On February 2, 2023 HLB Life Science, which holds the domestic rights to the targeted anticancer drug ‘Riboceranib’ and some of the profit rights in Europe and Japan, reported the company has applied for conditional product approval for adenoid cystic carcinoma (adenoid carcinoma) for the first time in Korea (Press release, HLB Life Science, FEB 2, 2023, View Source;word=&page=1&v=380 [SID1234649275]).

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HLB Life Science announced on the 2nd through a public notice that it has applied for conditional product approval of riboceranib for ‘patients with recurrent or metastatic adenoid cystic carcinoma’ to the Ministry of Food and Drug Safety.

Adenoid cystic carcinoma, commonly called salivary gland cancer, is a rare disease and an intractable cancer with no proper treatment other than repeated surgery or radiation therapy. It is a very difficult to differentiate in advance because it is histologically very diverse and complex, and it occurs multiple times throughout the salivary glands. It is a very difficult type of cancer to treat as it frequently relapses locally along the nerves or metastasizes remotely to the lungs, bones, intestines, and brain.

HLB’s US subsidiary Elevar Therapeutics and HLB Life Science have conducted a phase 2 clinical trial of riboceranib on 80 patients with adenoid cystic carcinoma in the US and Korea. According to the clinical results announced at the American Society of Cancer (ASCO) (Free ASCO Whitepaper) in June last year, the primary endpoint, objective response rate (ORR), showed 15.1% by the Response Evaluation Criteria (RECIST v1.1) based on the change in tumor size, and 51.7% by the CHOI evaluation criteria that measures the density along with the change in tumor size, confirming its high potential as a treatment for adenomatous polyposis.

The disease control rate (DCR) was 64.4%, and the duration of response (DOR) was 14.9 months. While many anticancer drugs showed low response rates of 0-10% in investigator-directed clinical results for adenomatous polyposis, riboceranib is evaluated to have confirmed high efficacy and safety in a strictly controlled large patient group.

If HLB Life Science receives conditional product approval from the MFDS, it plans to quickly supply riboceranib to major domestic hospitals for the treatment of adenomatous polyposis patients. At the same time, as it is a conditional approval, we plan to immediately begin preparing for phase 3 clinical trials to further prove the efficacy and safety of the new drug.

Rivoceranib was designated as an orphan drug and an orphan drug in development stage for adenoma of the thyroid gland in the United States (February 2021) and Korea (November 2022), respectively.

According to the MFDS guidelines, orphan drugs are subject to accelerated review, so they can be approved and launched with only phase 2 clinical trials, and can enjoy benefits such as clinical trial subsidies, market exclusivity, and conditional sales approval.

Separately, in the United States, Elevar is also finalizing a trial plan for phase 3 clinical trials after conditional approval, and HLB Life Sciences, which holds the Korean rights, has applied for product approval to the Korean MFDS first.

Han Yong-hae, CEO of HLB Life Science, said, "In the case of adenomatoid carcinoma, there is no standard treatment, so it is a representative cancer with high unmet medical needs among patients, and the rapid launch of a new drug is very important." He continued, "HLB Life Science and Elevar have been working together to apply for approval from the MFDS and the FDA, respectively, but HLB Life Science completed its preparations one step ahead and has now applied for approval of the new drug. We will do our best to receive conditional product approval from the MFDS, and we will establish a systematic system to ensure a fast and stable supply of riboceranib in the future."

On February 2, 2023 RefleXion Medical, a therapeutic oncology company, reported the U.S. Food and Drug Administration (FDA) has granted the first marketing clearance for its SCINTIX biology-guided radiotherapy, a cutting-edge treatment applicable for early and late-stage cancers (Press release, RefleXion, FEB 2, 2023, View Source [SID1234638894]). The company will host a live-stream event featuring both its co-founder and CEO discussing the breakthrough significance of SCINTIX therapy for cancer treatment.

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SCINTIX is the first and only radiotherapy that allows each cancer’s unique biology to autonomously determine where and how much radiation to deliver, second-by-second, during actual treatment delivery. This expands the RefleXion X1 into the only dual-treatment modality platform that can treat patients with indicated solid tumors of any stage. The SCINTIX biologic modality tracks tumor motion from all types of movement, including expected motion from internal processes such as breathing and digestion or unexpected movement by a patient. The X1 also has a state-of-the-art anatomic modality previously cleared by the FDA for solid tumors located anywhere in the body.

SCINTIX ushers in a new era of external-beam radiotherapy by harnessing data produced from the biologic process of cancer cells, which until now has been untapped. We are excited to be among the early adopters of SCINTIX and to help develop this therapy for all cancer patients, especially those with stage 4 disease, where treatment options often remain very limited.

