On February 26, 2024 FibroGen, Inc. (NASDAQ: FGEN) reported financial results for the fourth quarter and full year 2023 and provided an update on the company’s recent developments (Press release, FibroGen, FEB 26, 2024, View Source [SID1234640449]).

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"We are extremely excited about the company’s prospects in 2024," said Thane Wettig, Chief Executive Officer, FibroGen. "In this year alone, we will obtain data read-outs from our two late-stage pancreatic cancer trials, start a Phase 2 metastatic castration-resistant prostate cancer trial, file an immuno-oncology IND, and potentially receive approval for roxadustat in chemotherapy-induced anemia in China. Furthermore, the continued strength of our China business, accelerated realization of our corporate cost reduction program, and our strong balance sheet provide us a cash runway into 2026. These unique and exciting programs, combined with the quality of our talented colleagues, provide a strong foundation to create significant value for shareholders relative to our current valuation."

Upcoming Milestones:

Pamrevlumab

Topline data from the PanCAN Precision Promise℠ Phase 2/3 study of pamrevlumab in metastatic pancreatic cancer expected in 2Q 2024.
Topline data from the LAPIS Phase 3 study of pamrevlumab in locally advanced unresectable pancreatic cancer (LAPC) expected in 2Q 2024.
Roxadustat

Expect approval decision for roxadustat in chemotherapy-induced anemia (CIA) in China in mid-2024. If approved, FibroGen will receive a $10M milestone payment from AstraZeneca.
Oncology Pipeline

Additional data from Phase 1 monotherapy study of FG-3246 in metastatic castration-resistant prostate cancer (mCRPC) expected in 1Q 2024.
Anticipate the initiation of a Phase 2 study of FG-3246 in mCRPC in 2H 2024.
Anticipate the filing of two INDs: FG-3165 (anti-Gal9 antibody) in 1Q 2024 and FG-3175 (anti-CCR8 antibody) in 2025.
Recent Developments and Key Highlights of 2023:

Pamrevlumab

Announced graduation and completion of the pamrevlumab arm in Precision Promise℠, Pancreatic Cancer Action Network’s Phase 2/3 adaptive platform trial for metastatic pancreatic cancer.
Pamrevlumab, in Stage 1 of the trial, achieved a protocol pre-specified ≥ 35% predictive probability of success for the primary endpoint of overall survival at the completion of the trial.
Roxadustat

Regained all rights to roxadustat from AstraZeneca in the United States and other AstraZeneca territories, except China and South Korea.
Presented data from Phase 3 MATTERHORN study of roxadustat in patients with anemia of lower risk transfusion-dependent myelodysplastic syndromes at American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting.
Corporate

Thane Wettig appointed Chief Executive Officer.
Successful execution of cost reduction plan, resulting in a reduction of total annualized expenses of $120 million.
China:

Fourth quarter FibroGen’s net product revenue under U.S. GAAP from the sale of roxadustat in China was $23.5 million compared to $23.4 million in the fourth quarter of 2022.
Full year 2023 FibroGen’s net product revenue under U.S. GAAP from the sale of roxadustat in China was $100.9 million compared to $82.9 million in the full year 2022, an increase of 22%.
Fourth quarter total roxadustat net sales in China¹ by FibroGen and the distribution entity jointly owned by FibroGen and AstraZeneca (JDE) was $66.5 million, compared to $53.1 million in the fourth quarter of 2022, an increase of 25%.
Full year 2023 total roxadustat net sales in China¹ by FibroGen and the JDE was $284.1 million, compared to $208.8 million in the full year 2022, an increase of 36%, driven by over 41% growth in volume.
Roxadustat continues to be the number one brand based on value share in the anemia of CKD market in China and has secured renewal on the National Reimbursement Drug List.
For 2024, we anticipate FibroGen’s full year net product revenue under U.S. GAAP to range between $120 million to $135 million, representing full year roxadustat net sales in China¹ by FibroGen and the JDE to range between $300 million to $340 million.
Financial:

Total revenue for the fourth quarter of 2023 was $27.1 million, as compared to $34.4 million for the fourth quarter of 2022. Reduction primarily driven by the change in net product revenue assumptions under U.S. GAAP and drug product revenue shipment timing.
Total revenue for full year 2023 was $147.8 million as compared to $140.7 million in 2022.
Net loss for the fourth quarter of 2023 was $56.2 million, or $0.57 net loss per basic and diluted share, compared to a net loss of $66.2 million, or $0.70 net loss per basic and diluted share one year ago.
Net loss for the year was $284.2 million, or $2.92 net loss per basic and diluted share, compared to a net loss of $293.7 million, or $3.14 net loss per basic and diluted share one year ago.
At December 31, 2023, FibroGen had $248.1 million in cash – defined as cash, cash equivalents, investments, and accounts receivable.
We expect our cash, cash equivalents, investments, and accounts receivable to be sufficient to fund our operating plans into 2026.
Conference Call and Webcast Details

FibroGen will host a conference call and webcast today, Monday, February 26, 2024, at 5:00 PM Eastern Time to discuss financial results and provide a business update. Interested parties may access a live audio webcast of the conference call via the "Investor Relations" page of the Company’s website at www.fibrogen.com. To access the call by phone, please go to this link (registration link), and you will be provided with dial in details. To avoid delays, we encourage participants to dial into the conference call fifteen minutes ahead of the scheduled start time. A replay of the webcast will also be available for a limited time at the following link (webcast replay).

About Pamrevlumab

Pamrevlumab is a potential first-in-class antibody being developed by FibroGen to inhibit the activity of connective tissue growth factor (CTGF). Pamrevlumab is in Phase 3 clinical development for the treatment of locally advanced unresectable pancreatic cancer (LAPC) and in Phase 2/3 for the treatment of metastatic pancreatic cancer. The U.S. Food and Drug Administration has granted Orphan Drug Designation, and Fast Track designation to pamrevlumab for the treatment of patients with LAPC. Pamrevlumab has demonstrated a safety and tolerability profile that has supported ongoing clinical investigation in LAPC and metastatic pancreatic cancer. Pamrevlumab is an investigational drug and not approved for marketing by any regulatory authority. For information about our pamrevlumab studies please visit www.clinicaltrials.gov.

About Roxadustat

Roxadustat, an oral medication, is the first in a new class of medicines comprising HIF-PH inhibitors that promote erythropoiesis, or red blood cell production, through increased endogenous production of erythropoietin, improved iron absorption and mobilization, and downregulation of hepcidin. Roxadustat is in clinical development for chemotherapy-induced anemia (CIA) and a Supplemental New Drug Application (sNDA) has been accepted by the China Health Authority.

Roxadustat is approved in China, Europe, Japan, and numerous other countries for the treatment of anemia of CKD in adult patients on dialysis (DD) and not on dialysis (NDD). Several other licensing applications for roxadustat have been submitted by partners, Astellas and AstraZeneca, to regulatory authorities across the globe, and are currently under review. Astellas and FibroGen are collaborating on the development and commercialization of roxadustat for the potential treatment of anemia in territories including Japan, Europe, Turkey, Russia, and the Commonwealth of Independent States, the Middle East, and South Africa. AstraZeneca and FibroGen continue to collaborate on the development and commercialization of roxadustat in China.

On February 26, 2024 Fate Therapeutics, Inc. (NASDAQ: FATE), a clinical-stage biopharmaceutical company dedicated to bringing a first-in-class pipeline of induced pluripotent stem cell (iPSC)-derived cellular immunotherapies to patients with cancer and autoimmune disorders, reported business highlights and financial results for the fourth quarter and full year ended December 31, 2023 (Press release, Fate Therapeutics, FEB 26, 2024, View Source [SID1234640448]).

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"We have started the year with strong momentum across our iPSC product platform in oncology and autoimmunity, including the award of a grant by the California Institute of Regenerative Medicine to support Phase 1 clinical investigation of our off-the-shelf FT819 CAR T-cell program in systemic lupus erythematosus," said Scott Wolchko, President and Chief Executive Officer of Fate Therapeutics. "We have also treated the first patient with FT522, our off-the-shelf CAR NK cell program targeting CD19+ B cells, which is our first product candidate to incorporate our proprietary Alloimmune Defense Receptor technology that is designed to reduce or eliminate the need for administration of intense chemotherapy conditioning to patients. In addition, we have initiated the Phase 1 study of our FT825 / ONO-8250 CAR T-cell program in solid tumors, which incorporates seven synthetic controls of cell function including a novel cancer-specific binding domain targeting HER2. We are well positioned to generate initial clinical data across these off-the-shelf programs during 2024."

