On September 12, 2024 Medivir AB (NASDAQ Stockholm: MVIR), a pharmaceutical company focused on developing innovative treatments for cancer in areas of high unmet medical need, reported that detailed and mature data from the phase 1b/2a study of fostrox (fostroxacitabine) in combination with Lenvima (lenvatinib) for the treatment of advanced hepatocellular carcinoma (HCC) will be presented at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Congress in Barcelona, September 16, 2024 (Press release, Medivir, SEP 12, 2024, View Source;lenvima-at-esmo-conference-and-to-host-a-webcast-on-september-16-302246468.html [SID1234646546]).

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The abstract, titled "Fostrox (fostroxacitabine bralpamide) plus lenvatinib in patients with locally advanced unresectable or metastatic hepatocellular carcinoma (HCC) progressed on immunotherapy combinations. Results from a multi-center phase 1b/2a study." will be presented by Dr Hong Jae Chon, CHA Bundang Medical Center in Korea.

After the presentation Medivir will also host a webcast where Dr Chon and Dr Pia Baumann, Chief Medical Officer at Medivir, will present the data and answer questions. The webcast will take place on September 16, at 13.45 CET, and will be streamed via a link on the website: www.medivir.com/investors/presentations.

In addition to the presentation of phase 1b/2a data, Dr Chon with his experience of treating liver cancer patients, will provide additional context to the data by comparing with what can be expected with current clinical practice in second-line.

The poster and the presentation from the webcast will be available on Medivir’s website after the webcast.

For additional information, please contact:

Magnus Christensen, CFO, Medivir AB
Telephone: +46 8 5468 3100
E-mail: [email protected]

About fostrox

Fostrox is a liver-targeted inhibitor of DNA replication that delivers the cell-killing compound selectively to the tumor while minimizing the harmful effect on normal cells. This is achieved by coupling an active chemotherapy (troxacitabine) with a prodrug tail. This design enables fostrox to be administered orally and travel directly to the liver where the active substance is released locally in the liver. With this unique mechanism, fostrox has the potential to become the first liver-targeted, orally administered drug that can help patients with various types of liver cancer. A phase 1b monotherapy study with fostrox has been completed and a phase 1b/2a combination study in HCC is ongoing where it has shown encouraging anti-cancer efficacy with a good safety and tolerability profile.

About primary liver cancer

Primary liver cancer is the third leading cause of cancer-related deaths worldwide. Hepatocellular carcinoma (HCC) is the most common cancer that arises in the liver and it is the fastest growing cancer in the USA. Although existing therapies for advanced HCC can extend the lives of patients, treatment benefits are insufficient and death rates remain high. There are approximately 660,000 patients diagnosed with primary liver cancer per year globally and current five-year survival is less than 20 percent1,2. HCC is a heterogeneous disease with diverse etiologies, and lacks defining mutations observed in many other cancers. This has contributed to the lack of success of molecularly targeted agents in HCC. The limited overall benefit, taken together with the poor overall prognosis for patients with intermediate and advanced HCC, results in a large unmet medical need.

On September 12, 2024 Kazia Therapeutics Limited (NASDAQ: KZIA), an oncology-focused drug development company, reported that an agreement has been executed with QIMR Berghofer Medical Research Institute, one of Australia’s foremost cancer research centres, to obtain an exclusive license to certain intellectual property rights in relation to combination therapies consisting of PI3K inhibitor drugs, and one or more immunotherapy or PARP inhibitor drugs (PI3K combination) (Press release, Kazia Therapeutics, SEP 12, 2024, View Source [SID1234646545]).

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Under the license agreement, Kazia receives an exclusive, worldwide, sub-licensable and royalty-bearing licence to certain intellectual property for the development of any drugs or product candidates within the PI3K inhibitor class in combination with immunotherapy or PARP inhibitors. Paxalisib, Kazia’s lead product candidate, is a member of the PI3K inhibitor class.

The exclusive license agreement follows a collaboration between Kazia and QIMR Berghofer which began in December 2022 and has already led to the filing of supportive patents which include the use of paxalisib as an immune modulator in the treatment of diseases such as breast cancer.

The terms of the license include standard provisions for an upfront license fee and development milestones related to the initiation of Phase 1, Phase 2 trial, first Phase 3 trial, first product approval.

Commenting on the new license agreement, Kazia CEO, Dr John Friend said: "This is an exciting evolution in our partnership with QIMR Berghofer and an important milestone for not only Kazia’s development of paxalisib, but also the company’s commercial portfolio as we secure the licence of a significant cancer immunotherapy pathway. We are very pleased to have obtained the potential intellectual property rights around PI3K inhibitors, which is a significant step forward as we continue to explore cancer treatments beyond the brain, including novel therapeutics in solid tumours such as breast cancer."

Professor Fabienne Mackay, Director and CEO of QIMR Berghofer said: "We are pleased to enter this exclusive licence agreement with Kazia following what has been a successful research collaboration over the past two years. We look forward to progressing the clinical development pathway for PI3K inhibitor drugs such as paxalisib under this partnership in the hope of delivering tangible, life-changing benefits to patients."

