On February 23, 2022 Foresee Pharmaceuticals Co., Ltd. (6576.TWO) ("Foresee"), a Taiwan and US-based biopharmaceutical company and TRPharm, a leading Turkish healthcare company, reported that they have entered into an exclusive license agreement for the co-development and commercialisation in Turkey and certain MENA countries of Foresee’s FP-045 program, an oral allosteric Aldehyde Dehydrogenase 2 (ALDH2) activator for the treatment of Fanconi Anemia and other rare and severe diseases (Press release, Foresee Pharmaceuticals, FEB 23, 2022, View Source [SID1234608918]). Under the terms of this partnership, Foresee and TRPharm will collaborate to initially expedite the development of FP-045 in Fanconi Anemia. Turkey and the surrounding region has a high prevalence Fanconi Anemia as well as other rare diseases. Foresee will own all development data which will be used as part of its global development and registration dossier. Upon approval and commercialization in the territory, Foresee will receive significant royalties from the revenue in the territory. TRPharm will have exclusive rights to register and commercialize in the territory and will cover all related costs.

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Dr. Ben Chien, Founder, Chairman and CEO of Foresee commented: "We are very pleased to have established this collaboration with TRPharm, whose established track record of successful development, registrations and launches of rare disease products, including in hematology, makes us confident that they will make a remarkable partner for our FP-045 program. This is our second partnership with TRPharm and we look forward to continuing working together."

İsmet Ince, General Manager of TRPharm, commented "Our purpose in TRPharm is to be the platform where health meets innovative solutions and I can think of no better example for it than this collaboration on FP-045 with Foresee. We are thrilled to have this opportunity to blaze a trail in Fanconi Anemia, which has a particularly high prevalence in our core regions, and provide relief to the patients with significant unmet need. It is an even greater pleasure to be doing this with Foresee, an esteemed international biopharmaceutical company with whom we share common views and a history of productive partnership. This collaboration also marks a step change in our R&D approach and is the culmination of a unique set of capabilities which we have developed over many years. We are confident that they will contribute materially to making this collaboration a rapid success."

On February 23, 2022 The Life Raft Group reported that we continue highlighting recent clinical trials that are still recruiting in the U.S. which investigate potential treatments for GIST patients (Press release, The Life Raft Group, FEB 23, 2022, View Source [SID1234609028]). Our GIST Trials Database lists over 70 trials that are GIST-related and ongoing around the world . Currently there are 12 GIST-related trials recruiting in the United States. In our last article, we listed several for different types of GIST mutations. You can read the previous clinical trials update (December 2021) here:
View Source

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For further information about GIST trials including international trials, visit: The Life Raft Group Clinical Trials Website: View Source If you want to speak with someone about clinical trials, please contact [email protected].

For patients with advanced KIT/PDGFRa mutant GIST:

THE-630 Phase 1/2 – ""A Study of THE-630 in Patients With Advanced Gastrointestinal Stromal Tumors (GIST)" (View Source)

Site is at Dana-Farber Boston, MA Principal Investigator Suzanne George. Started January 3, 2022. The study will be conducted in two parts: a dose escalation phase, followed by an expansion phase. Plans to recruit 160 over a period of 42 months.

In the Phase 1 dose escalation phase oral THE-630 will be administered once daily in a continuous regimen. Primary objectives during the 28-day dose escalation phase are: 1. Number of patients with dose-limiting toxicities. 2. Determination of the recommended Phase 2 dose. 3. Determination of the maximum tolerable dose. 4. Safety analysis – number of participants with treatment emergent adverse events. For the subsequent expansion Phase 2 the primary objective is confirmed Objective Response Rate over a period of up to 24 months after first dose. In the escalation Phase 1 participants with metastatic or unresectable GIST will be admitted.

During the Expansion Phase 2 unresectable or metastatic GIST patients will be divided into 4 cohorts depending on prior TKI therapy. Cohort 4 will include patients who have progressed on or are intolerant to imatinib including patients in the adjuvant setting. The sponsor is Theseus Pharmaceuticals headquartered in Cambridge, MA. Theseus was founded in 2017 and emerged from stealth mode in 2021. On their web page Theseus describes THE-630 as "Our lead program, THE-630, is a pan-variant KIT inhibitor designed for patients with advanced gastrointestinal stromal tumors (GIST) whose cancer has developed resistance to earlier lines of kinase inhibitor therapy." Patients interested in this clinical trial can contact: Theseus Pharmaceuticals, 857-400-9491, [email protected]. NCT05160168

About Phase 1 trials: It is important to recognize that Phase I studies are held to find the highest dose
of the new treatment that can be given safely without causing severe side effects.

• The first few people in the study get a very low dose of the treatment and are watched very closely. If there are only minor side effects, the next feaw participants get a higher dose. This process continues until doctors find a dose that’s most likely to be and effective treatment while having an acceptable level of side effects.

• Safety is the main concern. The research team monitors participants and watches for severe side effects. Due to the small numbers of people in Phase I studies, rare side effects may not be seen until later phases of the trial when more people are receiving the treatment.

• While some people may benefit from being on one, disease response is not the main purpose of a Phase I trial.

Phase I trials carry the most potential risk, but these studies do help some patients. For those with life-threatening illnesses, weighing the potential risks and benefits carefully is key. Sometimes people choose to join Phase I trials when all other treatment options have already been tried.

