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4SC AG reported Resminostat Marketing Application Proceeding & Kinselby accepted as Tradename

On June 19, 2023 4SC AG (4SC, FSE Prime Standard: VSC) reported that the European Medicines Agency (EMA) has notified 4SC that it has accepted its Letter of Intent to Submit a MAA and has informed 4SC that its nominated trade name for resminostat – Kinselby – has been accepted by its Name Review Group (NRG) (Press release, 4SC, JUN 19, 2023, View Source [SID1234632780]).

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If approved by regulatory authorities, Kinselby (resminostat) would be the first HDAC inhibitor approved in Europe for CTCL and the first and only drug approved for maintenance therapy in this disease, making it the Company’s most valuable asset. The positive outcome to RESMAIN creates an opportunity to either sell, license or partner 4SC’s resminostat program for commercialization worldwide (excluding Japan), where Yakult Honsha Co. Ltd, was granted an exclusive license for the development and marketing of resminostat in Japan in 2011.

Jason Loveridge, CEO of 4SC: "This is another key milestone for 4SC in moving resminostat to market in the EU. Now we have an accepted tradename for resminostat we will continue preparation of our MAA and the commercialisation of Kinselby for the benefit of all stakeholders".

Arcellx Announces Clinical Hold for its iMMagine-1 Phase 2 Clinical Program

On June 19, 2023 Arcellx, Inc. (NASDAQ: ACLX), a biotechnology company reimagining cell therapy through the development of innovative immunotherapies for patients with cancer and other incurable diseases, reported that it has received notification from the U.S. Food and Drug Administration (FDA) that a clinical hold has been placed on its CART-ddBCMA investigational new drug (IND) for the treatment of patients with relapsed or refractory multiple myeloma (rrMM) (Press release, Arcellx, JUN 19, 2023, View Source [SID1234632779]).

The clinical hold was received on June 16, following a recent patient death. The company believes limitations on bridging therapy are a contributing factor and is working with FDA to amend the protocol to expand options for patients that are consistent with current clinical practice. The FDA has provided clearance to Arcellx to continue to dose patients who have undergone lymphodepletion.

"The safety and well-being of patients enrolled in our studies is our top priority," said Rami Elghandour, Arcellx’s Chairman and Chief Executive Officer." In coordination with our investigators, data safety monitoring board (DSMB), and our partners at Kite Pharma, we are working with FDA to address the clinical hold. The expansion of bridging therapy regimens is consistent with what’s currently available in clinical practice and is in the best interest of patients. Additionally, we continue to evaluate other potential improvements to the study. We remain confident that CART-ddBCMA is a potential best-in-class therapy for the treatment of patients with rrMM based on the clinical profile observed in the patients dosed to date across our studies. The drug product release characteristics from iMMagine-1 are consistent with those from our Phase 1 study. The manufacturing success rate remains 100% while ramping Lonza, our cell therapy manufacturer, to full scale. Fourteen clinical sites have been opened and study enrollment is tracking to our expectations. We look forward to resolving this matter expeditiously and to continue to advance our therapy to the benefit of patients suffering from rrMM."

About iMMagine-1
iMMagine-1 is a Phase 2 pivotal, open-label, multicenter clinical trial designed to evaluate CART-ddBCMA, a BCMA-specific CAR-modified T-Cell therapy utilizing Arcellx’s novel BCMA-targeted binding domain, for the treatment of adult patients with relapsed or refractory multiple myeloma. The primary objective of this study is to evaluate the overall response rate over a 24-month period. In addition to safety, secondary endpoints include depth of disease response, duration of response, and overall survival over a 24-month period.

About Multiple Myeloma
Multiple Myeloma (MM) is a type of hematological cancer in which diseased plasma cells proliferate and accumulate in the bone marrow, crowding out healthy blood cells and causing bone lesions, loss of bone density, and bone fractures. These abnormal plasma cells also produce excessive quantities of an abnormal immunoglobulin fragment, called a myeloma protein (M protein), causing kidney damage and impairing the patient’s immune function. Multiple myeloma is the third most common hematological malignancy in the United States and Europe, representing approximately 10% of all hematological cancer cases and 20% of deaths due to hematological malignancies. The median age of patients at diagnosis is 69 years with one-third of patients diagnosed at an age of at least 75 years. Because MM tends to afflict patients at an advanced stage of life, patients often have multiple co-morbidities and toxicities that can quickly escalate and become life-endangering.

