A Phase II Clinical Study on Precision Treatment of Rare Tumours in China Guided by Patient-Derived Tumor Organoids (PDO) and Next-Generation Sequencing (NGS)

The objective of this Phase II, open-label, multicenter, non-randomised controlled clinical trial is to guide precision treatment for patients with rare tumours based on Patient-Derived Organoids/Next-Generation Sequencing drug screening.

Start Date01 Jan 2025

Sponsor / Collaborator

Peking University Shenzhen Hospital

NCT06377852 / RecruitingPhase 3IIT

Comparing Oral Drug Dosing Strategies in Older Patients With Metastatic Breast Cancer to Maximize Tolerance and Reduce Discontinuation: The CDK4/6 Inhibitor Dosing Knowledge (CDK) Study

The purpose of this study is to generate evidence on an alternative dosing strategy for CDK4/6 inhibitors to help more patients with Metastatic Breast Cancer (MBC) (age ≥ 65 years) tolerate side effects and stay on treatment longer, to derive the most clinical benefit from these drugs.
The primary objective of the CDK Study is to compare time to treatment discontinuation (TTD) on the approved dosing for palbociclib (125 mg orally daily on days 1-21 of 28-day cycle) or ribociclib (600 mg orally daily on days 1-21 of 28-day cycle) vs. TTD using titrated dosing approach with the same schedule but starting at a lower dose of palbociclib (100 mg or 75 mg) or ribociclib (400 mg or 200 mg) and escalating the dose if well-tolerated in combination with provider/patient choice endocrine therapy (aromatase inhibitor (AI) or fulvestrant) in patients age 65 or older with HR+/HER2- MBC. The secondary and exploratory objectives will generate evidence needed to personalize treatment decisions by comparing patient-centric secondary outcomes and evaluating baseline factors.
Together with their treating physician, participants will choose the CDK4/6 inhibitor (palbociclib or ribociclib) and which endocrine therapy (aromatase inhibitor or fulvestrant) of their choice but will be randomized to either Arm 1 (indicated dosing) or Arm 2 (titrated dosing).

Start Date29 Oct 2024

Sponsor / Collaborator

American Society of Clinical Oncology, Inc.

[+1]

CTR20232765 / RecruitingPhase 3

一项在激素受体阳性和人表皮生长因子受体2阴性的局部晚期、不可切除或转移性乳腺癌患者中评估capivasertib联合CDK4/6抑制剂和氟维司群对比CDK4/6抑制剂和氟维司群的Ib/III期、开放标签、随机研究（CAPItello-292）

[Translation] A phase Ib/III, open-label, randomized study evaluating capivasertib plus a CDK4/6 inhibitor and fulvestrant versus a CDK4/6 inhibitor and fulvestrant in patients with hormone receptor-positive and human epidermal growth factor receptor 2-negative locally advanced, unresectable or metastatic breast cancer (CAPItello-292)

通过评估PFS，证明在HR+/HER2-局部晚期或转移性乳腺癌患者（总体人群）中capivasertib组相比对照组的优效性

[Translation]

Demonstrated superiority of capivasertib over control in patients with HR+/HER2- locally advanced or metastatic breast cancer (overall population) as assessed by PFS

Start Date29 Aug 2024

Sponsor / Collaborator

AstraZeneca AB

[+1]

100 Clinical Results associated with Palbociclib

100 Translational Medicine associated with Palbociclib

100 Patents (Medical) associated with Palbociclib

1,925

Literatures (Medical) associated with Palbociclib

31 Dec 2024·Future Science OA

Real-world effectiveness of palbociclib in HR+/HER2- metastatic breast cancer: a literature review

Review

Author: Kümler, Iben ; Christiansen, Emilie Adrian

Approximately 70% of newly diagnosed breast cancers are of the HR+/HER2- subtype. For the treatment of patients with HR+/HER2- metastatic breast cancer, current guidelines recommend the use of a CDK4/6 inhibitor (palbociclib, ribociclib or abemaciclib) in combination with endocrine therapy. In this review we assess existing literature concerning real-world effectiveness of palbociclib. Survival outcomes in terms of progression-free survival and overall survival are discussed and compared among the included real-world studies and in relation to the phase III PALOMA trials.

31 Dec 2024·Future Science OA

Surgical resection due to poor outcome of the immunotherapy of a relapsed mediastinal liposarcoma: a case report

Author: Li, Zhi-Xin ; Xu, Shu-Quan ; Wu, Lei-Lei ; Wang, Ming-Ji ; Xie, Dong

The feasibility of surgery after immunotherapy for mediastinal liposarcoma remains uncertain. Besides, the case of immunotherapy for liposarcoma is still lacking. We report a case of recurrence after resection of a left mediastinal liposarcoma. After recurrence, one course of pembrolizumab plus anlotinib hydrochloride showed no tumor shrinkage, and genetic testing showed CDK4 amplification and PD-L1 TPS <1%; therefore, the plan was changed to one course of pembrolizumab plus palbociclib, but the tumor still did not shrink. Thus, second tumor resection was performed. In addition, the postoperative pathology was still well-differentiated liposarcoma. The significance of immunotherapy in liposarcoma still needs to be further explored. In the absence of surgical contraindications, secondary surgery might be feasible.

