exchange Commission or an applicable exemption from such registration requirements.

This press release shall not constitute an offer to sell or the solicitation of an offer to buy any of the securities described herein. There shall not be any offer, solicitation of an offer to buy, or sale of securities in any state or jurisdiction in which such an offering, solicitation, or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction.

On January 2, 2024 Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a leader in the field of cellular metabolism pioneering therapies for rare diseases, reported that the company is scheduled to present at the 42nd Annual J.P. Morgan Healthcare Conference on Wednesday, January 10, 2024, at 7:30 a.m. PT (Press release, Agios Pharmaceuticals, JAN 2, 2024, View Source [SID1234638852]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

A live webcast of the presentation can be accessed under "Events & Presentations" in the Investors section of the company’s website at www.agios.com. A replay of the webcast will be archived on the company’s website for at least two weeks following the presentation.

On January 2, 2024 Actinium Pharmaceuticals, Inc. (NYSE AMERICAN: ATNM) (Actinium or the Company), a leader in the development of targeted radiotherapies, reported that five abstracts have been accepted for presentation at the 2024 Tandem Meetings | Transplantation & Cellular Therapy (TCT) Meetings of ASTCT (American Society for Transplantation and Cellular Therapy and CIBMTR (Center for International Blood and Marrow Transplant Research) being held February 21 – 24, 2024 in San Antonio, Texas (Press release, Actinium Pharmaceuticals, JAN 2, 2024, View Source [SID1234638851]). Two oral presentations will highlight results from the positive Phase 3 SIERRA trial of Iomab-B (131I-Apamistamab), representing 10 oral presentations of the SIERRA results since they were first reported at the 2023 Tandem Meetings.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Sandesh Seth, Actinium’s Chairman and CEO, said, "We are excited to continue to build recognition for Iomab-B and the SIERRA data with the transplant community at the upcoming TCT meetings. Over the last year, we have highlighted to the global transplant and hematology communities that an Iomab-B led transplant improves outcomes for patients with high-risk relapsed/refractory acute myeloid leukemia, including those with a TP53 mutation, who have limited treatment options and poor outcomes with current therapies. These results exemplify the unique capabilities of targeted radiotherapy and its potent, mutation-agnostic mechanism of action. We are highly encouraged by the positive receptivity by the medical and scientific communities to date and look forward to building on this momentum in 2024."

Oral Presentations:

Title: 131I-Apamistamab Improves Outcomes in Patients 65 Years and Older with Relapsed or Refractory AML

Date & Time: Saturday, February 24, 2024, at 11:45 AM

Title: Targeted Myeloablative Radiation Using 131I-Apamistamab Prior to Allogeneic Hematopoietic Cell Transplant for Patients with R/R AML Results in Robust Engraftment

Date & Time: Saturday, February 24, 2024, at 10:30 AM

Poster Presentations:

Dose-Response Demonstrated for Durable Complete Remission Following High-Dose Targeted Radiation with 131I-Apamistamab Prior to HCT in Patients with R/R AML

Low-Dose Targeted Radioimmunotherapy (Iomab-ACT) Achieves Lymphocyte and Monocyte Depletion Prior to CD19-Targeted CAR T-Cell Therapy for Relapsed of Refractory B-Cell ALL or DLBCL with Minimal CRS and Icans

131I-Apamistamab-Led Allogeneic Hematopoietic Cell Transplant for Patients with TP53 Mutated R/R AML Results in Significantly Improved Outcomes

The posters will be presented on Thursday, February 22, 2024, from 6:45 – 7:45 PM

About the TCT Tandem Meetings

The Tandem Meetings I Transplantation & Cellular Therapy Meetings of ASTCT and CIBMTR are the combined annual meetings of the American Society for Transplantation and Cellular Therapy (ASTCT) and the Center for International Blood & Marrow Transplant Research (CIBMTR). Administrators, clinicians, data manager / clinical research professionals, fellows-in-training, investigators, laboratory technicians, MD/PhDs, nurses, nurse practitioners, pharmacists, physician assistants, and other allied health professional attendees benefit from a full scientific program that addresses the most timely issues in hematopoietic cell transplantation and cellular therapy.

On January 2, 2024 Syndax Pharmaceuticals (Nasdaq: SNDX), a clinical stage biopharmaceutical company developing an innovative pipeline of cancer therapies, reported recent updates and anticipated 2024 milestones (Press release, Syndax, JAN 2, 2024, View Source [SID1234638850]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"With positive pivotal data readouts for both revumenib and axatilimab and subsequent presentations at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting, 2023 was truly a landmark year for Syndax," said Michael A. Metzger, Chief Executive Officer. "We are working with the FDA during their review of our regulatory submissions while diligently preparing for the potential launch of two first- and best-in-class products in the U.S. in 2024. We look forward to continuing to broadly expand revumenib and axatilimab beyond their first approvals and into additional indications which have the potential to deliver meaningful clinical benefit."

