On March 12, 2024 Black Diamond Therapeutics, Inc. (Nasdaq: BDTX), a clinical-stage oncology company focused on the development of MasterKey therapies to treat patients with genetically defined tumors, reported financial results for the fourth quarter and full year ended December 31, 2023, and provided a corporate update (Press release, Black Diamond Therapeutics, MAR 12, 2024, View Source [SID1234641060]).

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"The year 2023 was exciting, with generation of clinical data that positions BDTX-1535 as a potential first and best in class 4th generation EGFR TKI, and we recently began dosing first-line patients in a Phase 2 trial in NSCLC with non-classical mutations," said Mark Velleca, M.D., Ph.D., President and Chief Executive Officer of Black Diamond Therapeutics. "This year will be data rich across our pipeline, with Phase 2 data for BDTX-1535 in NSCLC, additional GBM results, and initial Phase 1 data for BDTX-4933 in non-G12C KRAS mutated NSCLC."

Recent Developments & Upcoming Milestones:

BDTX-1535:

In October 2023, Black Diamond presented a poster with updated clinical data at the European Organization for Research and Treatment of Cancer-National Cancer Institute-American Association for Cancer Research (EORTC-NCI-AACR) (Free EORTC-NCI-AACR Whitepaper) Symposium on Molecular Targets and Cancer Therapeutics from the dose escalation portion of the Phase 1 clinical trial of BDTX-1535 in patients with non-small cell lung cancer (NSCLC). The presentation included clinical data from 27 patients with advanced/metastatic NSCLC who received once daily doses ranging from 25mg to 400mg. These results demonstrated a favorable tolerability profile and durable responses in patients with NSCLC expressing both acquired resistance C797S and non-classical driver epidermal growth factor receptor (EGFR) mutations.
Following receipt of End of Phase 1 feedback from the U.S. Food and Drug Administration (FDA) in the fourth quarter of 2023, Black Diamond initiated a Phase 2 cohort in first-line (1L) patients with non-classical EGFR mutations in early 2024. (NCT05256290)
Phase 0/1 "window of opportunity" clinical trial of BDTX-1535 began enrollment in October 2023 to evaluate the pharmacokinetic, pharmacodynamic, and clinical response in patients with recurrent high-grade glioma (HGG) with EGFR alterations and/or fusions who are undergoing a planned surgical resection. The trial is sponsored by the Ivy Brain Tumor Center in Phoenix, Arizona. (NCT06072586)
Top-line results disclosed in December 2023 from the Phase 1 dose escalation trial of BDTX-1535 in patients with relapsed/recurrent glioblastoma (GBM) showed clinical activity in heavily pretreated patients. BDTX-1535 was shown to be generally well tolerated, with no new safety signals observed.
Black Diamond anticipates the following upcoming key milestones for BDTX-1535:
Oral presentation describing real world data of the evolving EGFR mutation landscape in patients with NSCLC, and the MasterKey profile of BDTX-1535 addressing a broad spectrum of mutations at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) annual meeting on April 7, 2024.
Phase 2 clinical data in patients with NSCLC and non-classical driver or acquired resistance EGFR mutations in the third quarter of 2024.
Phase 1 dose escalation data in patients with relapsed/recurrent GBM, and initial results from the investigator sponsored "window of opportunity" trial in patients with recurrent HGG are expected to be presented at a medical meeting in the second quarter of 2024.
BDTX-4933:

In October 2023, Black Diamond presented a poster at the EORTC-NCI-AACR (Free EORTC-NCI-AACR Whitepaper) Symposium on Molecular Targets and Cancer Therapeutics detailing preclinical data for the clinical-stage MasterKey RAF inhibitor BDTX-4933, a brain-penetrant oral inhibitor of oncogenic alterations in KRAS, NRAS and BRAF. Preclinical results showed that BDTX-4933 potently and selectively inhibited the proliferation of tumor cells expressing a range of KRAS, NRAS and BRAF mutations, suggesting clear differentiation compared to other RAF inhibitors.
A Phase 1 clinical trial for BDTX-4933 was initiated in the second quarter of 2023 in patients with BRAF and select RAS/MAPK mutation-positive cancers, with an emphasis on patients with KRAS mutant NSCLC. The trial is currently in dose escalation with an update anticipated in the fourth quarter of 2024. (NCT05786924)
Financial Highlights

