On August 21, 2024 Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for rare and ultrarare diseases, reported the grant of 37,975 restricted stock units of the company’s common stock to 12 newly hired non-executive officers of the company (Press release, Ultragenyx Pharmaceutical, AUG 21, 2024, View Source [SID1234646034]). The awards were approved by the compensation committee of the company’s board of directors and granted under the Ultragenyx Employment Inducement Plan, with a grant date of August 16, 2024, as an inducement material to the new employees entering into employment with Ultragenyx in accordance with Nasdaq Listing Rule 5635(c)(4).

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The restricted stock units vest over four years, with 25% of the underlying shares vesting on each anniversary of the grant date, subject to the employee being continuously employed by the company as of such vesting dates.

On August 21, 2024 Propanc Biopharma, Inc. (OTC Pink: PPCB) ("Propanc" or the "Company"), a biopharmaceutical company developing novel cancer treatments for patients suffering from recurring and metastatic cancer, reported that two scientific, peer reviewed journal articles published by the Company and its research partners reached 10 citations and 4,500 reads, respectively, in August, 2024 (Press release, Propanc, AUG 21, 2024, View Source [SID1234646032]). "This shows unprecedented interest in our field from researchers and among the broader scientific community," said Dr Kenyon, MD, MB, ChB, Propanc’s Chief Scientific Officer.

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The first article, which reached 10 citations, published in Scientific Reports, is entitled, "Pancreatic proenzymes treatment suppresses BXPC-3 pancreatic Cancer Stem Cell subpopulation and impairs tumour engrafting." From the publishers of Nature, it is an online, open access journal, which publishes from all areas of the natural and clinical sciences. According to an insider, Scientific Reports is published on the most prestigious site of the National Institute of Health (NIH) and considered top line coverage: View Source

Published data from the Company’s joint research and development program were generated in conjunction with the Universities of Jaén and Granada, Biosanitary Research Institute of Granada, and University Hospital, Spain. Cancer stem cells (CSCs) subpopulation within the tumour is responsible for metastasis and cancer relapse. Exposure of human pancreatic CSCs to proenzymes resulted in significant decrease of specific pancreatic CSC markers. Also, in vivo (in a living organism) anti-tumor, xenograft (tissue graft) studies demonstrated high anti-tumour efficacy against tumors induced by human pancreatic CSCs. They concluded that "proenzymes treatment is a valuable strategy to suppress the CSC population in solid pancreatic tumours."

The second article, achieved 4,500 reads, also published in Scientific Reports, entitled "A formulation of pancreatic proenzymes provides potent anti-tumor efficacy: A pilot study focused on pancreatic and ovarian cancer." Published data from the Company’s R&D program were conducted with vivoPharm Pty Ltd, Australia, and the Dove Clinic for Integrated Medicine, UK, along with the Company’s joint researchers. Highlights include anti-angiogenic (anti-tumor blood vessel formation) effects of proenzymes, along with analysis of epithelial to mesenchymal transition (EMT) markers performed on human cancer cells treated with proenzymes. The EMT is a mechanism by which cancerous cells become motile and invasive, as well as immortal, thus seeding new tumors. For online access: View Source

Additionally, clinical efficacy of a suppository formulation of pancreatic proenzymes in the context of a UK Pharmaceutical Specials Scheme, led by Dr Kenyon, where 19 from 46 patients (41.3%) with late-stage cancers, had a survival time significantly longer than expected. Mean survival of 9.0 months was significantly higher than mean life expectancy, 5.6 months, in a one-way ANOVA (analysis of variance) test (alpha = 0.05, P less than 0.05).

For diagnosed pancreatic and ovarian cancer patients, there is a 20% survival rate and a 5-year relative survival rate of 29%, respectively, according to the National Cancer Institute. The global market projection for pancreatic cancer drugs is $6.93 billion by 2030, according to Brainy Insights and for ovarian cancer drugs is $13.9 billion by 2029, according to iHealthcareAnalyst.

