On November 8, 2023 Verastem Oncology (Nasdaq: VSTM), a biopharmaceutical company committed to advancing new medicines for patients with cancer, reported financial results for the third quarter ending September 30, 2023 and highlighted recent progress (Press release, Verastem, NOV 8, 2023, View Source [SID1234637295]).

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"In the third quarter, we presented additional data with robust levels of response from a planned subgroup analysis of the RAMP 201 study supporting the role of avutometinib and defactinib as a potential treatment option for LGSOC regardless of a patient’s prior therapy. These data continue to build on the foundational proof of concept and support our plans to submit an application for Accelerated Approval in the first half of 2024," said Dan Paterson, President and Chief Executive Officer, Verastem Oncology. "As part of our broader development program, we were excited to share the initial, promising efficacy and safety data of the combination of avutometinib and sotorasib in G12C-mutant non-small cell lung cancer. In addition, we look forward to our synergistic collaboration with GenFleet Therapeutics that will provide us with the exclusive option to license three new programs to expand our pipeline. This collaboration along with our progress across our broader development platform, will allow us to further address the significant unmet medical needs across RAS pathway-driven cancers."

Third Quarter 2023 and Recent Highlights

Low-Grade Serous Ovarian Cancer (LGSOC)

The Company plans to submit an application for Accelerated Approval with the U.S. Food and Drug Administration (FDA) in the first half of 2024 for the combination of avutometinib and defactinib based on mature data from the Phase 2 registration-directed trial, RAMP 201, together with the results of the investigator-initiated FRAME trial. The Company also plans to have discussions with global regulatory authorities to bring the combination to additional regions.
The Company finalized the design of the Phase 3 confirmatory trial (RAMP 301) of avutometinib and defactinib in LGSOC versus standard of care (SOC) chemotherapy (pegylated liposomal doxorubicin, paclitaxel, topotecan) or hormone therapy (letrozole, anastrozole). The trial will enroll approximately 270 patients randomized to either the combination of avutometinib and defactinib or SOC. RAMP 301 is an international collaboration between The GOG Foundation, Inc. (GOG) and the European Network of Gynaecological Oncological Trial groups (ENGOT) sponsored by Verastem Oncology. RAMP 301 is the follow-up confirmatory study being conducted for full regulatory approval in recurrent LGSOC and is expected to begin enrollment in the fourth quarter of this year.
The results of a planned subgroup analysis of the Phase 2 RAMP 201 trial of avutometinib and defactinib were presented as a late-breaking abstract in an oral presentation at the Annual Global Meeting of the International Gynecologic Cancer Society (IGCS) in November. The data showed the combination demonstrated promising levels of response in recurrent LGSOC regardless of number and class of prior therapies including after poor response to prior therapy. In the combination arm, the observed overall response rate (ORR) was consistent across patients who received 1-3 (45.5%, 5/11, 95% CI 17-77) and ≥4 lines of therapy (44.4%, 8/18, 95% CI 22-69). Prior to enrollment in RAMP 201, only 2/23 (8.7%) patients responded to their last prior treatment in the recurrent setting, whereas the combination of avutometinib and defactinib yielded an ORR of 43.5% (10/23) in this subgroup. The safety profiles of avutometinib and defactinib were similar in the less and more heavily pretreated subgroups and both analyses were consistent with previously reported safety data.
The Company, in collaboration with the LGSOC Patient Impact Advisory Committee, a global collaboration among leaders in the medical community as well as patient advocacy groups including STAAR Ovarian Cancer Foundation, Cure Our Ovarian Cancer and the World Ovarian Cancer Coalition, announced results of the first-ever LGSOC Patient Impact Survey. The focus of this survey is to better understand and address the particular challenges with diagnosis, disease management and mental, physical, and emotional well-being experienced by people living with LGSOC. More information is available LetsTalkAboutLGSOC.com.
Other Programs

