On August 31, 2023 Twist Bioscience Corporation (NASDAQ: TWST), a company enabling customers to succeed through its offering of high-quality synthetic DNA using its silicon platform, reported a drug discovery agreement with Ono Pharmaceutical Co., Ltd. (Osaka, Japan; President and CEO: Gyo Sagara; "Ono") to discover and develop novel antibodies for the treatment of autoimmune diseases (Press release, Twist Bioscience, AUG 31, 2023, View Source [SID1234634817]).

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"Ono has a strong and proven track record in developing antibody candidates in areas of need, including the development of the anti-PD-1 antibody Opdivo, which has had a tremendous impact for patients and researchers," said Emily M. Leproust, Ph.D., CEO and co-founder of Twist Bioscience. "This collaboration bringing together our antibody discovery expertise with Ono’s experience in drug development has the potential to lead to future breakthrough treatment options for patients with autoimmune diseases."

Under the terms of the agreement, Twist will utilize the Twist Biopharma Solutions Library of Libraries to conduct research activities to discover novel antibodies against targets identified by Ono. Twist’s Library of Libraries is an expansive collection of synthesized antibody libraries based on naturally occurring sequences that harness innovative structural and developability features to cover a wide range of drug targets. Twist will receive research fees, success-based clinical and regulatory milestones, as well as royalties on product sales. As part of the strategic collaboration, Ono will also utilize the scientific expertise of the Biopharma Solutions team and Twist’s Premium Project Management services to evaluate new targets and generate comprehensive discovery campaigns. Ono will be responsible for the development, manufacturing and commercialization of any products resulting from the collaboration.

"We are actively working to create biologics like antibodies in order to address unmet medical needs for a wide range of diseases including autoimmune diseases. We are very pleased to collaborate with Twist at this time in the area of autoimmune diseases and are confident that their expertise in antibody discovery and their impressive collection of diverse and highly specific antibody libraries will bring us potential functional antibodies," said Dr. Toichi Takino, Senior Executive Officer / Executive Director, Discovery and Research of Ono. "We believe that this new collaboration will enhance our biologics drug discovery efforts and bring innovative drugs for patients as soon as possible."

On August 31, 2023 Nuvalent, Inc. (Nasdaq: NUVL), a clinical-stage biopharmaceutical company focused on creating precisely targeted therapies for clinically proven kinase targets in cancer, reported that James Porter, Ph.D., Chief Executive Officer, and Alexandra Balcom, Chief Financial Officer, will participate in fireside chats during the following September investor conferences (Press release, Nuvalent, AUG 31, 2023, View Source [SID1234634816]):

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2023 Wells Fargo Securities Healthcare Conference on Friday, September 8, 2023 at 10:15 a.m. ET in Boston;
Morgan Stanley 21st Annual Global Healthcare Conference on Wednesday, September 13, 2023 at 8:50 a.m. ET in NYC.
Live webcasts will be available in the investor section of the company’s website at www.nuvalent.com, and archived for 30 days following the presentations.

On August 31, 2023 CANbridge Pharmaceuticals, Inc. ("CANbridge," stock code 1228.HK), a global biopharmaceutical company, with a foundation in China, committed to the research, development and commercialization of transformative therapies for rare diseases and oncology indications, reported its financial results for the six months ended June 30, 2023 plus corporate updates (Press release, CANbridge Life Sciences, AUG 31, 2023, View Source [SID1234634815]).

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"On the product front, it had been a very productive first half year for CANbridge. We achieved multiple data and regulatory milestones that we believe will continue to drive both near- and long-term value for our stockholders," said James Xue, Ph.D., CANbridge founder, chairman, and CEO. "Regarding new data, we have encouraging updates on several programs, notably CAN106 for treating paroxysmal nocturnal hemoglobinuria (PNH), as well as CAN203, our gene therapy candidate for SMA. On the regulatory front, we are pleased to have received approval for LIVMARLI (maralixibat/CAN108) in China for treating Cholestatic Pruritus in patients with the rare liver disease Alagille syndrome (ALGS). Livmarli is the first and only approved product in China for the disease, thus marked a pivotal milestone for CANbridge, given the sizeable commercial opportunity and the potential to expand the Livmarli label to cover multiple other rare liver disease indications. In the second half year, we look forward to achieving continuous progress in developing our pipeline of leading therapies for rare diseases."

