On March 31, 2025 Corcept Therapeutics Incorporated (NASDAQ: CORT), a commercial-stage company engaged in the discovery and development of medications to treat severe endocrinologic, oncologic, metabolic and neurologic disorders by modulating the effects of the hormone cortisol, reported that ROSELLA, the company’s pivotal Phase 3 trial of relacorilant plus nab-paclitaxel in patients with platinum-resistant ovarian cancer, met its primary endpoint of improved progression-free survival, as assessed by blinded independent central review (PFS-BICR) (Press release, Corcept Therapeutics, MAR 31, 2025, https://ir.corcept.com/news-releases/news-release-details/primary-endpoint-met-corcepts-pivotal-phase-3-rosella-trial [SID1234651666]).

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In ROSELLA, patients treated with relacorilant in addition to nab-paclitaxel chemotherapy experienced a 30 percent reduction in risk of disease progression compared to patients treated with nab-paclitaxel alone (hazard ratio: 0.70; p-value: 0.008). Their median PFS-BICR was 6.5 months, compared to 5.5 months in patients who received nab-paclitaxel alone. At an interim evaluation of overall survival (OS), patients treated with relacorilant plus nab-paclitaxel had a significant improvement in OS, with a median OS of 16.0 months, compared to 11.5 months for patients receiving nab-paclitaxel alone (hazard ratio: 0.69; p-value: 0.012). Relacorilant was well-tolerated and no new safety signals were observed. As was the case in the company’s Phase 2 trial, safety and tolerability were comparable in the two groups.

Complete results from ROSELLA will be presented at a medical conference this year. Results from Corcept’s Phase 2 trial of relacorilant in patients with platinum-resistant ovarian cancer were published in the Journal of Clinical Oncology in June 2023.

The ROSELLA trial enrolled 381 patients with platinum-resistant ovarian cancer at sites in the United States, Europe, South Korea, Brazil, Argentina, Canada and Australia; biomarker selection was not required. Patients were randomized 1:1 to receive either relacorilant plus nab-paclitaxel or nab-paclitaxel alone. ROSELLA has dual primary endpoints — PFS-BICR and OS. A positive outcome is achieved if either endpoint is met.

"Patients with advanced ovarian cancer have few good treatment options and, unfortunately, patients with recurrent disease eventually develop resistance to available therapies. The ROSELLA results represent an important advancement in the development of a treatment for patients with platinum-resistant ovarian cancer," said Alexander B. Olawaiye, M.D., Director of gynecological cancer research at Magee-Women’s Hospital of the University of Pittsburgh and Principal Investigator in the ROSELLA trial.

"Platinum-resistant ovarian cancer poses a significant treatment challenge. The ROSELLA results demonstrate that relacorilant in combination with nab-paclitaxel has the potential to become a key strategy to help improve patient outcomes," said Domenica Lorusso, M.D., Ph.D., Director of the Gynaecological Oncology Unit at Humanitas Hospital San Pio X, Milan, and Full Professor of Obstetrics and Gynaecology, Humanitas University, Rozzano and investigator in the ROSELLA trial.

"The improvement in survival seen in ROSELLA, without an increased safety burden, brings us closer to delivering a new standard-of-care treatment for patients with platinum-resistant ovarian cancer," said Bill Guyer, PharmD, Corcept’s Chief Development Officer. "We deeply appreciate the patients and investigators who participated in the trial, and we look forward to presenting the trial’s full results in the coming months. We expect to submit our NDA in the third quarter and our MAA shortly thereafter."

The ROSELLA trial is being conducted in collaboration with The GOG Foundation, Inc. (GOG-F), the European Network of Gynaecological Oncological Trial groups (ENGOT), the Asia-Pacific Gynecologic Oncology Trials Group (APGOT), the Latin American Cooperative Oncology Group (LACOG) and the Australia New Zealand Gynaecological Oncology Group (ANZGOG).

About Relacorilant

Relacorilant, an oral therapy, is a selective glucocorticoid receptor (GR) antagonist that modulates cortisol activity by binding to the GR but not to the body’s other hormone receptors. Corcept is studying relacorilant in a variety of serious disorders in addition to ovarian cancer, including endogenous hypercortisolism (Cushing’s syndrome) and prostate cancer. Relacorilant is proprietary to Corcept and is protected by composition of matter, method of use and other patents. It has been designated an orphan drug by the FDA and the European Commission (EC) for the treatment of hypercortisolism and by the EC for the treatment of ovarian cancer.

