On July 27, 2021 SynOx Therapeutics Limited ("SynOx" or the "Company"), the late-stage clinical biopharmaceutical company developing emactuzumab for the treatment of Tenosynovial Giant Cell Tumours (TGCT), reported the appointment of a new Chairman and Chief Executive Officer and plans for a registrational trial of emactuzumab in the USA and EU (Press release, SynOx Therapeutics, JUL 27, 2021, View Source [SID1234585435]).

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Professor Ton Logtenberg has been appointed non-executive Chairman. Ton has over 25 years’ experience in the biopharmaceutical industry, including as Co-Founder and Chief Scientific Officer of Crucell N.V. (acquired by Johnson & Johnson for $2.4 billion) and Founder, President and CEO of Merus N.V (NASDAQ: MRUS).

Ton Logtenberg, Chairman of SynOx, said: "I am delighted to be joining SynOx as non-executive Chairman. SynOx’s mission is to focus on the unmet clinical needs of patients with TGCT, a group of rare tumours that form in the joints and is often a progressive disease that negatively impacts young adults in the prime of their life. SynOx is well funded and backed by world leading investors to enable the development of emactuzumab, a late-stage clinical asset with the potential to provide a safe and efficacious treatment for this debilitating disease."

Ray Barlow joins SynOx as CEO from Kiadis Pharma N.V. where he was Chief Business Officer up to the successful conclusion of its sale to Sanofi in April 2021. Ray has over 20 years’ experience in the biopharmaceutical industry gained through leadership positions in scientific, clinical, commercial, and executive roles in global pharmaceutical companies (AstraZeneca, J&J and Amgen), publicly listed biotech companies (Emergent BioSolutions Inc, Crucell N.V., e-Therapeutics PLC and Kiadis Pharma N.V.) and a number of private biotech companies.

Ray Barlow, Chief Executive Officer of SynOx, commented: "I am excited to be joining SynOx as CEO at this critical point in the Company’s journey to establish emactuzumab as a potentially best-in-class treatment for patients with TGCT on a global basis. Emactuzumab, an IgG1 CSF-1R targeted antibody, has already generated highly promising results as a monotherapy in over 60 patients with TGCT and we look forward to continuing to work with the regulatory agencies to enable the initiation of our upcoming registrational clinical trial (TANGENT) in the USA and EU."

Jacob Gunterberg, Partner at HealthCap, noted: "We are delighted to welcome Ton and Ray to the SynOx Therapeutics Board and leadership team. We are confident that they will bring significant energy and experience to our mission to establish emactuzumab as the therapeutic treatment of choice for patients with TGCT. We would like to thank Nick La Thangue for his contributions as CEO in the period before the transition to the new leadership."

Francesco De Rubertis, Partner at Medicxi, added: "We remain excited by the potential of emactuzumab as a best-in-class treatment for TGCT and are pleased to have Ton and Ray join the Company as we continue the development of the asset through confirmatory clinical trials."

On July 27, 2021 NexImmune, Inc. (Nasdaq: NEXI), a clinical-stage biotechnology company developing a novel approach to immunotherapy designed to orchestrate a targeted immune response by directing the function of antigen-specific T cells, reported it plans to report second quarter financial results and provide a corporate update on Monday, August 9, 2021, via press release, after market close (Press release, NexImmune, JUL 27, 2021, View Source [SID1234585303]).

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The press release will be accessible under the investor section of the NexImmune’s website at www.neximmune.com.

On July 27, 2021 Prescient Therapeutics reported that it has successfully finished a round of safety testing on its PTX-100 drug in a basket trial of patients with solid and blood cancers, revealing an "excellent" safety profile (Press release, Prescient Therapeutics, JUL 27, 2021, View Source;utm_medium=rss&utm_campaign=more-good-safety-results-as-prescient-therapeutics-continues-cancer-treatment-tests [SID1234585286]).

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On Tuesday, Prescient Therapeutics announced it had completed Phase 1b trials of its novel PTX-100 cancer treatment, with none of the ten patients in the study suffering serious adverse effects linked to the medication.