"Survival rates, especially for pancreatic and ovarian cancers, continue to remain low. As a result, there is a pressing market need for new therapies, but without severe, or serious side effects associated with standard treatments," said Dr Kenyon. "We look forward to advancing our lead asset, PRP, into early-stage clinical development to further improve the significant life extension I first observed using proenzymes treatment in the UK Pharmaceuticals Scheme."

About PRP:

PRP is a mixture of two proenzymes, trypsinogen and chymotrypsinogen from bovine pancreas, administered by intravenous injection. A synergistic ratio of 1:6 inhibits growth of most tumor cells. Examples include pancreatic, ovarian, kidney, breast, brain, prostate, colorectal, lung, liver, uterine, and skin cancers. Orphan Drug Designation status of PRP has been granted from the US Food and Drug Administration (FDA) for treatment of pancreatic cancer.

To view the Company’s "Mechanism of Action" video on the Company’s lead asset, PRP, please click on the following link: View Source

On August 20, 2024 Crown Bioscience, a global contract research organization (CRO) and a JSR Life Sciences company, reported a strategic partnership with Clinical Trial & Consulting Services (CTI), a renowned global full-spectrum research service provider, to bolster oncology-focused consulting services (Press release, Crown Bioscience, AUG 21, 2024, https://www.crownbio.com/about-us/news-and-events/crown-bioscience-partners-with-cti-to-support-consulting-services-in-oncology-drug-development [SID1234646030]). This collaboration unites decades of expertise from both organizations in oncology drug discovery and development, leveraging Crown Bioscience’s preclinical and translational models and CTI’s well-established track record in the clinical and regulatory space.

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Customers will benefit from combined expert guidance, supporting the transition of oncology compounds from discovery through early-phase clinical trials more rapidly and effectively. This alliance aims to determine the optimal and most expeditious path to the clinic, ensure long-term drug development success and growth, and accelerate the delivery of superior oncology therapeutics to patients.

From today, customers can access consulting services to ensure an integrated drug development approach, including:

Strategic preclinical development planning
Study data analysis, interpretation and advice
Regulatory guidance
De novo assay development
Grant review
Alex Slater, Senior Vice President of Commercial, commented: "Crown Bioscience has an established reputation for exceeding the typical services offered by a regular CRO. This initiative unites deep-seated expertise spanning all phases of drug development, marking a pivotal step towards delivering a true translational offer to our clients."

Ryan Gifford, Vice President of Global Laboratory Services, Business Development, shares: "We are delighted to collaborate with Crown Bioscience and view this as a valuable synergy. Crown Bioscience’s leadership in early-phase oncology research and suite of preclinical services complement CTI’s broad spectrum of capabilities. Together, we can offer enhanced support to our oncology clients and deepen our commitment to advancing cancer research."

On August 21, 2024 Chemomab Therapeutics Ltd. (Nasdaq: CMMB), (Chemomab), a clinical stage biotechnology company developing innovative therapeutics for fibro-inflammatory diseases with high unmet need, reported financial and operating results for the second quarter ended June 30, 2024, and provided a corporate update (Press release, Chemomab, AUG 21, 2024, View Source [SID1234646028]).

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"This past period has been an exciting time of transformation for Chemomab. The positive results of the Phase 2 SPRING trial represent a major milestone for the company and clearly establish clinical proof-of-concept for CM-101 in primary sclerosing cholangitis (PSC) and potentially other fibrotic diseases," said Adi Mor, PhD, co-founder, Chief Executive Officer and Chief Scientific Officer of Chemomab. "CM-101 achieved the primary and key secondary endpoints in the trial and is the first therapy to demonstrate broad, clinically relevant effects on the three main components of PSC. This is also the first PSC trial to show, after just 15 weeks of treatment, a statistically significant reduction in liver stiffness, a widely used and well validated measure for assessing disease progression in PSC."

"We believe these results provide strong support for advancing CM-101 to a Phase 3 PSC trial, which we are in the process of planning and will be discussing with the FDA during the End-of-Phase 2 meeting targeted for later this year. We anticipate two milestones early in 2025 – additional data from the open label extension portion of the SPRING trial and finalization of the PSC Phase 3 design reflecting our discussions with the FDA. Based on recent developments in related indications, we are optimistic that an accelerated approval design incorporating surrogate biomarkers may be feasible for CM-101."

