On February 24, 2025 Azitra, Inc. (NYSE American: AZTR), a clinical stage biopharmaceutical company focused on developing innovative therapies for precision dermatology, reported financial results for the full year ended December 31, 2024, and provided a business update (Press release, Azitra, FEB 24, 2025, View Source [SID1234650461]).

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FY 2024 and Recent Business Highlights

Initiated a Phase 1b clinical trial investigating ATR-12 in adult Netherton syndrome patients; Initial safety data from first set of Netherton syndrome patients expected in the first half of 2025 with topline data from the Phase 1b trial by year-end 2025
Received clearance from the U.S. Food and Drug Administration (FDA) for a first-in-human Phase 1/2 clinical study of ATR-04 for adults with moderate to severe EGFRi-associated dermal toxicity
FDA granted Fast Track designation to ATR-04, demonstrating that the FDA recognizes the unmet need for treatment of EGFRi-associated skin rash
Announced closing of $10.0 million and $5.0 million public offerings
Strengthened intellectual property (IP) portfolio with newly granted and allowed patents
"This is a very exciting time in the growth and evolution of Azitra as we seek to drive shareholder value through development of first-in-class drugs to treat dermatological diseases," said Francisco Salva, CEO of Azitra. "Azitra is currently advancing a therapeutic pipeline with multiple programs developed from our proprietary platform of engineered proteins delivered using topical live biotherapeutic products. Our initial focus is the development of genetically engineered strains of Staphylococcus epidermidis (S. epidermidis) to enable the delivery of critical missing natural proteins and disease-modifying proteins through the stratum corneum of the skin. This advantage could allow Azitra to address several dermatological conditions that are significantly underserved by current standards of care."

Salva continued, "Our lead product, ATR-12, is an engineered strain of S. epidermidis designed to treat Netherton syndrome, a rare, chronic skin disease with no approved treatment options. In August 2024, we initiated a Phase 1b clinical trial investigating ATR-12 in adult Netherton syndrome patients to assess multiple safety, tolerability, and efficacy endpoints. Initial safety data from this trial is expected in the first half of 2025 with topline results by year-end 2025."

Salva continued, "In addition to ATR-12, Azitra has made significant progress with our next most advanced product, ATR-04. ATR-04 is a live biotherapeutic product candidate containing an isolated, naturally derived S. epidermidis strain being developed for the treatment of EGFR inhibitor ("EGFRi") associated rash, which impacts approximately 150,000 patients in the United States annually, representing a market opportunity in excess of $1 billion. Our next milestone in the development of ATR-04 is the first patient dosed in a multicenter, randomized, controlled Phase 1/2 clinical trial in patients undergoing EGFR inhibitors with dermal toxicity, which we expect to occur in the first half of 2025."

Salva concluded, "We look forward to capitalizing on multiple value-building milestones during 2025, including clinical data from our ATR-12 program. These events are expected to provide key inflection points for the company and investors throughout the year as we continue to position Azitra as a leading and innovative company developing transformative drugs for underserved patients with life-altering dermatological diseases."

Pipeline and Upcoming Milestones
ATR-12 – Advancing Phase 1b Clinical Trial in Netherton Syndrome with Multiple Milestones Expected

In August 2024, initiated a Phase 1b clinical trial investigating ATR-12 in adult Netherton syndrome patients. Trial is designed to assess multiple safety, tolerability, and efficacy endpoints, providing a springboard for several potential value creating events during the year
Initial safety data from first set of Netherton syndrome patients in the first half of 2025
Topline data from the Phase 1b trial by year-end 2025
Azitra presented compelling preclinical data for ATR-12 in Netherton syndrome at the American Society of Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper) 2024 Annual Meeting. Among the findings presented at the conference, ATR-12 significantly reduced protease activity in skin samples compared to a Netherton syndrome model skin (p<0.01). Additionally, ATR-12 produced higher amounts of LEKTI subunit compared to topical application of LEKTI protein alone after 24 hours and resulted in deeper skin penetration of LEKTI.

