On March 31, 2025 CASI Pharmaceuticals, Inc. (NASDAQ:CASI), ("CASI" or the "Company"), a Cayman incorporated biopharmaceutical company focused on developing and commercializing innovative therapeutics and pharmaceutical products, reported business and financial results for the fourth quarter ended December 31, 2024, and provided an update on key highlights for 2024 (Press release, CASI Pharmaceuticals, MAR 31, 2025, https://feeds.issuerdirect.com/news-release.html?newsid=5642877492672387&symbol=CASI [SID1234652235]).

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Wei-Wu He, Ph.D., CASI’s Chairman and Chief Executive Officer, commented, "2024 was a transformative year for CASI as we strategically pivoted our company toward the development of CID-103 for organ transplant rejection and autoimmune disease. CID-103 is an anti-CD38 antibody with the potential to be a best-in-class treatment for a myriad of autoimmune diseases as well as antibody mediated rejection (AMR). We are encouraged by our clinical progress, achieving a milestone with the first Immune Thrombocytopenia (ITP) patient dosed in January 2025. Simultaneously, we are diligently working towards resolution of the FDA renal allograft AMR clinical hold."

Dr. He continued, "As we advance into 2025, we remain firmly committed to progressing CID-103 at an accelerated pace. We expect to reach multiple important milestones this year. Our focused approach and prudent capital allocation position CASI to deliver sustainable long-term value creation for both patients and shareholders."

Key Business Highlights

CASI reported the fourth quarter revenue of $13.4 million and full-year revenue of $28.5 million.

Received a proposal with respect to the acquisition of CASI’s China business from Dr. Wei-Wu He on June 21, 2024.

Completed a $15.0 million private placement financing with leading healthcare investors including Venrock Healthcare Capital Partners, Foresite Capital, Panacea Venture and Dr. Wei-Wu He, on July 15, 2024.

Pipeline and Program Updates

Received FDA IND clearance of ITP on May 15, 2024.

Received CTA approval from China’s NMPA on ITP on October 24, 2024.

First patient dosed in Phase 1/2 clinical trial in ITP on January 3, 2025. Patient enrollment and treatment continues.

Upcoming Milestones

Anticipate FDA feedback on clinical hold for AMR in Q2, 2025.

Target IND submission for Aplastic Anemia in Q2, 2025.

Report the interim data from the ongoing ITP phase 1 study in mid-2025.

Anticipate finalizing the equity transfer agreement regarding Precision Autoimmune Therapeutics ("PAT") in Q2 2025. Upon completion of this transaction, CASI will have the sole global ownership of all indications in CID-103.

Product and Pipeline Highlights

CID-103 (Anti-CD38 antibody)

CID 103 is a full human IgG1 anti-CD38 monoclonal antibody recognizing a unique epitope that has demonstrated an encouraging preclinical efficacy and safety profile compared to other anti-CD38 monoclonal antibodies, and which we have exclusive global rights. CID 103 is being developed for the treatment of patients with chronic Immune Thrombocytopenia (ITP), renal allograft antibody mediated rejection (AMR). In May 2024, we announced the clearance of IND application with the US FDA for the initiation of a phase 1/2 study of CID-103 in adults with ITP. In October 2024, the Center for Drug Evaluation (CDE) approved the Clinical Trial Application (CTA) for a phase 1/2 study of CID-103 in patients with chronic ITP in China. The Chinese ITP study is part of the global development program which has been approved by the US FDA in May 2024. In January 2025, CASI announced the first patient was enrolled and dosed in the ITP clinical study. Furthermore, we are making steady progress towards resolution of the FDA clinical hold.

EVOMELA (melphalan for injection)

On December 3, 2018, CASI received the NMPA approval for importation, marketing and sales in China for EVOMELA , and on August 12, 2019, CASI announced the commercial launch of EVOMELA in China. Prior to EVOMELA’s entry into the Chinese market, an average of 800 stem cell transplants per year were conducted in the multiple myeloma (MM) treatment setting. Following EVOMELA’s launch in August of 2019, CASI worked closely with KOLs to improve market awareness and expedite adoption in the Chinese market. In 2023, nearly 10,000 patients were treated with EVOMELA. In 2024, the launch of an undifferentiated generic formulation of melphalan for injection product by a Chinese domestic manufacture has presented challenges, resulting in a decline of EVOMELA sales.

