On September 3, 2024 Cogent Biosciences, Inc. (Nasdaq: COGT), a biotechnology company focused on developing precision therapies for genetically defined diseases, reported several updates from the company’s ongoing registration-directed clinical trials of its potential best-in-class KIT mutant inhibitor, bezuclastinib (Press release, Cogent Biosciences, SEP 3, 2024, View Source [SID1234646288]).

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Patient enrollment is now complete in Cogent’s Phase 3 PEAK trial evaluating bezuclastinib in combination with sunitinib for the treatment of patients with gastrointestinal stromal tumors (GIST). Based on strong global patient interest, a total of 413 patients were enrolled in the study. In addition, Cogent recently completed a pre-planned interim futility analysis, and the Independent Data Monitoring Committee (IDMC) recommended continuing the PEAK study without modification. This pre-specified analysis was based on an assessment of progression-free survival (PFS) as determined by independent central review and did not include the option for early stopping due to efficacy. 

Separately, based on significant patient interest in the ongoing SUMMIT trial in nonadvanced systemic mastocytosis (NonAdvSM), Cogent also announced today that it expects to complete enrollment in this study during Q1 2025, approximately three months earlier than originally projected.

"We are excited to announce these important updates to the PEAK and SUMMIT studies today," said Andrew Robbins, Cogent’s President and Chief Executive Officer. "Strong continued interest from patients around the world to participate in our bezuclastinib trials has allowed us to accelerate development and surpass our original enrollment timelines. Completing enrollment in our Phase 3 PEAK trial of bezuclastinib and sunitinib for second-line GIST patients several months ahead of schedule represents a significant milestone for the program and we are extremely grateful to the patients, families, caregivers, advocacy groups and clinical investigators for their participation in, and support of, the PEAK trial." 

PEAK is a randomized, open-label, global Phase 3 clinical trial evaluating bezuclastinib in combination with sunitinib vs. sunitinib alone in GIST patients previously treated with imatinib. The primary endpoint of the trial is median progression free survival (mPFS). PEAK is a registration study intended to support a New Drug Application (NDA) in GIST. 

SUMMIT is a randomized, blinded, global, registration-directed clinical trial evaluating bezuclastinib vs. placebo in NonAdvSM patients. The primary endpoint of the trial is mean improvement in patient symptoms measured at 24 weeks. SUMMIT is intended to be a registrational study designed to support a New Drug Application (NDA) in NonAdvSM. 

Appointment of Darara Dibabu as Vice President of Marketing
In addition to the updates to PEAK and SUMMIT trials, Cogent announced today that Mr. Dibabu has joined Cogent as the VP of Marketing. Mr. Dibabu has 25 years of experience in the biopharmaceutical industry, most recently as the Global Brand Lead of TUKYSA at Pfizer and SeaGen, where he led the global launch and marketing strategy of the product for metastatic breast cancer patients. Previously, he served in various roles of increasing responsibility at Seagen, Bayer and Merck. Mr. Dibabu holds a bachelor’s degree in Biology from the University of Southern California. In connection with Mr. Dibabu joining the company, he was granted an "inducement" equity award in accordance with Listing Rule 5635(c)(4) of the corporate governance rules of the Nasdaq Stock Market.  The award was approved by the Compensation Committee of Cogent’s Board of Directors, made up entirely of independent directors, as an inducement material to Mr. Dibabu’s employment. The award consists of nonqualified options to purchase 100,000 shares of Cogent common stock with a 10-year term, at an exercise price of $10.74 per share, and a 4-year vesting schedule with 25% vesting on the 1-year anniversary of Mr. Dibabu’s employment and the remainder vesting in equal monthly installments over the subsequent 36 months, provided Mr. Dibabu’s remains employed through each such vesting date.

On September 3, 2024 Biostar Pharma, Inc., the US subsidiary of Beijing Biostar Pharmaceuticals Co., Ltd. which is a synthetic biology driven biopharma company focusing on the discovery, development and commercialization of innovative oncology drugs, reported that subject recruitment of a US Phase 1 clinical study of Utidelone Capsule (UTD2), a company’s key pipeline product, for advanced solid tumors (NCT05681000) has been completed (Press release, Biostar, SEP 3, 2024, View Source [SID1234646322]).