Upcoming Milestones

CAN108 – Plan to launch Livmarli for patients with ALGS in China in Q1 2024.
CAN108 – Anticipate New Drug Application (NDA) approval for Livmarli’s use in ALGS patients aged one year and older in Taiwan and Hong Kong by the end of 2023.
CAN108 – Data from the Mirum-sponsored EMBARK Phase 2 trial in biliary atresia patients is expected in H2 2023.
CAN008 – Plan to report data from the Phase 2 clinical trial in H1 2024.
CAN106 – Begin enrolling a Phase 2 clinical trial in PNH in China that will commence in H2 2023 with data potentially ready in H2 2024.
Gene Therapy – Advance novel second-generation scAAV gene therapy for the treatment of spinal muscular atrophy (SMA)
Financial Highlights

Revenue increased by RMB8.4 million, or 24.2%, to RMB43.1 million for the six months ended 30 June 2023 from RMB34.7 million for the same period last year, mainly attributable to the increase in sales of Hunterase and Livmarli.
Research and development ("R&D") expenses decreased by approximately RMB15.3 million, or 9.7%, to RMB143.0 million for the six months ended 30 June 2023 from RMB158.3 million for the same period last year, primarily attributable to the decrease in upfront and milestone payments made to CANbridge’s licensing partners and in technical service fees, partially offset by the increase in depreciation and amortization costs.
Loss for the Reporting Period decreased by approximately RMB30.8 million, or 12.4%, to RMB218.2 million for the six months ended 30 June 2023 from RMB249.0 million for the same period last year, primarily attributable to the increase in revenue and decrease in selling and distribution expenses, R&D expenses and administrative expenses.
Recent Highlights

Hunterase (CAN101), an enzyme replacement therapy for treating MPS II, also known as Hunter syndrome, has been on the "First National List of Rare Diseases" since May 2018.

It was launched in May 2021 in mainland China as the first and only ERT for MPS II, and since then more new patients have been identified, up to 739 as of 30 June 2023.
Expanded relevant commercial insurance programs (Huiminbao) to 109 cities, covering a population of 586 million in China.
LIVMARLI is an oral, minimally absorbed reversible inhibitor of the ileal bile acid transporter (IBAT) being developed for treating rare cholestatic liver diseases. CANbridge has the exclusive rights to develop, commercialize, and under certain conditions, manufacture LIVMARLI in Greater China.

On May 29, 2023, the Chinese National Medical Products Administration (NMPA) approved CAN108, making Livmarli the first and only approved product in China to be marketed for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) aged one year and older.
CANbridge plans to launch Livmarli for ALGS in Q1 2024.
Completed enrollment of the EMBARK Phase 2 study of Livmarli in biliary atresia (BA) in China in May 2023. The clinical trial in BA is conducted by Mirum and supported by CANbridge under the license agreement with Mirum.
Expected to obtain NDA approval for Livmarli’s use in ALGS patients aged one year and older in Taiwan and Hong Kong by the end of 2023.
Plan to submit NDA for Livmarli’s use in PFIC patients in Taiwan and mainland China in the second half of 2023.
CAN106 is a novel, long-acting monoclonal antibody for treating complement-mediated diseases, including PNH, myasthenia gravis (MG) and various other complement-mediated diseases that are targeted by anti-C5 antibodies. PNH has been included on the "First National List of Rare Diseases" in China since May 2018.

Reported positive preliminary top-line Phase 1 data from the ongoing Phase 1b study of CAN106 in China for PNH. The results suggest complete blockade of complement function at safe and well-tolerated doses.
Based on the positive results from the Phase 1b study, CANbridge will begin enrolling a registrational clinical trial in PNH in China that will commence in the second half of 2023 with data amassed potentially in the second half of 2024.
CAN008, a glycosylated CD95-Fc fusion protein being developed for the treatment of glioblastoma multiforme (GBM).