ATR-04 – Addressing an Unmet Need in a Multi-billion Dollar Market Opportunity

In August, Azitra received clearance from the U.S. Food and Drug Administration (FDA) for a first-in-human Phase 1/2 clinical study of ATR-04 for moderate to severe EGFRi-associated dermal toxicity
In September, the FDA granted Fast Track designation to ATR-04, demonstrating that the FDA recognizes the unmet need for treatment of EGFRi-associated skin rash
Also in 2024, Azitra presented preclinical data at the Society of Investigative Dermatology (SID) and the European Academy of Dermatology and Venereology (EADV) annual meetings showing ATR-04 inhibits IL-36g and S. aureus, both of which are key drivers of the disease
Plan to initiate a multicenter, randomized, controlled Phase 1/2 clinical trial in patients undergoing EGFR inhibitors with dermal toxicity in first half of 2025
Financial Results for the Year Ended December 31, 2024
Service Revenue – Related Party: The Company generated $0.8 thousand of service revenue during the year ended December 31, 2024, compared to $0.7 million for fiscal year 2023.
Research and Development (R&D) expenses: R&D expenses for the year ended December 31, 2024, were $4.7 million compared to $3.6 million for fiscal year 2023.
General and Administrative (G&A) expenses: G&A expenses for the year ended December 31, 2024, were $6.3 million compared to $4.5 million for fiscal year 2023.
Net Loss was $9.0 million for the year ended December 31, 2024, compared to $11.3 million for fiscal year 2023.
Cash and cash equivalents: As of December 31, 2024, the Company had cash and cash equivalents of $4.6 million, which does not include gross proceeds of approximately $2.2 million from follow-on offerings in January and February, 2025.

On February 24, 2025 Ascendis Pharma A/S (Nasdaq: ASND) reported that company executives will participate in a fireside chat at the TD Cowen 45th Annual Health Care Conference on Tuesday, March 4, 2025, at 11:10 a.m. Eastern Time / 8:10 a.m. Pacific Time in Boston, Massachusetts (Press release, Ascendis Pharma, FEB 24, 2025, View Source [SID1234650460]).

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A live webcast of the presentation will be available via the Investors & News section of the Ascendis Pharma website at investors.ascendispharma.com. A webcast replay will also be available on this website shortly after conclusion of the event for 30 days.

On February 24, 2025 Anixa Biosciences, Inc. ("Anixa" or the "Company") (NASDAQ: ANIX), a biotechnology company focused on the treatment and prevention of cancer, reported that its pioneering CAR-T therapy for ovarian cancer, developed in collaboration with Moffitt Cancer Center ("Moffitt"), was prominently featured in Breaking Cancer News in an article titled "Navigating Uncharted Territory: CAR-T for Ovarian Cancer (Press release, Anixa Biosciences, FEB 24, 2025, View Source [SID1234650459])."

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The article highlights the groundbreaking clinical trial led by Dr. Robert Wenham, Chairman of the Department of Gynecologic Oncology at Moffitt Cancer Center, and Dr. Monica Avila, a leading gynecologic oncologist at Moffitt. The trial explores the use of chimeric antigen receptor-T cell (CAR-T) therapy to treat ovarian cancer, a disease with historically limited treatment options for advanced-stage patients.

The CAR-T therapy being studied in this trial, exclusively licensed to Anixa from The Wistar Institute, and developed through a partnership between Anixa and Moffitt, is designed to target the follicle-stimulating hormone receptor (FSHR), a protein uniquely expressed on ovarian cancer cells. This novel approach has the potential to revolutionize the treatment of ovarian cancer by leveraging the body’s immune system to directly attack tumors.

Dr. Amit Kumar, Chairman and CEO of Anixa Biosciences, commented, "We are honored to see our work recognized in Breaking Cancer News and to witness the promising impact this CAR-T therapy is having on patients. Our partnership with Moffitt Cancer Center exemplifies our commitment to developing novel and targeted immunotherapies that could transform the standard of care for some of the most challenging cancers, including ovarian cancer."

The article also highlights the potential expansion of the study to evaluate repeat dosing, given Gallagher’s response, and explores the broader implications of CAR-T therapy for solid tumors—an area of intense research and high unmet medical need.

On February 23, 2025 Innovent Biologics, Inc. ("Innovent") (HKEX: 01801), a world-class biopharmaceutical company that develops, manufactures and commercializes high quality medicines for the treatment of oncology, cardiovascular and metabolic, autoimmune, ophthalmology and other major diseases, reported that the New Drug Application (NDA) for ipilimumab injection (anti-CTLA-4 monoclonal antibody; R&D Code: IBI310) has been accepted by the Center for Drug Evaluation (CDE) of China’s National Medical Products Administration (NMPA) and granted Priority Review designation[1] in combination with sintilimab as neoadjuvant treatment for resectable microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) colon cancer (Press release, Innovent Biologics, FEB 23, 2025, View Source [SID1234650451]).