FT819 iPSC-derived CAR T-cell Program

CLIN2 Grant Awarded by CIRM to Fund FT819 Phase 1 Autoimmunity Study in SLE. In February, the Company was awarded $7.9 million by the California Institute for Regenerative Medicine (CIRM) to support clinical investigation of FT819 in patients with systemic lupus erythematosus (SLE). FT819 is the Company’s off-the-shelf CAR T-cell product candidate that incorporates several novel synthetic controls of cell function, including the integration of a novel CD19-targeted 1XX chimeric antigen receptor (CAR) construct into the T-cell receptor alpha constant (TRAC) locus that is intended to promote uniform CAR expression, enhance T-cell potency, and prevent graft-versus-host disease (GvHD). The multi-center, Phase 1 clinical trial for SLE is designed to evaluate the safety, pharmacokinetics, and anti-B-cell activity of a single dose of FT819 administered following a standard three-day chemotherapy conditioning regimen. The Company is currently conducting study start-up activities at multiple U.S. clinical sites.
Dose Escalation Ongoing in Phase 1 Study for B-cell Lymphoma. The Company’s landmark clinical trial of FT819 for the treatment of relapsed / refractory B-cell malignancies is the first-ever clinical investigation of a T-cell product candidate manufactured from a clonal master iPSC line. The Company is currently enrolling patients in single-dose treatment cohorts at 540 million cells and at 1.08 billion cells using a standard three-day chemotherapy conditioning regimen. Any further clinical development of FT819 for the treatment of B-cell malignancies will be determined by the Company based on safety and activity at these higher dose levels. Clinical data previously presented by the Company from the first 11 patients with relapsed / refractory B-cell lymphoma treated with a single dose of FT819 at up to 360 million cells showed anti-tumor activity including three complete responses and one partial response, CAR T-cell expansion that peaked in the peripheral blood between Days 8 and 11, and a favorable safety profile with no dose-limiting toxicities, no events of any grade of immune effector-cell associated neurotoxicity syndrome (ICANS) or GvHD, and no events of Grade 3 or greater cytokine release syndrome (CRS).
FT825 / ONO-8250 iPSC-derived CAR T-cell Program

Phase 1 Study Initiated with HER2-targeted CAR T-cell for Advanced Solid Tumors. In January, the Company initiated enrollment of a multi-center, Phase 1 clinical trial of FT825 / ONO-8250 under its collaboration with Ono Pharmaceutical Co., Ltd. (Ono). Designed using the Company’s iPSC product platform, FT825 / ONO-8250 incorporates seven synthetic controls of cell function including a novel cancer-specific H2CasMab-2 CAR targeting human epidermal growth factor receptor 2 (HER2). Preclinical data of FT825 / ONO-8250 presented at the 2023 Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) Annual Meeting demonstrated that the profile of its novel HER2-targeted antigen binding domain is unique and differentiated from that of trastuzumab, exhibiting similar potency with greater specificity for cancer cells expressing HER2. The Phase 1 study is designed to assess the safety, pharmacokinetics, and activity of a single dose of FT825 / ONO-8250 as monotherapy and in combination with monoclonal antibody therapy in patients with advanced solid tumors.
FT522 iPSC-derived CAR NK Cell Program

First Patient Treated with ADR-armed, CD19-targeted CAR NK Cell Product Candidate. FT522 is the Company’s first product candidate incorporating its proprietary Alloimmune Defense Receptor (ADR) technology, which is designed to reduce or eliminate the need for administration of intense chemotherapy conditioning to patients receiving cell therapies. The multi-center, Phase 1 clinical trial of FT522 in patients with relapsed / refractory B-cell lymphoma is currently enrolling patients in the first three-dose cohort at 300 million cells per dose of Regimen A, which includes administration of chemotherapy conditioning. Subject to clearance of dose-limiting toxicities at this first dose level of Regimen A, enrollment is expected to commence in the first three-dose cohort at 300 million cells per dose of Regimen B without administration of chemotherapy conditioning.
Preclinical Studies Ongoing to Support Expansion into Autoimmunity. The Company is conducting a preclinical assessment of the potential for FT522 to induce CD19+ B-cell depletion across a range of autoimmune diseases, including without administration of intense chemotherapy conditioning to patients. In a disseminated Nalm6 leukemia model comprised of CD19+ target cells resistant to T-cell killing, ADR-armed, CD19-targeted CAR NK cells exhibited robust killing in vivo of CD19+ target cells in the presence of alloreactive T cells, suggesting that FT522 has the potential to deplete CD19+ B cells without administration of intense chemotherapy conditioning to patients. Additional preclinical studies are ongoing with FT522 in combination with monoclonal antibody therapy to assess the potential depletion of both CD19+ B-cell and CD38+ plasma-cell autoantibody-producing lineages.
FT576 iPSC-derived CAR NK Cell Program