Cash Position: Black Diamond ended 2023 with approximately $131.4 million in cash, cash equivalents, and investments compared to $122.8 million as of December 31, 2022. Net cash used in operations was $66.7 million for the year ended December 31, 2023 compared to $85.1 million for the year ended December 31, 2022.
Research and Development Expenses: Research and development (R&D) expenses were $15.3 million for the fourth quarter of 2023, compared to $14.6 million for the same period in 2022. Research and development expenses were $59.4 million for the year ended December 31, 2023, compared to $64.4 million for the year ended December 31, 2022. The decrease in R&D expenses was primarily due to reduced spending on early discovery projects as we deepen our focus on our clinical-stage assets.
General and Administrative Expenses: General and administrative (G&A) expenses were $5.6 million for the fourth quarter of 2023, compared to $7.2 million for the same period in 2022, and $27.1 million for the year ended December 31, 2023, compared to $28.4 million for the year ended December 31, 2022. The decrease in G&A expenses was primarily due to a decrease in legal and other professional fees.
Net Loss: Net loss for the fourth quarter of 2023 was $19.4 million, as compared to $21.1 million for the same period in 2022. Net loss for the year ended December 31, 2023 was $82.4 million compared to $91.2 million for the year ended December 31, 2022.
Financial Guidance

Black Diamond ended 2023 with approximately $131.4 million in cash, cash equivalents and investments which the Company believes is sufficient to fund its anticipated operating expenses and capital expenditure requirements into the second quarter of 2025.

On March 12, 2024 Aligos Therapeutics, Inc. (Nasdaq: ALGS, "Aligos"), a clinical stage biopharmaceutical company focused on developing novel therapeutics to address unmet medical needs in liver and viral diseases, reported recent business progress and financial results for the fourth quarter and full year 2023 (Press release, Aligos Therapeutics, MAR 12, 2024, View Source [SID1234641059]).

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"Last year we built the foundation for a critical 2024 by closing a $92 million PIPE from healthcare-dedicated institutional investors and successfully completing our Phase 2a enabling activities for ALG-055009," stated Lawrence Blatt, Ph.D., MBA, Chairman & CEO of Aligos Therapeutics. "In addition, we presented unprecedented antiviral activity from our ALG-000184 program for chronic hepatitis B (CHB). With these important accomplishments in hand, we are well positioned to deliver important planned milestones in 2024, which include releasing topline data in subjects with metabolic dysfunction-associated steatohepatitis (MASH) in the ALG-055009 Phase 2a study (HERALD) and completing Phase 2 enabling activities for ALG-000184 by the end of the year. We look forward to sharing our achievements with our stakeholders throughout this year."

Recent Business Progress

Aligos Portfolio of Drug Candidates

ALG-055009: Potential best-in-class small molecule THR-β agonist for MASH

Phase 2a enabling activities, including completion of supporting nonclinical/Phase 1 studies and manufacturing of drug supply, were completed
The Phase 2a HERALD protocol was filed with the FDA in Q4 2023
First subject dosed expected in Q2 2024 and topline HERALD data anticipated in Q4 2024
ALG-000184: Potential best-in-class small molecule CAM-E for CHB

Dosing continues in the ongoing Phase 1a/1b ALG-000184-201 study, with multiple subjects now having been dosed for >1 year (total planned dosing duration is 96 weeks). Interim data readouts are planned to be presented this year at the following conferences: Asian Pacific Association for the Study of the Liver (APASL), European Association for the Study of the Liver (EASL), and American Association for the Study of Liver Diseases (AASLD)
Phase 2 enabling activities, including regulatory interactions and drug supply manufacturing, are underway
ALG-097558: Potential best-in-class small molecule pan-coronavirus protease inhibitor

The ongoing Phase 1 first in human study is nearing completion of dosing (Q2), with topline data to be presented at the European Congress of Clinical Microbiology and Infectious Diseases (ECCMID) Annual Meeting, occurring in April 2024
Phase 2 enabling activities are underway with financial support from the National Institute of Allergy and Infectious Diseases (NIAID)
Financial Results for the Fourth Quarter and Full Year 2023

Cash, cash equivalents and investments totaled $135.7 million as of December 31, 2023, compared with $125.8 million as of December 31, 2022. Additionally, in October 2023, we raised $92.1 million in gross proceeds in a private placement financing, before deducting placement agent’s fees and other expenses. Including the proceeds from the private placement financing, we continue to believe our cash balance provides sufficient cash to fund planned operations through the end of 2025.