01 Dec 2024·INTERNATIONAL IMMUNOPHARMACOLOGY

Pirfenidone inhibits CCL2-mediated Treg chemotaxis induced by palbociclib and fulvestrant in HR+/HER2− breast cancer

Article

Author: Zhang, Xianyu ; Li, Siwei ; Pang, Da

In human epidermal growth factor receptor 2-negative (HR+/HER2-) breast cancer, the most prevalent subtype, the pathological complete response (pCR) rate after neoadjuvant chemotherapy is less than 18 %, and the survival of patients with advanced-stage disease is approximately 34 %, highlighting the critical demand for more potent therapies. Recent research has underscored the substantial therapeutic benefits of the combination of CDK4/6 inhibitors and fulvestrant (Ful) in managing HR+/HER2- breast cancer. These therapeutics not only curtail tumor proliferation but also alter the tumor immune microenvironment, suggesting novel avenues for immunotherapy for this breast cancer subtype. Flow cytometry, PCR, WB, and RNA-seq experiments revealed that the combination of the CDK4/6 inhibitor palbociclib (Pal) with Ful upregulated CCL2 in tumor cells by inducing the SASP and activating the MAPK signaling pathway. CCL2 attracts Tregs to the tumor microenvironment, where it exerts an immunosuppressive effect. By administering the CCL2 inhibitor pirfenidone, we inhibited these effects and enhanced the antitumor efficacy of Pal + Ful. Our research revealed an immunosuppressive effect of CDK4/6 inhibitors and fulvestrant and suggested that CCL2 inhibitors may be a viable approach for treating patients with advanced HR+/HER2- breast cancer.

315

News (Medical) associated with Palbociclib

14 Nov 2024

·www.globenewswire.com

Celcuity Inc. Reports Third Quarter Financial Results and Provides Corporate Update