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This is China’s first NDA for a domestic CTLA-4 inhibitor and another milestone strengthening sintilimab’s leadership position in cancer immunotherapy. Immune checkpoint blockade (ICB) therapy targeting PD-1 and CTLA-4 has transformed oncology treatment, ipilimumab with sintilimab as a neoadjuvant treatment could increase R0 resection rate, achieve pathological complete response, and relieve the majority of patients from adjuvant chemotherapy burdens. This novel treatment is also expected to reduce recurrence rate and improve long-term prognosis, anticipated to benefit MSI-H/dMMR colon cancer patients upon NDA approval.

The NDA acceptance and Priority Review designation are based on results from a randomized, controlled, multicenter, pivotal Phase 3 clinical trial (NeoShot, NCT05890742) which evaluated the safety and efficacy of ipilimumab combined with sintilimab as neoadjuvant therapy and as compared with direct radical surgery for MSI-H/dMMR colon cancer. The primary endpoints are pathologic complete response (pCR) rate and event-free survival (EFS). Interim analysis by the Independent Data Monitoring Committee (IDMC) showed that the NeoShot trial has met its primary endpoint. Detailed results will be presented at future academic conferences or published in academic journals.

Previously, the results of a randomized controlled Phase 1b trial evaluating ipilimumab in combination with sintilimab as neoadjuvant treatment for MSI-H/dMMR colon cancer were presented orally at the 2024 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meetingi. Based on promising Phase 1b results, the CDE has granted Breakthrough Therapy Designation (BTD) for ipilimumab.

As of February 4, 2024, 101 patients were enrolled and randomized to receive ipilimumab plus sintilimab (n=52) or sintilimab alone (n=49).
In the per-protocol population, the pCR rate in the ipilimumab-plus-sintilimab arm was significantly higher than in the sintilimab-alone arm（80.0% vs 47.7%, p=0.0007）.
All patients in both treatment arms had R0 resection. With median follow-up of 5.65 months, no patient had disease recurrence.
At postoperative pathological evaluation, 3.9% of patients with ipilimumab plus sintilimab and 15.9% of patients with sintilimab alone were stage N+. The majority of patients could be relieved from adjuvant treatment according to clinical guidelines.
Ipilimumab -plus-sintilimab increased neither safety risk compared to sintilimab alone nor risk for subsequent surgery delay or cancellation.
The Principal Investigator of the NeoShot study, Prof. Ruihua Xu from Sun Yat-sen University Cancer Center, stated: "At present, R0 resection for certain locally advanced colon cancer patients remains a significant challenge, along with risks of extensive trauma and poor prognosis. The results of the FOxTROT study suggested that neoadjuvant chemotherapy is not effective in MSI-H/dMMR colon cancer, and the pCR rate is only around 5%ii. The NeoShot trial is the first randomized, controlled, Phase 3 clinical trial to show promising efficacy of dual checkpoint inhibition as neoadjuvant therapy in MSI-H/dMMR colon cancer. Results from the phase 1b study suggest that ipilimumab with sintilimab as short-term neoadjuvant treatment could increase R0 resection rate, achieve pathological complete response, and relieve patients from adjuvant chemotherapy burdens. This novel treatment is also expected to lower recurrence rate and improve long-term prognosis i.As this dual immunotherapy regimen has the potential to change clinical practice, we look forward to the NDA approval of this novel drug to benefit more MSI-H/dMMR colon cancer patients soon. "

Dr. Hui Zhou, Senior Vice President of Innovent, stated: "There is a huge unmet clinical need for neoadjuvant therapy of resectable MSI-H/dMMR colon cancer in China. Interim analysis showed that the NeoShot trial has met its primary endpoint. With Innovent’s highly efficient and high-quality clinical development, ipilimumab has become China’s first domestic CTLA-4 inhibitor to submit an NDA. We will actively cooperate with regulatory authorities to accelerate approval, and provide a new treatment option for patients with resectable MSI-H/dMMR colon cancer in China."