Additionally the study found PTX-100 was well tolerated by patients even at the highest administered dosage of 2,000 mg/m2.

Steven Yatomi-Clarke, Prescient Therapeutics’ Chief Executive, said he was "pleased" with the results, and noted there were other reasons for the company to celebrate.

Phase 1b testing was primarily designed to test for PTX-100’s safety profile, however clinical benefits were also observed in two of the study’s patients who have previously failed other treatments.

One of these patients had endured five failed treatments before joining the study, and each time was only able to control the disease for a few months before it advanced again.

Following the administration of PTX-100, the patient saw a reduction in their cancer burden and the disease has not progressed for 17 months.

The patient has undergone 24 treatment cycles with PTX-100 and is still taking the drug.

Another patient with cutaneous T cell lymphoma (CTCL) with K-Ras mutation also had aggressive disease and had failed three prior treatments. This patient had a partial response on the study, with reduced cancerous lesions and symptomatic relief. The patient was on therapy for 12 months, receiving 19 cycles of therapy.

"We are encouraged by the biological activity demonstrated by PTX-100 in certain patients on the basket trial with T cell lymphomas," Mr Yatomi-Clarke said.

"Whilst numbers are small, the observation is encouraging in that it indicates activity of PTX-100 as a monotherapy in patients where other therapies have failed."

"We look forward to exploring this in the expansion cohort study as we pursue the quickest route to market for PTX-100 in areas of unmet clinical need."

Positive results in OmniCAR testing
Earlier in July, separate immunogenicity testing on Prescient Therapeutics’ OmniCAR universal CAR-T platform provided similarly positive safety results.

The in-silico tests (meaning they were conducted by complex computer algorithms) found two binding components used in OmniCAR treatments, SpyTag and SpyCatcher, are unlikely to trigger adverse immune responses in patients.

However, Prescient Therapeutics did acknowledge that due to limitations with modern algorithms, in silico testing can often be over-predictive.

Even so, the results substantially de-risk the platform’s development and Mr Yatomi-Clarke said the results leave him confident patients’ immune systems won’t react dangerously to treatments.

OmniCAR is a next-generation CAR-T platform, designed to give medical practitioners greater control over traditional CAR-T treatments – which genetically re-engineer a patients’ own immune system to identify and attack cancerous cells.

The platform is also designed to make treatment safer for patients.

On July 27, 2021 Gateway for Cancer Research℠ reported that awards grant to the EORTC 1634- Brain Tumour Group (BTG) academic trial in Post-Pubertal Patients with Newly-Diagnosed Medulloblastoma (PersoMed-I) (Press release, EORTC, JUL 27, 2021, View Source [SID1234585279]).

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Medulloblastoma is a rare brain tumor. In the US, it affects 550 adults, and in Europe around 450 adults annually, with the majority being young adults. Adolescent and adult medulloblastomas are biologically distinct in comparison to pediatric medulloblastomas, which mandates age adapted treatment strategies. Adolescent patients all bear an intermediate to high prognostic risk, leading to poor patient survival and disability. We are still unaware of the most effective treatment with the lowest possible rate of side effects (long-term toxicity) for this age group.

Today, treatment consists of maximal surgical removal plus craniospinal radiotherapy and maintenance chemotherapy. Treatment toxicity is high and often includes decline of cognition, fertility, neurological function and hearing, connected to severe impairments of quality of life, social and professional function. There is therefore an unmet medical and scientific need to help treat adolescent and adult patients burdened by this rare brain tumour.

The EORTC 1634-BTG trial and connected translational research projects provide a unique opportunity to investigate a personalized medical therapy that can be applied to about 70% of adolescent and adult patients with medulloblastoma, all the while addressing highly relevant toxicity and efficacy aspects in this highly under-investigated population, with an eminent output for affected patients in view of increasing survival rates and patient re-integration socially and professionally, alike.

