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 3, 2024 Primrose Bio, a leading biotechnology company specializing in technologies that improve the manufacturing of next-generation therapeutics, and ExPLoRNA Therapeutics, a pioneering biotechnology company advancing mRNA technologies, vaccines, and therapeutics, reported a strategic partnership aimed at jointly developing and marketing products for mRNA medicines (Press release, ExploRNA Therapeutics, SEP 3, 2024, View Source [SID1234646291]).

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The landscape of mRNA medicines is evolving, marked by an expanding pipeline of drug candidates, a diverse range of targeted therapeutic indications, and advancements in drug design and delivery. Consequently, the pharmaceutical industry is confronted with pressing challenges in manufacturing. To effectively address these challenges, innovative solutions are necessary.

Primrose Bio’s expertise in engineering high-performance RNA polymerases (Prima RNApols) enhances both the yield and fidelity of mRNA synthesis, effectively reducing double-stranded RNA (dsRNA) and lowering costs. This advancement meets the rigorous manufacturing demands for a wide range of mRNA-based applications in therapeutics and vaccines.

ExPLoRNA Therapeutics specializes in innovations of mRNA as a therapeutic platform, focusing on novel cap analogs vital for successful protein production. Their technology significantly increases protein production compared to standard cap analogs, limits double-stranded RNA formation, and brings down IVT cost, offering promising advancements in therapeutic effectiveness.

Together, Primrose Bio and ExPLoRNA will co-develop and offer RNA polymerases and capping compounds, co-optimized to enhance mRNA quality, manufacturing performance, and therapeutic safety and efficacy. This partnership provides mRNA drug developers with superior manufacturing solutions and innovative design options through advanced capping chemistry.

"We are excited to work with ExPLoRNA to bring industry-leading mRNA cap analogs to the market and pairing them with Primrose Bio’s RNA polymerases," said Helge Zieler, CEO of Primrose Bio. "This partnership will result in broader manufacturing and design options for companies developing mRNA therapeutics and vaccines, facilitating access to the most efficient capping chemistry available."

"Through joining forces, we’ll advance the mRNA as a modality from the first applications to the next generation," said Jacek Jemielity, CEO of ExPLoRNA Therapeutics. "We aim at creating the best synergies between our cap analogs and novel polymerases to surpass current manufacturing process and what is achievable with mRNA today."

On September 3, 2024 Precision BioSciences, Inc. (Nasdaq: DTIL), an advanced gene editing company utilizing its novel proprietary ARCUS platform to develop in vivo gene editing therapies for sophisticated gene edits, including gene elimination, gene insertion, and gene excision, reported maturity of a $13 million convertible note from its previously announced strategic transaction with Imugene Limited (ASX: IMU) for azercabtagene zapreleucel (azer-cel) in oncology (Press release, Precision Biosciences, SEP 3, 2024, View Source [SID1234646306]). The proceeds to Precision BioSciences include $9.75 million in cash and $3.25 million in Imugene ordinary shares.

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"We are pleased that Imugene continues to advance azer-cel toward a pivotal trial in LBCL for patients with significant unmet need," said Michael Amoroso, President and Chief Executive Officer of Precision BioSciences. "This marks one year since our strategic decision to focus our in-house programs exclusively on in vivo gene editing and further bolsters our balance sheet, enabling us to generate Phase 1 clinical data across multiple in vivo gene editing programs starting in the first half of 2025. In the near term, we are focused on progressing our pipeline towards the clinic and remain on track to file applications with multiple global regulatory bodies for PBGENE-HBV for chronic hepatitis B later this year."

In August 2023, Precision announced a strategic transaction with Imugene. Under the agreement, Imugene assumed global rights to azer-cel for cancer, the ongoing clinical execution for azer-cel in large B-cell lymphoma (LBCL) patients who have relapsed following autologous CAR T treatment as well as Precision BioSciences’ CAR T capability, including manufacturing. In exchange, Precision received upfront economics valued at $21 million (all figures in USD) consisting of $8 million in cash and a $13 million convertible note. The convertible note matured on August 30, 2024 and resulted in payment to Precision of $9.75 million in cash and $3.25 million in Imugene stock. Precision is also eligible for additional payments based on azer-cel clinical milestones as well as milestones for other future nominated cancer research programs.

As of June 30, 2024, Precision had approximately $123.6 million in cash and cash equivalents, prior to receipt of the payment from Imugene. Existing cash and cash equivalents, upfront and potential near-term cash from CAR T transactions, along with expected operational receipts, continued fiscal and operating discipline, availability of Precision’s at-the-market (ATM) facility, and marketable securities are expected to extend Precision’s cash runway into the second half of 2026.