The Company announced a discovery and development collaboration with GenFleet Therapeutics ("GenFleet") to advance three oncology discovery programs targeting RAS pathway-driven cancers. The collaboration, which builds on the strengths of both companies in oncology small molecule drug development, enables Verastem Oncology to partner its clinical development and regulatory expertise with GenFleet’s accomplished discovery capabilities. Verastem Oncology has the exclusive rights to obtain a license to each of the compounds after successful completion of pre-determined milestones in Phase 1 trials and adds to the Company’s ability to become a leader in the treatment of RAS pathway-driven cancers.
The Company presented initial safety, pharmacokinetics and recommended Phase 2 dose (RP2D) in the RAMP 203 trial evaluating the safety, tolerability and efficacy of avutometinib in combination with sotorasib in patients with KRAS G12C-mutant non-small cell lung cancer (NSCLC) at the 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) in October. The confirmed ORR was 25% (3/12) across efficacy-evaluable patients and seen in both KRAS G12C inhibitor resistant (14.3%; 1/7) and naïve (40%; 2/5) patients. Avutometinib 4.0 mg PO BIW 21/28 days + sotorasib 960 mg PO QD 28/28 days was selected as RP2D based on dose limiting toxicity assessment. Enrollment of patients with KRAS G12C-mutant NSCLC who are either naïve to or previously treated with a KRAS G12C inhibitor is ongoing in the expansion phase of RAMP 203.
Dose escalation is ongoing in the RAMP 204 Phase 1/2 clinical trial of avutometinib with Mirati’s KRAZATI (adagrasib) in KRAS G12C-mutant NSCLC. The study is in its second dose cohort after successfully completing the first dose cohort.
Enrollment is ongoing in the RAMP 205 Phase 1b/2 clinical trial evaluating avutometinib and defactinib in combination with SOC chemotherapy (GEMZAR (gemcitabine) and ABRAXANE) in patients with metastatic adenocarcinoma of the pancreas. The trial is supported by the Company’s receipt of the first "Therapeutic Accelerator Award" from the Pancreatic Cancer Action Network (PanCAN).
Corporate Updates

The Company strengthened its executive team in advance of the Company’s potential commercial launch of avutometinib in combination with defactinib in LGSOC and to support the advancement of its broader development programs in RAS pathway-driven cancers. The appointments include Mike Crowther to Chief Commercial and Business Strategy Officer, David Mitchell to Senior Vice President, Head of Regulatory Affairs and the promotion of Dan Calkins to Chief Financial Officer from Vice President of Finance.
Third Quarter 2023 Financial Results

Verastem Oncology ended the third quarter of 2023 with cash, cash equivalents and investments of $165.7 million. Total operating expenses for the three months ended September 30, 2023 (the "2023 Quarter") were $21.3 million, compared to $17.7 million for the three months ended September 30, 2022 (the "2022 Quarter").

Research & development expenses for the 2023 Quarter were $13.9 million, compared to $11.3 million for the 2022 Quarter. The increase of $2.6 million, or 23.0%, primarily resulted from a $2.0 million upfront payment made to GenFleet pursuant to the discovery and development collaboration agreement and increases in contract research organization costs.

Selling, general & administrative expenses for the 2023 Quarter were $7.4 million, compared to $6.4 million for the 2022 Quarter. The increase of $1.0 million, or 15.6%, was primarily related to increased personnel costs, including non-cash stock compensation, additional costs in anticipation of a potential launch of avutometinib and defactinib in LGSOC, and increased travel and other costs.

Net loss for the 2023 Quarter was $20.0 million, or $0.75 per share (basic and diluted), compared to net loss of $18.1 million, or $1.10 per share (basic and diluted, each as adjusted for the Company’s reverse stock split) for the 2022 Quarter.

For the 2023 Quarter, non-GAAP adjusted net loss was $19.0 million, or $0.71 per share (diluted), compared to non-GAAP adjusted net loss of $16.6 million, or $1.01 per share (diluted, as adjusted for the Company’s reverse stock split) for the 2022 Quarter. Please refer to the GAAP to Non-GAAP Reconciliation accompanying this press release.