Gateway for Cancer Research awarded over $500K to the EORTC-1634BTG study, marking the first collaboration between the two organisations. "EORTC is proud to count Gateway for Cancer Research as their partner in tackling unmet patient-centred needs in cancer clinical research. Their support contributes to an important international randomised clinical trial that will pave new avenues in neuro-oncology for adolescent and adult patients", commented Dr. Denis Lacombe, EORTC CEO.

"Gateway for Cancer Research and EORTC are deeply committed to advancing research that ultimately changes the standard of care for cancer patients worldwide," said Michael Burton, president and CEO, Gateway for Cancer Research. "We are proud to partner with EORTC to fund this promising clinical trial, and we are confident that our collaboration will truly accelerate progress for the patients we are privileged to serve."

The study

The EORTC1634-BTG PersoMed I study is a European based study (50 sites in 9 countries) with an intergroup collaboration in Australia. It will be the first prospective randomized trial in post-pubertal and adult patients with medulloblastoma. In view of novel combination therapies, it will use a targeted therapy in combination with radio-chemotherapy in a randomized setting, based on evaluation of the genetic subtype of medulloblastoma, and will therefore be personalized. The study will prospectively investigate molecular subtypes in an adult population, addressing the area of better characterization of cancers through biomarkers, and will implement a dose reduction of radio-chemotherapy in its experimental arms, focusing on treatment de-escalation. It will unify the pediatric and the adult neuro-oncology trial world and be the first trial worldwide that includes pediatric and adult patients in a prospective setting, also addressing patient feedback.

The primary objective of EORTC 1634-BTG is to compare PFS (progression free survival) of a personalized intensity-modulated therapy (experimental arm; sonidegib) vs. standard therapy (modified NOA-07) in the Sonic Hedgehog (SHH)-dependent subgroup. It therefore aims to improve PFS, translating into a higher survival rate and clinically relevant functional improvements for the affected patients.

In addition, by decreasing toxicity in its risk-adapted setting, the study will help to decrease short- and long-term toxicity burden and thereby help to re-integrate affected patients in their social and professional lives. The study also implements effective interventions that enable symptom management during and after treatment and empower patients to better handle their disease and become actively involved in their care decisions. This is reflected in tight toxicity management plans, patient education and monitoring of patient-reported outcomes.

Secondary objectives include additional efficacy objectives as well as toxicity of treatment. As patient reported outcomes are highly important in a setting where young patients in the middle of their lives are affected, short- and long-term health-related quality of life (HR-QoL), neurocognitive function, social outcome and endocrine function will be assessed in the study.

A total of 205 patients will be recruited over a period of 3 years. Patient follow-up duration until primary objective, after LPI (last patient in), is estimated at 4.6 year to provide the targeted number of events for the study analysis. The overall duration of this study is 9 years and is coordinated by Professor Peter Hau (Universitaetsklinikum Regensburg, Germany).

On the study’s importance, Professor Peter Hau commented: "The EORTC 1634-BTG trial is the first randomised trial in adults with medulloblastoma worldwide. It both aims to decrease treatment toxicity and increase efficacy in targeted subpopulations of patients with medulloblastoma and will thereby be the first randomised trial ever in medulloblastoma that uses a targeted therapy. In addition to its immediate output, it will also generate a wealth of clinical, imaging and biological data that will help to develop the field further after the trial has concluded."

On July 27, 2021 Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development of novel therapies for serious rare and ultra-rare genetic diseases, reported that it will host a conference call on Monday, August 2, 2021 at 5pm ET to discuss its financial results and corporate update for the quarter ended June 30, 2021 (Press release, Ultragenyx Pharmaceutical, JUL 27, 2021, https://ir.ultragenyx.com/news-releases/news-release-details/ultragenyx-host-conference-call-second-quarter-2021-financial [SID1234585263]).

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The live and replayed webcast of the call will be available through the company’s website at View Source To participate in the live call by phone, dial (855) 797-6910 (USA) or (262) 912-6260 (International) and enter the passcode 3654725. The replay of the call will be available for one year.