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This study is an open-label, dose-escalation Phase 1 clinical study, and conducted in several institutions across the US including Sarah Cannon Research Institute (Florida Cancer Specialists & Research Institute), University of Southern California, and the Washington University in St. Louis. The primary objective of this study is to determine the maximum tolerated dose (MTD) and dose-limiting toxicity (DLT); the secondary objective is to evaluate the efficacy and pharmacokinetic profile of UTD2 monotherapy in patients with advanced solid tumors.

Five dose cohorts were explored in this study. After confirming the DLT (diarrhea) in the highest dose cohort, 75mg/m2/day x 5 days was determined as MTD. A total of 18 patients with more than 10 different types of advanced solid tumors were enrolled. The median age was 63 years old, and 2/3 of the patients had received ≥ 3 lines of prior treatments. As of now, efficacy evaluations were completed in 12 patients, and 1 complete response (CR, ovarian cancer), 1 partial response (PR, ovarian cancer), and 8 stable disease (SD, prostate cancer, testicular cancer, non-small cell lung cancer, pancreatic cancer, appendix adenocarcinoma, soft tissue sarcoma, etc.) were obtained with a clinical benefit rate of 83%. The results further demonstrated the broad-spectrum anti-tumor property of Utidelone. As for treatment duration, more than half of these patients received ≥3 cycles of treatments, and the maximum duration of response reached 36 weeks. In terms of safety, most treatment-related adverse events (TRAEs) were grade 1-2, and the most frequently seen TRAEs (incidence ≥ 20%, including all grades) were diarrhea (55.6%), fatigue (33.3%), and neutropenia (22.2%), all of which were manageable. The preliminary results of this study were also presented at the 2024 ASCO (Free ASCO Whitepaper) meeting.

Dr. Li Tang, Chairman of Biostar Pharma commented: "This is the first study of UTD2 that has completed enrollment globally. We successfully achieved the primary objectives and observed encouraging efficacy and safety profiles. We are very grateful for the joint engagement and dedication of our US clinical institutions and investigators. We are confident that this blockbuster product will substantially change the administration regimen of microtubule inhibitors. We will advance the following pipelines for UTD2 at full speed and hopefully bring it to the market to benefit global cancer patients as soon as possible".

Biostar is actively carrying out clinical studies of UTD2 in China and abroad. A pivotal study in China for advanced breast cancer led by Professor Xu Binghe, Cancer Hospital of Chinese Academy of Medical Sciences, is in recruitment. The preliminary findings of this study were also presented at the ASCO (Free ASCO Whitepaper) meeting in 2024; the China Phase 2 study for advanced solid tumors including ovarian cancer and cholangiocarcinoma, etc. is in initiation. In addition, US FDA has granted an Orphan Drug Designation (ODD) to UTD2 for the treatment of advanced gastric cancer, and Biostar is preparing for IND filing of a Phase 2/3 multi-reginal clinical study (MRCT) led by Professor Ruihua Xu of Sun Yat-sen University Cancer Center in both China and US simultaneously.

Utidelone is a new-generation genetically engineered microtubule inhibitor. Utidelone Injection (UTD1) has been launched in China in 2021 for the treatment of metastatic breast cancer (MBC) progressed after at least one anthracycline- or taxane-containing chemotherapy regimen. The phase 3 study data showed that UTD1 is the only one to achieve both PFS and OS benefits for heavily pretreated MBC patients, and the results were twice orally presented at ASCO (Free ASCO Whitepaper) annual meetings. Utidelone has similar mechanism of action with that of taxanes while demonstrating multiple advantages, including better anti-tumor activity, broader anti-tumor spectrum, better safety profile with very low hematologic toxicity, effective against multidrug-resistant tumors with less prone to develop drug resistance, capability of crossing the blood-brain barrier so as to prevent and treat brain tumor, and high oral bioavailability. Multiple indication expansion clinical studies for UTD1 are also in progress in China and US, including but not limited to MRCT Phase 3 study for non-small-cell lung cancer (NSCLC), Phase 2 pivotal study for breast cancer brain metastasis in US and NSCLC brain metastasis in China, and Phase 3 study for breast cancer neoadjuvant in China.