Net losses for the three months ended December 31, 2023 were $27.9 million or basic and diluted net loss per common share of $(0.22), compared to net losses of $21.9 million or basic and diluted net loss per common share of $(0.51) for the three months ended December 31, 2022.

Net losses for the year ended December 31, 2023 were $87.7 million or basic and diluted net loss per common share of $(1.36), compared to net losses of $96.0 million or basic and diluted net loss per common share of $(2.25) for the year ended December 31, 2022.

Research and development (R&D) expenses for the three months ended December 31, 2023 were $22.3 million, compared with $19.1 million for the same period of 2022. The increase was primarily due third party expenses due to the milestone payments made as a result of dosing the first patient in a clinical trial. Total R&D stock-based compensation expense incurred for the three months ended December 31, 2023 was $1.5 million, compared with $1.9 million for the same period of 2022.

R&D expenses for the year ended December 31, 2023, were $73.0 million, compared with $85.1 million for the same period of 2022. The decrease was primarily due to a decrease in third-party expenses mainly related to our discontinued STOPs and ASO programs in 2022, partially offset by increases in our ongoing activities and related expenditures associated with our CAM clinical trial activities and MASH program, as well as the milestone payment to Katholieke Universiteit Leuven (KU Leuven) under its collaboration agreement related to the coronaviruses and the dosing of the first patient in a Phase 1 clinical trial. Total R&D stock-based compensation expense incurred for the year ended December 31, 2023 was $6.8 million, compared with $8.0 million for the same period of 2022.

General and administrative (G&A) expenses for the three months ended December 31, 2023 were $6.4 million, compared with $7.1 million for the same period of 2022. The decrease in G&A expenses for this comparative period is primarily due to a decrease in facility costs. Total G&A stock-based compensation expense incurred for the three months ended December 31, 2023 was $1.1 million, compared with $1.6 million for the same period of 2022.

G&A expenses for the year ended December 31, 2023, were $30.6 million, compared with $26.4 million for the same period of 2022. This was due to an increase in third-party expenses primarily due to higher legal and patent attorney costs, partially offset by a decrease in facility expenses. Total G&A stock-based compensation expense incurred for the year ended December 31, 2023 was $5.8 million, compared with $6.7 million for the same period of 2022.

On March 12, 2024 Ratio Therapeutics Inc. (Ratio), an emerging pharmaceutical company employing innovative technologies to develop best-in-class radiopharmaceuticals for cancer treatment and monitoring, reported an expanded manufacturing agreement with PharmaLogic, a world-class contract development and manufacturing organization (CDMO) specializing in radiopharmaceutical production and distribution (Press release, Ratio Therapeutics, MAR 12, 2024, View Source [SID1234641054]). The collaboration aims to significantly accelerate the development and commercialization of Ratio’s pipeline of next-generation radiotherapies, representing a critical component in advancing Ratio’s fibroblast activation protein-alpha (FAP)-targeted radiotherapeutic candidate.

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"We are excited to partner with PharmaLogic, an industry-leading CDMO with over 15 years of experience in radiopharmaceutical manufacturing and supply chain solutions" said Dr. Matthias Friebe, Chief Technology Officer of Ratio. "With advanced facilities, substantial capacity, and an impressive track record of quality and compliance, PharmaLogic has demonstrated their ability to reliably produce highly complex radiopharmaceutical products. Their expertise in sophisticated technologies such as accelerated radiochemistry and automation will ensure efficient, scalable production of our novel pipeline therapies to meet future commercial demands. This collaboration represents a major step towards Ratio Therapeutics’ mission of discovering and rapidly transitioning innovative radiopharmaceuticals to the clinic, aiming to transform treatment paradigms and improve patient outcomes."

"We are honored to collaborate with Ratio Therapeutics and support their pipeline of precision-targeted radiotherapies," said D. Scott Holbrook, Chief Strategy Officer and General Manager at PharmaLogic. "Ratio is at the forefront of developing radiopharmaceutical solutions that have the potential to significantly improve patient outcomes. By leveraging our combined expertise and manufacturing capabilities, we aim to make these cutting-edge therapies readily available to patients in need."