Kazia’s preclinical research collaboration with QIMR Berghofer Medical Research Institute is investigating the use of paxalisib in solid tumours. The ongoing research project is led by Professor Sudha Rao, a leading expert in transcriptional biology, particularly as it applies to the function of the immune system in cancer. Prof Rao is the principal investigator of preclinical studies where paxalisib and KEYTRUDA combination is used in Triple Negative Breast Cancer, and paxalisib and LYNPARZA (Olaparib) combination in advanced breast cancer.

Professor Rao’s team has demonstrated in preclinical studies that the combination of paxalisib with checkpoint inhibitor blockade resulted in highly consistent and statistically significant signals of efficacy including overall tumour volume, metastases, and inflammatory markers. Furthermore, the addition of paxalisib to immunotherapy was observed to reinvigorate the immune cells within the tumour microenvironment by restoring immune killing function while inhibiting "pro-tumour" immune cells. Further data is expected to be presented at future scientific meetings in 2025.

Commenting on the research, Prof Rao said: "The immune system plays a critical role in fighting against cancer. We urgently need treatments that can make cancer cells visible, and at the same time increase the utility of immunotherapy for metastatic breast cancers. In that sense, paxalisib is an exciting PI3K inhibitor because it not only has been observed to inhibit primary tumour burden but was also observed to reinvigorate the immune system of cancer patients. We look forward to providing a preliminary update in the near future on our findings of using paxalisib in breast cancer."

On September 12, 2024 TC BioPharm (Holdings) PLC ("TC BioPharm" or the "Company") (NASDAQ: TCBP) a clinical stage biotechnology company developing platform allogeneic gamma-delta T cell therapies for cancer and other indications, reported the first patient has completed the full dose regimen in the ACHIEVE Phase 2b trial ongoing in the UK with no drug related Adverse Events seen in any of the re start patients (Press release, TC Biopharm, SEP 12, 2024, https://www.prnewswire.com/news-releases/tcbp-announces-first-patient-completed-the-full-fose-regimen-in-achieve-clinical-trial-302246114.html [SID1234646544]). As previously stated, patients are eligible to receive up to 4 total doses of TCB under the ACHIEVE protocol. Based on the amended dosing, this patient received approximately one billion cells in the TCB008 dosing regimen.

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One patient in Cohort A has successfully completed the full dosing regimen having received all 4 doses. Preliminary safety data indicate the 5mL dose of TCB008 is well tolerated, with no TCB008-related Adverse Events. These data outputs are indicative of TCB008’s safety profile, in support of the ACHIEVE study safety objectives and endpoints. New patients will continue to be identified, screened, and enrolled into the study.

It’s expected that a further three patients will have received their 4th and final dose by the end of September. 14 patients in Cohort A are initially expected to receive TCB008 and, pending confirmation of primary endpoints, a further 10 patients will be recruited into the cohort for a total of 24 patients.

"We are extremely pleased to announce that the full dosing regimen is completed by the first patient to receive the increased dose of TCB008 under the ACHIEVE trial. Including the fully-dosed first patient in the ACHIEVE Phase 2b trial, we have observed no drug related Adverse Events in any of the restart patients," said Bryan Kobel, Chief Executive Officer of TC BioPharm. "This initial feedback speaks to the safety and tolerability of TCBP’s drug. An additional three patients will receive their 4th and final dose by the end of September. While we are able to see safety and tolerability in the immediate aftermath of dosing for ACHIEVE to update investors, the measurement for efficacy and the release of that data will be after completion of the cohort and the data clean per regulatory mandate. We are seeing strong recruitment and enrollment at our active sites and expect to be able to announce a full data set in the first half of 2025 inclusive of primary and secondary endpoints"

The ACHIEVE UK clinical trial is an open-label, phase II study designed to evaluate the efficacy and effectiveness of TCB008 in patients with AML or MDS/AML, with either refractory or relapsed disease.

On September 12, 2024 City of Hope, one of the largest cancer research and treatment organizations in the United States, and Biopharmaceutical Research Company (BRC), a specialty pharmaceutical company developing proprietary cannabinoid therapeutics, reported that the first patient has received BRC-001, an experimental supportive care therapy for breast cancer patients (Press release, City of Hope, SEP 12, 2024, View Source [SID1234646543]). City of Hope researchers are evaluating whether this cannabinoid therapeutic candidate could address joint pain (arthralgia) that is often a side effect of aromatase inhibitors, a type of hormone therapy used to treat postmenopausal women with breast cancer.

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Aromatase inhibitors such as letrozole, anastrozole or exemestane are prescribed as standard of care for postmenopausal women with estrogen-receptor positive breast cancer to decrease breast cancer recurrence and mortality. About half of patients who take aromatase inhibitors experience joint pain, and many of these patients report severe symptoms. A significant number of patients discontinue cancer treatment due to this side effect. Currently, there is no approved treatment for aromatase inhibitor-induced arthralgia (AIIA). Patients often use over-the-counter, anti-inflammatory medications and other treatment options with limited efficacy for short-term pain management.

