On February 25, 2025 Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company developing precision genetic medicines through base editing, reported fourth quarter and full year 2024 financial results and reiterated anticipated milestones across the company’s hematology and genetic disease franchises (Press release, Beam Therapeutics, FEB 25, 2025, View Source [SID1234650517]).

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"We are incredibly proud of the significant progress and momentum across our hematology and liver-targeted genetic disease franchises over the last year," said John Evans, chief executive officer of Beam. "On the heels of the initial positive results from the BEACON trial for BEAM-101 in patients with sickle cell disease, we recently achieved our adult enrollment target in the study and enrolled our first adolescent patients. Additionally, we remain on track to deliver initial data for our lead in vivo program, BEAM-302 in alpha-1 antitrypsin deficiency, in the first half of 2025, where we have the potential for a one-time treatment to address both the lung and liver manifestations of disease. With a strong financial position and important catalysts on the horizon, we are well equipped to continue driving forward our mission of providing life-long cures to patients suffering from serious diseases."

Fourth Quarter 2024 and Recent Progress

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The company has achieved its adult enrollment target for the BEACON Phase 1/2 clinical trial of BEAM-101, an investigational genetically modified cell therapy for the treatment of sickle cell disease (SCD). In addition, multiple adolescent patients have cleared screening and enrolled in the trial.
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In December, Beam presented initial results for the BEACON trial at the 66th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting, which demonstrated that treatment with BEAM-101 induced robust and durable increases in fetal hemoglobin and reductions in sickle hemoglobin, rapid neutrophil and platelet engraftment, and normalized or improved markers of hemolysis. The presentation subsequently received the "Best of ASH (Free ASH Whitepaper)" distinction, and an encore of the data was presented at the 2025 Tandem Meetings of ASTCT and CIBMTR.
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Also at ASH (Free ASH Whitepaper), Beam presented proof-of-concept data in non-human primates for its Engineered Stem Cell Antibody Evasion (ESCAPE) platform demonstrating engraftment of base-edited cells using only antibody conditioning and no chemotherapy. The company initiated Phase 1-enabling preclinical toxicology studies for ESCAPE in December.
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The company continues to advance global regulatory and site activation activities for BEAM-302, an in vivo base editor being developed for the potential treatment of alpha-1 antitrypsin deficiency (AATD), with sites now open in the United Kingdom, New Zealand, Australia and Netherlands.
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In January, Beam activated the first clinical trial site for the Phase 1/2 clinical trial of BEAM-301, an in vivo base editor being developed for the potential treatment of glycogen storage disease type 1a (GSD1a).

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In December, Beam appointed Sravan Emany as chief financing officer. In addition, Chirfi Guindo, chief marketing officer of Human Health at Merck & Co., Inc., was appointed to its board of directors.
Key 2025 Anticipated Milestones

Hematology Franchise

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In the BEACON Phase 1/2 clinical trial of BEAM-101 in adults with severe SCD, Beam expects to present updated data in mid-2025.
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Beam expects to dose 30 patients in the BEACON trial by mid-2025.
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The company expects to initiate a Phase 1 healthy volunteer clinical trial of BEAM-103, the ESCAPE monoclonal antibody, by the end of 2025.
Liver-targeted Genetic Disease Franchise

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Beam expects to report initial data from multiple cohorts from the Phase 1/2 study of BEAM-302 in AATD in the first half of 2025.
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Patient dosing in the Phase 1/2 clinical trial of BEAM-301 in GSD1a is expected to commence in early 2025.
Fourth Quarter and Full Year 2024 Financial Results