Use of Non-GAAP Financial Measures

To supplement Verastem Oncology’s condensed consolidated financial statements, which are prepared and presented in accordance with generally accepted accounting principles in the United States (GAAP), the Company uses the following non-GAAP financial measures in this press release: non-GAAP adjusted net loss and non-GAAP adjusted net loss per share. These non-GAAP financial measures exclude certain amounts or expenses from the corresponding financial measures determined in accordance with GAAP. Management believes this non-GAAP information is useful for investors, taken in conjunction with the Company’s GAAP financial statements, because it provides greater transparency and period-over-period comparability with respect to the Company’s operating performance and can enhance investors’ ability to identify operating trends in the Company’s business. Management uses these measures, among other factors, to assess and analyze operational results and trends and to make financial and operational decisions. Non-GAAP information is not prepared under a comprehensive set of accounting rules and should only be used to supplement an understanding of the Company’s operating results as reported under GAAP, not in isolation or as a substitute for, or superior to, financial information prepared and presented in accordance with GAAP. In addition, these non-GAAP financial measures are unlikely to be comparable with non-GAAP information provided by other companies. The determination of the amounts that are excluded from non-GAAP financial measures is a matter of management judgment and depends upon, among other factors, the nature of the underlying expense or income amounts. Reconciliations between these non-GAAP financial measures and the most comparable GAAP financial measures for the three and nine months ended September 30, 2023, and 2022 are included in the tables accompanying this press release, after the unaudited condensed consolidated financial statements.

About Avutometinib (VS-6766)

Avutometinib is a RAF/MEK clamp that induces inactive complexes of MEK with ARAF, BRAF and CRAF potentially creating a more complete and durable anti-tumor response through maximal RAS pathway inhibition. Avutometinib is currently in late-stage development.

In contrast to other MEK inhibitors, avutometinib blocks both MEK kinase activity and the ability of RAF to phosphorylate MEK. This unique mechanism allows avutometinib to block MEK signaling without the compensatory activation of MEK that appears to limit the efficacy of other inhibitors. The U.S. Food and Drug Administration granted Breakthrough Therapy designation for the combination of Verastem Oncology’s investigational RAF/MEK clamp avutometinib, with defactinib, its FAK inhibitor, for the treatment of all patients with recurrent low-grade serous ovarian cancer (LGSOC) regardless of KRAS status after one or more prior lines of therapy, including platinum-based chemotherapy.

Verastem Oncology is currently conducting clinical trials with its RAF/MEK clamp avutometinib in RAS- driven tumors as part of its (Raf And Mek Program). RAMP 201 is a registration-directed trial of avutometinib in combination with defactinib in patients with recurrent LGSOC. Verastem Oncology has established clinical collaborations with Amgen and Mirati to evaluate LUMAKRAS (sotorasib) and KRAZATI (adagrasib) in combination with avutometinib in KRAS G12C mutant NSCLC as part of the RAMP 203 and RAMP 204 trials, respectively. As part of the "Therapeutic Accelerator Award" Verastem Oncology received from PanCAN, Verastem Oncology is conducting RAMP 205, a Phase 1b/2 clinical trial evaluating avutometinib and defactinib with gemcitabine/nab-paclitaxel in patients with front-line metastatic pancreatic cancer.

On November 8, 2023 SQZ Biotechnologies Company (OTC: SQZB), focused on unlocking the full potential of cell therapies, reported third quarter 2023 financial results and recent portfolio updates (Press release, SQZ Biotech, NOV 8, 2023, View Source [SID1234637294]).

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"We have taken difficult, but necessary, steps to reduce our operating expenses as we explore strategic alternatives," said Howard Bernstein, M.D., Ph.D., Interim Chief Executive Officer and Member of the Board of Directors. "The Management Team and the Board firmly believe in the technology and therapeutic potential and are committed to exploring a variety of potential paths."

