On February 28, 2024 G1 Therapeutics, Inc. (Nasdaq: GTHX), a commercial-stage oncology company, reported a corporate and financial update for the fourth quarter and full year ended December 31, 2023 (Press release, G1 Therapeutics, FEB 28, 2024, View Source [SID1234640581]).

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"The strong fourth quarter 2023 vial volume growth of COSELA, which has continued through the beginning of 2024, highlights not only the importance of this unique drug to oncologists treating people living with extensive-stage small cell lung cancer, but also the significant addressable market still available to us as we drive continued penetration and growth," said Jack Bailey, Chief Executive Officer of G1 Therapeutics. "Looking ahead, our primary clinical focus is on completing our ongoing trials of trilaciclib in metastatic triple negative breast cancer, including our pivotal PRESERVE 2 trial. We remain confident in the potential of trilaciclib in this trial given the robust long term survival benefit observed in prior trials and the increased statistical power for the final analysis. If successful, we would work closely with the U.S. Food and Drug Administration to expeditiously file for label expansion and bring this therapy to patients as quickly as possible."

Fourth Quarter 2023 and Recent Highlights

Financial

Recognized $13.9 Million and $46.3 Million in Net COSELA Revenue for the Fourth Quarter and Full Year 2023: Net COSELA revenue and vial volume grew 29% and 19%, respectively, during the fourth quarter over the third quarter of 2023 despite the impact of a platinum-based chemotherapy shortage. G1 recognized total revenues of $14.9 million and $82.5 million for the fourth quarter and full year of 2023, respectively.
Cash Runway Extends into 2025: G1 ended 2023 with cash, cash equivalents, and marketable securities of $82.2 million.
Clinical

Final Analysis of the Phase 3 PRESERVE 2 Trial in Metastatic Triple Negative Breast Cancer (mTNBC) is Estimated to Occur in the Third Quarter of 2024: G1 announced that the Independent Data Monitoring Committee (DMC) recommended continuation of the Phase 3 PRESERVE 2 trial, evaluating trilaciclib in combination with gemcitabine and carboplatin for 1L treatment of mTNBC, to the final analysis. The DMC did not express any concerns or recommend any other changes to the study. The final analysis will be conducted on the intent-to-treat (ITT) population. G1 remains blinded to all data. (See February 12, 2024 press release here)
Initial Efficacy Results from Ongoing Phase 2 Antibody-Drug Conjugate (ADC) Trial Suggest Improved Overall Survival (OS) Among Patients Receiving Trilaciclib in Combination with a TROP2 ADC: The preliminary data provided in January 2024 from the ongoing Phase 2 trial of trilaciclib in combination with the ADC sacituzumab govitecan (SG) in patients with mTNBC patients suggested clinically meaningful improvements in OS among patients receiving trilaciclib in combination with SG compared to SG alone (using historical data from the ASCENT study), including (1) median OS of 17.9 months with trilaciclib versus 12.1 months for SG alone and (2) estimated 12-month survival of 59% of patients receiving trilaciclib in combination with SG, representing a ~20% improvement over SG alone. The Company expects to provide updated OS results from this study mid-2024. (See January 8, 2024 J.P. Morgan update press release here)
Medical

Presented New Post Hoc Analyses Indicating that Patients Who Previously Received Trilaciclib Experienced Improved OS with Subsequent Anticancer Therapies (SACT): G1 presented new data at the 2023 San Antonio Breast Cancer Symposium (SABCS) from patients with mTNBC who participated in G1’s Phase 2 trial. These data indicate that patients who received trilaciclib with their cytotoxic chemotherapy during the trial and then received SACT after trilaciclib discontinuation exhibit statistically significant and clinically meaningful improvements in median OS (32.7 months versus 12.8 months; p=0.001). Additionally, median OS for patients who received prior trilaciclib was improved from the time they started their first SACT compared to patients who did not receive prior trilaciclib (14.0 months versus 5.8 months; p=0.001). (See December 5, 2023 SABCS press release here)
Presented Four Posters During the 2023 ASCO (Free ASCO Whitepaper) Quality Care Symposium: These provide new real-world evidence indicating that trilaciclib administered prior to chemotherapy in patients with extensive-stage small cell lung cancer (ES-SCLC) lowers the rate of hospitalization and cytopenia events and may improve survival. (See October 27, 2023 ASCO (Free ASCO Whitepaper) Quality Care Symposium press release here)
COSELA Recommended as a Myeloid Supportive Agent in the Updated ASCO (Free ASCO Whitepaper) SCLC Guidelines: The SCLC guidelines provide evidence-based recommendations to practicing clinicians on the management of patients with SCLC. (See October 18, 2023 ASCO (Free ASCO Whitepaper) Guidelines press release here)
Fourth Quarter and Full Year 2023 Financial Results

As of December 31, 2023, cash, cash equivalents and marketable securities totaled $82.2 million, compared to $145.1 million as of December 31, 2022.