The IDMC (independent data monitoring committee) completed an interim analysis and review of the ongoing Phase 2 study of CAN008 in China in patients with newly diagnosed GBM and recommended the study to continue without any changes to the current trial design.
CAN103 is an ERT for treating Gaucher Disease (GD) and GD has been included on the "First National List of Rare Diseases" in China since May 2018.

Completed Part A of the ongoing Phase 1/2 clinical trial in China and initiated Part B in the first quarter of 2023, with the latter to serve as a potential registrational trial.
CAN103 is the first clinical stage ERT being developed for GD in China.
In Gene Therapy, CANbridge advanced its world-class gene therapy platform, focusing on adeno-associated virus (AAV) as a gene delivery vehicle, with potential as a one-time durable therapy for many genetic diseases. Programs in Fabry disease and SMA have been included on the "First National List of Rare Diseases" since May 2018.

Appointed Jason West, Ph.D., as Vice President, Head of Gene Therapy Research. Dr. West possesses expertise in areas such asgene therapy development, platform innovation and clinical candidate development.
Presented preclinical data at the 2023 American Society of Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper) Annual Meeting. The data shared evidenced the potential of the novel, second-generation scAAV in treating SMA as it expresses a codon optimized (co)-hSMN1 from an endogenous hSMN1 promoter. Low-dose intracerebroventricular delivery of the gene therapy was able to achieve superior potency, efficacy and safety in mice with SMA, compared to the benchmark vector, scAAV-CMVen/CB-hSMN1, which is similar to the FDA-approved gene therapy vector for SMA.
CANbridge will also host a conference call to discuss the 2023 interim results and recent corporate progress. To register for the English session, please click here. To register for the Chinese session, please click here. This webcast will be English, and translation will be provided on the Chinese channel.

On August 31, 2023 Compugen Ltd. (Nasdaq: CGEN) (TASE: CGEN), a clinical-stage cancer immunotherapy company and a pioneer in computational target discovery, reported that management will present virtually at the H.C. Wainwright 25th Annual Global Investment Conference (Press release, Compugen, AUG 31, 2023, https://www.prnewswire.com/news-releases/compugen-to-present-at-the-hc-wainwright-25th-annual-global-investment-conference-301914569.html [SID1234634814]). The presentation will be available on demand on the Investor Relations section of Compugen’s website at www.cgen.com from Monday, September 11, 2023, 7:00 AM ET for 30 days.

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On August 31, 2023 Can-Fite BioPharma Ltd. (NYSE American: CANF) (TASE: CANF), a biotechnology company advancing a pipeline of proprietary small molecule drugs that address oncology, inflammatory and liver diseases, reported financial results for the six months ended June 30, 2023 (Press release, Can-Fite BioPharma, AUG 31, 2023, View Source [SID1234634813]).

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Clinical Progress

Pivotal Phase III Advanced Liver Cancer Study—Can-Fite’s pivotal Phase III liver cancer study, Liveration, which continues enrollment is designed to assess Namodenoson in the treatment of patients with advanced hepatocellular carcinoma (HCC) and underlying Child Pugh B7 (CPB7) who have not responded to one or two other lines of therapy. The primary endpoint is overall survival. An interim analysis will be conducted by an Independent Data Monitoring Committee (IDMC) after 50% of the planned 450 patients are enrolled and treated.

Breakthrough Abstract Award–Can-Fite was recently granted a prestigious Breakthrough Abstract Award by the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Conquer Cancer Foundationfor the development of a novel approach to treat advanced liver cancer with the A3 adenosine receptor agonist, Namodenoson.

Exploratory Phase II Pancreatic Cancer Study—Can-Fite is preparing an open-label Phase II exploratory trial to assess the safety and efficacy of Namodenoson in the treatment of patients with pancreatic cancer who have received at least one previous systemic therapy. In pre-clinical studies, Namodenoson demonstrated a robust anti-growth effect against pancreatic carcinoma, reaching 90% growth inhibition. The mechanism of action entails de-regulation of the Wnt signal transduction pathway, a key modulator of pancreatic carcinoma cell growth.