About Platinum-Resistant Ovarian Cancer

Ovarian cancer is the fifth most common cause of cancer death in women. Patients whose disease returns less than six months after receiving platinum-containing therapy have "platinum-resistant" disease. There are few treatment options for these women. Median overall survival following recurrence is approximately 12 months with single-agent chemotherapy. Approximately 20,000 women with platinum-resistant disease are candidates to start a new therapy each year in the United States, with at least an equal number in Europe.

On March 31, 2025 Circle Pharma, a clinical-stage biopharmaceutical company focused on developing cell-permeable macrocycle therapeutics, reported that Chief Executive Officer David Earp, JD, Ph.D., will present at the following investor conferences (Press release, Circle Pharma, MAR 31, 2025, View Source;utm_medium=rss&utm_campaign=circle-pharma-to-present-at-the-upcoming-investor-conferences [SID1234651665]):

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24th Annual Needham Virtual Healthcare Conference
April 9, 2025

11:45 – 12:25 pm ET

Stifel 2025 Virtual Targeted Oncology Forum
April 9, 2025

1:30 p.m. – 1:55 p.m. ET

On March 31, 2025 Celcuity Inc. (Nasdaq: CELC), a clinical-stage biotechnology company pursuing development of targeted therapies for oncology, reported financial results for the fourth quarter ended December 31, 2024 and other recent business developments (Press release, Celcuity, MAR 31, 2025, View Source [SID1234651664]).

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"We expect 2025 to be a transformational year for Celcuity as we anticipate reporting several clinical data readouts, including primary analysis for the PIK3CA wild-type cohort of the VIKTORIA-1 trial. We expect to share topline data in Q2 2025," said Brian Sullivan, CEO and co-founder of Celcuity. "In addition, we anticipate reporting topline data for the Phase 1b/2 trial in metastatic castration resistant prostate cancer in late Q2 2025, and topline data for the PIK3CA mutant-type cohort of the VIKTORIA-1 trial in Q4 2025. We also remain on track to enroll the first patient for our VIKTORIA-2 Phase 3 trial in Q2 2025."

Fourth Quarter 2024 Business Highlights and Other Recent Developments

● The VIKTORIA-1 Phase 3 clinical trial evaluating gedatolisib in combination with fulvestrant with and without palbociclib in adults with HR+, HER2- advanced breast cancer who have received prior treatment with a CDK4/6 inhibitor is 100% enrolled for the PIK3CA wild-type cohort. We expect to provide topline data in Q2 2025.

○ Enrollment for the PIK3CA mutant cohort remains on track and topline data is expected in Q4 of 2025.

● The VIKTORIA-2 Phase 3 open-label randomized study evaluating the efficacy and safety of gedatolisib in combination with fulvestrant plus a CDK4/6 inhibitor, either ribociclib or palbociclib, in comparison to fulvestrant plus a CDK4/6 inhibitor as a first-line treatment for patients with HR+/HER2- advanced breast cancer who are endocrine therapy resistant remains on track to enroll its first patient in Q2 2025.

○ A safety run-in study preceding the initiation of the Phase 3 portion of the study will be conducted in 12-36 participants to assess the safety profile of gedatolisib in combination with ribociclib and fulvestrant.
○ Site selection activities are completed. We expect to activate approximately 200 sites across North America, Europe, Latin America, and Asia-Pacific.
○ The Phase 3 portion of the study is expected to enroll approximately 638 patients.

● The Phase 1b/2 clinical trial, evaluating gedatolisib in combination with darolutamide for the treatment of patients with metastatic castration resistant prostate cancer ("mCRPC"), is ongoing and remains on track to report preliminary data in late Q2 2025.

○ The study is expected to enroll up to 54 patients with mCRPC whose disease progressed while on or after treatment with an androgen receptor signaling inhibitor.

● In December 2024, Celcuity presented overall survival data from a Phase 1b trial, which evaluated gedatolisib in combination with palbociclib and either letrozole or fulvestrant, in patients with HR+, HER2- advanced or metastatic breast cancer during a poster session at the 2024 San Antonio Breast Cancer Symposium (SABCS). Median overall survival was 77.3 months among patients with HR+, HER2- advanced breast cancer who were treatment-naïve in the advanced setting and 33.9 months among patients previously treated with a CDK4/6 inhibitor.

○ Results compare favorably to published data for currently available first- or second-line standard-of-care treatment regimens for patients with HR+, HER2- advanced breast cancer and highlight the promising clinical development strategy of simultaneously inhibiting the ER, CDK4/6, and PAM (PI3K/AKT/mTOR) signaling pathways.

Fourth Quarter and Full Year 2024 Financial Results

Unless otherwise stated, all comparisons are for the fourth quarter and full year ended December 31, 2024, compared to the fourth quarter and full year ended December 31, 2023.