Total revenues for the fourth quarter of 2023 were $14.9 million, including $13.9 million in net product sales of COSELA and license revenue of $1.0 million, primarily related to supply and manufacturing services from Simcere and patent and clinical trial costs reimbursed primarily by EQRx and Simcere, compared to $10.3 million in total revenues in the fourth quarter of 2022. Total revenues for the full year 2023 were $82.5 million, including net product revenue of $46.3 million from sales of COSELA and license revenue of $36.2 million, compared to total revenues of $51.3 million in the prior year.

Operating expenses for the fourth quarter of 2023 were $23.8 million, compared to $41.1 million for the fourth quarter of 2022. GAAP operating expenses include stock-based compensation expense of $3.2 million for the fourth quarter of 2023, compared to $4.4 million for the fourth quarter of 2022. Operating expenses for the full year 2023 were $122.0 million, compared to $187.5 million for the prior year. Stock-based compensation expense for the full year 2023 was $14.5 million, compared to $20.6 million for the prior year.

Cost of goods sold expense for the fourth quarter of 2023 was $1.3 million, compared to $1.0 million for the fourth quarter of 2022, primarily due to an increase in product sales. Cost of goods sold expense for the full year 2023 was $7.2 million, compared to $3.7 million for the prior year.

Research and development (R&D) expenses for the fourth quarter of 2023 were $7.4 million, compared to $16.6 million for the fourth quarter of 2022. The decrease in R&D expenses was primarily due to a decrease in the Company’s clinical program costs. R&D expenses for the full year 2023 were $43.7 million, compared to $83.3 million for the prior year.

Selling, general, and administrative (SG&A) expenses for the fourth quarter of 2023 were $15.2 million, compared to $23.6 million for the fourth quarter of 2022. The decrease in SG&A expenses was primarily due to decreases in commercialization activities, personnel costs, and medical affairs. SG&A expenses for the full year 2023 were $71.1 million, compared to $100.4 million for the prior year.

The net loss for the fourth quarter of 2023 was $10.9 million, compared to $33.6 million for the fourth quarter of 2022. Net loss for the full year 2023 was $48.0 million, compared to a net loss of $147.6 million for the prior year. The basic and diluted net loss per share for the fourth quarter of 2023 was $(0.21), compared to $(0.73) for the fourth quarter of 2022. The basic and diluted net loss per share for the full year 2023 was $(0.93) compared to $(3.38) for the prior year.

2024 Financial Guidance

G1 today provided full year 2024 financial guidance. The Company expects to generate between $60 million and $70 million in COSELA net revenue in 2024. G1’s product revenue guidance is based on expectations for continued acceleration of sales performance of COSELA in the U.S. Additionally, the Company believes that its current cash runway is sufficient to fund its operations into 2025.

Webcast and Conference Call

G1 will host a webcast and conference call at 8:30 a.m. ET today to provide a corporate and financial update for the fourth quarter and full year ended December 31, 2023.

Please note the following process to access the call via telephone: To register and receive a dial in number and unique PIN to access the live conference call, please follow this link to register online. While not required, it is recommended to join 10 minutes prior to the start of the event. A live and archived webcast will be available on the Events & Presentations page of the Company’s website: www.g1therapeutics.com. The webcast will be archived on the same page for 90 days following the event.

About COSELA (trilaciclib) for Injection

COSELA (trilaciclib) was approved by the U.S. Food and Drug Administration on February 12, 2021.

Indication

COSELA (trilaciclib) is indicated to decrease the incidence of chemotherapy-induced myelosuppression in adult patients when administered prior to a platinum/etoposide-containing regimen or topotecan-containing regimen for extensive-stage small cell lung cancer.

Important Safety Information

COSELA is contraindicated in patients with a history of serious hypersensitivity reactions to trilaciclib.

Warnings and precautions include injection-site reactions (including phlebitis and thrombophlebitis), acute drug hypersensitivity reactions, interstitial lung disease (pneumonitis), and embryo-fetal toxicity.

