On March 15, 2024 aTyr Pharma, Inc. (Nasdaq: LIFE) ("aTyr" or the "Company"), a clinical stage biotechnology company engaged in the discovery and development of first-in-class medicines from its proprietary tRNA synthetase platform, reported fourth quarter and full year 2023 results and provided a corporate update (Press release, aTyr Pharma, MAR 15, 2024, View Source [SID1234641208]).

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"Throughout 2023 we made meaningful progress with our clinical development program for our lead therapeutic candidate, efzofitimod, in interstitial lung disease (ILD)," said Sanjay S. Shukla, M.D., M.S., President and Chief Executive Officer of aTyr. "Our primary focus for 2024 is completing enrollment in our global pivotal Phase 3 EFZO-FIT study in patients with pulmonary sarcoidosis, a major form of ILD, which is anticipated in the second quarter."

"We ended 2023 with more than $100 million in cash, restricted cash, cash equivalents and investments. Based on our current cash position and operational plans, we believe our financial resources are sufficient to fund the Company’s operations through the filing of a Biologics License Application (BLA) for efzofitimod in pulmonary sarcoidosis."

Fourth Quarter 2023 and Subsequent Period Highlights

Continued enrollment in the global pivotal Phase 3 EFZO-FIT study to evaluate the efficacy and safety of efzofitimod in patients with pulmonary sarcoidosis. This is a randomized, double-blind, placebo-controlled, 52-week study consisting of three parallel cohorts randomized equally to either 3.0 mg/kg or 5.0 mg/kg of efzofitimod or placebo dosed intravenously monthly for a total of 12 doses. The study intends to enroll up to 264 patients with pulmonary sarcoidosis. The study is currently enrolling at more than 90 centers in 9 countries. A positive data and safety monitoring board review assessed that the study could continue unmodified. Based on current enrollment projections, the Company anticipates completing enrollment in the study in the second quarter of 2024.
Announced an Individual Patient Expanded Access Program (EAP) for efzofitimod for patients with pulmonary sarcoidosis. The EAP has been initiated based on blinded EFZO-FIT study investigator and patient participant feedback. The program is designed to allow access for patients who complete the Phase 3 EFZO-FIT study and wish to receive treatment with efzofitimod outside of the clinical trial.
Continued enrollment in the Phase 2 EFZO-CONNECT study to evaluate the efficacy, safety and tolerability of efzofitimod in patients with SSc-ILD. This proof-of-concept study is a randomized, double-blind, placebo-controlled, 28-week study consisting of three parallel cohorts randomized 2:2:1 to either 270 mg or 450 mg of efzofitimod or placebo dosed intravenously monthly for a total of 6 doses. The study intends to enroll up to 25 patients with SSc-ILD and is open for enrollment at multiple centers in the U.S.
Poster for efzofitimod accepted for presentation at the upcoming American Thoracic Society (ATS) 2024 International Conference. The conference is scheduled to take place May 17 – 22, 2024, in San Diego, CA.
Poster 8837 – Efzofitimod is an Immunomodulator of Myeloid Cell Function and Novel Therapeutic Candidate for Interstitial Lung Diseases on Sunday, May 19, 2024, at 2:15 p.m. PDT.
Presented two posters highlighting the importance of neuropilin-2 (NRP2) in immune regulation at the Keystone Symposia on Myeloid Cell Diversity. The findings further demonstrate that efzofitimod modulates myeloid cells via the NRP2 receptor to promote a unique anti-inflammatory mechanism and validates the role of NRP2 in the immune system by the activity of an NRP2 blocking antibody in preclinical models.
Announced Wayne A. I. Frederick, M.D., President Emeritus of Howard University, as an advisor to the Company. Dr. Frederick is a distinguished physician executive with extensive knowledge on disparities in healthcare and will advise the Company on its efzofitimod program in ILD.
Poster for ATYR0750 accepted for presentation at the upcoming Gordon Research Conference Fibroblast Growth Factors in Development and Disease. The conference is scheduled to take place March 24 – 29, 2024, in Galveston, TX.
Poster – Alanyl-tRNA Synthetase Fragment Binds to FGFR4 and Induces Morphological Changes and Downstream Signaling in Liver Cells with Functional Similarities to FGF2.

Year Ended 2023 Financial Highlights and Cash Position

Cash & Investment Position: Cash, cash equivalents, restricted cash and investments as of December 31, 2023, were $101.7 million. Based on the Company’s current operational plans and existing cash, the Company maintains its prior guidance and believes its cash runway will be sufficient to fund the Company’s operations through the filing of a BLA for efzofitimod in pulmonary sarcoidosis.
R&D Expenses: Research and development expenses were $42.3 million for the year ended 2023, which consisted primarily of clinical trial costs for the Phase 3 EFZO-FIT and Phase 2 EFZO-CONNECT studies, manufacturing costs for the efzofitimod program and research and development costs for the efzofitimod and discovery programs.
G&A Expenses: General and administrative expenses were $13.0 million for the year ended 2023.