FOLOTYN (Pralatrexate)

On July 31, 2023, CASI entered into a tripartite assignment agreement with Mundipharma International Corporation Limited ("MICL"), Mundipharma Medical Company (MMCo), and Acrotech Biopharma Inc. (Acrotech) for the commercialization of FOLOTYN (Pralatrexate) in China. FOLOTYN (Pralatrexate) is a dihydrofolate reductase inhibitor indicated for the treatment of patients with relapsed or refractory peripheral T-cell lymphoma (PTCL). This product was approved by both the FDA and China’s NMPA for PTCL. CASI announced the first patient was dosed with FOLOTYN in China on February 15, 2024.

Fourth Quarter and Full-Year 2024 Financial Results

Revenue for the fourth quarter of 2024 was $13.4 million, representing a 94% increase compared to $6.9 million in the same period last year. The quarterly growth reflects a successful execution of commercial strategy implemented in the second half of the year. Full-year revenue was $28.5 million, compared to $33.9 million in 2023, a decrease of 16%. The annual performance was impacted by intensified competition following the market entry of domestically produced injectable melphalan, which created pricing pressure throughout the year.

R&D expenses for the fourth quarter was $3.7 million, a 61% increase from $2.3 million in the same period last year. The increase is primarily for the development of CID-103, supporting the company’s focused pivot toward opportunities in organ transplant rejection and autoimmune indications. Full-year R&D expenses was $8.9 million, compared to $9.9 million in 2023, representing a decrease of 10%. The reduction primarily resulted from lower amortization expenses following the decision to write off generic portfolio at the end of 2023.

G&A expenses for the fourth quarter of 2024 was $7.1 million, compared to $6.4 million in the same period in 2023, a 11% increase. This quarter-over-quarter increase is primarily attributable to legal expenses associated with ongoing arbitration proceedings with Juventas. Full-year G&A expenses was $23.6 million, compared to $25.4 million in 2023, representing a decrease of 7%. This year-over-year improvement demonstrates the effectiveness of our operational efficiency initiatives and cost control measures, reinforcing our commitment to disciplined financial management.

Net loss for the year ended December 31, 2024 was $39.3 million compared to $26.3 million for the year ended December 31, 2023.

As of December 31, 2024, CASI had cash and cash equivalents of $13.5 million compared to cash and cash equivalents plus short term investments of $29.1 million as of December 31, 2023.

Further information regarding the Company, including its Annual Report on Form 20-F for the year ended December 31, 2024, can be found at www.casipharmaceuticals.com. The Company will provide a hard copy of its annual report containing the audited consolidated financial statements, free of charge, to its shareholders upon request. Requests should be directed to Investor Relations Department, CASI Pharmaceuticals, Inc., Rm 1701-1702, China Central Office Tower 1, No.81 Jianguo Road Chaoyang District, Beijing, 100025, China.

On March 31, 2025 Curis, Inc. (NASDAQ: CRIS), a biotechnology company focused on the development of emavusertib (CA-4948), an orally available, small molecule IRAK4 inhibitor, reported its business update and financial results for the quarter ended December 31, 2024 (Press release, Curis, MAR 31, 2025, View Source [SID1234651668]).

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Operational Highlights

Emavusertib (IRAK4 Inhibitor)

TakeAim Lymphoma

Curis successfully concluded meetings with both the U.S. Food and Drug Administration (FDA) and the Committee for Medicinal Products for Human Use of the European Medicines Agency (EMA) on the suitability of using the ongoing TakeAim Lymphoma study (NCT 03328078) to support a potential accelerated approval path in PCNSL.
Curis also announced that emavusertib has been granted Orphan Drug Designation for PCNSL in both the US and Europe.
Curis provided a data update for 27 patients enrolled in the ongoing TakeAim Lymphoma study in relapsed/refractory (R/R) PCNSL as of January 2, 2025 (data cutoff).
In 20 BTKi-experienced patients:
13 patients had change in tumor burden data available as of data cutoff;
9 of these 13 patients demonstrated a reduction in tumor burden, including 6 objective responses, 4 complete responses (CR) and 2 partial responses (PR), with 3 of 4 CRs lasting more than six months.
In 7 BTKi-naïve patients:
6 patients had change in tumor burden data available as of data cutoff;
5 of these 6 patients demonstrated a reduction in tumor burden, including 5 objective responses, 1 CR and 4 PRs.
TakeAim Leukemia