The most common adverse reactions (>10%) were fatigue, hypocalcemia, hypokalemia, hypophosphatemia, aspartate aminotransferase increased, headache, and pneumonia.

This information is not comprehensive. Please click here for full Prescribing Information. View Source

To report suspected adverse reactions, contact G1 Therapeutics at 1-800-790-G1TX or call FDA at 1-800-FDA-1088 or visit www.fda.gov/medwatch.

On February 28, 2024 CymaBay Therapeutics, Inc. (NASDAQ: CBAY), a clinical-stage biopharmaceutical company focused on developing therapies for liver and other chronic diseases with high unmet need, reported corporate updates and financial results for the year and fourth quarter ended December 31, 2023 (Press release, CymaBay Therapeutics, FEB 28, 2024, View Source [SID1234640580]).

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"2023 was a seminal year for CymaBay with critical achievements in the development of our investigational therapeutic, seladelpar. The Phase 3 RESPONSE data presented in 2023 and recently published in the New England Journal of Medicine, indicate that seladelpar has the potential to raise the bar in PBC second-line treatment and improve quality of life for people living with this debilitating condition," said Sujal Shah, President and CEO of CymaBay. "Our team moved at speed to submit seladelpar to regulatory agencies and with an updated breakthrough therapy designation were able to secure FDA priority review. These accomplishments were recognized with the recent announcement of the pending acquisition of CymaBay by Gilead. I am incredibly proud of the team and everything that has been achieved in 2023 to help bring seladelpar to people living with PBC, and believe that through Gilead, seladelpar can reach a broad range of people that may benefit in 2024 and beyond."

2023 and Recent Corporate Highlights

Pending Acquisition by Gilead:

On February 11, 2024, CymaBay entered into a definitive agreement with Gilead Sciences, Inc. (Gilead) under which Gilead will acquire CymaBay for $32.50 per share in cash or a total equity value of $4.3 billion. The transaction is anticipated to close during the first quarter of 2024, subject to the receipt of regulatory approvals and the satisfaction of other customary closing conditions.
Regulatory Updates and Launch Readiness:

In February 2024, the U.S. Food and Drug Administration (FDA) accepted a New Drug Application (NDA) for seladelpar, an investigational treatment for the management of primary biliary cholangitis (PBC) including pruritus in adults without cirrhosis or with compensated cirrhosis (Child Pugh A) who are inadequate responders or intolerant to ursodeoxycholic acid. The FDA has granted priority review and set a Prescription Drug User Fee Act target action date of August 14, 2024. The agency has notified the company that it is not currently planning to hold an advisory committee meeting to discuss the application.
CymaBay has received validation of its application to the U.K. Medicines Healthcare products Regulatory Agency (MHRA) for seladelpar for the treatment of PBC. Seladelpar has also been submitted to the European Medicines Agency (EMA) and validation of the application is anticipated in H1 2024. Review by both agencies is anticipated to be completed in 2025.
In the U.S., this application was further supported by the Breakthrough Therapy Designation for seladelpar, that was updated by the FDA in October 2023, with clinical results that indicate seladelpar may provide meaningful improvement over existing therapy based on a reduction in alkaline phosphatase (ALP) and improvement in pruritus in patients without cirrhosis or with compensated cirrhosis.
In preparation for the potential launch of seladelpar in the U.S. in 2024, CymaBay hired, trained and deployed a medical affairs team with a range of expertise across PBC, rare disease and liver diseases to drive PBC education. Our pre-commercial launch planning efforts in the U.S. accelerated in the fourth quarter as we built strong commercial strategy, marketing, market access, commercial operations and analytics teams with experience in launching new treatments in rare diseases and competitive therapeutics areas and markets.
Clinical Development:

In mid-2023, CymaBay announced initiation of the IDEAL study, a 52-week, placebo-controlled, randomized, Phase 3 study. The IDEAL study aimed to enroll 75 patients with PBC who have an incomplete response or intolerance to ursodeoxycholic acid (UDCA), with ALP levels greater than the upper limit of normal (ULN) but less than 1.67x ULN, and total bilirubin less than or equal to 2x ULN. To ensure IDEAL is well powered to effectively assess both liver biochemistry and pruritus impact we are now doubling the study size to 150 patients with a 2:1 ratio of patients receiving seladelpar vs. placebo. The primary outcome measure is the ALP composite of normalization and a greater than or equal to 15% decrease in ALP at 52 weeks. A key secondary endpoint is evaluating the change in pruritus Numerical Rating Scale (NRS) at six months in subjects with moderate to severe pruritus at baseline.
Enrollment in the long-term ASSURE study continues. ASSURE is an open-label study of seladelpar in patients with PBC intended to collect additional long-term safety and efficacy data to further support registration. There are now over 300 patients taking seladelpar 10 mg daily, through the study including those from prior studies of seladelpar and patients who have completed RESPONSE.
In 2023, CymaBay initiated AFFIRM, a randomized, placebo-controlled confirmatory study to evaluate the effect of seladelpar 10 mg daily on clinical outcomes in patients with compensated cirrhosis due to PBC. The AFFIRM study is planned to enroll approximately 192 patients with PBC who have compensated cirrhosis (Child-Pugh A or Child-Pugh B) based on prespecified clinical criteria. Patients will be randomly assigned using a 2:1 ratio to seladelpar or placebo for a fixed duration of three years. The primary outcome measure is the time to the first occurrence of clinical events (all-cause death, liver transplant, hospitalization for other serious liver-related events, and progression to Child-Pugh C decompensated cirrhosis). Additional key outcomes include overall survival, liver transplant-free survival, and time to hospitalization for serious liver-related events.
Presentations and Publications:

The pivotal Phase 3 RESPONSE study was presented at The Liver Meeting 2023 of the American Association for the Study of Liver Diseases, in Boston Massachusetts, and later published online in the New England Journal of Medicine in February 2024. The clinical data includes:
RESPONSE was a double-blind, placebo-controlled, global study of one-year duration that randomized 193 PBC patients in a 2:1 ratio to seladelpar 10 mg or placebo, once daily. Eligible patients had an inadequate response or intolerance to ursodeoxycholic acid (UDCA) with serum alkaline phosphatase (ALP) ≥ 1.67× the upper limit of normal (ULN) after at least 12 months of treatment.
The primary endpoint was a composite of ALP and total bilirubin previously accepted by the U.S. Food and Drug Administration (FDA) and European Medicines Agency (EMA) for registration studies in PBC. The composite endpoint was achieved in 61.7% of patients on seladelpar vs. 20.0% on placebo (p<0.0001).
The key secondary endpoint of ALP normalization occurred in 25% of patients receiving seladelpar vs. 0% for patients on placebo (p<0.0001). The average decrease in ALP for patients on seladelpar was -133.9 U/L vs. -16.9 U/L for patients on placebo (p<0.0001).
The study also measured the impact on patient-reported pruritus (itching), one of the most challenging symptoms experienced by people with PBC, as a key secondary endpoint using the daily numerical rating scale (NRS; 0-10). The pruritus endpoint was met at Month 6 among patients with baseline NRS > 4 reporting decreases of 3.2 points with seladelpar (n=65) vs. 1.7 for patients on placebo (n=20; p<0.005). Notably, these improvements were sustained through Month 12 (p<0.005). A statistically significant reduction in pruritus was also observed at Month 6 and at Month 12 for patients in the intent-to-treat population, which includes all patients irrespective of their NRS score at baseline.
CymaBay published a post-hoc analysis of the Phase 3 ENHANCE study in the open access journal Hepatology, demonstrating the impact of seladelpar on serum interleukin-31 (IL-31) levels and its correlation with pruritus improvement in people with PBC.
Financial Updates:

Held $416.2 million in cash, cash equivalents and investments as of December 31, 2023.

Fourth Quarter and Year Ended December 31, 2023, Financial Results:

Collaboration revenue recognized for the year ended December 31, 2023 was $31.1 million and was associated with the collaboration and license agreement with Kaken Pharmaceutical Co., Ltd. (Kaken) entered into in January 2023, to develop and commercialize seladelpar in Japan. As reported earlier, $31.0 million of this revenue was recognized upon completion of the initial technology transfer to Kaken in the second quarter of 2023. Of the $34.2M upfront payment received from Kaken, $2.7 million remains deferred as of December 31, 2023 and will be recognized upon completion of CymaBay’s ongoing clinical data delivery and CMC development performance obligations.
Research and development expenses for the three months ended December 31, 2023 and 2022 were $22.8 million, and $16.2 million, respectively. Research and development expenses for the years ended December 31, 2023, and 2022 were $80.8 million and $68.0 million, respectively. Research and development expenses for the three months and year ended December 31, 2023 increased compared to the corresponding periods in 2022 driven by higher clinical activities supporting our clinical studies and higher spend supporting our regulatory filings.
General and administrative expenses for the three months ended December 31, 2023 and 2022 were $19.8 million and $7.2 million, respectively. General and administrative expenses for the years ended December 31, 2023 and 2022 were $51.9 million and $25.1 million, respectively. General and administrative expenses for the three months and year ended December 31, 2023 were higher than the corresponding periods in 2022 driven by investments to prepare for potential commercialization of seladelpar in PBC as well as increase in other corporate expenses.
Net loss for the three months ended December 31, 2023 and 2022 was $41.9 million and $26.6 million, or ($0.35) and ($0.30) per share, respectively. Net loss for the year ended December 31, 2023 and 2022 was $105.4 million and $106.0 million, or ($0.99) and ($1.21) per share, respectively. Net loss for the three months ended December 31, 2023 was higher than the three months ended December 31, 2022 primarily due to higher operating expenses. Net loss in the year ended December 31, 2023 was slightly lower than the corresponding period in 2022 due primarily to $31.0 million of collaboration revenue related to the Kaken upfront payment during the second quarter of 2023 and higher interest income earned on our investments and other income due to refundable tax credits, offset in part by an increase in operating expenses and interest expense from the Abingworth development financing arrangement.

On February 28, 2024 Crinetics Pharmaceuticals, a clinical-stage pharmaceutical company focused on the discovery, development and commercialization of novel therapeutics for rare endocrine diseases and endocrine-related tumors, reported that it has agreed to sell 8,333,334 shares of its common stock at a price of $42.00 per share to a select group of institutional and accredited healthcare specialist investors in an oversubscribed private placement (Press release, Crinetics Pharmaceuticals, FEB 28, 2024, View Source [SID1234640579]). Crinetics anticipates the gross proceeds from the private placement to be approximately $350 million, before deducting any offering-related expenses. The private placement is expected to close on or about March 1, 2024, subject to the satisfaction of customary closing conditions.

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The financing includes participation from new and existing institutional investors, including Adage Capital Partners L.P., Driehaus Capital Management, EcoR1 Capital, First Light Asset Management, GordonMD Global Investments LP, Invus, Janus Henderson Investors, Paradigm BioCapital, Perceptive Advisors, Rock Springs Capital, as well as multiple leading mutual funds.

Leerink Partners, Piper Sandler, Baird, Citizens JMP, H.C. Wainwright & Co., and LifeSci Capital are acting as placement agents to the Company in connection with the private placement.

Crinetics intends to use the net proceeds from the private placement to fund research and development of its clinical-stage product candidates, other research programs, working capital and general corporate purposes.

The offer and sale of the securities to be sold in the private placement have not been registered under the Securities Act of 1933, as amended (the "Securities Act"), or any state or other applicable jurisdiction’s securities laws, and such securities may not be offered or sold in the United States absent registration or an applicable exemption from the registration requirements of the Securities Act and applicable state or other jurisdictions’ securities laws. Crinetics has agreed to file a registration statement with the U.S. Securities and Exchange Commission (the "SEC") registering the resale of the shares of common stock issued in the private placement no later than the 40th day after the pricing of the private placement.

This press release shall not constitute an offer to sell or the solicitation of an offer to buy, nor shall there be any sale of these securities in any jurisdiction in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of any such jurisdiction.

On February 28, 2024 Black Diamond Therapeutics, Inc. (Nasdaq: BDTX), a clinical-stage oncology company developing MasterKey therapies that target families of oncogenic mutations in patients with genetically defined cancers, reported that its Chief Executive Officer, Mark A. Velleca, M.D., Ph.D., will participate in a panel discussion on lung cancer at the TD Cowen 44th Annual Health Care Conference on Wednesday, March 6, 2024, at 9:10 a.m. ET in Boston, MA (Press release, Black Diamond Therapeutics, FEB 28, 2024, View Source [SID1234640578]).

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A live webcast of the panel can be accessed by visiting the investor relations section of the Company’s website at: www.blackdiamondtherapeutics.com. A replay of the webcast will also be available and archived for 90 days following the event.

On February 28, 2024 BioLineRx Ltd., a commercial stage biopharmaceutical company pursuing life-changing therapies in oncology and rare diseases, reported that the first patient has been dosed in the randomized CheMo4METPANC Phase 2 combination clinical trial evaluating the company’s CXCR4 inhibitor motixafortide, the PD-1 inhibitor cemiplimab, and standard of care chemotherapies gemcitabine and nab-paclitaxel, versus gemcitabine and nab-paclitaxel alone, in first-line pancreatic cancer (PDAC) (Press release, BioLineRx, FEB 28, 2024, View Source [SID1234640577]). The investigator-initiated trial is being conducted in collaboration with Columbia University and is the first large, multi-center, randomized study evaluating motixafortide with a PD-1 inhibitor and first-line PDAC chemotherapies.