The PIK3CA wild-type cohort of the Phase 3 VIKTORIA-1 trial is 100% enrolled; expect to report topline data for this cohort in late Q1 2025 or Q2 2025Approximately $264 million in cash, cash equivalents and investments at end of Q3 2024 expected to fund current clinical development program activities through 2026 Management to host webcast and conference call today, November 14, 2024, at 4:30 p.m. ET MINNEAPOLIS, Nov. 14, 2024 (GLOBE NEWSWIRE) -- Celcuity Inc. (Nasdaq: CELC), a clinical-stage biotechnology company pursuing development of targeted therapies for oncology, today announced financial results for the third quarter ended September 30, 2024 and other recent business developments. “Enrollment in our VIKTORIA-1 study remains robust and on-track. The PIK3CA wild-type cohort is 100% enrolled, and enrollment in the PIK3CA mutant cohort is on plan,” said Brian Sullivan, CEO and co-founder of Celcuity. “Based on our current forecast of reaching the event thresholds that will trigger primary analysis in both the PIK3CA wild-type and mutant cohorts, we expect to report topline data for the PIK3CA wild-type cohort sometime in late Q1 2025 or during Q2 2025 and to report topline data for the PIK3CA mutant cohort in the second half of 2025.” Third Quarter 2024 Business Highlights and Other Recent Developments The VIKTORIA-1 Phase 3 clinical trial expects to provide topline data for the PIK3CA wild-type cohort in late Q1 2025 or during Q2 2025 and for the PIK3CA mutant cohort in the second half of 2025. VIKTORIA-1 is 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.The PIK3CA wild-type cohort is 100% enrolled and enrollment of the PIK3CA mutant cohort is on-track relative to plan. The VIKTORIA-2 Phase 3 clinical trial remains on track to enroll its first patient in Q2 2025. The VIKTORIA-2 study is a global Phase 3 open-label randomized clinical trial 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.Prior to the initiation of the Phase 3 portion of the trial, a safety run-in study will be conducted in 12-36 participants to assess the safety profile of gedatolisib in combination with ribociclib and fulvestrant.Site qualification activities to support activation of up to 200 sites across North America, Europe, Latin America, and Asia are on track. 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 expected to report preliminary data in Q2 2025. Overall survival data from the B2151009 Phase 1b clinical trial will be presented at the San Antonio Breast Cancer Symposium (SABCS), taking place December 10-13, 2024. Details of the poster presentation are as follows: Abstract Title: Overall survival in patients with HR+/HER2- advanced breast cancer treated in a phase 1b trial evaluating gedatolisib in combination with palbociclib and endocrine therapy (SESS-1510)Presentation Number: P4-08-25Date/Time: Thursday, December 12, 5:30 PM CST Additional nonclinical data further characterizing the mechanism of action of gedatolisib and its effect on breast cancer cell metabolic functions will also be presented at the SABCS. Abstract Title: Mechanism of action of gedatolisib in combination with fulvestrant and/or palbociclib in estrogen receptor positive breast cancer models (SESS-989)Abstract Title: Different effects of gedatolisib versus single-node PI3K/AKT/mTOR pathway inhibitors on breast cancer cell metabolic functions (SESS-997) In October, Cancers published results of nonclinical studies in gynecological cancer cell line models highlighting the differences between single-node inhibitors of the PI3K/AKT/mTOR pathway and gedatolisib. The published manuscript is available online and on the publications section of Celcuity’s website. Third Quarter 2024 Financial Results Unless otherwise stated, all comparisons are for the third quarter ended September 30, 2024, compared to the third quarter ended September 30, 2023. Total operating expenses were $30.1 million for the third quarter of 2024, compared to $18.9 million for the third quarter of 2023. Research and development (R&D) expenses were $27.6 million for the third quarter of 2024, compared to $17.5 million for the prior-year period. Of the approximately $10.1 million increase in R&D expenses, $6.3 million primarily related to activities supporting the VIKTORIA-1 Phase 3 trial, the Phase 1b/2 trial and the initiation of the VIKTORIA-2 Phase 3 trial, and $3.8 million was related to increased employee and consulting expenses. General and administrative (G&A) expenses were $2.5 million for the third quarter of 2024, compared to $1.4 million for the prior-year period. Employee and consulting related expenses accounted for $0.9 million of the increase. Professional fees and other administrative expenses accounted for the remaining increase of approximately $0.2 million. Net loss for the third quarter of 2024 was $29.8 million, or $0.70 loss per share, compared to a net loss of $18.4 million, or $0.83 loss per share, for the third quarter of 2023. Non-GAAP adjusted net loss for the third quarter of 2024 was $27.6 million, or $0.65 loss per share, compared to non-GAAP adjusted net loss of $17.3 million, or $0.78 loss per share, for the third quarter of 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 third quarter of 2024 was $20.6 million, compared to $12.7 million for the third quarter of 2023. At September 30, 2024, Celcuity reported cash, cash equivalents and short-term investments of $264.1 million. 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 third quarter 2024 financial results and provide a corporate update. To participate in the teleconference, domestic callers should dial 1-800-717-1738 or 1-646-307-1865. A live webcast presentation can also be accessed using this weblink: https://viavid.webcasts.com/starthere.jsp?ei=1688854&tp_key=0b14db1255. A replay of the webcast will be available on the Celcuity website following the live event. About Celcuity Celcuity is a clinical-stage biotechnology company focused on development of targeted therapies for treatment of multiple solid tumor indications. The company's lead therapeutic candidate is gedatolisib, a potent, pan-PI3K and mTOR inhibitor. Its mechanism of action and pharmacokinetic properties are highly differentiated from other currently approved and investigational therapies that target PI3K or mTOR alone or together. A Phase 3 clinical trial, VIKTORIA-1, evaluating gedatolisib in combination with fulvestrant with or without palbociclib in patients with HR+/HER2- advanced breast cancer is currently enrolling patients. More detailed information about the VIKTORIA-1 study can be found at ClinicalTrials.gov. A Phase 1b/2 clinical trial, CELC-G-201, evaluating gedatolisib in combination with darolutamide in patients with metastatic castration resistant prostate cancer, is enrolling patients. A Phase 3 clinical trial, VIKTORIA-2, evaluating gedatolisib plus a CDK4/6 inhibitor and fulvestrant as first-line treatment for patients with HR+/HER2- advanced breast cancer is expected to begin enrolling patients in the second quarter of 2025. Celcuity is headquartered in Minneapolis. Further information about