Conference Call and Webcast Details

aTyr will host a conference call and webcast today at 5:00 p.m. EDT / 2:00 p.m. PDT to discuss its financial results and provide a corporate update. Interested parties may access the call by registering here in order to obtain a dial in, personalized passcode and webcast information. Links to a live audio webcast and replay may be accessed on the aTyr website Events page at: View Source An audio replay will be available for at least 90 days following the event.

About Efzofitimod

Efzofitimod is a first-in-class biologic immunomodulator in clinical development for the treatment of interstitial lung disease (ILD), a group of immune-mediated disorders that can cause inflammation and fibrosis, or scarring, of the lungs. Efzofitimod is a tRNA synthetase derived therapy that selectively modulates activated myeloid cells through neuropilin-2 to resolve inflammation without immune suppression and potentially prevent the progression of fibrosis. aTyr is currently investigating efzofitimod in the global Phase 3 EFZO-FIT study in patients with pulmonary sarcoidosis, a major form of ILD, and in the Phase 2 EFZO-CONNECT study in patients with systemic sclerosis (SSc, or scleroderma)-related ILD. These forms of ILD have limited therapeutic options and there is a need for safer and more effective, disease-modifying treatments that improve outcomes.

On March 15, 2024 Soligenix, Inc. (Nasdaq: SNGX) (Soligenix or the Company), a late-stage biopharmaceutical company focused on developing and commercializing products to treat rare diseases where there is an unmet medical need, reported its recent accomplishments and financial results for the year ended December 31, 2023 (Press release, Soligenix, MAR 15, 2024, View Source [SID1234641206]).

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"Our primary focus in 2024 continues to be advancing our multiple clinical programs in our rare disease pipeline," stated Christopher J. Schaber, PhD, President and Chief Executive Officer of Soligenix. "Our collaborative discussions continue with the U.S. Food and Drug Administration (FDA) regarding the design of a second, confirmatory Phase 3 pivotal study evaluating HyBryte (synthetic hypericin) in the treatment of cutaneous T-cell lymphoma (CTCL), where we successfully demonstrated statistically significant results in the first Phase 3 clinical trial. We also continue to engage in discussions with the European Medicines Agency to explore potential marketing approval and partnership in Europe. Recently, we shared successful preliminary top-line results of our ongoing Phase 2a clinical trial of SGX302 (synthetic hypericin) for the treatment of mild-to-moderate psoriasis. Following the clearance of the Investigational New Drug (IND) application for a Phase 2a clinical trial with SGX945 (dusquetide) in Behçet’s Disease and the recent receipt of "Fast Track" designation from the FDA, we anticipate initiating this study in the second half of 2024. Under our Public Health Solutions business segment, we recently announced publication of the preclinical efficacy of a novel, single-vial, bivalent vaccine providing 100% protection against both Sudan ebolavirus (SUDV) and Marburg marburgvirus (MARV) infections. The published paper describes the potency of the bivalent formulation against both lethal viruses, demonstrating 100% protection in the most rigorous non-human primate (NHP) challenge models."

Dr. Schaber continued, "With approximately $8.4 million in cash at December 31, 2023, not including our non-dilutive government funding, we are managing cash burn very carefully in order to achieve our near-term milestones. We recently received $0.6 million, net of transaction costs, from the state of New Jersey’s (NJ) Technology Business Tax Certificate Transfer Program. This is our fourteenth year participating in the program, over that time we have received approximately $9.4 million in non-dilutive capital. We continue to evaluate a number of strategic options, including but not limited to, partnership and merger and acquisition opportunities."

Soligenix Recent Accomplishments

On February 8, 2024, the Company announced the formation of a Medical Advisory Board to provide medical/clinical strategic guidance to the Company as it advances the clinical development of SGX945 (dusquetide) for the treatment of Behçet’s Disease. To view this press release, please click here.
On January 8, 2024, the Company announced its SGX945 development program for the treatment of oral lesions of Behçet’s Disease has received "Fast Track" designation from the FDA. To view this press release, please click here.
On January 4, 2024, the Company announced positive preliminary top-line results of its ongoing Phase 2a trial of SGX302 for the treatment of mild-to-moderate psoriasis. To view this press release, please click here.
On January 2, 2024, the Company announced publication describing the preclinical efficacy of a novel, single-vial, bivalent vaccine providing 100% protection against both SUDV and MARV infections in NHP models. This vaccine candidate had been previously demonstrated to be stable to high temperature storage for at least 2 years at 40 degrees Celsius (104 degrees Fahrenheit). To view this press release, please click here.
On December 1, 2023, the Company announced publication of an article describing the potential use of HyBryte in the treatment of CTCL in Frontiers in Drug Discovery. To view this press release, please click here.
On November 30, 2023, the Company announced the FDA had cleared the IND application for a Phase 2a clinical trial entitled, "Pilot Study of SGX945 (Dusquetide) in the Treatment of Aphthous Ulcers in Behçet’s Disease." To view this press release, please click here.
Financial Results – Year Ended December 31, 2023