"Cannabis-derived therapeutics are one of the most promising treatments to address underserved pain conditions. BRC has developed a proprietary therapy with unique properties to address joint pain in breast cancer patients taking aromatase inhibitors. We look forward to providing a therapy for these patients that allows them to complete a full course of treatment," said George Hodgin, CEO and founder of BRC, the industry leader in federally compliant cannabinoid therapeutics development.

"This research to alleviate or eliminate side effects, such as joint pain, is part of City of Hope’s commitment to provide compassionate, individualized care," said Lisa Yee, M.D., City of Hope professor of breast surgery and lead investigator of the trial. "We hope this work will yield innovative, FDA-approved therapies to help patients complete their lifesaving treatments."

The clinical trial entitled "High Cannabidiol (CBD) Standardized Extract for Aromatase Inhibitor-Induced Arthralgia – A randomized controlled double blind clinical trial" is a randomized, placebo-controlled study at City of Hope’s Los Angeles cancer center. It is designed to assess the safety, tolerability and efficacy of a botanically derived proprietary formulation developed by BRC (BRC-001) when taken orally to address the symptoms of AIIA.

Yuman Fong, M.D., Sangiacomo Family Chair in Surgical Oncology at City of Hope, added, "Cannabis-derived medicines hold promise in alleviating symptoms of cancer and cancer therapy. They are potentially alternatives to opiates for pain relief, and alternatives to benzodiazepines for relief of anxiety and insomnia. We look forward to working with BRC in conducting trials to bring these medicines forward to help patients suffering from cancer."

On September 12, 2024 HotSpot Therapeutics, Inc., a biotechnology company pioneering the discovery and development of oral, small molecule allosteric therapies targeting regulatory sites on proteins referred to as "natural hotspots," reported the presentation of initial clinical data for HST-1011, an investigational oral, selective inhibitor of Casitas B-lineage lymphoma proto-oncogene (CBL-B) (Press release, HotSpot Therapeutics, SEP 12, 2024, View Source [SID1234646542]). Data from the Phase 1 monotherapy dose escalation portion of the study are being shared in a proffered oral presentation at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Congress 2024.

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"These initial data provide the first comprehensive insight into the clinical experience with a CBL-B inhibitor, a novel immuno-oncology mechanism with the potential to meaningfully transform the treatment paradigm for patients with solid tumors," said Timothy Reilly, Ph.D., Chief Development Officer of HotSpot. "Critically, HST-1011 demonstrated important linkages between drug exposures, target engagement, and proximal and distal measures of pharmacology, and we observed initial signs of clinical benefit, in a heavily pre-treated, advanced solid tumor patient population. We believe these data provide a strong foundation for the continued evaluation of HST-1011 in dedicated patient populations and for the further assessment of HST-1011 in combination with a PD-1 inhibitor, cemiplimab."

The Phase 1/2 study of HST-1011 (NCT05662397) is an open-label clinical study designed to evaluate HST-1011 alone and subsequently in combination with Regeneron’s anti-PD-1 therapy, Libtayo (cemiplimab), in adult patients with advanced solid tumors that are relapsed on or are refractory to anti-PD(L)-1 or standard of care therapies. The results being presented at ESMO (Free ESMO Whitepaper) include data from 28 patients in the monotherapy dose escalation portion of the study. The presentation describes the following data:

Patients had a diverse range of solid tumor types and were heavily pre-treated, with a median of four prior lines of therapy, including 89% who had been treated previously with immunotherapy.
HST-1011 was generally well tolerated across a range of twice-weekly doses, with no dose-limiting toxicities. The most frequent adverse events were gastrointestinal in nature, which were generally mitigated by the adoption of a prophylactic pre-treatment regimen.
Encouraging linkages were observed between pharmacokinetics (PK), target engagement, pharmacodynamics (PD) and initial signals of clinical activity. Despite the heavily pre-treated, advanced cancer patient population, indications of clinical benefit, including tumor stasis or shrinkage, were observed in 10 of the 28 patients across a range of tumor types and dose levels.
These encouraging data provide support for the ongoing evaluation of HST-1011 (NCT05662397).

Presentation details are as follows:

Title: First-in-Human (FIH) Phase 1 Data of HST-1011, an Oral CBL-B Inhibitor, in Patients with Advanced Solid Tumors
Speaker: Rachel E. Sanborn, M.D., Earle A. Chiles Research Institute, Providence Cancer Institute, Portland, OR
Session Name: Proffered Paper session: Investigational immunotherapy
Session Date and Time: Fri., Sep. 13, 2024, 16:00-17:30 CEST
Presentation Time: 16:50-17:00 CEST
Location: Burgos Auditorium – Hall 5, Fira Barcelona Gran Via
Presentation Number: 991O