Celcuity can be found at www.celcuity.com. Follow us on LinkedIn and Twitter. Forward-Looking Statements This press release contains statements that constitute "forward-looking statements" including, but not limited to, the design of our clinical trials; the timing of initiating and enrolling patients in, and receiving results and data from, our clinical trials; the costs and expected results from any ongoing or planned clinical trials; the market opportunity for gedatolisib; revenue expectations; our strategy, marketing and commercialization plans, including the benefits of strategic decisions regarding studies and trials; other expectations with respect to Celcuity's lead product candidate, gedatolisib, and its CELsignia platform; our anticipated use of cash; and the strength of our balance sheet. In some cases, you can identify forward-looking statements by terminology such as "may," "should," "expects," "plans," "anticipates," "believes," "estimates," "predicts," "potential," "intends" or "continue," and other similar expressions that are predictions of or indicate future events and future trends, or the negative of these terms or other comparable terminology. Forward-looking statements are subject to numerous risks, uncertainties, and conditions, many of which are beyond the control of Celcuity. These include, but are not limited to, unforeseen delays in our clinical trials, our ability to obtain and maintain regulatory approvals to commercialize our products, and the market acceptance of such products, the development of therapies and tools competitive with our products, our ability to access capital upon favorable terms or at all, and those risks set forth in the Risk Factors section in Celcuity's Annual Report on Form 10-K for the year ended December 31, 2023 filed with the Securities and Exchange Commission on March 27, 2024. Readers are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof. Celcuity undertakes no obligation to update these statements for revisions or changes after the date of this press release, except as required by law. View source version of release on GlobeNewswire.com Contacts: Celcuity Inc. Brian Sullivan, bsullivan@celcuity.com Vicky Hahne, vhahne@celcuity.com (763) 392-0123 ICR HealthcarePatti Bank, patti.bank@icrhealthcare.com(415) 513-1284 Celcuity Inc.Condensed Balance Sheets September 30, 2024 December 31, 2023 (unaudited) Assets Current Assets: Cash and cash equivalents$12,603,289 $30,662,774 Investments 251,455,468 149,919,974 Other current assets 8,379,682 10,007,849 Total current assets 272,438,439 190,590,597 Property and equipment, net 338,787 228,782 Operating lease right-of-use assets 259,744 400,019 Total Assets$273,036,970 $191,219,398 Liabilities and Stockholders' Equity: Current Liabilities: Accounts payable$9,158,778 $5,076,699 Operating lease liabilities 175,226 184,950 Accrued expenses 16,974,725 8,927,094 Total current liabilities 26,308,729 14,188,743 Operating lease liabilities 95,699 225,922 Note payable, non-current 96,923,914 37,035,411 Total Liabilities 123,328,342 51,450,076 Total Stockholders' Equity 149,708,628 139,769,322 Total Liabilities and Stockholders' Equity$273,036,970 $191,219,398 Celcuity Inc.Condensed Statements of Operations(unaudited) Three Months Ended September 30, Nine Months Ended September 30, 2024 2023 2024 2023 Operating expenses: Research and development$27,587,483 $17,488,236 $70,732,017 $42,512,811 General and administrative 2,472,416 1,409,801 6,104,803 3,988,248 Total operating expenses 30,059,899 18,898,037 76,836,820 46,501,059 Loss from operations (30,059,899) (18,898,037) (76,836,820) (46,501,059) Other (expense) income Interest expense (3,343,989) (1,372,132) (7,005,284) (3,929,140)Interest income 3,612,099 1,865,629 8,716,040 5,499,555 Other income, net 268,110 493,497 1,710,756 1,570,415 Net loss before income taxes (29,791,789) (18,404,540) (75,126,064) (44,930,644)Income tax benefits - - - - Net loss$(29,791,789) $(18,404,540) $(75,126,064) $(44,930,644) Net loss per share, basic and diluted$(0.70) $(0.83) $(1.96) $(2.05) Weighted average common shares outstanding, basic and diluted 42,793,047 22,117,626 38,299,548 21,920,147 Cautionary Statement Regarding Non-GAAP Financial Measures This press release contains references to non-GAAP adjusted net loss and non-GAAP adjusted net loss per share. Management believes these non-GAAP financial measures are useful supplemental measures for planning, monitoring, and evaluating operational performance, as they exclude stock-based compensation expense, non-cash interest expense, and non-cash interest income from net loss and net loss per share. Management excludes these items because they do not impact Celcuity’s cash position, which management believes better enables Celcuity to focus on cash used in operations. However, non-GAAP adjusted net loss and non-GAAP adjusted net loss per share are not recognized measures under GAAP and do not have a standardized meaning prescribed by GAAP. As a result, management’s method of calculating non-GAAP adjusted net loss and non-GAAP adjusted net loss per share may differ materially from the method used by other companies. Therefore, non-GAAP adjusted net loss and non-GAAP adjusted net loss per share may not be comparable to similarly titled measures presented by other companies. Investors are cautioned that non-GAAP adjusted net loss and non-GAAP adjusted net loss per share should not be construed as alternatives to net loss, net loss per share or other statements of operations data (which are determined in accordance with GAAP) as an indicator of Celcuity’s performance or as a measure of liquidity and cash flows. Celcuity Inc.Reconciliation of GAAP Net Loss to Non-GAAP Adjusted Net Loss andGAAP Net Loss Per Share to Non-GAAP Adjusted Net Loss Per Share Three Months Ended September 30, Nine Months Ended September 30, 2024 2023 2024 2023 GAAP net loss$(29,791,789) $(18,404,540) $(75,126,064) $(44,930,644)Adjustments: Stock-based compensation Research and development(1) 1,190,424 660,706 3,000,641 1,954,689 General and administrative(2) 740,777 447,931 1,672,418 1,704,213 Non-cash interest expense(3) 730,741 520,794 1,891,139 1,523,699 Non-cash interest income(4) (473,584) (480,520) (1,112,420) (439,331)Non-GAAP adjusted net loss$(27,603,431) $(17,255,629) $(69,674,286) $(40,187,374) GAAP net loss per share - basic and diluted$(0.70) $(0.83) $(1.96) $(2.05)Adjustment to net loss (as detailed above) 0.05 0.05 0.14 0.22 Non-GAAP adjusted net loss per share$(0.65) $(0.78) $(1.82) $(1.83) Weighted average common shares outstanding, basic and diluted 42,793,047 22,117,626 38,299,548 21,920,147 (1) To reflect a non-cash charge to operating expense for Research and Development stock-based compensation.(2) To reflect a non-cash charge to operating expense for General and Administrative stock-based compensation.(3) To reflect a non-cash charge to other expense for amortization of debt issuance and discount costs and PIK interest related to the issuance of a note payable.(4) To reflect a non-cash adjustment to other income for accretion on investments.