Soligenix’s revenues for the year ended December 31, 2023 was $0.8 million as compared to $0.9 million for the year ended December 31, 2022. Revenues primarily relate to government contracts and grants awarded in support of HyBryte, our therapeutic candidate for early-stage CTCL; SGX943, our therapeutic candidate for treatment of emerging and/or antibiotic-resistant infectious diseases; and CiVax, our vaccine candidate for the prevention of COVID-19.

Soligenix’s net loss was $6.1 million, or ($0.79) per share, for the year ended December 31, 2023, as compared to $13.8 million, or ($4.81) per share, for the year ended December 31, 2022. The decrease in net loss was primarily due to decreases in operating expenses and interest expense and an increase in other income.

Research and development expenses were $3.3 million as compared to $7.9 million for the years ended December 31, 2023 and 2022, respectively. The decrease was primarily due to the decrease in manufacturing and regulatory costs associated with the HyBryte new drug application filing.

General and administrative expenses were $4.5 million and $6.7 million for the years ended December 31, 2023 and 2022, respectively. This decrease in general and administrative expenses is primarily attributable to a reduction in legal and consulting expenses.

As of December 31, 2023, the Company’s cash position was approximately $8.4 million.

On March 15, 2024 SELLAS Life Sciences Group, Inc. (NASDAQ: SLS) ("SELLAS" or the "Company"), a late-stage clinical biopharmaceutical company focused on the development of novel therapies for a broad range of cancer indications, reported that it has entered into definitive agreements with two existing institutional investors for the purchase and sale of 13,029,316 shares of its common stock (or common stock equivalents in lieu thereof) in a registered direct offering and warrants to purchase up to an aggregate of 13,029,316 shares of common stock in a concurrent private placement (together with the registered direct offering, the "Offering") at a combined purchase price of $1.535 per share and accompanying warrant, priced at-the-market under Nasdaq rules (Press release, Sellas Life Sciences, MAR 15, 2024, View Source [SID1234641205]). The warrants will have an exercise price of $1.41 per share, will be immediately exercisable upon issuance and will expire 5.5 years from issuance.

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The closing of the Offering is expected to occur on or about March 19, 2024, subject to the satisfaction of customary closing conditions. The gross proceeds from the Offering are expected to be approximately $20 million, before deducting placement agent fees and other estimated offering expenses. The Company intends to use the net proceeds from the Offering for research and development activities, working capital and general corporate purposes.

A.G.P./Alliance Global Partners is acting as sole placement agent for the Offering.

The registered direct offering of the shares of common stock (or common stock equivalents in lieu thereof) is being made pursuant to an effective shelf registration statement on Form S-3 (File No. 333-255318) previously filed with the U.S. Securities and Exchange Commission (the "SEC"). A prospectus supplement describing the terms of the proposed Offering will be filed with the SEC and will be available on the SEC’s website located at View Source Electronic copies of the prospectus supplement may be obtained, when available, from A.G.P./Alliance Global Partners, 590 Madison Avenue, 28th Floor, New York, NY 10022, or by telephone at (212) 624- 2060, or by email at [email protected].

The private placement of the warrants will be made in reliance on an exemption from registration under Section 4(a)(2) of the Securities Act and/or Regulation D thereunder. Accordingly, the securities issued in the concurrent private placement 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 and such applicable state securities laws.

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

On March 15, 2024 Oncternal Therapeutics, Inc. (Nasdaq: ONCT), a clinical-stage biopharmaceutical company focused on the development of novel oncology therapies, reported that it will participate in a virtual fireside chat on the Treatment Landscape & New Treatment Options for Prostate Cancer (Press release, Oncternal Therapeutics, MAR 15, 2024, View Source [SID1234641204]).

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Oncternal’s President and CEO, James Breitmeyer, M.D., Ph.D. will join Oppenheimer Senior Research Biotech Analyst, Hartaj Singh and a prostate cancer Key Opinion Leader on Tuesday, March 19th, 2024 at 11:00 AM ET. Dr. Breitmeyer will discuss the development of Oncternal’s novel dual-acting androgen receptor inhibitor, ONCT-534, the ongoing Phase 1/2 clinical trial ONCT-534-101, and the potential positioning of ONCT-534 within the treatment paradigm of advanced prostate cancer.

To join this call, please contact your Oppenheimer institutional salesperson. A replay of the event will be available online at investor.oncternal.com and it will be archived there for at least 30 days.