Compared to taxanes, which are difficult for oral formulation development, Utidelone is not susceptible to P-glycoprotein thus cannot be pumped out of the cancer cell by P-glycoprotein and has the advantage for higher oral bioavailability. By utilizing its synthetic biology technology platform, Biostar developed Utidelone Capsule, and its efficacy and safety have been confirmed in both US and China’s studies. Utidelone Capsule will significantly improve the convenience of administration, compliance of patients, decrease in treatment cost and ease of combination therapy with other oral anti-cancer drugs, meaning more suitable for adjuvant and maintenance therapy.

On September 3, 2024 Corvus Pharmaceuticals, Inc. (NASDAQ: CRVS), a clinical-stage biopharmaceutical company, reported that Richard A. Miller, M.D., president and chief executive officer, and Jeffrey Arcara, chief business officer, will conduct one-on-one meetings with investors and present a corporate overview at the H.C. Wainwright 26th Annual Global Investment Conference, which is being held in New York from September 9-11, 2024 (Press release, Corvus Pharmaceuticals, SEP 3, 2024, View Source [SID1234646290]). The Company’s presentation will be on Tuesday, September 10 at 12:00pm ET.

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A webcast of the presentation will be available live and for 90 days following the event. The webcast may be accessed via the investor relations section of the Corvus website.

On September 3, 2024 Jazz Pharmaceuticals plc (Nasdaq: JAZZ) ("Jazz Pharmaceuticals") reported that Jazz Investments I Limited, its wholly-owned subsidiary (the "Issuer"), intends to offer, subject to market conditions and other factors, $850 million aggregate principal amount of exchangeable senior notes due 2030 (the "notes") in a private offering (the "offering") to qualified institutional buyers pursuant to Rule 144A under the Securities Act of 1933, as amended (the "Securities Act") (Press release, Jazz Pharmaceuticals, SEP 3, 2024, View Source [SID1234646323]). The Issuer also expects to grant the initial purchasers of the notes an option, exercisable within a period of 13 days from and including the date the notes are first issued, to purchase up to an additional $150 million aggregate principal amount of notes.

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The notes will be exchangeable under certain conditions. The Issuer will settle exchanges by paying cash up to the aggregate principal amount of the notes to be exchanged. The remainder, if any, of the Issuer’s exchange obligation in excess of the aggregate principal amount of the notes, will be settled in cash, ordinary shares of Jazz Pharmaceuticals ("ordinary shares") or a combination of cash and ordinary shares, at the Issuer’s election. The interest rate, initial exchange rate and other terms of the notes will be determined at the time of pricing of the offering.

The notes will be general unsecured obligations of the Issuer and will accrue interest payable semiannually in arrears. The Issuer’s obligations under the notes will be fully and unconditionally guaranteed on a senior unsecured basis by Jazz Pharmaceuticals; will rank pari passu in right of payment with the Issuer’s existing 2.000% exchangeable senior notes due 2026; will be effectively subordinated to the Issuer’s guarantees of the indebtedness under Jazz Pharmaceuticals’ credit agreement (the "credit agreement") and Jazz Pharmaceuticals’ 4.375% senior secured notes due 2029 (the "senior secured notes") to the extent of the value of the assets securing such guarantees; and will be structurally subordinated to the indebtedness and guarantees under the credit agreement and the senior secured notes of Jazz Pharmaceuticals’ other subsidiaries that are borrowers or have provided guarantees of such indebtedness.

Jazz Pharmaceuticals, together with its consolidated subsidiaries ("Jazz"), expects to use a portion of the net proceeds to prepay up to approximately $350 million of the term loans outstanding under the credit agreement and the remainder for general corporate purposes. If the initial purchasers exercise their option to purchase additional notes, Jazz expects to use the net proceeds from the sale of the additional notes for further prepayments of the term loans.