Financial StatementPhase 1Phase 3Phase 2

12 Nov 2024

·www.globenewswire.com

Olema Oncology Reports Third Quarter 2024 Financial Results and Provides Corporate Update

Presented compelling new preclinical data demonstrating anti-tumor activity for OP-3136, a novel KAT6 inhibitor, with enhanced activity of palazestrant combinations at EORTC-NCI-AACR Symposium on Molecular Targets and Cancer Therapeutics (ENA 2024) New clinical data for palazestrant (OP-1250) in combination with ribociclib to be presented at the San Antonio Breast Cancer Symposium (SABCS) in DecemberIND submission for OP-3136 expected before year end; clinical study to initiate early 2025Cash, cash equivalents, and marketable securities of $214.8 million as of September 30, 2024 SAN FRANCISCO, Nov. 12, 2024 (GLOBE NEWSWIRE) -- Olema Pharmaceuticals, Inc. (“Olema”, “Olema Oncology”, Nasdaq: OLMA), a clinical-stage biopharmaceutical company focused on the discovery, development, and commercialization of targeted therapies for breast cancer and beyond, today reported financial results for the third quarter ended September 30, 2024, and provided a corporate update. “We look forward to presenting updated data from our ongoing Phase 2 clinical study of palazestrant in combination with ribociclib in frontline metastatic breast cancer patients at SABCS in December. The OPERA-01 Phase 3 clinical trial of palazestrant as a monotherapy in second/third-line patients continues to advance and we remain on track for top-line readout in 2026,” said Sean P. Bohen, M.D., Ph.D., President and Chief Executive Officer of Olema Oncology. “At ENA 2024, we presented three new, robust preclinical data sets. Palazestrant demonstrated combinability and enhanced tumor suppression with both everolimus and capivasertib. OP-3136, our potent and selective KAT6 inhibitor, demonstrated robust anti-tumor activity as a single agent, as well as synergy and enhanced anti-tumor activity in combination with palazestrant. These data reinforce our belief in the potential of OP-3136 as an exciting new therapy for breast and other cancers, and we remain on track to submit the IND application before year end.” Recent Progress Continued enrollment of patients in OPERA-01, the pivotal Phase 3 clinical trial of palazestrant as a monotherapy in second/third-line ER+/HER2- metastatic breast cancer.Presented preclinical data for OP-3136 and palazestrant at the 36th EORTC-NCI-AACR Symposium on Molecular Targets and Cancer Therapeutics (ENA 2024) in Barcelona, Spain.Initiated Phase 1b/2 clinical study of palazestrant in combination with everolimus.Successfully completed Investigational New Drug (IND)-enabling studies for OP-3136. Anticipated Upcoming Milestones Present updated Phase 2 data showing palazestrant in combination with ribociclib at the San Antonio Breast Cancer Symposium (SABCS) in December 2024.Submit the IND application for OP-3136 to the U.S. Food and Drug Administration (FDA) before year-end; initiate the Phase 1 clinical study for OP-3136 in early 2025. Third Quarter 2024 Financial ResultsCash, cash equivalents, and marketable securities as of September 30, 2024, were $214.8 million. Net loss for the quarter ended September 30, 2024, was $34.6 million, as compared to $21.5 million for the quarter ended September 30, 2023. The increase in net loss for the third quarter was primarily related to increased spending on clinical development and research activities as a result of late-stage clinical trials for palazestrant and the advancement of our KAT6 inhibitor program, as well as general and administrative (G&A) activities. The increase was partially offset by higher interest income earned from marketable securities. GAAP research and development (R&D) expenses were $33.2 million for the quarter ended September 30, 2024, as compared to $19.5 million for the quarter ended September 30, 2023. The increase in R&D expenses was primarily related to increased spending on clinical operations and development-related activities as we continue to advance palazestrant through late-stage clinical trials, research-related activities associated with the advancement of our KAT6 inhibitor program, and personnel related costs, including an increase in non-cash stock-based compensation expense of $1.5 million. Non-GAAP R&D expenses were $28.9 million for the quarter ended September 30, 2024, which excluded $4.3 million non-cash stock-based compensation expense. Non-GAAP R&D expenses were $16.7 million for the quarter ended September 30, 2023, excluding $2.8 million non-cash stock-based compensation expense. A reconciliation of GAAP to non-GAAP financial measures used in this press release can be found at the end of this press release. GAAP G&A expenses were $4.4 million for the quarter ended September 30, 2024, as compared to $3.9 million for the quarter ended September 30, 2023. The increase in G&A expenses was primarily due to increased spending on corporate-related costs and an increase in non-cash stock-based compensation expense of less than $0.1 million. Non-GAAP G&A expenses were $3.0 million for the quarter ended September 30, 2024, excluding $1.3 million non-cash stock-based compensation expense. Non-GAAP G&A expenses were $2.6 million for the quarter ended September 30, 2023, excluding $1.3 million non-cash stock-based compensation expense. A reconciliation of GAAP to non-GAAP financial measures used in this press release can be found at the end of this press release. About Palazestrant (OP-1250)Palazestrant (OP-1250) is a novel, orally available small molecule with dual activity as both a complete estrogen receptor (ER) antagonist (CERAN) and selective ER degrader (SERD). It is currently being investigated in patients with recurrent, locally advanced or metastatic ER-positive (ER+), human epidermal growth factor receptor 2-negative (HER2-) breast cancer. In clinical studies, palazestrant completely blocks ER-driven transcriptional activity in both wild-type and mutant forms of metastatic ER+ breast cancer and has demonstrated anti-tumor efficacy along with attractive pharmacokinetics and exposure, favorable tolerability, CNS penetration, and combinability with CDK4/6 inhibitors. Palazestrant has been granted U.S. Food and Drug Administration (FDA) Fast Track designation for the treatment of ER+/HER2- metastatic breast cancer that has progressed following one or more lines of endocrine therapy with at least one line given in combination with a CDK4/6 inhibitor. It is being evaluated both as a single agent in an ongoing Phase 3 clinical trial, OPERA-01, and in Phase 1/2 combination studies with CDK4/6 inhibitors (palbociclib and ribociclib), a PI3Ka inhibitor (alpelisib), and an mTOR inhibitor (everolimus). For more information on OPERA-01, please visit www.opera01study.com. About OP-3136OP-3136 is a novel, orally available small molecule that potently and selectively inhibits KAT6, an epigenetic target that is dysregulated in breast and other