Dose Escalation Ongoing in Phase 1 Multiple Myeloma Study. The Company’s multi-center, Phase 1 clinical trial of FT576, its BCMA-targeted CAR NK cell product candidate, is currently accruing patients with relapsed / refractory multiple myeloma in three-dose treatment cohorts as monotherapy as well as in combination with CD38-targeted monoclonal antibody. Using a standard three-day chemotherapy conditioning regimen, the Company has treated six patients at 1 billion cells per dose, with no dose-limiting toxicities and no reports of any grade of CRS, ICANS or GvHD. The study is currently enrolling patients at 2.5 billion cells per dose. Any further clinical development of FT576 for the treatment of multiple myeloma will be determined by the Company based on safety and activity at these higher dose levels.
Fourth Quarter 2023 Financial Results

Cash & Investment Position: Cash, cash equivalents and investments as of December 31, 2023 were $316.2 million.
Total Revenue: Revenue was $1.7 million for the fourth quarter of 2023, which was derived from the Company’s conduct of preclinical development activities for a second collaboration candidate targeting an undisclosed solid tumor antigen under its collaboration with Ono.
Total Operating Expenses: For the fourth quarter of 2023, GAAP operating expenses were $49.8 million, including research and development expenses of $31.8 million and general and administrative expenses of $17.9 million. Such amounts included $9.5 million of non-cash stock-based compensation expense.
Shares Outstanding: Common shares outstanding were 98.6 million, and preferred shares outstanding were 2.8 million, as of December 31, 2023. Each preferred share is convertible into five common shares.
Today’s Conference Call and Webcast
The Company will conduct a conference call today, Monday, February 26, 2024 at 5:00 p.m. ET to review financial and operating results for the quarter and full year ended December 31, 2023. In order to participate in the conference call, please register using the conference link here. The live webcast can be accessed under "Events & Presentations" in the Investors section of the Company’s website at www.fatetherapeutics.com. The archived webcast will be available on the Company’s website beginning approximately two hours after the event.

On February 26, 2024 Exact Sciences Corp. (Nasdaq: EXAS), a leading provider of cancer screening and diagnostic tests, reported that company management will participate in the following conference and invited investors to participate by webcast (Press release, Exact Sciences, FEB 26, 2024, View Source [SID1234640447]).

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TD Cowen 44th Annual Health Care Conference, Boston
Fireside chat on Monday, March 4, 2024 at 1:30 p.m. ET

The webcast can be accessed in the investor relations section of Exact Sciences’ website at www.exactsciences.com.

On February 26, 2024 Day One Biopharmaceuticals (Nasdaq: DAWN) ("Day One" or the "Company"), a clinical-stage biopharmaceutical company dedicated to developing and commercializing targeted therapies for people of all ages with life-threatening diseases, reported its fourth quarter and full year 2023 financial results and highlighted recent corporate achievements (Press release, Day One, FEB 26, 2024, View Source [SID1234640446]).

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"We have a monumental year ahead of us at Day One with the upcoming PDUFA date for tovorafenib," said Jeremy Bender, Ph.D., chief executive officer of Day One. "Our team is trained and ready to deliver our first expected commercial medicine to children in need of new treatment options. We also continue to advance our Phase 3 front-line trial with tovorafenib and are actively exploring other potential additions to our pipeline for children and adults living with cancer."

Program Highlights

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In October 2023, Day One announced that the U.S. Food and Drug Administration (FDA) accepted its New Drug Application (NDA) for Priority Review of tovorafenib. The FDA has assigned a Prescription Drug User Fee Act (PDUFA) target action date of April 30, 2024. The Company anticipates being eligible for a Priority Review Voucher upon potential approval of tovorafenib.

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In November 2023, Nature Medicine published the registrational Phase 2 FIREFLY-1 trial results investigating tovorafenib in patients with BRAF-altered, relapsed or progressive pediatric low-grade glioma (pLGG).

