More good safety results as Prescient Therapeutics continues cancer treatment tests

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]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

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.

EORTC 1634-Brain Tumour Group trial in Post-Pubertal Patients with Newly-Diagnosed Medulloblastoma (PersoMed-I) receives support from Gateway for Cancer Research?

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]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

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."

Ultragenyx to Host Conference Call for Second Quarter 2021 Financial Results and Corporate Update

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]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

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.

Prescient Therapeutics (ASX:PTX) expands trial of PTX-100

On July 27, 2021 Prescient Therapeutics (PTX) reported that it is progressing the trial of its cancer-blocking drug PTX100 following successful results from Phase 1b of the trial (Press release, Prescient Therapeutics, JUL 27, 2021, View Source;utm_medium=rss&utm_campaign=prescient-therapeutics-asxptx-expands-trial-of-ptx-100 [SID1234585261]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

The cancer treatment company is trialling PTX-100 which is a first-in-class compound with the ability to block an important cancer growth enzyme known
as geranylgeranyl transferase-1.

Phase 1b of the trial assessed the safety of PTX-100 in patients with solid and haematological cancers and PTX said the results have exhibited an "excellent" safety profile, being well tolerated up to and including the highest dose.

While several adverse events occurred for some patients during the trial, including nose bleeds, a bone infection, hip fracture and a reduction in platelets, PTX said none of these events were related to the drug.

Ten patients were enrolled in the Phase 1b basket trial, half of which had solid tumours and the other half had multiple myeloma and T cell lymphomas (TCL).

The company was particularly encouraged by clinical benefit observed in two patients with TCL with aggressive disease who had undergone previous therapies that were unable to control the disease.

Both patients experienced a reduction in cancer burden, with one patient with peripheral T cell lymphoma (PTCL) experiencing no disease progression for 17 months so far.

Prescient’s CEO and Managing Director Steven Yatomi-Clarke said the results from Phase 1b are significant for two reasons.

"Firstly, that this drug may have utility in fragile patients unable to tolerate therapies with high toxicities.

"Secondly, because the low-toxicity profile of PTX-100 opens up possibilities to add it as a combination agent with various other cancer therapies, depending on the cancer and line of therapy."

Looking forward, Prescient said it will progress the development of PTX-100 as a monotherapy in an expansion cohort study in relapsed and refractory TCL, with a particular focus on PTCL.

The company plans to enrol between 8-12 patients in the expanded cohort and said costs will be within its current budget.

Additionally, PTX said if the expansion cohort is successful in demonstrating the benefit of PTX-100 in PTCL, it’s possible development could advance directly to a separate registration study.

Curis to Release Second Quarter 2021 Financial Results and Hold Conference Call on August 3, 2021

On July 27, 2021 Curis, Inc. (NASDAQ: CRIS), a biotechnology company focused on the development of innovative therapeutics for the treatment of cancer, reported that the Company will release its second quarter 2021 financial results on Tuesday, August 3, 2021, after the close of US markets. Management will host a conference call on the same day at 4:30 pm ET (Press release, Curis, JUL 27, 2021, View Source [SID1234585252]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

To access the live conference call, please dial (888) 346-6389 from the United States or (412) 317-5252 from other locations, shortly before 4:30 pm ET. The conference call can also be accessed on the Curis website at www.curis.com in the ‘Investors’ section. A replay of the financial results conference call will be available on the Curis website shortly after completion of the call.