In December 2024, the Company announced data from the TakeAim Leukemia study (NCT 04278768) in R/R Acute Myeloid Leukemia (AML) at the 66th ASH (Free ASH Whitepaper) annual meeting for 21 patients with a FLT3 mutation who had received fewer than 3 lines of prior therapy and were treated with emavusertib as monotherapy at the Recommended Phase 2 Dose (RP2D) of 300 mg BID.
19 patients were response-evaluable:
10 of 19 patients achieved objective response, including 6 CRs, 2 CRs with incomplete hematological recovery or partial hematological recovery (CRi/CRh) and 2 morphologic leukemia-free state (MLFS);
7 of the 10 objective responses were reported at the first assessment.
2 patients were not response-evaluable, as they discontinued treatment prior to first disease assessment (death occurred at Day 8 and Day 13, respectively).
Ema-Ven-Aza Triplet Study in Frontline AML

The Company initiated a Phase 1 clinical study of emavusertib in combination with venetoclax and azacitidine (ema-ven-aza) in frontline AML (CA-4948-104, 2023-505828-58). The study is currently being conducted in Spain, Germany, and Italy to assess the safety and tolerability of different dosing regimens by adding emavusertib to a patient’s ven-aza regimen after they have achieved a CR on ven-aza but remain positive for minimal residual disease. The first dosing cohort was completed and well tolerated, with no unexpected adverse events. As a result, the external Clinical Safety Review Committee recommended escalation to the next dosing cohort. Enrollment for this cohort is currently ongoing.

Corporate

On March 28, 2025, Curis priced a registered direct offering of common stock and concurrent private placement of pre-funded warrants and warrants ("March 2025 Offerings") with gross proceeds of approximately $10.0 million. The offerings are expected to close concurrently on March 31, 2025, subject to the satisfaction of customary closing conditions.

In October 2024, Curis completed a registered direct offering and concurrent private placement with net proceeds of approximately $10.8 million.

"Curis had a very productive 2024. We started the year seeing early responses in our PCNSL trial. As more patients enrolled, and we continued to see positive data, we initiated discussions with the FDA and EMA to discuss the possibility of pursuing an accelerated approval path for emavusertib. We are pleased to announce today that we have received supportive feedback from both agencies. Over the next 12-18 months, we will be focused on enrolling 30-40 additional patients to support the regulatory filing for accelerated approval," said James Dentzer, Chief Executive Officer of Curis.

Additional details of the Company’s discussions with EMA and FDA and the March 2025 Offerings were reported on Form 8-K filed by the Company with the SEC on March 28, 2025.

Fourth Quarter 2024 Financial Results

For the year ended December 31, 2024, Curis reported a net loss of $43.4 million, or $6.88 per share on both a basic and diluted basis, as compared to a net loss of $47.4 million, or $8.96 per share on both a basic and diluted basis in 2023. For the fourth quarter of 2024, Curis reported a net loss of $9.6 million or $1.25 per share on both a basic and diluted basis as compared to a net loss of $11.7 million or $2.03 on both a basic and diluted basis for the same period in 2023.

Revenues, net were $10.9 million and $10.0 million for the years ended December 31, 2024 and 2023, respectively. Revenues are comprised of royalty revenues related to Genentech and Roche’s net sales of Erivedge. Revenues were $3.3 million and $2.7 million for the fourth quarters of 2024 and 2023, respectively.

Research and development expenses were $38.6 million and $39.5 million for the years ended December 31, 2024 and 2023, respectively. The decrease was primarily attributable to lower clinical and consulting costs, partially offset by higher manufacturing costs. Research and development expenses were $9.0 million and $10.0 million for the fourth quarters of 2024 and 2023, respectively.

General and administrative expenses were $16.8 million and $18.6 million for the years ended December 31, 2024 and 2023, respectively. The decrease was primarily attributable to lower consulting, legal, facility, insurance and employee-related costs. General and administrative expenses were $3.4 million and $4.9 million for the fourth quarters of 2024 and 2023, respectively.