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In the fourth quarter of 2023, Day One continued its commercial preparedness for the approval and launch of tovorafenib with the hiring of 18 sales representatives in the U.S.

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The pivotal Phase 3 FIREFLY-2/LOGGIC clinical trial evaluating tovorafenib as a front-line therapy in patients aged 6 months to 25 years with pLGG continues to enroll in the United States, Canada, Europe, Australia and Asia, with more than 80 sites activated.

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Patient enrollment continues in the Phase 1b/2 substudy (102b) of the FIRELIGHT-1 trial evaluating the combination of tovorafenib with the Company’s investigational MEK inhibitor, pimasertib.

Corporate Highlights and Upcoming Milestones

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Elly Barry, MD, has been promoted to Chief Medical Officer where she will lead the execution and expansion of Day One’s clinical development programs. Most recently, Dr. Barry was Head of Clinical Development at Day One where she played an integral role in the execution of the Company’s clinical programs. Prior to joining the Company in 2021, Dr. Barry was Global Clinical Lead for Pediatric Oncology at Pfizer, as well as Head of Pfizer’s Pediatric Oncology Leadership Team where she oversaw multiple oncology clinical programs. She has replaced Raphaël Rousseau, MD, PhD, who has transitioned into a strategic advisory consulting role into the second quarter of 2024.

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Day One welcomed seasoned biotechnology veterans Habib Dable and Dr. William Grossman to its Board of Directors. Both individuals bring deep expertise and leadership in oncology to the Company’s Board.

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The recommended Phase 2 dose and schedule in the FIRELIGHT-1 clinical trial is expected in 2H 2024.

Fourth Quarter and Full Year 2023 Financial Highlights

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Cash Position: Cash, cash equivalents and short-term investments totaled $366.3 million on December 31, 2023. Based on Day One’s current operating plan, management believes it has sufficient capital resources to fund anticipated operations into 2026.

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R&D Expenses: Research and development expenses were $37.3 million and $130.5 million for the fourth quarter and full year ended December 31, 2023, respectively, as compared to $26.0 million and $85.6 million for the same periods in 2022. The increase was primarily due to additional employee compensation costs, a milestone payment, as well as clinical trial and manufacturing activities related to Day One’s lead product candidate, tovorafenib.

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G&A Expenses: General and administrative expenses were $22.2 million and $75.5 million for the fourth quarter and full year ended December 31, 2023, respectively, as compared to $16.7 million and $61.3 million for the same periods in 2022. The increase was primarily due to additional employee compensation costs, an ongoing commercial buildout, and professional service expenses to support company growth.

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Net Loss: Net loss totaled $54.5 million for the fourth quarter of 2023 with non-cash stock compensation expense of $10.8 million, compared to $40.1 million for the fourth quarter of 2022 with non-cash stock compensation expense of $6.8 million. Net loss was $188.9 million for the year ended December 31, 2023, with non-cash stock compensation expense of $39.3 million, compared to $142.2 million for the year ended December 31, 2022, with non-cash stock compensation expense of $27.2 million.

Upcoming Events

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44th Annual TD Cowen Health Care Conference
o
Management will participate in a fireside chat on Tuesday, March 5 at 9:50 a.m. ET. A live and archived audio webcast of the discussion will be available by visiting the Events & Presentations section of the Company’s website.

About Tovorafenib

Tovorafenib is an investigational, oral, brain-penetrant, highly selective type II RAF kinase inhibitor designed to target a key enzyme in the MAPK signaling pathway, which is being investigated in primary brain tumors or brain metastases of solid tumors. Tovorafenib is currently under evaluation in two pivotal clinical trials for pLGG. Tovorafenib is also being evaluated as a combination therapy for adolescent and adult patient populations with recurrent or progressive solid tumors with MAPK pathway aberrations (FIRELIGHT-1).

Tovorafenib has been granted Breakthrough Therapy and Rare Pediatric Disease designations by the FDA for the treatment of patients with pLGG harboring an activating RAF alteration. Tovorafenib has also received Orphan Drug designation from the FDA for the treatment of malignant glioma, and from the European Commission for the treatment of glioma.

On February 26, 2024 Cogent Biosciences, Inc. (Nasdaq: COGT), a biotechnology company focused on developing precision therapies for genetically defined diseases, reported a business update and announced financial results for the fourth quarter and full year of 2023 (Press release, Cogent Biosciences, FEB 26, 2024, View Source [SID1234640445]).