Third Quarter 2023 and Recent Portfolio Updates

Clinical Programs:

SQZ Activating Antigen Carriers ("AAC") Platform in Oncology

Of the five patients treated, 1 patient experienced a complete response and 2 patients experienced stable disease
Interim clinical data reported at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) Annual Meeting 2023
SQZ Enhanced Antigen Presenting Cell ("eAPC") Platform in Oncology

Completed enrollment for highest-dose cohort of monotherapy dose escalation trial
Observed BOR of Stable Disease in 40 Percent (8 of 20) of Treated Patients
Interim clinical data reported at the SITC (Free SITC Whitepaper) Annual Meeting 2023
Corporate:

The Company is actively seeking potential partnerships, collaborations and strategic alternatives to advance its oncology programs and platforms
On September 29, 2023, the Board of Directors of SQZ approved a reduction in the Company’s workforce by approximately 80 percent. The decision was based on cost-reduction initiatives intended to reduce the Company’s ongoing operating expenses while it pursues strategic alternatives
Third Quarter 2023 Financial Highlights

Revenue for the quarter ended September 30, 2023, was $0.0 compared to $3.5 million for the same period in 2022
Research and development expenses for the quarter ended September 30, 2023, were $10.5 million compared to $19.6 million for the same period in 2022
General and administrative expenses for the quarter ended September 30, 2023, were $5.6 million compared to $6.9 million for the same period in 2022
Restructuring charges for the quarter ended September 30, 2023 were $7.7 million, driven primarily by a non-cash impairment charge to its facility right of use asset
Net loss for the quarter ended September 30, 2023, was $23.6 million, compared to $22.6 million for the same period in 2022
As of September 30, 2023, the Company had cash and cash equivalents of $10.2 million

On November 8, 2023 Takeda (TSE:4502/NYSE:TAK) reported that the U.S. Food and Drug Administration (FDA) has approved FRUZAQLA (fruquintinib), an oral targeted therapy for adults with metastatic colorectal cancer (mCRC) who have been previously treated with fluoropyrimidine-, oxaliplatin-, and irinotecan-based chemotherapy, an anti-VEGF therapy, and, if RAS wild-type and medically appropriate, an anti-EGFR therapy (Press release, Takeda, NOV 8, 2023, View Source [SID1234637293]). FRUZAQLA is the first and only selective inhibitor of all three VEGF receptor kinases approved in the U.S. for previously treated mCRC regardless of biomarker status.1,2 This approval was received under Priority Review more than 20 days ahead of the scheduled PDUFA date of November 30, 2023.

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"We are encouraged to see the continued progress in providing new options to patients."

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"There is a pressing need for new treatments for individuals with metastatic colorectal cancer, who have had limited options and continue to face poor outcomes. FRUZAQLA is the first novel chemotherapy-free treatment option approved for patients in the U.S. regardless of biomarker status in more than a decade," said Teresa Bitetti, president of the Global Oncology Business Unit at Takeda. "For far too long, healthcare providers and patients have had limited options when selecting a therapy for metastatic colorectal cancer. FRUZAQLA has the potential to offer a significant survival benefit to patients without negatively impacting their quality of life."

The approval of FRUZAQLA is based on data from two large Phase 3 trials: the multi-regional FRESCO-2 trial, data from which were published in The Lancet, along with the FRESCO trial conducted in China, data from which were published in JAMA. The trials investigated FRUZAQLA plus best supportive care versus placebo plus best supportive care in patients with previously treated mCRC. Both FRESCO and FRESCO-2 met their primary and key secondary efficacy endpoints and showed consistent benefit among a total of 734 patients treated with FRUZAQLA. Safety profiles were consistent across trials.

"Patients with metastatic disease are often fragile and fatigued – due to both their condition as well as the therapies they have been exposed to. An oral, chemotherapy-free option that offers a survival benefit despite treatment with prior therapies is a critical need for treating metastatic colorectal cancer," said Cathy Eng, M.D., FACP, at Vanderbilt University Medical Center. "Colorectal cancer is a highly heterogeneous disease, making it difficult to bring advancements to patients whose cancer has metastasized. I look forward to being able to offer a new solution to appropriate patients."

In the United States, approximately 153,000 new cases of CRC will be diagnosed in 2023, representing 7.8% of all new cancer cases.3,4 Approximately 70% of patients with CRC will experience metastatic disease, whether at diagnosis or after treatment. Metastases are the main cause of CRC-related mortality.5,6

"We have witnessed firsthand the physical and emotional toll metastatic colorectal cancer has on patients, their families and their care teams," said Michael Sapienza, Chief Executive Officer, at Colorectal Cancer Alliance. "We are encouraged to see the continued progress in providing new options to patients."