Ratio, in collaboration with Lantheus and PharmaLogic, is currently conducting a Phase I trial evaluating the efficacy of a novel FAP-targeted imaging biomarker, copper-64[Cu-64]-labeled LNTH-1363S (formerly RTX-1363S) for PET imaging in adult healthy volunteers. The company also recently announced a $50M Series B financing which will significantly drive the clinical advancement of Ratio’s first FAP-targeted therapeutic candidate into clinical trials this year.

On March 11, 2024 ImmuneOnco Biopharmaceuticals (Shanghai) Co., Ltd. reported that the Phase Ib/II clinical study of the company developed bispecific antibody-receptor recombinant protein drug targeting both CD47 and CD20 (Project No.: IMM0306) in combination with lenalidomide (Study No.: IMM0306-003) for the treatment of advanced relapsed/refractory follicular lymphoma (FL) was discussed at the SRC meeting and the Phase II recommended dose was determined, So the project entered Phase II clinical trial officially (Press release, ImmuneOnco Biopharma, MAR 11, 2024, View Source [SID1234655697]). This is another milestone achievement in the rapid development of ImmuneOnco.

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Previously, IMM0306 obtained permission from China’s NMPA and the US FDA to conduct clinical trials, and obtained patent authorization in China, the United States, Japan and Europe, consolidating ImmuneOnco’s leading position in the research echelon of CD47 target drug development and bispecific antibodies. Recently, the Safety Review Committee (SRC) unanimously approved 1.6mg/kg QW as the RP2D dose of IMM0306 in combination with lenalidomide for the treatment of advanced relapsed/refractory follicular lymphoma (FL) to enter Phase II for further development. In the phase I clinical study in relapsed or refractory B-cell lymphoma, IMM0306 monotherapy showed encouraging efficacy and a favorable safety profile, with no DLTs observed in any of the eight dose groups. Five cases of CR and five cases of PR were observed in four dose groups, starting from 0.8 mg/kg to 2.0 mg/kg, and the ORR of relapsed and refractory FL reached 41%. At the same time, all patients did not have significant cytokine storm toxicity.

Dr. Tian, Wenzhi, founder and chairman of ImmuneOnco, said:

"We are very pleased to see that our IMM0306 in combination with lenalidomide in patients with advanced relapsed/refractory follicular lymphoma (FL) has determined the recommended Phase II dose. Data from the Phase I clinical study showed a favorable safety and clinical efficacy of monotherapy, especially in the combination escalation study of lenalidomide, in which 11 patients with FL/MZL who failed at least one prior line of therapy were enrolled, and ORR of 75% was observed (2 patients with CR and 4 patients with PR). We firmly believe that the development of second-line follicular lymphoma (FL) and diffuse large B-cell lymphoma (DLBCL) will be extremely competitive in the market. We will continue to promote the research of IMM0306 projects and strive to bring good news to cancer patients as soon as possible. "

Dr. Lu, Qiying, Chief Medical Officer/Senior Vice President of ImmuneOnco, said:

"Today, it is important for our company to determine the recommended dose in a Phase II clinical study of IMM0306, one of our main products, in combination with lenalidomide in patients with advanced relapsed/refractory follicular lymphoma (FL). At present, outstanding efficacy has been observed in multiple indications (including indolent lymphoma dominated by FL and MZL and DLBCL, the most common aggressive lymphoma in clinical practice), indicating that the product has broad clinical development prospects. IMM0306 Phase I data was postered at ASCO (Free ASCO Whitepaper) 2023 conference attracted the attention of many global researchers. At present, we have carried out a comprehensive layout of this product, not just a single-agent clinical development project, but also a combined LEN project to fully develop NHL indications, not only for FL, MZL, DLBCL and other indications. In the current combination escalation study with lenalidomide, in FL/MZL patients who have failed at least first-line therapy, 75% ORR was observed (2 CR patients, 4 PR patients), and the company will accelerate the clinical development of this product. We look forward to bringing new treatment options to cancer patients."

On March 11, 2024 GenScript reported its 2023 Annual Results Presentation (Presentation, GenScript, MAR 11, 2024, https://www.genscript.com/gsfiles/IPO/2023%20Annual%20Results.pdf?1153641870 [SID1234644733]).

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