Jazz Pharmaceuticals also expects to repurchase up to $150 million of its ordinary shares from purchasers of the notes in privately negotiated transactions with or through one of the initial purchasers or its affiliate concurrently with the pricing of the offering (the "concurrent ordinary share repurchases"). Jazz Pharmaceuticals expects the purchase price per ordinary share repurchased in such concurrent ordinary share repurchases to equal the closing price per ordinary share on the date of the offering. To the extent Jazz Pharmaceuticals effects any such concurrent ordinary share repurchases, it will pay for such repurchases with existing cash on hand and such repurchases will be effected as part of Jazz Pharmaceuticals’ share repurchase program announced in July 2024. Accordingly, any such concurrent ordinary share repurchases will reduce the remaining amount authorized under the share repurchase program. No assurance can be given as to how much, if any, of Jazz Pharmaceuticals’ ordinary shares will be repurchased or the terms on which they will be repurchased. The concurrent ordinary share repurchases could increase, or reduce the size of any decrease in, the market price of the ordinary shares, including concurrently with the pricing of the notes, resulting in a higher effective exchange price for the notes. This press release is not an offer to repurchase the ordinary shares, and the offering of the notes is not contingent upon the repurchase of any ordinary shares.

None of the notes, the guarantee or the ordinary shares issuable upon exchange of the notes, if any, have been registered under the Securities Act or the securities laws of any other jurisdiction, and, unless so registered, may not be offered or sold in the United States absent registration or an applicable exemption from, or in a transaction not subject to, the registration requirements of the Securities Act and other applicable securities laws.

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

On September 2, 2024 Theolytics, a clinical-stage biotechnology company developing next-generation oncolytic viral therapies, reported that it has been awarded £2M from Innovate UK’s prestigious Transforming Cancer Therapeutics grant funding competition to support the clinical development of its lead candidate THEO-260 (Press release, Theolytics, SEP 2, 2024, View Source [SID1234646268]). This non-dilutive funding adds to the £19M raised earlier this year from a strong investor syndicate comprising M Ventures, Taiho Ventures, Epidarex Capital, Oxford Science Enterprises, Sound Bioventures and Oxford University Innovation.

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Innovate UK, part of UK Research and Innovation, invests in innovative projects that are positioned to advance next-generation immunotherapies for cancer.

The grant will primarily support the Company’s upcoming Phase 1 multi-centre, open-label first-in-human trial to evaluate safety and tolerability of THEO-260 in patients with advanced-stage platinum-resistant ovarian cancer and determine the recommended Phase 2 dose. This trial is expected to begin in the coming months.

It will further allow for comprehensive analysis to elucidate and evidence THEO-260’s unique mechanism of action to destroy cancer cells and immune suppressive stromal cells, whilst also inducing T cell activation, through a comprehensive biomarker study, including the development of novel biomarker tests. Proceeds will also support the evolution of Theolytics as it continues to expand its footprint and reach as a clinical-stage oncology biotech company.

"This competitive award from Innovate UK in the category of "Transforming Cancer Therapeutics" reflects the novelty of our research as well as our ability to deliver operational excellence as we move forward to the clinic. This will allow us to precisely demonstrate the mechanism of action of THEO-260, which we anticipate will provide key information to enhance further development of this immunotherapeutic oncolytic virus and our wider platform," said Miriam Bazan Peregrino, Theolytics’ VP Translational Development.

About THEO-260

THEO-260 is a next-generation oncolytic adenovirus for the treatment of ovarian cancer. Positioned to tackle the complex, immune suppressed nature of advanced solid tumours. It demonstrates effective killing of cancer cells and cancer-associated fibroblasts (CAFs), whilst inducing immune activation in advanced preclinical models, including extensive panels of ovarian cancer patient samples. THEO-260 will be evaluated in Phase I clinical trials by intravenous and intraperitoneal delivery to ovarian cancer patients.