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Cash Position: Cash, cash equivalents and marketable securities were $850.7 million as of December 31, 2024, compared to $1.2 billion as of December 31, 2023.
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Research & Development (R&D) Expenses: R&D expenses were $101.4 million for the fourth quarter of 2024 and $367.6 million for the full year ended December 31, 2024, compared to $140.1 million for the fourth quarter of 2023 and $437.4 million for the full year ended December 31, 2023.
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General & Administrative (G&A) Expenses: G&A expenses were $28.7 million for the fourth quarter of 2024 and $111.5 million for the full year ended December 31, 2024, compared to $43.3 million for the fourth quarter of 2023 and $116.8 million for the full year ended December 31, 2023.
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Net Income (Loss): Net loss attributable to common stockholders was $90.4 million, or $1.09 per share, for the fourth quarter of 2024 and $376.7 million, or $4.58 per share, for the year ended December 31, 2024, compared to net income attributable to common stockholders of $142.8 million, or $1.77 per basic share and $1.73 per diluted share, for the fourth quarter of 2023 and net loss attributable to common stockholders of $132.5 million, or $1.72 per share, for the year ended December 31, 2023.
Cash Runway

Beam expects that its cash, cash equivalents and marketable securities as of December 31, 2024, will enable the company to fund its anticipated operating expenses and capital expenditure requirements into 2027. This expectation includes funding directed toward reaching each of the key anticipated milestones for BEAM-101, ESCAPE, BEAM-301 and BEAM-302 described above.

On February 25, 2025 LIXTE Biotechnology Holdings, Inc. ("LIXTE" or the "Company") (Nasdaq: LIXT and LIXTW), a clinical stage pharmaceutical company, reported it has added the Robert H. Lurie Comprehensive Cancer Center (Lurie Cancer Center) of Northwestern University as a second site in a clinical trial combining the Company’s proprietary compound LB-100 with GSK’s Dostarlimab to treat ovarian clear cell cancer (Press release, Lixte Biotechnology, FEB 25, 2025, View Source [SID1234650537]).

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Emily M. Hinchcliff, MD, MPH, will lead the clinical trial at Lurie Cancer Center, a renown Chicago-based National Cancer Institute-designated Comprehensive Cancer Center, which is located at Northwestern Memorial Hospital’s downtown medical campus. Patient recruitment is underway, and the first patient has been dosed.

"Clinical trials testing potentially effective therapies are essential to move our field forward, with many recent great successes," said Dr. Hinchcliff. "We are pleased to be participating in this important clinical trial to assess whether adding LIXTE’s LB-100 to GSK’s Dostarlimab will enhance the effectiveness of immunotherapy in the treatment of ovarian clear cell carcinoma, a disease of high unmet need."

Bas van der Baan, LIXTE’s Chief Executive Officer, said, "The addition of Lurie Cancer Center is a positive step in expanding the patient population and accelerating this clinical trial, which was initiated in January 2024 at The University of Texas MD Anderson Cancer Center. The trial is directed by lead clinical investigator Amir Jazaeri, MD, Professor of Gynecologic Oncology."

On February 25, 2025 Novita Pharmaceuticals, Inc. ("Novita"), a privately held, clinical-stage pharmaceutical company dedicated to developing novel cancer drugs through its proprietary fascin inhibitor technology, reported additional results from its Phase 2 study (NCT05023486) evaluating NP-G2-044 in combination with SOC anti-PD-1 therapy in patients with advanced solid tumors resistant to prior anti-PD-1 therapy at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Immuno-oncology (AACR IO) Annual Meeting (Press release, Novita Pharmaceuticals, FEB 25, 2025, View Source [SID1234650555]). The data was presented in a poster presentation titled "Phase 2 Study of NP-G2-044, a Novel Fascin Inhibitor, in Combination with Anti-PD-1 Therapy in Patients with Solid Tumors Resistant to Prior Anti-PD-1 Therapy." Findings indicate that NP-G2-044 provides a novel therapeutic opportunity when combined with ICIs.