Other income, net was $1.2 million and $0.9 million for the years ended December 31, 2024 and 2023, respectively. The increase was attributable to a decrease in expense related to the sale of future royalties partially offset by a decrease in interest income. Other expense, net was $0.6 million for the fourth quarter of 2024 and other income, net was $0.5 million for the fourth quarter of 2023.

As of December 31, 2024, Curis’s cash and cash equivalents totaled $20.0 million, and the Company had approximately 8.5 million shares of common stock outstanding.

Cash Runway Guidance

Curis expects its cash and cash equivalents as of December 31, 2024, together with the expected proceeds from the March 2025 Offerings will enable the Company to fund its planned operations into the fourth quarter of 2025.

Conference Call Information

Curis management will host a conference call today, March 31, 2025, at 8:30 a.m. ET, to discuss the business update and these financial results.

To access the live conference call, please dial (800)-836-8184 from the United States or (646)-357-8785 from other locations, shortly before 8:30 a.m. ET. The conference call can also be accessed here on the Curis website in the Investors section.

On March 31, 2025 Oncotelic Therapeutics, Inc. (OTCQB:OTLC) ("Oncotelic," the "Company," or "We"), a leader in RNA-based therapeutics, reported the publication of its latest research paper, titled, "Positive Prognostic Overall Survival Impacts of Methylated TGFB2 and MGMT in Adult Glioblastoma Patients (Press release, Oncotelic, MAR 31, 2025, View Source [SID1234651690])." The paper, authored by Sanjive Qazi, Michael Potts, Scott Myers, Stephen Richardson, and Vuong Trieu, is available online at: View Source

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To facilitate deeper exploration and discussion of this research by the research community, Oncotelic is introducing its proprietary communication platform powered by PDAOAI. PDAOAI enables users to query this paper and dozens of referenced articles through a single interactive interface. Scientists and clinicians are invited to engage at: View Source

A Simple Summary

Glioblastoma (GBM) is one of the most aggressive brain tumors in adults. It is well established that methylation of the O-6-methylguanine-DNA methyltransferase (MGMT) gene is predictive of overall survival (OS) benefits in patients receiving standard temozolomide and radiotherapy. Transforming growth factor beta (TGFB) is a family of cytokines involved in vital cellular processes and the regulation of growth factors.

The study’s novel discovery demonstrates that high TGFB2 gene methylation correlates with an improved OS risk, surpassing the predictive value of MGMT and TGFB1 methylation when controlling for age and sex. Several genes and pathways linked to TGFB2 methylation, including immune mechanisms such as T-cell activation, antigen processing, and Toll-like receptor pathways, were identified as improving survival outcomes in GBM patients. Of note, mucosa-associated lymphoid tissue lymphoma translocation protein, also referred to as MALT1, mRNA negatively impacted survival rates, suggesting a potential avenue for targeted therapies.

"The complexity of the publication was simplified into a concise statement by our PDAOAI platform, demonstrating the power of this platform for scientific communication: The findings underscore the importance of TGFB2 methylation as a prognostic marker in GBM treatment. High levels of TGFB2 methylation are associated with improved overall survival, particularly in young adult males. This suggests that TGFB2 methylation could be a valuable biomarker for risk stratification and therapeutic targeting in GBM, potentially guiding treatment decisions and improving patient outcomes." – Dr. Vuong Trieu, CEO of Oncotelic and co-author of the study.

"Our findings present an actionable opportunity to improve GBM patient outcomes by integrating sophisticated predictive analytical platforms and tools with clinical data. By uncovering insightful methylation patterns and elucidating gene-expression profiles at the biochemical pathway level, we expand our capacity to identify potential therapeutic targets. This approach supports the development of tailored treatment strategies through our nano-technology drug delivery platform. Ultimately, these insights enhance innovation in targeted therapies, driving improved clinical efficacy and patient survival rates in this devastating disease." – Dr. Sanjive Qazi, lead researcher

"This is the first paper we have published that utilized our proprietary AI technology. We are excited to see our technology being applied in real-world scenarios, and we look forward to the advancements it can potentially bring in the future." – Scott Myers, Product Manager