About Ipilimumab

Ipilimumab (R&D code: IBI310) is a fully human monoclonal antibody injection independently developed by Innovent. Ipilimumab can specifically bind cytotoxic T lymphocyte-associated antigen 4 (CTLA-4), thereby blocking CTLA-4 mediated T cell inhibition, promoting T cell activation and proliferation, improving tumor immune response, and achieving anti-tumor effects. iii

The NDA for ipilimumab in combination with sintilimab as neoadjuvant treatment for resectable microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) colon cancer is under the NMPA review and has been granted Priority Review designation.

About Sintilimab

Sintilimab, marketed as TYVYT (sintilimab injection) in China, is a PD-1 immunoglobulin G4 monoclonal antibody co-developed by Innovent and Eli Lilly and Company. Sintilimab is a type of immunoglobulin G4 monoclonal antibody, which binds to PD-1 molecules on the surface of T-cells, blocks the PD-1 / PD-Ligand 1 (PD-L1) pathway, and reactivates T-cells to kill cancer cells.iv

In China, sintilimab has been approved and included in the updated NRDL for seven indications. The updated NRDL reimbursement scope for TYVYT (sintilimab injection) includes:

For the treatment of relapsed or refractory classic Hodgkin’s lymphoma after two lines or later of systemic chemotherapy;
For the first-line treatment of unresectable locally advanced or metastatic non-squamous non-small cell lung cancer lacking EGFR or ALK driver gene mutations;
For the treatment of patients with EGFR-mutated locally advanced or metastatic non-squamous non-small cell lung cancer who progressed after EGFR-TKI therapy;
For the first-line treatment of unresectable locally advanced or metastatic squamous non-small cell lung cancer;
For the first-line treatment of unresectable or metastatic hepatocellular carcinoma with no prior systematic treatment;
For the first-line treatment of unresectable locally advanced, recurrent or metastatic esophageal squamous cell carcinoma;
For the first-line treatment of unresectable locally advanced, recurrent or metastatic gastric or gastroesophageal junction adenocarcinoma.
Furthermore, sintilimab’s eighth indication, in combination with fruquintinib for the treatment of patients with advanced endometrial cancer with pMMR tumors that have failed prior systemic therapy and are not candidates for curative surgery or radiation, was conditional approved by the NMPA in December 2024. And the NDA for sintilimab in combination with ipilimumab as neoadjuvant treatment for resectable MSI-H/dMMR colon cancer is under the NMPA review and has been granted Priority Review designation.

In addition, two clinical studies of sintilimab have met their primary endpoints:

Phase 2 study of sintilimab monotherapy as second-line treatment of esophageal squamous cell carcinoma;
Phase 3 study of sintilimab monotherapy as second-line treatment for squamous non-small cell lung cancer with disease progression following platinum-based chemotherapy.

On February 23, 2025 Agenus Inc. (Nasdaq: AGEN), a leader in immuno-oncology, reported that BOT/BAL will be featured in two presentations at the upcoming American Association for Cancer Research (AACR) (Free AACR Whitepaper) IO Annual Meeting that will take place on February 23-26 in Los Angeles, California (Press release, Agenus, FEB 23, 2025, View Source [SID1234650450]). An oral presentation will highlight interim data from the ongoing Phase 2 study of botensilimab and balstilimab (BOT/BAL) in combination with MiNK Therapeutics’ iNKT cell therapy, AgenT-797, in patients with refractory (2L+) gastric cancer (NCT06251973). A Trial-in-Progress (TiP) poster will feature data from the ongoing Phase 1/2 study of BOT/BAL in first-line MSS colorectal cancer (NCT06268015).

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Presentation Details:

Abstract Title: First-line botensilimab and balstilimab optimization in microsatellite stable colorectal cancer (MSS-CRC) without liver metastasis (BBOpCo)

Session : Poster Session A

Session Date and Time: Monday, February 24th , 1:45-4:45 p.m. PST

Abstract Title: Biomarker analysis from phase 2 study of AgenT-797 (invariant natural killer T-cells), botensilimab (a Fc-enhanced CTLA-4 Inhibitor) with balstilimab (anti-PD-1) in PD-1 refractory gastroesophageal cancer (GEC)

Session : Proffered Papers, Session 2

Session Date and Time: Monday, February 24th , 1:39-1:45 p.m. PST