About CART-ddBCMA
CART-ddBCMA is Arcellx’s BCMA-specific CAR-modified T-cell therapy utilizing the company’s novel BCMA-targeting binding domain for the treatment of patients with relapsed or refractory multiple myeloma. CART-ddBCMA is currently in a Phase 2 study. Arcellx’s proprietary binding domains are novel synthetic proteins designed to bind specific therapeutic targets. CART-ddBCMA has been granted Fast Track, Orphan Drug, and Regenerative Medicine Advanced Therapy Designations by the U.S. Food and Drug Administration.

Sirnaomics Advances STP705 for Squamous Cell Carcinoma In Situ into Late-Stage Clinical Development

On June 19, 2023 Sirnaomics Ltd. (the "Company"; stock code: 2257, together with its subsidiaries, the "Group" or "Sirnaomics"), a leading biopharmaceutical company in discovery and development of RNAi therapeutics, reported the advancement of STP705 for the treatment of Squamous Cell Carcinoma in situ (isSCC) into late-stage clinical development after encouraging Phase IIa and Phase IIb clinical results were shared with the U.S. Food and Drug Administration (FDA) in an End of Phase-2 meeting (Press release, Sirnaomics, JUN 19, 2023, View Source [SID1234632777]). The FDA provided Sirnaomics guidance to move forward with late-stage clinical development because of the efficacious data provided as well as the widespread prevalence of Squamous Cell Carcinoma (SCC) lesions.

Sirnaomics is well-positioned currently to advance STP705 into a confirmatory clinical study for treatment of Squamous Cell Carcinoma in situ (isSCC). We are preparing to move forward in 2023 with a well-designed single dosage study as a sub-group of subjects in a large Phase III clinical study. Positive results will provide the basis for completion of this large registration Phase III trial. Sirnaomics is also studying STP705 for Basal Cell Carcinoma (BCC), which will be the next candidate to move into late-stage development pending the FDA’s review.

STP705 has been studied in isSCC and BCC in more than 100 participants. The safety data generated from prior clinical studies for both types of cancers has shown that STP705 was safe without grade 3 or higher adverse events. The preliminary efficacy data with complete histological clearance of cancer cells was observed in majority of the treatment groups.

"Moving STP705 for the treatment of isSCC into late-stage clinical development is a major milestone for our clinical program and for a dermatology/oncology application," said Dr. Patrick Lu, Founder, Chairman of the Board, Executive Director, President and Chief Executive Officer of Sirnaomics. "According to a 2020 research report from JAMA Dermatology, among patients with isSCC, the cumulative risk of developing an invasive SCC was 11.7% in men and 6.9% in women. Given the widespread prevalence of SCC lesions and a tremendous unmet need, Sirnaomics is dedicated to taking on the challenge for development of a novel therapeutic product with RNAi-based technology."

"Based on the guidance from the type B meeting with the FDA, we currently have a clear path moving forward for late-stage development for STP705 as an innovative drug for treatment of isSCC. Our data has demonstrated excellent safety and efficacy for the treatment of isSCC, and we look forward to advancing this program to late-stage development," said Dr. Michael Molyneaux, M.D., Executive Director and Chief Medical Officer of Sirnaomics.

About STP705
Sirnaomics’ leading product candidate, STP705, is a siRNA (small interfering RNA) therapeutic that takes advantage of a dual-targeted inhibitory property and polypeptide nanoparticle (PNP)-enhanced delivery to directly knock down both TGF-β1 and COX-2 gene expression. The product candidate has received multiple IND approvals from both the U.S. Food and Drug Administration (FDA) and the Chinese National Medical Products Administration (NMPA), including treatments of cholangiocarcinoma, non-melanoma skin cancer and hypertrophic scar. There are currently three product pipeline programs prioritized by STP705: a late-stage clinical development for Squamous Cell Carcinoma in situ (isSCC), completion of a Phase II for Basal Cell Carcinoma (BCC) and a Phase I for the fat remodeling. For other indications, STP705 has received Orphan Drug Designation for the treatment of cholangiocarcinoma (CCA) and primary sclerosing cholangitis (PSC).