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"Pancreatic ductal adenocarcinoma (PDAC) has had limited responses to traditional immunotherapy, resulting in a poor prognosis for patients and an urgent need for new treatment approaches," said Philip Serlin, Chief Executive Officer of BioLineRx. "We are encouraged by our early pilot data and look forward to continuing to advance the expanded, randomized Phase 2 CheMo4METPANC trial for patients living with this cancer."

Findings from the single-arm pilot phase of the CheMo4METPANC trial will be shared by Dr. Manji at the 10th Annual Immuno-Oncology (IO) 360° Summit in Brooklyn, New York on Thursday, February 29, 2024. The findings were previously presented during an oral presentation at the American Association of Cancer Research (AACR) (Free AACR Whitepaper) Special Conference on Pancreatic Cancer in Boston, Massachusetts, September 28, 2023. As of July 2023, 7 of the 11 patients (64%) in the pilot phase experienced a partial response (PR) of which 5 (45%) were confirmed PRs at the time of the data cut; one patient experienced resolution of the hepatic (liver) metastatic lesion; and three patients (27%) experienced stable disease, resulting in a disease control rate of 91%.

Motixafortide, BioLineRx’s lead therapeutic candidate, was approved by the U.S. Food & Drug Administration (FDA) in September 2023, in combination with filgrastim (G-CSF), to mobilize hematopoietic stem cells for collection and subsequent autologous transplantation in patients with multiple myeloma, under the brand name APHEXDA. Motixafortide is also being evaluated in a Phase 1 clinical trial evaluating motixafortide as a monotherapy and in combination with natalizumab for CD34+ hematopoietic stem cell (HSC) mobilization for gene therapies in sickle cell disease (SCD).

About CheMo4METPANC Phase 2 Clinical Trial
The multi-center CheMo4METPANC Phase 2 clinical trial (ClinicalTrials.gov Identifier: NCT04543071) is a randomized, investigator-initiated clinical trial in first line metastatic pancreatic cancer. Sponsored by Columbia University, and supported equally by BioLineRx and Regeneron, the study is evaluating the combination of CXCR4 inhibitor motixafortide, PD-1 inhibitor cemiplimab, and standard of care chemotherapies gemcitabine and nab-paclitaxel, versus gemcitabine and nab-paclitaxel alone, in 108 patients. The trial’s primary endpoint is progression free survival (PFS). Secondary objectives include safety, response rate, disease control rate, duration of clinical benefit and overall survival.

About Pancreatic Cancer
Pancreatic cancer has a low rate of early diagnosis and a poor prognosis. In the United States in 2024, an estimated 66,000 adults will be diagnosed with the disease, which accounts for approximately 3% of all cancers in the U.S. and about 7% of all cancer deaths.1 Worldwide, an estimated 496,000 people were diagnosed with the disease in 2020. In the U.S., if the cancer is detected at an early stage when surgical removal of the tumor is possible, the 5-year relative survival rate is 44%. About 12% of people are initially diagnosed at this stage. If the cancer has spread to surrounding tissues or organs, the 5-year relative survival rate is 15%. For the 52% of patients who are initially diagnosed with metastatic cancer, the 5-year relative survival rate is 3%.2 In particular, hepatic (liver) metastases are a critical risk factor driving poor prognoses for patients with metastatic PDAC. These data highlight the need for the development of new therapeutic options.

About Motixafortide in Cancer Immunotherapy
Motixafortide inhibits CXCR4, a chemokine receptor and a well validated therapeutic target that is over-expressed in many human cancers including pancreatic ductal adenocarcinoma (PDAC). Motixafortide leverages the expression of the CXCR4 receptor on different immune cells and potentiates the immune system against the tumor. Among CXCR4-expressing immune cells, some exhibit anti-tumoral activity, such as effector T cells and some exhibit pro-tumoral activity and support tumor growth. By blocking the CXCR4 receptor, motixafortide was shown in a Phase 2 study in pancreatic cancer patients to enhance anti-tumoral activity and to ameliorate the pro-tumoral activities by modulating the effector/suppressor cell ratio towards a proinflammatory profile.