The data from FRESCO and FRESCO-2 also supported the EU marketing authorization application (MAA) for fruquintinib, which was validated and accepted for review by the European Medicines Agency (EMA) in June 2023. A submission to the Japan Pharmaceuticals and Medical Devices Agency (PMDA) also took place in September 2023.

About FRUZAQLA (fruquintinib)
FRUZAQLA (fruquintinib) is a selective oral inhibitor of VEGFR -1, -2 and -3. VEGFR inhibitors play a pivotal role in blocking tumor angiogenesis. FRUZAQLA was designed to have enhanced selectivity that limits off-target kinase activity, allowing for high drug exposure, sustained target inhibition, and flexibility for the potential use as part of combination therapy. FRUZAQLA has demonstrated a manageable safety profile and is being investigated in combinations with other anti-cancer therapies. Fruquintinib was approved for marketing by the China National Medical Products Administration (NMPA) in September 2018 and commercially launched in China in November 2018 under the brand name ELUNATE. In addition, a marketing authorization application (MAA) from the European Medicines Agency (EMA) was validated and accepted for review in June 2023, and a submission to the Japan Pharmaceuticals and Medical Devices Agency (PMDA) took place in September 2023. Takeda has the exclusive worldwide license to further develop, commercialize, and manufacture fruquintinib outside of mainland China, Hong Kong and Macau. Fruquintinib is developed and marketed in China by HUTCHMED.

IMPORTANT SAFETY INFORMATION

WARNINGS AND PRECAUTIONS

Hypertension occurred in 49% of 911 patients with mCRC treated with FRUZAQLA, including Grade 3-4 events in 19%, and hypertensive crisis in three patients (0.3%). Do not initiate FRUZAQLA unless blood pressure is adequately controlled. Monitor blood pressure weekly for the first month and at least monthly thereafter as clinically indicated. Initiate or adjust anti-hypertensive therapy as appropriate. Withhold, reduce dose, or permanently discontinue FRUZAQLA based on severity of hypertension.
Hemorrhagic Events including serious, fatal events can occur with FRUZAQLA. In 911 patients with mCRC treated with FRUZAQLA, 6% of patients experienced gastrointestinal hemorrhage, including 1% with a Grade ≥3 event and 2 patients with fatal hemorrhages. Permanently discontinue FRUZAQLA in patients with severe or life-threatening hemorrhage. Monitor the International Normalized Ratio (INR) levels in patients receiving anticoagulants.
Infections. FRUZAQLA can increase the risk of infections, including fatal infections. In 911 patients with mCRC treated with FRUZAQLA, the most common infections were urinary tract infections (6.8%), upper respiratory tract infections (3.2%) and pneumonia (2.5%); fatal infections included pneumonia (0.4%), sepsis (0.2%), bacterial infection (0.1%), lower respiratory tract infection (0.1%), and septic shock (0.1%). Withhold FRUZAQLA for Grade 3 or 4 infections, or worsening infection of any grade. Resume FRUZAQLA at the same dose when the infection has resolved.
Gastrointestinal Perforation occurred in patients treated with FRUZAQLA. In 911 patients with mCRC treated with FRUZAQLA, 1.3% experienced a Grade ≥3 gastrointestinal perforation, including one fatal event. Permanently discontinue FRUZAQLA in patients who develop gastrointestinal perforation or fistula.
Hepatotoxicity. FRUZAQLA can cause liver injury. In 911 patients with mCRC treated with FRUZAQLA, 48% experienced increased ALT or AST, including Grade ≥3 events in 5%, and fatal events in 0.2% of patients. Monitor liver function tests (ALT, AST, and bilirubin) before initiation and periodically throughout treatment with FRUZAQLA. Temporarily hold and then reduce or permanently discontinue FRUZAQLA depending on the severity and persistence of hepatotoxicity as manifested by elevated liver function tests.
Proteinuria. FRUZAQLA can cause proteinuria. In 911 patients with mCRC treated with FRUZAQLA, 36% experienced proteinuria and 2.5% of patients experienced Grade ≥3 events. Monitor for proteinuria before initiation and periodically throughout treatment with FRUZAQLA. For proteinuria ≥2g/24 hours, withhold FRUZAQLA until improvement to ≤Grade 1 proteinuria and resume FRUZAQLA at a reduced dose. Discontinue FRUZAQLA in patients who develop nephrotic syndrome.
Palmar-Plantar Erythrodysesthesia (PPE) occurred in 35% of 911 patients treated with FRUZAQLA, including 8% with Grade 3 events. Based on severity of PPE, withhold FRUZAQLA and then resume at the same or reduced dose.
Posterior Reversible Encephalopathy Syndrome (PRES), a syndrome of subcortical vasogenic edema diagnosed by characteristic finding on MRI, occurred in one of 911 patients treated with FRUZAQLA. Perform an evaluation for PRES in any patient presenting with seizures, headache, visual disturbances, confusion, or altered mental function. Discontinue FRUZAQLA in patients who develop PRES.
Impaired Wound Healing. In 911 patients with mCRC treated with FRUZAQLA, 1 patient experienced a Grade 2 event of wound dehiscence. Do not administer FRUZAQLA for at least 2 weeks prior to major surgery. Do not administer FRUZAQLA for at least 2 weeks after major surgery and until adequate wound healing. The safety of resumption of FRUZAQLA after resolution of wound healing complications has not been established.
Arterial Thromboembolic Events. In 911 patients with mCRC treated with FRUZAQLA, 0.8% of patients experienced an arterial thromboembolic event. Initiation of FRUZAQLA in patients with a recent history of thromboembolic events should be carefully considered. In patients who develop arterial thromboembolism, discontinue FRUZAQLA.
Allergic Reactions to FD&C Yellow No. 5 (Tartrazine) and No. 6 (Sunset Yellow FCF). FRUZAQLA 1 mg capsules contain FD&C Yellow No. 5 (tartrazine), which may cause allergic-type reactions (including bronchial asthma) in certain susceptible persons. FRUZAQLA 1 mg contains FD&C Yellow No. 6 (sunset yellow FCF), which may cause allergic reactions.
Embryo-Fetal Toxicity. Based on findings in animal studies and its mechanism of action, FRUZAQLA can cause fetal harm when administered to pregnant women. Advise pregnant women of the potential risk to a fetus. Advise females of childbearing potential and males with female partners of childbearing potential to use effective contraception during treatment with FRUZAQLA and for 2 weeks after the last dose.
ADVERSE REACTIONS