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"We are very pleased with the findings generated to date in our Phase 2 trial of NP-G2-044 both as a monotherapy and in combination with anti-PD-1 immune checkpoint inhibitors (ICIs) in ICI-resistant patients with advanced and metastatic solid tumors," said Jillian Zhang, Ph.D., President & Chief Scientific Officer of Novita. "The strong safety and durable efficacy we have observed with our first-in-class fascin inhibitor further support the simultaneous inhibition of metastasis and enhancement of cancer immunotherapy as a promising and innovative approach in cancer treatment with broad applications for many solid tumors. We look forward to sharing additional data from the Phase 2 expansion cohort of NP-G2-044 in combination with ICI in the second half of 2025."

Among the 45 patients treated with NP-G2-044 as of the last data cutoff (Oct. 2024), 80% had progressed on prior anti-PD-(L)1 therapies. The anti-PD-1 Combination RP2D for NP-G2-044 was 1600 mg QD with 4-week cycles. The primary endpoint was objective response rate (ORR), and secondary endpoints included progression-free survival (PFS), metastasis-free interval (MFI), overall survival (OS), safety, and tolerability.

Key highlights include:

A Disease Control Rate of 76% (includes patients with Stable Disease and Objective Responses)
An ORR of 21% (95% CL 9-38.9%) including 4 patients with Partial Response (PR) and 3 patients with Complete Responses (CR) including Pathologic Complete Response
Results indicate durable responses and tumor control in a significant proportion of patients across at least seven cancer types, including cases converted from ICI-non-responsive to ICI-responsive.
Long lasting objective responses have been observed.
Notable outcomes include a CR in a cervical cancer patient, target lesion CR in an endometrial cancer patient, pathological CRs in a pancreatic cancer patient and a patient with gastroesophageal junction adenocarcinoma and PRs in cutaneous squamous cell carcinoma, non-small cell lung cancer, and cholangiocarcinoma.
Seven patients are still on treatments, with the longest duration of 18+ months in an endometrial cancer patient and a patient with pancreatic cancer.
An amendment to the study is underway to open additional cohorts. These new cohorts aim to further evaluate the combination of NP-G2-044 with anti-PD-1 therapy across patient populations and solid tumor subtypes, providing a broader understanding of its therapeutic potential. Future analysis will also explore biomarkers for response prediction and mechanisms of resistance, guiding personalized approaches in treatment-resistant cancer. Novita is on track to begin enrollment in its pivotal Phase 3 study of NP-G2-044 + PLD in platinum resistant ovarian cancer in the third quarter of 2025.

On February 25, 2025 BeiGene, Ltd. (NASDAQ: ONC; HKEX: 06160; SSE: 688235), a global oncology company that intends to change its name to BeOne Medicines Ltd., reported that the Company will participate in fireside chats at two upcoming investor conferences (Press release, BeiGene, FEB 25, 2025, View Source [SID1234650518]):

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TD Cowen 45th Annual Health Care Conference on Monday, March 3, 2025 at 9:10 am ET; and
Leerink Partners Global Biopharma Conference on Monday, March 10, 2025 at 1:00 pm ET
Live webcasts of these events can be accessed from the investors section of BeiGene’s website at View Source, View Source, View Source Archived replays will be available for 90 days following the events.

On February 25, 2025 Merck (NYSE: MRK), known as MSD outside of the United States and Canada, reported the U.S. Food and Drug Administration (FDA) has accepted for priority review a new supplemental Biologics License Application (sBLA) seeking approval for KEYTRUDA (pembrolizumab), Merck’s anti-PD-1 therapy, for the treatment of patients with resectable locally advanced head and neck squamous cell carcinoma (LA-HNSCC) as neoadjuvant treatment, then continued as adjuvant treatment in combination with standard of care radiotherapy with or without cisplatin and then as a single agent (Press release, Merck & Co, FEB 25, 2025, View Source [SID1234650538]). The FDA has set a Prescription Drug User Fee Act (PDUFA), or target action, date of June 23, 2025.