On March 31, 2025 Exact Sciences Corp. (Nasdaq: EXAS), a leading provider of cancer screening and diagnostic tests, reported the launch of Cologuard Plus, the most accurate noninvasive colorectal cancer screening (CRC) test reported in studies to date.* FDA-approved for average-risk patients 45+ and covered by Medicare, the Cologuard Plus test detects 95% of colorectal cancers at 94% specificity in the U.S. screening population (Press release, Exact Sciences, MAR 31, 2025, View Source [SID1234651669]). This performance means fewer unnecessary follow-up colonoscopies—up to a 40% reduction compared to the original Cologuard test2—and greater confidence in results.

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Building on the trust and success of Cologuard—used for more than 19 million screenings over the past decade—Cologuard Plus delivers even greater performance while maintaining the convenience of at-home collection. Backed by pivotal data from the 20,000+ person BLUE-C study published in the New England Journal of Medicine, the Cologuard Plus test sets a new standard for noninvasive CRC screening:

Unmatched Noninvasive CRC Accuracy*:95% sensitivity for CRC detection, versus 71% sensitivity with fecal immunochemical test (FIT).1
Fewer False Positives: 40% reduction compared to the original Cologuard test.2
High Precancerous Lesion Detection:43% sensitivity for advanced precancerous lesions and 74% sensitivity for high-grade dysplasia—the type of precancerous growths most likely to become cancer.1
Greater Confidence in Negative Results:A negative Cologuard Plus result means a 99.98% chance the patient does not have colorectal cancer—offering peace of mind to both patients and clinicians.1
Access & Coverage:Covered by Medicare and included in the U.S. Preventive Services Taskforce (USPSTF) guidelines as a recommended stool-based screening option.
Recognized for Quality Care:Satisfies three years of colorectal cancer screening quality measure credit, helping health care professionals meet screening goals while improving patient outcomes.
"Cologuard Plus builds on the proven performance of Cologuard," said Jake Orville, Executive Vice President and General Manager, Screening. "Cologuard transformed colorectal cancer screening—driving an estimated 77% of the nationwide increase in CRC screening participation from 2018 to 20213 and enabling more than 19 million screenings to date. Cologuard Plus delivers key enhancements to help improve patient care and streamline health care delivery, bringing us closer to eradicating this highly preventable and treatable disease."

Driving Better Outcomes Through Early Detection and Adherence

Colorectal cancer is highly treatable when caught early—survivable in about 90% of cases.‡ Yet, nearly 48 million Americans remain unscreened.4 Routine screening not only detects colorectal cancer when it’s most treatable but also prevents it by identifying precancerous growths so they can be removed.5

"Early detection helps save lives, and clinicians need highly accurate tests that their patients will complete," said Dr. Paul Limburg, Chief Medical Officer, Screening. "Cologuard Plus delivers unprecedented performance in a noninvasive test—detecting more cancers while significantly reducing false positives. Combined with strong patient adherence, it gives health care providers confidence that more patients will get screened and receive accurate results to drive better outcomes."

Effective screening depends on patient adherence, and Cologuard Plus is designed to remove barriers to testing. A large national sample of Cologuard orders shows that 71% of patients complete their test within an average of 28 days,6 significantly outperforming adherence rates seen in separate meta-analyses for FIT (42%) or colonoscopy referrals (38%).7

Follow-up adherence is also strong—79% of patients who receive a positive Cologuard result complete a colonoscopy, and 83% of patients complete repeat screening three years later.8,9 These adherence rates are critical in detecting cancer early and ensuring patients get the care they need.

Like the original Cologuard test, the Cologuard Plus test is shipped directly to a patient’s home and integrates with the ExactNexus technology platform. This platform simplifies ordering, result delivery, and patient navigation—a feature proven to improve test completion rates.10 As Exact Sciences works to expand patient access to the Cologuard Plus test, the original Cologuard test will remain available. Nationwide, more than 96% of patients aged 45 and older have no out-of-pocket costs for screening with the Cologuard test.11§

* Based on relative comparison to published reports; not direct evidence from head-to-head comparisons with all other screening tests.
† 94% specificity was determined for adults with no colorectal neoplasia age-weighted to the U.S. population
‡ Based on 5-year survival.
§ Exact Sciences estimate based on historical patient billing as of November 2024. Exceptions for coverage may apply; only patients’ insurers can confirm how the Cologuard test would be covered.