On February 23, 2022 Immunetune, a preclinical-stage biotech developing next-generation DNA vaccines against cancer and infectious diseases, reported the publication of preclinical data on its novel adjuvant in the scientific journal Vaccine (Press release, ImmuneTune, FEB 23, 2022, View Source [SID1234608861]). The published findings underline the ability of Immunetune’s proprietary adjuvant PyroVant to increase the immune response against its neoantigen cancer vaccine and the resulting profound impact on tumor growth and survival.

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The publication on PyroVant in the journal Vaccine describes the development of a novel, first-in-class genetic adjuvant that greatly improves immune responses elicited by DNA cancer vaccines. Unlike other adjuvants, this DNA-encoded adjuvant induces pyroptosis, a form of inflammatory cell death that results in the release of a plethora of inflammatory molecules that together attract and stimulate immune cells crucial for the generation of an anti-tumor T cell response. When given simultaneously with a DNA vaccine encoding tumor antigens, PyroVant increases tumor-specific T cell numbers up to 8-fold, in a dose-dependent fashion. Subsequently, in a preclinical model of melanoma, the addition of PyroVant to this DNA vaccine significantly delayed tumor growth and increased survival.

"It is important to have these results peer-reviewed by the scientific community and share our mechanistic insights into this first-in-class genetic adjuvant to increase the potential impact of cancer vaccines," said Jeroen van Bergen, Chief Scientific Officer of Immunetune. "We are especially excited to have incorporated PyroVant in our neoantigen cancer vaccine NeoVAC, which is currently moving towards clinical development."

The experiments were performed in close collaboration with the Tumor Immunology group (Prof. Ossendorp) of the Department of Immunology at the Leiden University Medical Center (LUMC) in the Netherlands. Immunetune has submitted a patent application on PyroVant and has recently completed GLP toxicity studies of its DNA-based cancer vaccines in combination with PyroVant. These studies showed that both vaccine and PyroVant were well tolerated with no test article-related observations on survival, body weight and clinical pathology across the toxicity and biodistribution studies. Immunetune is now moving its lead cancer vaccine NeoVAC towards clinical studies.

The full publication is titled ‘Pyroptosis-inducing active caspase-1 as a genetic adjuvant in anti-cancer DNA vaccination’ and can be accessed on the website of the journal Vaccine here.

On February 23, 2022 Nichi-Iko Pharmaceutical Co., Ltd. has recently reported that it received approval for a biosimilar of bevacizumab BS intravenous infusion 100 mg/400 mg, whose marketing authorization application was filed based on the clinical pharmacokinetic (PK) study data conducted in Japan in addition to the global Phase 3 study data conducted by mAbxience (Press release, mAbxience, FEB 23, 2022, View Source [SID1234608885]).

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Bevacizumab BS is highly similar to the reference medicinal product and data has shown a comparable quality, safety and efficacy to Avastin. Bevacizumab BS is indicated for the treatment of advanced or recurrent colorectal cancer that is not curatively resectable and advanced or recurrent non-small cell lung cancer that is not resectable except for squamous cell carcinoma.

On February 23, 2022 Avidity Biosciences, Inc. (Nasdaq: RNA), a biopharmaceutical company committed to delivering a new class of RNA therapeutics called Antibody Oligonucleotide Conjugates (AOCs), reported the appointment of Steve Hughes, M.D. as chief medical officer (Press release, Avidity Biosciences, FEB 23, 2022, View Source [SID1234608919]). Dr. Hughes has over 20 years of experience building and leading clinical development and medical affairs teams at prominent biopharmaceutical companies.

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"Steve brings a unique combination of extensive experience in the RNA field as well as a deep understanding of rare disease drug development," said Sarah Boyce, president and chief executive officer. "Steve’s unparalleled leadership in developing RNA-based treatments across therapeutic areas will be invaluable as we expand our AOC platform and execute on our goal of having three programs in the clinic by the end of this year."

Dr. Hughes has contributed to over 50 clinical trials for more than 25 drugs across multiple therapeutic areas including cardiovascular, neurology and several rare diseases. He has been closely involved in multiple registrational regulatory filings, including playing a key role in the approval of three rare disease products in the last six years.

"I’m thrilled to be joining the talented team at Avidity. The AOC platform is a ground-breaking technology that significantly broadens the potential for RNA therapies," said Steve Hughes, M.D., chief medical officer at Avidity. "I look forward to contributing to Avidity’s progress in the clinic during this pivotal time as we work to advance our clinical programs and deliver potentially transformative medicines to people living with rare diseases that have limited or no therapeutic options."

Dr. Hughes joins Avidity from Arcturus Therapeutics where he continues to be a strategic clinical advisor. Prior to that, he held the position of CMO where he provided leadership and direction to clinical operations, clinical development, clinical sciences, data management, biometrics, and drug safety. Prior to Arcturus, he served as the Chief Medical Officer of Organovo where he led the non-clinical and clinical development teams developing bioprinted tissues for the treatment of hepatic and renal diseases. Dr. Hughes’s previous positions include the Chief Clinical Development Officer at Ionis Pharmaceuticals and positions at Biogen, CSL Behring and Sanofi. Dr. Hughes is board certified in pharmaceutical medicine and received his medical degree from Imperial College, London. He also has an MBA from Imperial College Business School.