Dr. Mor continued, "We were pleased to welcome existing and major new investors, including OrbiMed, HBM Partners and Sphera, who participated in our successful $10 million PIPE financing. The proceeds generated by this financing will enable us to achieve our upcoming milestones in early 2025 and fund the operations of the company through the beginning of 2026."

Dr. Mor concluded, "As a first-in-class molecule with a promising novel target relevant to multiple fibro-inflammatory diseases, CM-101 has long been of interest to potential biopharma partners. As expected, the positive data reported from the Phase 2 SPRING trial provides us opportunities to further advance the optimal path forward for CM-101, including potentially partnering to accelerate timelines for CM-101 development in PSC and other indications."

Second Quarter 2024 and Recent Updates

On July 30, 2024, Chemomab announced the closing of a private placement that resulted in gross proceeds of approximately $10 million. Existing investors such as OrbiMed and new investors including HBM Partners and Sphera Biotech Master Fund participated in the financing, which extends the company’s cash runway through early 2026.

On July 25, 2024, Chemomab reported topline results from the CM-101 Phase 2 SPRING trial in patients with PSC. CM-101 met the primary study endpoint, demonstrating a favorable safety profile over the 15-week treatment period. CM-101-treated patients with moderate/advanced disease showed improvements on a wide range of disease-related secondary endpoints, including assessments of changes from baseline relative to placebo at Week 15 in liver stiffness; in liver fibrosis biomarkers, including the Enhanced Liver Fibrosis (ELF) score and PRO-C3 levels; in total bilirubin and liver function tests; in pruritis (itch) and in markers of inflammation. Dose-dependent responses were observed for multiple disease-related biomarkers. A consistent pattern of greater improvement on the secondary endpoints was observed in the study arm receiving the higher 20 mg/kg dose of CM-101 and in the prespecified subgroup of PSC patients with moderate/advanced disease. The open label extension portion of the Phase 2 SPRING trial is continuing, with results expected to be reported in early 2025.

On June 18, 2024, Chemomab announced new scientific publications reinforcing the clinical potential of CM-101 in PSC. A proteomic analysis of patient samples further confirmed that the company’s novel CCL24 target is associated with disease severity and progression in PSC.

On June 6, 2024, Chemomab participated in multiple data presentations at EASL 2024 and a Gordon Research Conference supporting the clinical potential of CM-101 as a novel treatment for PSC. The findings support CM-101’s mode of action in liver fibrosis and could help in characterizing its anti-fibrotic drug effects and potentially serve as a translational tool in future PSC clinical trials.

On April 18, 2024, Chemomab announced a new peer-reviewed publication reinforcing the clinical association of its novel CCL24 target with disease severity and mortality in patients with systemic sclerosis.

On April 10, 2024, Chemomab hosted an expert PSC webinar featuring Christopher Bowlus, MD, of UC Davis Health; Ricky Safer, founder and CEO of PSC Partners Seeking a Cure and Massimo Pinzani, MD, PhD, of the UCL Institute for Liver and Digestive Health and UPMC ISMETT.
Second Quarter 2024 Financial Highlights

Cash Position: Cash, cash equivalents and short-term bank deposits were $12.8 million as of June 30, 2024, compared to $19.9 million as of December 31, 2023. On July 30, 2024, Chemomab successfully closed a $10 million private investment.

Research and Development (R&D) Expenses: R&D expenses were $2.9 million for the second quarter of 2024, compared to $5.0 million for the second quarter of 2023. The decrease in R&D expenses in the second quarter of 2024 compared to the second quarter of 2023 primarily resulted from the completion of the double-blinded portion of the company’s CM-101 Phase 2 PSC trial.

General and Administrative (G&A) Expenses: G&A expenses were $0.8 million for the second quarter of 2024, compared to $3.2 million for the second quarter of 2023. The decrease in G&A expenses primarily reflected reductions in headcount, consulting fees and other cost savings.