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"We have entered 2024 in a very strong position," said Andrew Robbins, Cogent’s President and Chief Executive Officer. "We are now actively enrolling three registration-directed clinical trials of bezuclastinib across NonAdvSM, AdvSM and 2nd-line GIST patients, all of which are on track to deliver top-line results in 2025. With our recent fundraising, we are funded into 2027, and based on the clinical data we have presented across these programs to date, believe strongly that bezuclastinib has the opportunity to become the best-in-class KIT mutant inhibitor for these three indications."

Q4 2023 and Recent Business Highlights

In February 2024, reported positive Part 1b data from the Company’s ongoing Phase 2 SUMMIT trial at the 2024 American Academy of Allergy Asthma & Immunology Annual Meeting (AAAAI) meeting. SUMMIT is a registration-directed, randomized, double-blind, placebo-controlled, global, multicenter, clinical trial of bezuclastinib in patients with nonadvanced systemic mastocytosis (NonAdvSM). Key highlights include:

Announced the 100 mg Recommended Phase 2 Dose (RP2D)
Demonstrated a well-tolerated safety profile, generally consistent with placebo
Introduced Cogent’s MS2D2, a novel, refined patient reported outcomes measure (PROM) designed to assess symptomatic severity and improvement
Reported a 51% week 12 mean improvement in total symptom score (TSS) for bezuclastinib, including 70% of patients achieving ≥50% improvement in symptom severity
Reported a 49% week 12 mean improvement in quality of life (MC-QoL) for bezuclastinib

In February 2024, closed a $225 million oversubscribed private investment in public equity (PIPE) financing. Cogent sold approximately 17 million shares of its common stock at a price of $7.50 per share, representing a premium of approximately 37% to its closing price on February 13, 2024. The financing also included Series B non-voting convertible Preferred Stock convertible into approximately 13 million shares of its common stock.

In December 2023, reported positive clinical data from the Phase 2 SUMMIT and APEX trials at the American Society of Hematology (ASH) (Free ASH Whitepaper) annual meeting. Highlights from APEX, a registration-directed trial evaluating bezuclastinib in patients with advanced systemic mastocytosis (AdvSM), include:

Reported an encouraging safety and tolerability profile with no related cognitive impairment or bleeding events
Achieved impressive effects in key biomarkers of disease burden
Achieved 56% Overall Response Rate (ORR) in TKI-naïve patients, including 86% ORR by Pure Pathological Response (PPR) criteria

In December 2023, presented new preclinical data highlighting the potential best-in-class potency and selectivity of Cogent’s ErbB2 inhibitor and announced a third Cogent discovery stage PI3Kα inhibitor program at the San Antonio Breast Cancer Symposium (SABCS).

The novel EGFR-sparing, brain-penetrant ErbB2 inhibitor demonstrated a superior efficacy profile, reaching 80% brain penetrance with potent coverage of key mutations
The novel, H1047R mutant-selective PI3Kα inhibitor demonstrated high clinical target engagement without metabolic dysfunction commonly associated with molecules in the class

In November 2023, reported updated clinical data from the lead-in portion of the ongoing PEAK Phase 3 trial evaluating bezuclastinib in combination with sunitinib in patients with GIST at the Connective Tissue Oncology Society (CTOS) annual meeting. Positive lead-in data was first presented at the 2023 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) annual meeting in June.

Safety and tolerability data from 42 patients enrolled in Part 1a and Part 1b were consistent with results shared at the 2023 ASCO (Free ASCO Whitepaper) annual meeting, demonstrating the combination of bezuclastinib and sunitinib was well tolerated with an adverse event profile similar to sunitinib monotherapy
Updated clinical activity from a subset of 2nd-line GIST patients demonstrated a 33% confirmed overall response rate (ORR) with ongoing median duration of therapy greater than 14 months. Together with clinical data previously reported from a Phase 1/2 trial, 4 of 10 evaluable 2nd-line GIST patients treated with the combination have reached confirmed partial response status

In October 2023, presented updated preclinical data from the Company’s next-generation selective fibroblast growth factor receptor 2 (FGFR2) program in a poster presentation at the 2023 AACR (Free AACR Whitepaper)-NCI-EORTC AACR-NCI-EORTC (Free AACR-NCI-EORTC Whitepaper) International Conference on Molecular Targets and Cancer Therapeutics (EORTC-NCI-AACR) (Free ASGCT Whitepaper) (Free EORTC-NCI-AACR Whitepaper).