Medivir’s patent application for fostrox approved in China

On June 19, 2023 Medivir AB (Nasdaq Stockholm: MVIR), a pharmaceutical company focused on developing innovative treatments for cancer in areas of high unmet medical need, reported that the company’s patent application, covering both substance claims for fostroxacitabine bralpamide (fostrox) and its use for liver cancer treatment, has recently been approved by the Chinese patent authority (Press release, Medivir, JUN 19, 2023, View Source [SID1234632776]).

Corresponding patents have already been granted in the USA, the EU, Japan and a large number of other countries, mainly in Asia. The patent is valid until 2035 with extensions of up to 5 years expected in key markets. Primary liver cancer (HCC) is one of the most common forms of cancer with over 800,0001) new patients globally per year. More than 50% of these are estimated to be in China

"This important patent approval in the largest HCC market, combined with our exciting clinical data from the ongoing phase 1b/2a study with fostrox, are key components to enable in-depth discussions with potential partners in China and other countries in Asia," says Jens Lindberg, CEO at Medivir.

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

About fostrox
Fostrox is a pro-drug designed to selectively treat liver cancers and to minimize side effects. It has the potential to become the first liver-targeted and orally administered drug for patients with HCC and other forms of liver cancer. Fostrox has completed a phase 1b monotherapy study, and a combination study in HCC currently ongoing.

About primary liver cancer
Primary liver cancer is the third leading cause of cancer-related deaths worldwide and hepatocellular carcinoma (HCC) is the most common cancer that arises in the liver. Although existing therapies for advanced HCC can extend the lives of patients, treatment benefits are insufficient and death rates remain high. There are 42,000 patients diagnosed with primary liver cancer per year in the US and current five-year survival is

11 percent. 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.

First Patient Dosed in Phase II STARBURST Study of TLX250-CDx Exploring Indication Expansion

On June 18, 2023 Telix Pharmaceuticals Limited (ASX: TLX, Telix, the Company) reported that the first patient has been dosed in a Phase II study of the Company’s carbonic anhydrase- (CAIX)-targeting PET/CT[1] imaging agent TLX250-CDx (89Zr-DFO-girentuximab), exploring this potential target across a broad range of cancer indications (Press release, Telix Pharmaceuticals, JUN 18, 2023, View Source [SID1234632771]).

The STARBURST study (ClinicalTrials.gov Identifier: NCT05563272) is a prospective, open label Phase II "basket" study to investigate CAIX expression in patients with a diverse range of solid tumours – including breast, cervix, colorectal, gastric, esophageal, head and neck, lung, ovarian, pancreatic and vulval cancers[2] – for potential diagnostic and therapeutic applications.

CAIX is a protein overexpressed on the surface of clear cell renal cell carcinoma (ccRCC), the cancer target in Telix’s highly successful Phase III ZIRCON study.[3] It is also expressed to varying degrees in many other advanced-stage solid tumours with poor prognosis. Tumours that express CAIX are typically hypoxic, more aggressive and feature a tumour micro-environment (TME) that can be resistant to therapy, particularly immunotherapies.

STARBURST is exploring these tumour types in the refractory setting to assess whether tumour sites can be targeted, both for imaging and potentially therapeutic purpose. The study builds on encouraging preliminary data from two investigator-initiated studies in triple-negative breast cancer and non-muscle-invasive bladder cancer, which demonstrate the potential of TLX250-CDx in these disease settings with unmet medical need. The half-life of 89Zr means that imaging these tumours with TLX250-CDx will enable predictive dosimetry for therapeutic radionuclides, effectively serving as a theranostic "scouting" study for future studies harnessing therapeutic radionuclides.

Principal Investigator for the STARBURST study, Dr Jackson Kiser, Medical Director Molecular Imaging at Carilion Clinic in Roanoke, Virginia (U.S.) stated, "CAIX is a cancer target that has now been validated in Telix’s Phase III ZIRCON study in ccRCC, and so it is very interesting to explore expanding the potential utility of the same investigational agent in a series of other tumour types known to express this important target."

Dr Colin Hayward, Telix Chief Medical Officer, said, "Building on the success of ZIRCON and positive preliminary results in investigator-initiated studies of TLX250-CDx in bladder and breast cancer,[4] dosing a first patient in the STARBURST study is a strategically important milestone for Telix. This study will add value and clinical insight to the platform and enable Telix to assess the potential of CAIX as a biomarker as we continue to scout the theranostic potential of targeting CAIX beyond renal cancer. We would like to thank Dr Kiser and his clinical team at Carilion Clinic, as well as the patients who will contribute to the study."