cancers. In preclinical studies, OP-3136 has demonstrated significant anti-proliferative activity in ER+ breast cancer models and is combinable and synergistic with endocrine therapies including palazestrant and CDK4/6 inhibitors. Olema has successfully completed IND-enabling studies in support of a potential Investigational New Drug (IND) application with the FDA and expects to initiate Phase 1 clinical trials for OP-3136 in early 2025. About Olema OncologyOlema Oncology is a clinical-stage biopharmaceutical company committed to transforming the standard of care and improving outcomes for women living with cancer. Olema is advancing a pipeline of novel therapies by leveraging our deep understanding of endocrine-driven cancers, nuclear receptors, and mechanisms of acquired resistance. Our lead product candidate, palazestrant (OP-1250), is a proprietary, orally available complete estrogen receptor (ER) antagonist (CERAN) and a selective ER degrader (SERD), currently in a Phase 3 clinical trial called OPERA-01. In addition, Olema is developing a potent KAT6 inhibitor (OP-3136). Olema is headquartered in San Francisco and has operations in Cambridge, Massachusetts. For more information, please visit us at www.olema.com. Non-GAAP Financial InformationThe results presented in this press release include both GAAP information and non-GAAP information. As used in this release, non-GAAP R&D expense is defined by Olema as GAAP R&D expense excluding stock-based compensation expense, and non-GAAP G&A expense is defined by Olema as GAAP G&A expense excluding stock-based compensation expense. We use these non-GAAP financial measures to evaluate our ongoing operations and for internal planning and forecasting purposes. We believe that non-GAAP financial information, when taken collectively, may be helpful to investors because it provides consistency and comparability with past financial performance. However, non-GAAP financial information is presented for supplemental informational purposes only, has limitations as an analytical tool, and should not be considered in isolation or as a substitute for financial information presented in accordance with GAAP. Other companies, including companies in our industry, may calculate similarly titled non-GAAP measures differently or may use other measures to evaluate their performance, all of which could reduce the usefulness of our non-GAAP financial measures as tools for comparison. Investors are encouraged to review the related GAAP financial measures and the reconciliation of these non-GAAP financial measures to their most directly comparable GAAP financial measures and not rely on any single financial measure to evaluate our business. Forward Looking StatementsStatements contained in this press release regarding matters that are not historical facts are “forward-looking statements” within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934. Words such as “anticipate,” “believe,” “could,” “expect,” “goal,” “may,” “potential,” “upcoming,” “will” and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances) are intended to identify forward-looking statements. These statements include those related to the timelines for initiation and enrollment for potential clinical studies and for results of clinical trials of palazestrant (OP-1250) and OP-3136, each as a monotherapy and in combination trials, Olema’s financial condition and resources, results of operations, cash position, potential beneficial characteristics including but not limited to safety, tolerability, activity, efficacy and therapeutic effects of palazestrant, the potential of palazestrant to advance the standard of care for women living with cancer, combinability with other drugs, palazestrant, or OP-3136, and the sufficiency and timing of Olema’s preclinical program, including the potential beneficial characteristics of its KAT6 inhibitor compounds and the timing of a potential IND application and advancement into clinical development for OP-3136. Because such statements deal with future events and are based on Olema’s current expectations, they are subject to various risks and uncertainties, and actual results, performance or achievements of Olema could differ materially from those described in or implied by the statements in this press release. These forward-looking statements are subject to risks and uncertainties, including, without limitation, those discussed in the section titled “Risk Factors” in Olema’s Quarterly Report on Form 10-Q for the quarter ended September 30, 2024, and future filings and reports that Olema makes from time to time with the U.S. Securities and Exchange Commission. Except as required by law, Olema assumes no obligation to update these forward-looking statements, including in the event that actual results differ materially from those anticipated in the forward-looking statements. Olema Pharmaceuticals, Inc. Condensed Consolidated Balance Sheets Data (in thousands) September 30,December 31, 2024 2023 Cash, cash equivalents and marketable securities $214,763$261,807 Total assets 230,173 276,945 Total current liabilities 30,700 21,621 Total liabilities 31,262 23,050 Total stockholders’ equity 198,911 253,895 Total liabilities and stockholders’ equity $ 230,173$ 276,945 Olema Pharmaceuticals, Inc. Condensed Consolidated Statements of Operations (In thousands, except share and per share data) Three Months Ended September 30, Nine Months Ended June 30, 2024 2023 2024 2023 Operating expenses: Research and development (1)$33,226 $19,453 $92,218 $60,268 General and administrative (2) 4,395 3,889 13,272 14,277 Total operating expenses 37,621 23,342 105,490 74,545 Loss from operations (37,621) (23,342) (105,490) (74,545) Other income: Interest income 2,928 1,919 9,388 4,774 Other income (expense) 138 (79) 195 (112) Total other income 3,066 1,840 9,583 4,662 Net loss$ (34,555)$ (21,502) $ (95,907)$ (69,883) Net loss per share, basic and diluted$(0.60)$(0.48) $(1.80)$(1.66) Weighted average shares used to compute net loss per share, basic and diluted 57,262,803 44,977,161 53,194,081 41,999,978 Reconciliation of GAAP to Non-GAAP Information (In thousands) Three Months Ended September 30, Nine Months Ended June 30, 2024 2023 2024 2023 (1) Research and development reconciliation GAAP research and development (3)$33,226 $19,453 $92,218 $60,268 Less: share-based compensation expense 4,280 2,801 11,925 8,858 Non-GAAP research and development$ 28,946 $ 16,652 $ 80,293 $ 51,410 (2) General and administrative reconciliation GAAP general and administrative$4,395 $3,889 $13,272 $14,277 Less: share-based compensation expense 1,346 1,304 4,334 4,047 Non-GAAP general and administrative$ 3,049 $ 2,585 $ 8,938 $ 10,230 (3) Research and development expenses for the nine-months ended September 30, 2024 include a $5.0 million milestone payment in connection to the Aurigene Agreement. IR and Media ContactCourtney O’Konek, Vice President, Corporate Communicationsmedia@olema.com