The poster showed Cogent’s internally-developed FGFR2 inhibitor exhibits low nM potency on WT FGFR2 and FGFR2 mutations and is selective against the kinome and a panel of channels and receptors.
Exploratory pharmacokinetics (PK) studies conducted across species showed CGT4859 to be a low-clearance compound with high oral bioavailability. Further, in an AN3 CA model, CGT4859 demonstrated dose-responsive tumor growth inhibition with complete regressions at 5 mg/kg PO and was well-tolerated
Projected Near-Term Milestones

Bezuclastinib – Systemic Mastocytosis (SM)

Complete enrollment in SUMMIT Part 2 in the second quarter of 2025 and deliver top-line results by the end of 2025

Provide additional safety, tolerability and patient-reported outcomes data from the open label extension portion of SUMMIT Part 1 during 2024

Complete enrollment in the registration-directed APEX Phase 2 trial in patients with Advanced Systemic Mastocytosis (AdvSM) by the end of 2024
Bezuclastinib – Gastrointestinal Stromal Tumors (GIST)

Complete enrollment in the global, randomized Phase 3 PEAK trial in 2nd-line GIST patients by the end of 2024

Provide additional clinical results from the PEAK lead-in trial, including longer duration safety and tolerability results along with updated efficacy measures, including objective response rate (ORR) and progression free survival (PFS) during 2024
CGT4859 (FGFR2 inhibitor)

Initiate a Phase 1 trial of the first Cogent-discovered pipeline program, designed as a potent, selective, reversible FGFR2 inhibitor with best-in-class potential in the second half of 2024
Preclinical Pipeline

Initiate IND-enabling studies for lead candidate from potent, selective ErbB2 program, highlighted by potential best-in-class brain penetrant properties

Select lead candidate and initiate IND-enabling studies from ongoing PI3Ka program, designed to potently and selectively target the H1047R driver mutation, which affects >30,000 cancer patients each year
Upcoming Investor Conference

Leerink Healthcare Conference on Tuesday, March 12 at 2:20 p.m. ET.

A live webcast can be accessed on the Investors & Media page of Cogent’s website at investors.cogentbio.com/events. A replay will be available approximately two hours after completion of the event and will be archived for up to 30 days.
Fourth Quarter and Full Year 2023 Financial Results

Cash and Cash Equivalents: As of December 31, 2023, Cogent had cash, cash equivalents and marketable securities of $273.2 million. Cogent believes this year-end balance, together with the gross proceeds from the $225.0 million oversubscribed private placement, which closed February 16, 2024, will be sufficient to fund its operating expenses and capital expenditure requirements into 2027, including through clinical readouts from the ongoing SUMMIT, PEAK and APEX registration-directed trials.

R&D Expenses: Research and development expenses were $48.7 million for the fourth quarter of 2023 and $173.8 million for the year ended December 31, 2023, as compared to $36.7 million for the fourth quarter of 2022 and $121.6 million for the year ended December 31, 2022. The increase is driven by progress on the APEX, SUMMIT and PEAK trials and the continued development of the research pipeline. During the quarter Cogent also completed and paid for bulk manufacturing campaigns of bezuclastinib to continue supporting the company’s clinical trials. R&D expenses include non-cash stock compensation expense of $4.1 million for the fourth quarter of 2023 and $14.6 million for the year ended December 31, 2023, as compared to $2.4 million for the fourth quarter of 2022 and $8.5 million for the year ended December 31, 2022.

G&A Expenses: General and administrative expenses were $9.5 million for the fourth quarter of 2023 and $34.4 million for the year ended December 31, 2023, as compared to $7.0 million for the fourth quarter of 2022 and $26.2 million for the year ended December 31, 2022. G&A expenses include non-cash stock compensation expense of $4.8 million for the fourth quarter of 2023 and $16.0 million for the year ended December 31, 2023, as compared to $2.6 million for the fourth quarter of 2022 and $9.9 million for the year ended December 31, 2022.

Net Loss: Net loss was $54.4 million for the fourth quarter of 2023 and $192.4 million for the year ended December 31, 2023, as compared to a net loss of $39.6 million for the fourth quarter of 2022 and $140.2 million for the year ended December 31, 2022.