Net Loss: Net loss was $3.6 million, or a net loss of approximately $0.01 per basic and diluted ordinary share for the second quarter of 2024, compared to $8.0 million, or a net loss of approximately $0.04 per basic and diluted ordinary share for the second quarter of 2023. The weighted average number of ordinary shares outstanding, basic and diluted, was 286,080,133 (equal to approximately 14.3 million ADSs) for the second quarter of 2024.

Liquidity and Capital Resources: Chemomab believes its existing liquidity resources as of June 30, 2024, together with the additional funds of approximately $10 million raised in July 2024, will enable the Company to fund its operations through the beginning of 2026.

Number of issued and outstanding shares: Following completion of its July 2024 financing, the Company had 18,508,057 ADSs (representing 370,161,140 ordinary shares) issued and outstanding and 25,121,231 ADSs (representing 502,424,620 ordinary shares) outstanding on a fully diluted basis.

On August 21, 2024 Bristol Myers Squibb (NYSE: BMY) reported that the U.S. Food and Drug Administration (FDA) has accepted the supplemental Biologics License Application (sBLA) for Opdivo (nivolumab) plus Yervoy (ipilimumab) as potential first-line treatment for adult patients with unresectable hepatocellular carcinoma (HCC), based on results from the Phase 3 CheckMate -9DW trial (Press release, Bristol-Myers Squibb, AUG 21, 2024, View Source [SID1234646027]). The FDA assigned a Prescription Drug User Fee Act (PDUFA) goal date of April 21, 2025.

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"HCC is the most common form of liver cancer and is often diagnosed when surgery is no longer an option. With the number of individuals diagnosed with HCC in the United States increasing over the last decade, new treatment options are urgently needed," said Dana Walker, M.D., M.S.C.E., vice president, global program lead, gastrointestinal and genitourinary cancers, Bristol Myers Squibb. " Opdivo plus Yervoy showed superior survival benefit compared to other available treatment options, and we look forward to working with the FDA to advance our application to potentially bring a new first-line treatment option to patients."

The filing was based on the results from the Phase 3 CheckMate -9DW study in which the combination demonstrated a statistically significant and clinically meaningful improvement in overall survival (OS) compared to investigator’s choice of lenvatinib or sorafenib. The combination of Opdivo plus Yervoy has been an established second-line treatment for patients with advanced HCC, and these results support the combination becoming a potential new treatment option in the first-line setting. The safety profile for the combination of Opdivo plus Yervoy remained consistent with previously reported data and was manageable with established protocols, with no new safety signals identified. Results were presented at the 2024 American Society of Oncology (ASCO ) Annual Meeting .

Bristol Myers Squibb thanks the patients and investigators involved with the Phase 3 CheckMate -9DW clinical trial.

About CheckMate -9DW

CheckMate -9DW is a Phase 3 randomized, open-label trial evaluating the combination of Opdivo plus Yervoy compared to investigator’s choice of lenvatinib or sorafenib monotherapy in patients with unresectable or advanced hepatocellular carcinoma who have not received prior systemic therapy.

668 patients were randomized to receive Opdivo plus Yervoy ( Opdivo 1mg/kg plus Yervoy 3 mg/kg Q3W for up to four doses, followed by Opdivo monotherapy 480 mg for a maximum duration of 2 years) infusion, or single agent lenvatinib or sorafenib as oral capsules in the control arm. The primary endpoint of the trial is overall survival and key secondary endpoints include objective response rate and time to symptom deterioration.

About Hepatocellular Carcinoma

Liver cancer is the third most frequent cause of cancer death worldwide. Hepatocellular carcinoma (HCC) is the most common type of primary liver cancer and accounts for 75%-85% of all liver cancers. HCC is often diagnosed in an advanced stage, where effective treatment options are limited and are usually associated with poor outcomes.

Up to 70% of patients experience recurrence within five years, particularly those still considered to be at high risk after surgery or ablation. While most cases of HCC are caused by hepatitis B virus (HBV) or hepatitis C virus (HCV) infections, metabolic syndrome and nonalcoholic steatohepatitis (NASH) are rising in prevalence and expected to contribute to increased rates of HCC.