Phase 1Financial StatementPhase 2Phase 3

12 Nov 2024

·www.globenewswire.com

Celcuity Inc. To Present at Upcoming Stifel and Jefferies Investor Conferences

MINNEAPOLIS, Nov. 12, 2024 (GLOBE NEWSWIRE) -- Celcuity Inc. (Nasdaq: CELC), a clinical-stage biotechnology company pursuing development of targeted therapies for oncology, today announced that Brian Sullivan, Chief Executive Officer, and Co-founder of Celcuity, will present and be available for one-on-one investor meetings at the following investor conferences: A fireside chat at the Stifel 2024 Healthcare Conference at 1:15 p.m. ET on Tuesday, November 19, 2024. A live webcast will be available using this weblink: https://wsw.com/webcast/stifel96/celc/2061562; andA fireside chat at the Jefferies London Healthcare Conference at 11:30 a.m. GMT/6:30 a.m. ET on Thursday, November 21, 2024. A live webcast will be available using this weblink: https://wsw.com/webcast/jeff315/celc/1814988 About Celcuity Celcuity is a clinical-stage biotechnology company focused on development of targeted therapies for treatment of multiple solid tumor indications. The company's lead therapeutic candidate is gedatolisib, a potent, pan-PI3K and mTOR inhibitor. Its mechanism of action and pharmacokinetic properties are highly differentiated from other currently approved and investigational therapies that target PI3K or mTOR alone or together. A Phase 3 clinical trial, VIKTORIA-1, evaluating gedatolisib in combination with fulvestrant with or without palbociclib in patients with HR+/HER2- advanced breast cancer is currently enrolling patients. More detailed information about the VIKTORIA-1 study can be found at ClinicalTrials.gov . A Phase 1b/2 clinical trial, CELC-G-201, evaluating gedatolisib in combination with darolutamide in patients with metastatic castration resistant prostate cancer, is enrolling patients. A Phase 3 clinical trial, VIKTORIA-2, evaluating gedatolisib plus a CDK4/6 inhibitor and fulvestrant as first-line treatment for patients with HR+/HER2- advanced breast cancer is expected to begin enrolling patients in the second quarter of 2025. Celcuity is headquartered in Minneapolis. Further information about Celcuity can be found at www.celcuity.com . Follow us on LinkedIn and Twitter . Contacts: Celcuity Inc. Brian Sullivan, bsullivan@celcuity.com Vicky Hahne, vhahne@celcuity.com (763) 392-0123 ICR Westwicke Maria Yonkoski, maria.yonkoski@westwicke.com (203) 682-7167

Phase 3

100 Deals associated with Palbociclib

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KEGG	Wiki	ATC	Drug Bank
D10372	Palbociclib	L01XE33	DB09073

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Metastatic breast cancer	JP	Pfizer Japan, Inc.	27 Sep 2017
Hormone receptor positive HER2 negative breast cancer	US	Pfizer Inc.	19 Feb 2016
ER-positive/HER2-negative Breast Cancer	US	Pfizer Inc.	03 Feb 2015

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Human Papillomavirus-Related Squamous Cell Carcinoma	Phase 3	US	Pfizer Inc.	06 Apr 2022
Squamous Cell Carcinoma of Head and Neck	Phase 3	US	Pfizer Inc.	06 Apr 2022
Breast cancer recurrent	Phase 3	AT	Pfizer Inc.	27 Aug 2019
Breast cancer recurrent	Phase 3	FR	Pfizer Inc.	27 Aug 2019
Breast cancer recurrent	Phase 3	HU	Pfizer Inc.	27 Aug 2019
Breast cancer recurrent	Phase 3	IT	Pfizer Inc.	27 Aug 2019
Breast cancer recurrent	Phase 3	ES	Pfizer Inc.	27 Aug 2019
Breast cancer recurrent	Phase 3	CH	Pfizer Inc.	27 Aug 2019
HR Positive/HER2 Negative/Node positive breast cancer	Phase 3	AT	Pfizer Inc.	27 Aug 2019
HR Positive/HER2 Negative/Node positive breast cancer	Phase 3	FR	Pfizer Inc.	27 Aug 2019