The most common adverse reactions (incidence ≥20%) following treatment with FRUZAQLA included hypertension, palmar-plantar erythrodysesthesia (hand-foot skin reactions), proteinuria, dysphonia, abdominal pain, diarrhea, and asthenia.

DRUG INTERACTIONS: Avoid concomitant administration of FRUZAQLA with strong or moderate CYP3A inducers.

USE IN SPECIFIC POPULATIONS

Lactation: Advise women not to breastfeed during treatment with FRUZAQLA and for 2 weeks after the last dose.
To report SUSPECTED ADVERSE REACTIONS, contact Takeda Pharmaceuticals at 1-844-662-8532 or the FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

Please see FRUZAQLA (fruquintinib) full Prescribing Information View Source

About CRC
CRC is a cancer that starts in either the colon or rectum. According to the International Agency for Research on Cancer, CRC is the third most prevalent cancer worldwide, associated with more than 935,000 deaths in 2020.7 In the U.S., it is estimated that 153,000 patients will be diagnosed with CRC and 53,000 deaths from the disease will occur in 2023.3 In Europe, CRC was the second most common cancer in 2020, with approximately 520,000 new cases and 245,000 deaths. In Japan, CRC was the most common cancer, with an estimated 148,000 new cases and 60,000 deaths in 2020.7 Although early-stage CRC can be surgically resected, metastatic CRC remains an area of high unmet need with poor outcomes and limited treatment options. Some patients with metastatic CRC may benefit from personalized therapeutic strategies based on molecular characteristics; however, most patients have tumors that do not harbor actionable mutations.8,9,10,11,12