Recent Company Updates and Planned 2024 Milestones

Revumenib:

The Company announced the submission of a New Drug Application (NDA) under the U.S. Food and Drug Administration (FDA) Real-time Oncology Review (RTOR) program to the FDA for revumenib, a first-in-class menin inhibitor, for the treatment of adult and pediatric relapsed or refractory (R/R) KMT2A-rearranged (KMT2Ar) acute leukemia on December 29, 2023. As per RTOR guidance, the submission is not deemed complete until the FDA issues a PDUFA date, which Syndax expects to receive in the first quarter. RTOR allows for close engagement between the sponsor and the FDA throughout the submission process and enables the FDA to review individual modules of a drug application rather than requiring a complete application prior to initiating its review.
The Company expects to complete enrollment in the AUGMENT-101 pivotal trial cohort of patients with R/R mutant nucleophosmin (mNPM1) acute myeloid leukemia (AML) later this quarter or early in the second quarter. The Company plans to report topline data in the fourth quarter of 2024, which could support a regulatory filing for revumenib in an additional indication of R/R mNPM1 AML.
At the 65th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting in December 2023, the Company highlighted positive results in multiple presentations from the Phase 1 and 2 portions of the AUGMENT-101 trial, including the pivotal AUGMENT-101 results that were featured as a late-breaking oral presentation.
Investigators presented data from multiple Phase 1 combination trials of revumenib in mNPM1 and KMT2Ar acute leukemia across the treatment landscape at the ASH (Free ASH Whitepaper) Annual Meeting and the Company’s corporate event. The trials are expanding to validate the recommended Phase 2 dose and the Company expects to have additional data on the trials in the second half of 2024. These include:
BEAT AML: Evaluating the combination of revumenib with venetoclax and azacitidine in front-line AML patients. This trial is being conducted as part of the Leukemia & Lymphoma Society’s Beat AML Master Clinical Trial.
SAVE: Evaluating the all-oral combination of revumenib with venetoclax and decitabine/cedazuridine in R/R AML or mixed phenotype acute leukemias. The trial is being conducted by investigators from the MD Anderson Cancer Center and continues to accrue patients.
AUGMENT-102: Evaluating the combination of revumenib with fludarabine and cytarabine in patients with R/R acute leukemias.
The Company plans to initiate a trial of revumenib in combination with 7+3 cytarabine and daunorubicin chemotherapy followed by maintenance treatment in newly diagnosed patients with mNPM1 or KMT2Ar acute leukemias this quarter.
The Company plans to initiate a pivotal trial of revumenib in combination with venetoclax and azacitidine in newly diagnosed patients with mNPM1 or KMT2Ar acute leukemias by the end of 2024.
Enrollment is ongoing in a Phase 1 proof-of-concept clinical trial of revumenib in patients with unresectable metastatic microsatellite stable colorectal cancer. The Company expects to provide an update on the trial in the first half of 2024.
Axatilimab:

The Biologics License Application (BLA) for axatilimab, an anti-CSF-1R antibody, in adult and pediatric patients six years or older with chronic graft-versus-host disease (cGVHD) after failure of at least two prior lines of systemic therapy was submitted to the FDA on December 28, 2023.
Results from the pivotal Phase 2 AGAVE-201 trial were featured in the Plenary Scientific Session at the 65th ASH (Free ASH Whitepaper) Annual Meeting in December 2023.
Syndax announced today that it has exercised its option under the Company’s 2021 collaboration agreement with Incyte to co-commercialize axatilimab in the U.S.
Syndax announced today the randomized, double-blind and placebo-controlled Phase 2 trial to assess the efficacy, safety and tolerability of axatilimab in patients with idiopathic pulmonary fibrosis (IPF) is open for enrollment.
Additionally, Incyte plans to initiate two combination trials with axatilimab in cGVHD in mid-2024, including a Phase 2 combination trial with ruxolitinib and a Phase 3 combination trial with steroids.
Corporate

In the fourth quarter of 2023, Syndax issued 12,432,431 shares of its common stock at $18.50 per share. Additionally in the quarter, the Company sold 2,719,744 shares from its ATM facility. Aggregate net proceeds from these offerings were approximately $258.1 million after deducting underwriting discounts and commissions and estimated offering expenses payable by Syndax. With these proceeds, the Company now believes that it has sufficient cash runway to fund its clinical and commercial operations through 2026.
About Revumenib

Revumenib is a potent, selective, small molecule inhibitor of the menin-KMT2A binding interaction that is being developed for the treatment of KMT2A-rearranged, also known as mixed lineage leukemia rearranged or MLLr, acute leukemias including ALL and AML, and NPM1-mutant AML. Positive topline results from the Phase 2 AUGMENT-101 trial in R/R KMT2Ar acute leukemia showing the trial met its primary endpoint were recently presented at the 65th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and data from the Phase 1 portion of AUGMENT-101 in acute leukemia was published in Nature. Revumenib was granted Orphan Drug Designation by the FDA and European Commission for the treatment of patients with AML, and Fast Track designation by the FDA for the treatment of adult and pediatric patients with R/R acute leukemias harboring a KMT2A rearrangement or NPM1 mutation. Revumenib was granted BTD by the FDA for the treatment of adult and pediatric patients with R/R acute leukemia harboring a KMT2A rearrangement.

About Axatilimab

Axatilimab is an investigational monoclonal antibody that targets colony stimulating factor-1 receptor, or CSF-1R, a cell surface protein thought to control the survival and function of monocytes and macrophages. In pre-clinical models, inhibition of signaling through the CSF-1 receptor has been shown to reduce the number of disease-mediating macrophages along with their monocyte precursors, which has been shown to play a key role in the fibrotic disease process underlying diseases such as chronic graft-versus-host disease (GVHD) and idiopathic pulmonary fibrosis (IPF). Positive topline results from the Phase 2 AGAVE-201 trial showing the trial met its primary endpoint were recently presented at the 65th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Phase 1/2 data of axatilimab in chronic GVHD were published in the Journal of Clinical Oncology. Axatilimab was granted Orphan Drug Designation by the U.S. Food and Drug Administration for the treatment of patients with chronic GVHD and IPF. In September 2021, Syndax and Incyte entered into an exclusive worldwide co-development and co-commercialization license agreement for axatilimab. Axatilimab is being developed under an exclusive worldwide license from UCB entered into between Syndax and UCB in 2016.