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT03280303 (POLARIS, Pubmed \| Oncologist)	Not Applicable	Hormone receptor positive HER2 negative breast cancer First line \| Second line hormone receptor-positive \| human epidermal growth factor receptor 2-negative	1,250	Palbociclib + Endocrine Therapy in 1L setting	oahxexzkho(ptcvjoiewh) = ukieohfngf tfrszjbsdl (csmqjjicqh, 42.0 - not estimable) View more	Positive	30 Oct 2024
				Palbociclib + Endocrine Therapy in ≥2L setting	oahxexzkho(ptcvjoiewh) = jvqwzysivn tfrszjbsdl (csmqjjicqh, 31.2 - 40.8) View more
NCT04191499 (INAVO120, FDA) Manual	Phase 2/3	PIK3CA mutation/HR-positive/HER2-negative Breast Cancer Hormone Receptor Positive \| HER2 Negative \| PIK3CA Mutation	325	Inavolisib 9 mg + Palbociclib + Fulvestrant	qfiyfafbxn(gjcqcgpnhp) = kftjrxrfyx mpwdycbseu (tcyuooawcf, 11.3 - 20.5) View more	Positive	10 Oct 2024
				Placebo + Palbociclib + Fulvestrant	qfiyfafbxn(gjcqcgpnhp) = ygigffxyyj mpwdycbseu (tcyuooawcf, 5.6 - 9.3) View more
Phase II trial (ASTRO)	Phase 2	Metastatic breast cancer ctDNA	35	Palbociclib	lkgpojwghd(jcgadizxly) = kjrhrxqhsr eyjlahvwev (rfuwqosuwg ) View more	Positive	01 Oct 2024
NCT02028507 (PEARL \| GEICAM/2013-02 PEARL, CTgov)	Phase 3	Metastatic breast cancer \| Hormone receptor positive HER2 negative breast cancer	693	Palbociclib+Exemestane (Cohort 1: Palbociclib Plus Exemestane)	hjueizxhdx(xnxpjxjuec) = gzdzkiatph zjinttwved (gezvufsxzs, ozcrlqarjy - xzdqnvyane) View more	-	25 Sep 2024
				Fulvestrant+Palbociclib (Cohort 2: Palbociclib Plus Fulvestrant)	hjueizxhdx(xnxpjxjuec) = kndvhbijey zjinttwved (gezvufsxzs, bkognzubsn - muxkykvzti) View more
NCT02913430 (CTgov)	Early Phase 1	Metastatic breast cancer	7	Fulvestrant+Palbociclib (Fulvestrant + Palbociclib)	qxdjzwvfix(jjxsamtizc) = adkecizkie sumpzeumhc (autexnjtpt, okcmwhpzct - fdtqmuboay) View more	-	23 Sep 2024
				Tamoxifen+Palbociclib (Tamoxifen + Palbociclib)	qxdjzwvfix(jjxsamtizc) = lrderkliyc sumpzeumhc (autexnjtpt, vfotmwtxzt - kuyzcxkgra) View more
NCT05399329 (ESMO2024) Manual	Not Applicable	Hormone receptor positive HER2 negative breast cancer Second line \| First line HER2 Negative \| Hormone Receptor Positive	693	Palbociclib + Endocrine Therapy (1L)	qyuwbkgaln(zxnwbwwmjb) = duuekvddvi kioynsvntj (nrphvjplrd, 21.4 - 30.4) View more	Positive	16 Sep 2024
				Palbociclib + Endocrine Therapy (2L)	qyuwbkgaln(zxnwbwwmjb) = coccseyrgm kioynsvntj (nrphvjplrd, 11.7 - 18.3) View more
ESMO2024	Not Applicable	-	-	Palbociclib + Endocrine Therapy	cxzmxihyfg(grlclvkzyr) = hbgpeltkqw atsyqkwpga (lsiamowlgc ) View more	-	16 Sep 2024
				palbociclib (ECOG <2)	cxzmxihyfg(grlclvkzyr) = zaroqtldbi atsyqkwpga (lsiamowlgc ) View more
NCT03425838 (SONIA-trial, ESMO2024)	Phase 3	Advanced breast cancer	329	Palbociclib levels below median	jqdgvgowfv(wfgybyiquc): HR = 1.0 (90% CI, 0.76 - 1.35)	Negative	16 Sep 2024
				Palbociclib levels above median
NCT01864746 (PENELOPE-B, ESMO2024)	Phase 3	Neoadjuvant HR+ \| HER2-	1,250	Palbociclib 125mg	zqsbaamjkh(gjfhxcnliv) = zrqocxpgfp wgkgbkbrdb (oliidgzgaj )	Negative	16 Sep 2024
				Placebo	zqsbaamjkh(gjfhxcnliv) = ywnoawhvmv wgkgbkbrdb (oliidgzgaj )
ESMO2024	Not Applicable	Hormone receptor positive HER2 negative breast cancer	-	1st-line Palbociclib+Aromatase Inhibitors	jsjrpkwrcr(odabqefxzm) = jhnbfbcjfx kkfbeymvjx (vbjtntbadw, 49.1 - 53.3) View more	-	16 Sep 2024

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