Total operating expenses were $36.4 million for the fourth quarter of 2024, compared to $19.7 million for the fourth quarter of 2023. Operating expenses for the full year 2024 were $113.3 million, compared to $66.2 million for the full year 2023.

Research and development ("R&D") expenses were $33.5 million for the fourth quarter of 2024, compared to $18.1 million for the prior-year period. Of the approximately $15.4 million increase in R&D expenses, $9.9 million primarily related to costs supporting ongoing activities for the VIKTORIA-1 Phase 3 trial and the CELC-G-201 Phase 1b/2 trial, along with the commencement of the VIKTORIA-2 Phase 3 trial. The remaining $5.5 million primarily relates to increased employee and consulting expenses.

R&D expenses for the full year 2024 were $104.2 million, compared to $60.6 million for the prior year. Of the approximately $43.6 million increase in R&D expenses, $30.7 million was related to costs supporting ongoing activities for the VIKTORIA-1 Phase 3 trial and the CELC-G-201 Phase 1b/2 clinical trial, along with the commencement of the VIKTORIA-2 Phase 3 trial. The remaining $12.9 million increase in R&D expenses was primarily related to increased employee and consulting expenses.

General and administrative ("G&A") expenses were $3.0 million for the fourth quarter of 2024, compared to $1.6 million for the prior year period. Of the approximately $1.4 million increase, $1.1 million was related to increased employee-related expenses, and $0.3 million was related to professional fees, expanding infrastructure costs and other administrative expenses.

G&A expenses for the full year 2024 were $9.1 million, compared to $5.6 million for the prior year. Of the approximately $3.4 million increase in G&A expenses, $2.6 million was related to increased employee-related expenses, and $0.8 million was related to professional fees, expanding infrastructure costs, and other administrative expenses.

Net loss for the fourth quarter of 2024 was $36.7 million, or $0.85 loss per share, compared to a net loss of $18.8 million, or $0.65 loss per share, for the fourth quarter of 2023. Net loss for the full year 2024 was $111.8 million, or $2.83 loss per share, compared to a net loss of $63.8 million, or $2.69 loss per share, in 2023. Non-GAAP adjusted net loss for the fourth quarter of 2024 was $32.3 million, or $0.75 loss per share, compared to non-GAAP adjusted net loss of $17.6 million, or $0.61 loss per share, for the fourth quarter of 2023. Non-GAAP adjusted net loss for the full year 2024 was $101.9 million, or $2.58 loss per share, compared to non-GAAP adjusted net loss of $57.8 million, or $2.44 loss per share, for 2023. Non-GAAP adjusted net loss excludes stock-based compensation expense, non-cash interest expense, and non-cash interest income. Because these items have no impact on Celcuity’s cash position, management believes non-GAAP adjusted net loss better enables Celcuity to focus on cash used in operations. For a reconciliation of financial measures calculated in accordance with generally accepted accounting principles in the United States ("GAAP") to non-GAAP financial measures, please see the financial tables at the end of this press release.

Net cash used in operating activities for the fourth quarter of 2024 was $27.8 million, compared to $18.5 million for the fourth quarter of 2023. Net cash used in operating activities for the full year 2024 was $83.5 million, compared to $53.8 million for the full year 2023. Cash, cash equivalents and short-term investments were approximately $235.1 million at the end of fiscal year 2024 and are expected to fund current clinical development program activities through 2026.

Webcast and Conference Call Information

The Celcuity management team will host a webcast/conference call at 4:30 p.m. ET today to discuss the fourth quarter and full year 2024 financial results and provide a corporate update. To participate in the teleconference, domestic callers should dial 1-800-717-1738 and international callers should dial 1-646-307-1865. A live webcast presentation can also be accessed using this weblink: View Source;tp_key=f99d4186f3. A replay of the webcast will be available on the Celcuity website following the live event.

On March 31, 2025 Black Diamond Therapeutics, Inc. (Nasdaq: BDTX), a clinical-stage oncology company developing MasterKey therapies that target families of oncogenic mutations in patients with cancer, reported its participation in upcoming investor conferences. Presentation details with President and Chief Executive Officer, Mark Velleca, M.D., Ph.D., are as follows (Press release, Black Diamond Therapeutics, MAR 31, 2025, View Source [SID1234651663]):

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24th Annual Needham Virtual Healthcare Conference presentation at 3:00pm ET on Monday, April 7, 2025
Stifel 2025 Virtual Targeted Oncology Forum fireside chat at 1:00pm ET on Tuesday, April 8, 2025
Webcasts will be available at the start of the presentations on the investor relations section of the Company’s website, www.blackdiamondtherapeutics.com. Replays of the presentations will also be available and archived on the site for 90 days.