ASCO Recognition—Can-Fite’s pancreatic cancer program received recognition from ASCO (Free ASCO Whitepaper) when its study titled "Effects of Namodenoson on Pancreatic Carcinoma: Preclinical Evidence" was published in the Journal of Clinical Oncology supplement of the 2023 ASCO (Free ASCO Whitepaper) Annual Meeting Proceedings.

Preparatory Work for Pivotal Phase III Psoriasis Study; Can Fite Received Green Light from FDA and EMA— Following positive responses from the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA)for its registration plan and pivotal Phase III study protocol for Piclidenoson in the treatment of moderate to severe psoriasis, the Company is preparing for study initiation. The FDA requested two Phase III studies and also encouraged the Company to enroll adolescent patients due to Piclidenoson’s strong safety profile demonstrated over its development history and prior clinical studies. Can-Fite has submitted to the FDA a pediatric plan to allow the registration of Piclidenoson for the treatment of adolescents. Inclusion of adolescents for the psoriasis Indication is expected to broaden the market.

Development for Treatment of Lowe Syndrome, a Rare Genetic Disease—Researchers at the University of Naples Federico II and The Telethon Institute of Genetics and Medicine (TIGEM) in Italy found Piclidenoson to be effective in pre-clinical studies for the treatment of Lowe Syndrome. Can-Fite and Fondazione Telethon signed an agreement outlining their collaboration for the development of Piclidenoson for the treatment Lowe Syndrome, a rare genetic disease with no treatment available, and an estimated $100 million treatment market in the U.S. alone.

"With two ongoing studies and two more about to commence in large treatment markets with unmet needs, we continue to advance our late-stage development pipeline to bring our potentially game-changing drugs to market," stated Can-Fite CEO & CFO Motti Farbstein.

Dr. Pnina Fishman, Can-Fite’s CSO and Executive Chairman added, "We are particularly excited about our entry into the rare genetic disease field with the discovery of Piclidenoson’s efficacy in Lowe Syndrome in pre-clinical studies. Given Piclidenoson’s very favorable safety profile, we believe it will be a good candidate for directly entering advanced stage trials in children and adolescents living with Lowe Syndrome. Rare genetic diseases can have a more direct rapid path to regulatory approval with smaller trial sizes due to the pressing need in a small, unserved patient population. "

Financial Results

Revenues for the six months ended June 30, 2023 were $0.39 million compared to revenues of $0.41 million during the six months ended June 30, 2022. Revenues for the six months ended June 30, 2023 and June 30, 2022 comprised of recognition of a portion of advance payments received under distribution agreements with Gebro, CKD, Cipher and Ewopharma.

Research and development expenses for the six months ended June 30, 2023 were $3.41 million compared with $3.27 million for the same period in 2022. Research and development expenses for the first half of 2023 comprised primarily of expenses associated with the completion of the Phase 3 study of Piclidenoson for the treatment of psoriasis and two ongoing studies for Namodenoson, a Phase 3 study in the treatment of advanced liver cancer and a Phase 2b study for SLD. The increase is primarily due to an increase in expenses associated with Namodenoson.

General and administrative expenses were $1.47 million for the six months ended June 30, 2023 compared to $1.57 million for the same period in 2022. The decrease is primarily due to the decrease in public and investor relations expenses and in directors and officer’s insurance policy premium. We expect that general and administrative expenses will remain at the same level through 2023.

Financial income, net for the six months ended June 30, 2023 was $0.27 million compared to financial expense, net of $0.18 million for the same period in 2022. The decrease in financial expense, net was mainly due to revaluation of our short-term investment and increase in interest income from deposits in 2023.

Net loss for the six months ended June 30, 2023 was $4.22 million compared with a net loss of $4.62 million for the six months ended June 30, 2022. The decrease in net loss for the six months ended June 30, 2023 was primarily attributable to the increase in finance income, net.

As of June 30, 2023, Can-Fite had cash and cash equivalents and short term deposits of $9.60 million as compared to $7.98 million at December 31, 2022. The decrease in cash during the six months ended June 30, 2023 is due to the ongoing operations of the Company.