About the Phase 3 FRESCO-2 Trial
The FRESCO-2 study is a multi-regional clinical trial conducted in the U.S., Europe, Japan and Australia investigating FRUZAQLA (fruquintinib) plus best supportive care vs placebo plus best supportive care in patients with previously treated metastatic CRC (NCT04322539). The study met its primary and key secondary endpoints, demonstrating that treatment with FRUZAQLA resulted in statistically significant and clinically meaningful improvement in overall survival (OS) and progression-free survival (PFS). The safety profile of FRUZAQLA in FRESCO-2 was consistent with previously reported FRUZAQLA studies. Results from the study were presented at ESMO (Free ESMO Whitepaper) in September 2022 and subsequently published in The Lancet.13,14

The Phase 3 FRESCO and FRESCO-2 trials supported the marketing authorization application (MAA) from the European Medicines Agency (EMA) for fruquintinib, which was validated and accepted for review in June 2023. A submission to the Japan Pharmaceuticals and Medical Devices Agency (PMDA) also took place in September 2023.

On November 8, 2023 Gilead Sciences, Inc. (Nasdaq: GILD) reported that its executives will be speaking at the following investor conferences (Press release, Gilead Sciences, NOV 8, 2023, View Source [SID1234637292]):

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Jefferies London Healthcare Conference on Wednesday, November 15 at 9:00am Greenwich Mean Time
Evercore ISI HealthCONx Conference on Tuesday, November 28 at 1:20pm Eastern Time
Piper Sandler Healthcare Conference on Wednesday, November 29 at 9:00am Eastern Time

The live webcasts can be accessed at the company’s investors page at investors.gilead.com. The replays will be available for at least 30 days following the presentation.

On November 8, 2023 CASI Pharmaceuticals Inc. (Nasdaq: CASI), a biopharmaceutical company focused on developing and commercializing innovative therapeutics and pharmaceutical products, reported a major milestone in its partnership with Juventas Cell Therapy Ltd. (Juventas) (Press release, CASI Pharmaceuticals, NOV 8, 2023, View Source [SID1234637291]). The China National Medical Products Administration (NMPA) has granted market approval for Juventas’ investigational cell therapy, Inaticabtagene Autoleucel (CNCT 19), for the treatment of relapsed and refractory B-cell acute lymphoblastic leukemia (r/r B-ALL) in China.

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Inaticabtagene Autoleucel is a CD19 CAR-T cell therapy product comprised of a unique CD19 scFv（HI19a）structure and utilizes leading CMC manufacturing techniques. Inaticabtagene Autoleucel has demonstrated a high level of efficacy, with durable remissions, and substantially improved safety profile with reduced CAR-T related toxicities in the pivotal clinical study for the treatment of adults with r/r B-ALL.

This approval is based on the clinical results from a single-arm, multi-center, pivotal study of 39 adult patients with r/r B-ALL in China. The 9.3-month follow up data demonstrated very high durable response, overall response rate ("ORR"): 82.1%, complete response rate ("CR"): 66.7% within 3 month after infusion and median duration of response (DoR) has not reached. The safety profile shown the decreased severity of CAR T-Cell related adverse events in patients with r/r B-ALL. Given the currently available treatment options in China, Inaticabtagene Autoleucel will be the first treatment CAR-T therapy option with a positive benefit-risk ratio for adult patients with r/r B-ALL and potentially be a best-in-class CAR-T product.

This pivotal development not only marks a significant achievement for CASI and Juventas but also represents a remarkable advancement in the field of hematology-oncology and cell therapy. CNCT19 is the first CD19-directed CAR-T product with Chinese independent intellectual property rights, making it a trailblazer in the Chinese biopharmaceutical landscape. It is also the first commercialized cell therapy product in China designed to treat B-ALL.

Dr. Wei-Wu He, Chairman and CEO of CASI, expressed his enthusiasm for the commercial and clinical significance of CNCT19. He stated, "The approval of CNCT19 represents a transformative moment not only for CASI and Juventas but for all B-ALL patients in China. We are committed to making this groundbreaking therapy accessible to those in need and aspire to extend its reach globally. CNCT19 offers new hope to patients battling relapsed B-ALL, and this partnership between CASI and Juventas aims to ensure that this innovative therapy reaches those in need across China and beyond."