Terence Williams, M.D., Ph.D.
Chair of City of Hope’s Department of Radiation Oncology

"SCINTIX ushers in a new era of external-beam radiotherapy by harnessing data produced from the biologic process of cancer cells, which until now has been untapped," said Terence Williams, M.D., Ph.D., chair of City of Hope’s Department of Radiation Oncology. "We are excited to be among the early adopters of SCINTIX and to help develop this therapy for all cancer patients, especially those with stage 4 disease, where treatment options often remain very limited.

"With SCINTIX, the X1 machine and the tumor communicate continuously via a live data stream produced during patient treatment," continued Dr. Williams. "This precision should enable us to treat less surrounding tissue and may enable the treatment of more tumors in the same course of therapy."

The FDA cleared SCINTIX biology-guided radiotherapy to treat patients with lung and bone tumors. These tumors may arise from primary cancers or from metastatic lesions spread from other cancers in the body. Previously granted Breakthrough Device designation by the FDA for treating lung tumors, the breakthrough nature of SCINTIX technology lies in its ability to detect and then treat multiple moving tumors. Initially cleared for use with the radiopharmaceutical fludeoxyglucose F18—commonly known as FDG—the company plans to adapt SCINTIX therapy to work with the full array of novel radiopharmaceuticals under development for different cancer types.

"From its inception, the novel nature of SCINTIX therapy demanded that we fundamentally rethink how to design a machine capable of delivering cancer treatment to patients that were not considered candidates for radiotherapy because of the extent of their disease," said Todd Powell, CEO and president of RefleXion. "Likewise, our regulatory pathway encompassed unprecedented hurdles as medical device and pharmaceutical teams within the FDA worked together to create a new classification regulation for this breakthrough device and therapy.

"With initial clearance for SCINTIX therapy behind us, we will begin full commercialization and patient treatments in the coming weeks."

SCINTIX therapy (formerly referred to as BgRT) is delivered through the RefleXion X1 machine, which combines positron emission tomography (PET) with a linear accelerator (LINAC) to deliver a radiation dose that tracks the cancer’s motion. Immediately prior to treatment, the patient is injected with a radiopharmaceutical that interacts with cancer cells to produce signals or emissions. The X1 continuously constructs a map from the detected emissions data that determines where to aim beamlets of radiation. This crosstalk between the tumor and the X1 requires the system to rotate at 60 rpm—making it the first and only radiotherapy machine to spin at this speed.

Autonomous radiotherapy was just a concept over a decade ago, but now for the first time in the history of cancer treatment, the individual biology of the cancer itself guides its own destruction. Today, I want to acknowledge and thank the many talented people who have contributed over the years to translate SCINTIX therapy to the clinic.

Sam Mazin, Ph.D.
Founder and CTO of RefleXion

"Autonomous radiotherapy was just a concept over a decade ago, but now for the first time in the history of cancer treatment, the individual biology of the cancer itself guides its own destruction," said Sam Mazin, Ph.D., founder and CTO of RefleXion. "Today, I want to acknowledge and thank the many talented people who have contributed over the years to translate SCINTIX therapy to the clinic."

Combined, tumors in the lung or bone, including those that metastasize from other primary cancers, arise in approximately 430,000 patients.[1] In metastatic cancer, where patients can present with both lung and bone tumors, radiotherapy is often not considered as a treatment option due to the cumbersome clinical workflow and toxicity of treating more than one tumor in a treatment session.

RefleXion is hosting a brief live stream event from its headquarters in Hayward, Calif., about the SCINTIX therapy clearance. The live stream can be accessed on RefleXion’s YouTube channel today at 10:30 AM PST.

On February 2, 2023 Theralase Technologies Inc. ("Theralase" or the "Company") (TSXV: TLT) (OTCQB: TLTFF), a clinical stage pharmaceutical company focused on the research and development of light activated Photo Dynamic Compounds ("PDCs") and their associated drug formulations, used to safely and effectively destroy various cancers, bacteria and viruses, reported that its Phase II Bacillus Calmette Guerin ("BCG") Unresponsive Non-Muscle Invasive Bladder Cancer ("NMIBC") Carcinoma In-Situ ("CIS") clinical study ("Study II") (Interim) data has been accepted by the American Urology Association ("AUA") for a moderated poster presentation at it’s annual meeting (Press release, Theralase, FEB 2, 2023, View Source [SID1234627414]).

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The AUA annual meeting will take placebetweenApril 28th to May 1st, 2023 in Chicago, Illinois with the poster presented for general viewing and discussion within one of the moderated poster sessions, during the AUA annual meeting.