On March 31, 2025 Theriva Biologics (NYSE American: TOVX), a diversified clinical-stage company developing therapeutics designed to treat cancer and related diseases in areas of high unmet need, reported that a second Independent Data Monitoring Committee (IDMC) review of data from the VIRAGE Phase 2b clinical trial in newly-diagnosed metastatic pancreatic ductal adenocarcinoma (PDAC) found that that VCN-01 was well tolerated in combination with standard-of-care chemotherapy (gemcitabine/nab-paclitaxel) and the adverse event (AE) profile was as expected for the patient population and the medications being studied (Press release, Theriva Biologics, MAR 31, 2025, View Source [SID1234651691]).

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The IDMC, composed of key opinion leaders in pancreatic cancer and oncolytic viruses, reviewed clinical data from the complete safety population of 101 patients enrolled across 5 sites in the U.S. and 9 sites in Spain. The VCN-01 AE profile was consistent with that observed in prior clinical trials. The most common VCN-01 related AEs (pyrexia, flu-like illness, vomiting, nausea, and elevated transaminases) were transient and reversible. These AEs were observed to be less frequent and of reduced CTCAE grade after the second VCN-01 dose (administered on day 92) compared to the first VCN-01 dose (administered on day 1). The IDMC noted that the overall type and number of AEs in the VCN-01 treatment group was as expected for the pancreatic cancer population, the duration of treatment, and the administration of an oncolytic virus. VIRAGE patient enrollment was completed in September 2024 and topline clinical outcomes data are anticipated in Q2 2025.

"This second positive IDMC review of VCN-01 safety data from a larger number of patients affirms the feasibility of repeated VCN-01 dosing in metastatic PDAC patients" said Steven A. Shallcross, Chief Executive Officer of Theriva Biologics. "We are now working towards the release of topline clinical outcomes data in Q2 2025. If positive, these data, in combination with the previously reported feedback from the FDA and EMA, will guide the design of a potential Phase 3 registrational trial for discussion with regulatory agencies later this year."

About VIRAGE
VIRAGE is a two-arm, Phase 2b open-label, randomized, controlled, multicenter clinical trial in patients with histologically confirmed, newly-diagnosed metastatic PDAC. Patients have been enrolled at 5 sites in the U.S> and 9 sites in Spain. In both the control and VCN-01 treatment arms, patients receive gemcitabine/nab-paclitaxel standard-of-care chemotherapy in repeated 28-day cycles until disease progression. In the VCN-01 treatment arm only, patients are also administered intravenous VCN-01 seven-days prior to starting the first and fourth cycles of gemcitabine/nab-paclitaxel treatment (study days 1 and ~92 respectively). Primary endpoints for the trial include overall survival and VCN-01 safety/tolerability. Additional endpoints include progression free survival, objective response rate, and measures of VCN-01 biodistribution, replication, and immune response. More information about the trial is available on Clinicaltrials.gov (NCT05673811), through the Spanish Clinical Trials Registry and European Union Drug Regulating Authorities Clinical Trials Database (EudraCT Number: 2022-000897-24).

About VCN-01
VCN-01 is a systemically administered oncolytic adenovirus designed to selectively and aggressively replicate within tumor cells and degrade the tumor stroma that serves as a significant physical and immunosuppressive barrier to cancer treatment. This unique mode-of-action enables VCN-01 to exert multiple antitumor effects by (i) selectively infecting and lysing tumor cells; (ii) enhancing the access and perfusion of co-administered chemotherapy products; and (iii) increasing tumor immunogenicity and exposing the tumor to the patient’s immune system and co-administered immunotherapy products. Systemic administration enables VCN-01 to exert its actions on both the primary tumor and metastases. VCN-01 has been administered to 142 patients in clinical trials of different cancers, including PDAC (in combination with chemotherapy), head and neck squamous cell carcinoma (with an immune checkpoint inhibitor), ovarian cancer (with CAR-T cell therapy), colorectal cancer, and retinoblastoma (by intravitreal injection).