On March 31, 2025 Biomea Fusion, Inc. ("Biomea" or "Biomea Fusion" or "the Company") (Nasdaq: BMEA), a clinical-stage diabetes and obesity medicines company, reported fourth quarter and full year 2024 financial results and corporate highlights (Press release, Biomea Fusion, MAR 31, 2025, View Source [SID1234651662]).

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"2024 was a transformative year for Biomea, marked by the advancement of icovamenib into late-stage development and compelling clinical data that reinforced our confidence in its potential to reshape diabetes treatment, particularly for patients with severe insulin deficiency," said Mick Hitchcock, Ph.D., Interim Chief Executive Officer and Board Member of Biomea Fusion. "As we move into this next phase, the Board made a strategic decision to align leadership with the company’s evolution, and I’m honored to step in and contribute decades of experience in late-stage development, regulatory strategy, and commercialization to help guide Biomea forward. This transition reflects the continued confidence in our menin inhibitor program and the strength of our covalent small molecule platform. We remain fully committed to advancing icovamenib and delivering on our mission to transform diabetes treatment through this disease-modifying therapy. With key data readouts and regulatory milestones ahead, 2025 is set to be a pivotal year for the company."

In March 2025, the Company announced a leadership transition, appointing Board member Mick Hitchcock, Ph.D., as Interim Chief Executive Officer, succeeding Thomas Butler.

In January 2025, we announced plans to position Biomea as a dedicated diabetes and obesity medicines company. Building on our most recent clinical trial results, our strategic focus for icovamenib is now exclusively centered on metabolic disorders. As a result, we are terminating all ongoing oncology trials involving icovamenib and will conclude the BMF-500 study in patients with relapsed/refractory acute leukemia with FLT3 gene mutations following the dose escalation phase. Biomea will seek strategic partnerships to advance its oncology portfolio and the capabilities of its FUSION System, while reallocating internal resources to accelerate our metabolic disease programs.

In October 2024, we announced the formation of our Global Scientific Advisory Board, comprised of 22 internationally recognized experts in beta cell science and diabetes therapeutics. This board will work closely with our leadership team as we continue to explore menin biology and beta cell regeneration, and advance the clinical development of icovamenib as a novel, disease-modifying treatment targeting a root cause of diabetes.

RECENT DIABETES AND OBESITY PROGRAM UPDATES

COVALENT-111 (Icovamenib for Type 2 Diabetes ("T2D"))

Study Results:

In the dose expansion portion of the COVALENT-111 study, icovamenib demonstrated statistically significant reductions in HbA1c in the prespecified per protocol patient population, with notable effects in the severe insulin deficient patients.
In this group, icovamenib achieved a 1.47% reduction in HbA1c at Week 26 following 12 weeks of treatment with 100 mg once a day ("QD").
Severe insulin deficient patients also experienced the largest mean increase in C-peptide index levels, with a 53% mean increase from baseline by Week 26, indicating enhanced endogenous insulin production.
In a broader subset of insulin deficient patients, icovamenib treatment led to a 1.0% reduction in HbA1c at Week 26 following 12 weeks of treatment with 100 mg QD.
The data showed that icovamenib preferentially increased insulin secretion in insulin-deficient patients, supporting its potential as a targeted therapy for individuals with severe insulin deficiency, a population with limited treatment options and the highest risk profile.
Across all dosing groups in the severe insulin deficient subgroup, there was a strong correlation between increases in C-peptide and reductions in HbA1c, consistent with the proposed mechanism through beta cell restoration.
HbA1c reductions were durable at 26 weeks, 3 months post last dose, further supporting the long-lasting effect of icovamenib on glycemic control.
Icovamenib was generally well tolerated, with no treatment discontinuations due to adverse events, no hypoglycemic episodes, and no drug-related serious adverse events reported.
Preclinical Findings:

In preclinical experiments, including in ex vivo human islets, icovamenib was able to enhance the activity of GLP-1 RA-based therapies, potentially leading to increased insulin secretion and improved glycemic control in patients with diabetes. These effects were associated with an increase in the expression levels of the GLP-1 receptors ("GLP-1R").
Overall results showed synergy of the combination therapy, which may allow lower doses of GLP-1-based therapies to achieve glycemic targets potentially reducing side effects and improving the tolerability of GLP-1 based therapies.
Anticipated 2025 Milestones:

Planned U.S. Food and Drug Administration ("FDA") discussions regarding Phase II/III trial designs and the advancement of icovamenib into late-stage clinical development in the first half of 2025.
52-week data from the COVALENT-111 Phase II study anticipated in the second half of 2025.
COVALENT-112 (Icovamenib for Type 1 Diabetes ("T1D"))

Anticipated 2025 Milestones:

Initial open label data from the Phase II study is expected in the second half of 2025.
BMF-650 (Oral small molecule GLP-1 RA)

Preclinical Progress:

Preclinical studies evaluating the properties of our investigational, next-generation, oral small molecule GLP-1 RA (BMF-650) demonstrated positive early preclinical activity, including improved glucose-stimulated insulin secretion, reduction in blood glucose concentration, and appetite suppression in cynomolgus monkeys.
In comparison to a leading oral GLP-1 RA, BMF-650 exhibited higher bioavailability and a less variable pharmacokinetic profile, which may translate to improved tolerability and enable successful dose escalation in patients.
Human donor islet studies confirmed that BMF-650 significantly enhanced glucose-stimulated insulin secretion, aligning with findings from animal models.
In cynomolgus monkey studies, BMF-650 demonstrated robust improvements in glucose control and insulin secretion, consistent with its effects in human donor islets.
Appetite suppression studies revealed that daily oral dosing of BMF-650 significantly reduced food intake during peak drug concentration, with sustained effects throughout the day for a six-day study period.
Anticipated 2025 Milestones:

Submission of the Investigational New Drug ("IND") application for BMF-650 is planned for the second half of 2025.
ONCOLOGY PROGRAM

COVALENT-103 Study (BMF-500):

Preliminary Phase I data for BMF-500 in relapsed/refractory acute leukemia patients with FLT3 gene mutations having failed gilteritinib indicated clinical activity with evidence of responses, including a first complete response with incomplete hematologic recovery (CRi) and reductions in bone marrow blasts in five of six evaluable FLT3 mutated patients.
Pharmacokinetic and pharmacodynamic analyses confirmed dose-proportional on-target FLT3 inhibition and good compartmental penetration, and BMF-500 showed a favorable safety and tolerability profile with no dose-limiting toxicities observed.
Anticipated 2025 Milestones:

After the completion of the dose escalation of BMF-500 in relapsed/refractory acute leukemia patients with FLT3 gene mutations, we intend to conclude our oncology study with BMF-500 and explore strategic partnerships.
FOURTH QUARTER AND FULL YEAR 2024 FINANCIAL RESULTS

Cash, Cash Equivalents, and Restricted Cash: As of December 31, 2024, the Company had cash, cash equivalents and restricted cash of $58.6 million, compared to $177.2 million as of December 31, 2023.

Net Income/Loss: The Company reported a net loss attributable to common stockholders of $29.3 million for the three months ended December 31, 2024, compared to a net loss of $34.9 million for the same period in 2023. Net loss attributable to common stockholders was $138.4 million for the year ended December 31, 2024, compared to a net loss of $117.3 million for the same period in 2023.

Research and Development ("R&D") Expenses: R&D expenses were $25.2 million for the three months ended December 31, 2024, compared to $30.9 million for the same period in 2023. The decrease of $5.6 million was primarily due to the decrease in compensation and related expenses, manufacturing related expenses, and clinical related expenses. R&D expenses were $118.1 million for the year ended December 31, 2024, compared to $102.5 million for the same period in 2023. The increase of $15.5 million was primarily due to an increase in clinical development of icovamenib, consultants, advisors and other professional services to support our clinical studies, discovery research and overall research and development programs.

General and Administrative ("G&A") Expenses: G&A expenses were $4.8 million for the three months ended December 31, 2024, compared to $6.5 million for the same period in 2023. The decrease of $1.6 million was primarily due to the decrease in compensation and related expenses. G&A expenses were $26.0 million for the year ended December 31, 2024, compared to $23.6 million for the same period in 2023. The increase of $2.4 million was primarily due to increased personnel-related expenses, including stock-based compensation, due to an increase in headcount, as well as an increase in professional and consulting services to support the growth of the Company.

About Icovamenib
Icovamenib is an investigational, orally bioavailable, potent, and selective covalent inhibitor of menin. The molecule was built using Biomea Fusion’s FUSION System and is designed to regenerate insulin-producing beta cells with the aim to cure diabetes. Icovamenib’s proposed mechanism of action in diabetes is to enable the proliferation, preservation, and reactivation of a patient’s own healthy, functional, insulin-producing beta cells. As the potentially first disease-modifying therapy for T1D and T2D, icovamenib could become an important addition and complement to the diabetes treatment landscape once it has successfully completed clinical studies.