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The sBLA is based on data from the Phase 3 KEYNOTE-689 trial. Results from a pre-specified first interim analysis, which will be presented at an upcoming medical meeting, showed that in patients with resectable LA-HNSCC, the KEYTRUDA perioperative treatment regimen demonstrated a statistically significant and clinically meaningful improvement in event-free survival (EFS) compared to adjuvant radiotherapy (with or without cisplatin) alone. The study also showed a statistically significant improvement in major pathological response (mPR), a key secondary endpoint, for patients in the KEYTRUDA arm compared with adjuvant radiotherapy alone. The safety profile of KEYTRUDA was consistent with that observed in previously reported studies; no new safety signals were identified.

"The standard of care for patients with resectable locally advanced head and neck squamous cell carcinoma has remained the same for over two decades, representing a significant unmet need for new treatment options," said Dr. Marjorie Green, senior vice president and head of oncology, global clinical development, Merck Research Laboratories. "Based on the compelling results of the KEYNOTE-689 trial, we hope to reduce the risk of recurrence and disease progression in earlier stages of disease. We look forward to working with the FDA to potentially bring KEYTRUDA to these patients as soon as possible."

This review is being conducted under Project Orbis, an initiative of the FDA Oncology Center of Excellence that provides a framework for coordinated submission and review of oncology drugs among its international partners. Health authorities in Israel, Canada, Australia, Singapore, Brazil and Switzerland will review this application as part of Project Orbis.

KEYTRUDA is currently approved as monotherapy and in combination regimens for appropriate patients with metastatic or with unresectable, recurrent HNSCC in the U.S., Europe, China, Japan and other countries around the world. For more information, please see the "Selected KEYTRUDA (pembrolizumab) Indications in the U.S." section below.

About KEYNOTE-689

KEYNOTE-689 is a randomized, active-controlled, open-label Phase 3 trial (ClinicalTrials.gov, NCT03765918) evaluating KEYTRUDA as neoadjuvant treatment and KEYTRUDA in combination with standard of care radiotherapy (with or without cisplatin) as adjuvant treatment in treatment-naïve patients with newly diagnosed, stage III or IVA resectable, locally advanced, head and neck squamous cell carcinoma. Efficacy outcomes are classified by programmed cell death ligand 1 (PD-L1) combined positive score (CPS) status. The primary endpoint is EFS, and key secondary endpoints include overall survival, major pathological response, pathological complete response and safety. The study enrolled an estimated 704 patients who were randomized (1:1) to receive either:

KEYTRUDA (200 mg intravenously [IV] every three weeks [Q3W] for two cycles) as neoadjuvant therapy prior to surgery, followed by either KEYTRUDA (200 mg IV Q3W for 15 cycles) plus standard of care radiotherapy with cisplatin (100 mg/m2 IV Q3W for three cycles) as adjuvant therapy following surgery for high-risk patients or KEYTRUDA (200 mg IV Q3W for 15 cycles) plus standard of care radiotherapy without cisplatin as adjuvant therapy following surgery for low-risk patients; or
No neoadjuvant therapy prior to surgery, followed by either standard of care radiotherapy with cisplatin (100 mg/m2 IV Q3W for three cycles) as adjuvant therapy following surgery for high-risk patients or standard of care radiotherapy without cisplatin as adjuvant therapy following surgery for low-risk patients.
About head and neck cancer

Head and neck cancer describes a number of different tumors that develop in or around the throat, larynx, nose, sinuses and mouth. Most head and neck cancers are squamous cell carcinomas that begin in the flat, squamous cells that make up the thin surface layer of the structures in the head and neck. Locally advanced head and neck squamous cell carcinoma (LA-HNSCC) is cancer that has grown outside the original location, but has not yet spread to distant parts of the body. There are several factors that greatly increase the risk of developing head and neck cancer, including tobacco and alcohol use and human papillomavirus (HPV). It is estimated there were more than 891,500 new cases of head and neck cancer diagnosed and over 458,100 deaths from the disease in 2022 globally. In the U.S., it is estimated there will be more than 58,450 new cases of head and neck cancer diagnosed and more than 12,230 deaths from the disease in 2024.