On March 15, 2024 Merck (NYSE: MRK), known as MSD outside of the United States and Canada, reported that the Phase 3 KEYNOTE-A18 trial, also known as ENGOT-cx11/GOG-3047, investigating KEYTRUDA, Merck’s anti-PD-1 therapy, in combination with chemoradiotherapy (CRT) met its primary endpoint of overall survival (OS) for the treatment of newly diagnosed patients with high-risk locally advanced cervical cancer (Press release, Merck & Co, MAR 15, 2024, View Source [SID1234641202]).

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At a pre-specified interim analysis conducted by an independent Data Monitoring Committee, KEYTRUDA in combination with concurrent CRT showed a statistically significant and clinically meaningful improvement in OS versus concurrent CRT alone. The safety profile of KEYTRUDA in this trial was consistent with that observed in previously reported studies; no new safety signals were identified. Results will be presented at an upcoming medical meeting and shared with regulatory authorities worldwide.

As previously reported, KEYNOTE-A18 met its other primary endpoint of progression-free survival (PFS) in 2023. These PFS data were presented at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Congress 2023 and supported the U.S. Food and Drug Administration’s approval of KEYTRUDA in combination with CRT for the treatment of patients with FIGO (International Federation of Gynecology and Obstetrics) 2014 Stage III-IVA cervical cancer in January 2024.

"This is the first Phase 3 trial in which an immunotherapy-based regimen has shown a statistically significant and clinically meaningful improvement in overall survival compared to chemoradiotherapy alone," said Dr. Gursel Aktan, vice president, global clinical development, Merck Research Laboratories. "Building on the positive progression-free survival findings from this study, these results underscore our commitment to exploring the role of KEYTRUDA across different types of cancers in earlier stages of disease, where there is a greater potential for better outcomes."

"These findings are important for patients and the medical community alike and reinforce previous data from the KEYNOTE-A18 trial, now showing this regimen has the potential to extend the lives of patients with locally advanced cervical cancer," said Prof. Domenica Lorusso, the study’s overall principal investigator, lead investigator for ENGOT, and professor of Obstetrics and Gynecology at Humanitas University.

In the U.S., KEYTRUDA has two additional approved indications in cervical cancer: in combination with chemotherapy, with or without bevacizumab, for the treatment of patients with persistent, recurrent, or metastatic cervical cancer whose tumors express PD-L1 [Combined Positive Score (CPS) ≥1] as determined by an FDA-approved test; and as a single agent, for the treatment of patients with recurrent or metastatic cervical cancer with disease progression on or after chemotherapy whose tumors express PD-L1 (CPS ≥1) as determined by an FDA-approved test.

About KEYNOTE-A18/ENGOT-cx11/GOG-3047

KEYNOTE-A18, also known as ENGOT-cx11/GOG-3047, is a randomized, double-blind Phase 3 trial (ClinicalTrials.gov, NCT04221945) sponsored by Merck and conducted in collaboration with the European Network for Gynecological Oncology Trial (ENGOT) groups and the GOG Foundation, Inc. (GOG) investigating KEYTRUDA in combination with CRT (cisplatin and external beam radiotherapy [EBRT] followed by brachytherapy [BT]) compared to placebo plus concurrent CRT for the treatment of newly diagnosed high-risk (stage IB2-IIB with lymph node-positive disease, and stage III-IVA with and without lymph node-positive disease) locally advanced cervical cancer where patients are treated with definitive intent. The primary endpoints are PFS and OS, and secondary endpoints include complete response rate, objective response rate and safety. The trial enrolled 1,060 patients with cervical cancer who had not previously received any definitive surgery, radiation, or systemic therapy for cervical cancer. Patients were randomized (1:1) to receive either:

KEYTRUDA (200 mg intravenously [IV]) every three weeks (Q3W) for five cycles concurrent with cisplatin (40 mg/m2 IV) weekly for five cycles (an optional sixth infusion could be administered per local practice) and radiotherapy (EBRT followed by BT), followed by KEYTRUDA (400 mg IV) every six weeks (Q6W) for 15 cycles;
Placebo IV Q3W for five cycles concurrent with cisplatin (40 mg/m2 IV) weekly for five cycles (an optional sixth infusion could be administered per local practice) and radiotherapy (EBRT followed by BT), followed by placebo IV Q6W for 15 cycles.
About cervical cancer

Cervical cancer forms in the cells lining the cervix, which is the lower part of the uterus. All women are at risk for cervical cancer, and it is most frequently diagnosed between the ages of 35 and 44. While screenings and prevention have resulted in declining cervical cancer rates, the disease continues to affect many people in the U.S. and around the world. Cervical cancer is the fourth most common cancer in women globally. In the U.S., it is estimated there will be approximately 13,820 new patients diagnosed with invasive cervical cancer and about 4,360 deaths from the disease in 2024.