The poster titled: "A Phase II Clinical Study of Intravesical Photo Dynamic Therapy ("PDT") in Patients with BCG-Unresponsive NMIBC (interim analysis)"will be presented by the lead author, Girish Kulkarni M.D., Ph.D., FRCSC, Divisions of Urology and Surgical Oncology, Department of Surgery, Princess Margaret Cancer Centre, University Health Network, Professor, University of Toronto, (Toronto, Ontario, Canada).

The poster will include interim clinical data and analysis collected through the enrollment and treatment of patients at all Canadian and US clinical study site by their respective principal investigators for Study II.

Arkady Mandel MD, Ph.D., D.Sc., Interim Chief Executive Officer and Chief Scientific Officer, Theralase stated, "I am delighted that Theralase will have the opportunity to showcase its latest reported interim clinical data and analysis for Study II at the AUA annual meeting to leading uro-oncologists from around the world. The interim analysis of the clinical data collected from Study II to date supports that early results show complete response rates in 53% of patients evaluated at 90 days and 28 % of patients evaluated at 450 days. Based on the clinical data collected to date, PDT could represent a viable treatment option with an acceptable safety profile."

About AUA

Founded in 1902, with more than 20,000 members worldwide, the AUA is a premier urologic association, providing invaluable support to the urologic community with the mission is to promote the highest standards of urological clinical care through education, research and the formulation of health care policy.

About Study II

Study II utilizes the therapeutic dose of TLD-1433 (0.70 mg/cm2) activated by the proprietary TLC-3200 medical laser system. Study II is focused on enrolling and treating approximately 100 to 125 BCG-Unresponsive NMIBC CIS patients in up to 20 clinical study sites located in Canada and the United States.

About TLD-1433

TLD-1433 is a patented PDC with 12 years of published peer reviewed preclinical research and is currently under investigation in Study II.

On February 2, 2023 Takeda reported its financial report for the Quarter Ended December 31, 2022 (Presentation, Takeda, FEB 2, 2023, View Source [SID1234626983]).

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On February 2, 2023 4D pharma plc (AIM: DDDD, NASDAQ: LBPS), a pharmaceutical company leading the development of Live Biotherapeutic products (LBPs), a novel class of drug derived from the microbiome, reported updated clinical data from Part B of its signal finding study of MRx0518 in combination with MSD’s anti-PD-1 therapy, KEYTRUDA (pembrolizumab) in patients with solid tumors that have progressed on a prior immune checkpoint inhibitor (ICI) (Press release, 4d Pharma, FEB 2, 2023, View Source [SID1234626952]).

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The primary efficacy endpoint for Part B of the study is more than three out of 30 patients per tumor group achieving clinical benefit, defined as complete response, partial response, or stable disease for at least six months. In March 2022, it was announced that 4 patients in the RCC cohort had experienced durable stable disease.

At the time 4D pharma plc was placed into administration it was agreed that patient recruitment into the study would be placed on temporary pause but patients who had already started treatment would be allowed to continue. There were 2 Part B RCC patients on treatment at the time of administration.

Today, 4D pharma is pleased to announce that one of these patients is experiencing a partial response. Part B of the study has to date enrolled 22 patients with RCC, of which 5 out of 20 evaluable patients have achieved clinical benefit. To date, Part B of the study has enrolled 51 patients of up to a total of 120 patients with RCC, non-small cell lung cancer, bladder cancer, and head and neck squamous cell carcinoma. MRx0518 continues to be safe and well tolerated and 5 patients are continuing on treatment.

The study is being conducted in heavily pre-treated metastatic patients with solid tumors who have previously experienced clinical benefit on prior ICI therapy and subsequently developed progressive disease. The study is being conducted in collaboration with MSD (Merck & Co., Inc., Kenilworth, NJ, USA).

"Despite recent setbacks in the microbiome field experienced by other companies and the limitations on patient recruitment as a result of the administration, we are delighted that patients continue to benefit from the trial treatment. We continue to build on the body of evidence for the future development of MRx0518 and 4D’s approach to Live Biotherapeutics" commented Dr. Alex Stevenson, Chief Scientific Officer, 4D pharma. "Together with the clinical, safety and biomarker data we have generated in the wider MRx018 programme, it is clear that there is an opportunity to take MRx0518 into earlier lines of treatment to improve outcomes with immunotherapy. While the safety and tolerability of MRx0518 lends itself to multiple combinations and indications, the data we have generated in RCC suggests that this provides our most viable route to market."

4D pharma is in discussion regarding next steps for this study and future pivotal studies with partners and Key Opinion Leaders.

KEYTRUDA is a registered trademark of Merck Sharp & Dohme Corp., a subsidiary of Merck & Co., Inc., Kenilworth, NJ, USA.

On 24 June 2022, David Pike and James Clark of Interpath Advisory were appointed as administrators of 4D Pharma plc. The administrators make no statement or representation in respect of the announcement.