NBM-BMX received USFDA approval to proceed Phase Ib/II clinical trial in newly diagnosed glioblastoma multiforme (GBM).

On May 22, 2023 NatureWise reported that its new drug, NBM-BMX, received USFDA approval on January 4th, 2023 to proceed in Phase Ib/II clinical trials in newly diagnosed GBM (Press release, NatureWise Biotech & Medicals, MAY 22, 2023, View Source [SID1234631916]).

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Glioblastoma multiforme is one of the most aggressive, invasive, and deadly malignancies. More than 60% of newly diagnosed GBM patients do not respond to standard treatment. Our studies demonstrated that NBM-BMX is a new therapeutic approach with the potential for addressing this need for safer, more effective treatment options for GBM.

ImmunityBio to Participate in the Jefferies Global Healthcare Conference

On May 22, 2023 ImmunityBio, Inc. (NASDAQ: IBRX), a clinical-stage immunotherapy company, reported that Executive Chairman and Global Chief Scientific and Medical Officer Dr. Patrick Soon-Shiong, M.D., will participate in a fireside chat at the Jefferies Global Healthcare Conference on Wednesday, June 7, 2023, at 10:00 am Eastern Time (ET) at the Marriott Marquis, New York (Press release, ImmunityBio, MAY 22, 2023, View Source [SID1234631915]).

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An audio webcast of the presentation will be accessible under the "Events & Presentations" section of the Company’s investor relations website at ir.immunitybio.com. Management will be available during the conference for one-on-one meetings.

Rencofilstat (CRV431) A novel drug candidate for NASH, Fibrosis and HCC

On May 22, 2023 Hepion Pharmaceuticals presented its corporate presentation (Presentation, Hepion Pharmaceuticals, MAY 22, 2023, View Source [SID1234631914]).

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First patient dosed in clinical trial of LentinexHP in late-stage colorectal cancer

On May 22, 2023 GlycaNova AS, the Norwegian biotechnology company focused on structurally intact beta-glucans for human and animal health, reported that the first patient has been dosed in a pioneering clinical trial of LentinexHP, a proprietary immuno-modulatory beta-glucan, administered as a food supplement alongside chemotherapy in patients with late-stage colorectal cancer (Press release, GlycaNova, MAY 22, 2023, View Source;utm_medium=rss&utm_campaign=latest-news-from-clinical-trial [SID1234631913]).

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LentinexHP is a novel beta-glucan derived from the shiitake mushroom and is produced by GlycaNova’s proprietary manufacturing process, which enables production of beta-glucans with high biological activity and high structural integrity, maintaining the triple helix structure found in nature.

Beta-glucans have been widely studied in East Asia, where medicinal mushrooms have been used in traditional medicine for thousands of years. In 1985, a beta-glucan from shiitake mushrooms was approved in Japan as an adjuvant for stomach cancer therapy and beta-glucans have also been approved in China for treating multiple cancer types1.

The primary endpoint of the LentinexHP trial is quality of life of patients with stage 4 metastatic colorectal cancer and secondary endpoints include overall survival after one year. Colorectal cancer is the third most prevalent cancer worldwide, according to the International Agency for Research on Cancer2, and remains an area of high unmet need with limited treatment options.

The trial is being conducted at the Specialised Hospital for Active Treatment in Oncology (SHATO) in Haskovo, Bulgaria, and is of a randomised, parallel group, placebo-controlled design of 36 patients. All patients are expected to be dosed by the autumn of this year and the final read out from the study is expected around the end of 2024.

Patients on the trial will be randomly assigned to receive either 2ml of LentinexHP, or placebo, taken orally, twice daily for 12 weeks. Quality of life questionnaires and other assessments will be completed after six and 12 weeks. The primary endpoint of quality of life will be assessed at week 12, and further secondary endpoints of quality of life will be assessed at various times after 12 weeks. The secondary endpoint of overall survival will be measured after one year.

The rationale for conducting the trial is that the combination of a biologically active but non-toxic dietary supplement, LentinexHP, brings the potential to reduce resistance to chemotherapy and to reduce toxicity to non-cancerous cells thereby improving patient outcomes. The Company has previously demonstrated3 that the mode of action of LentinexHP is to stimulate a number of immune system responses thereby augmenting the effects of chemotherapy.

GlycaNova selected stage 4 colorectal cancer for the trial owing to earlier studies showing activity of its beta-glucans against solid tumours including colorectal cancer cells and because of the poor outcomes and limited treatment options in this cancer type, which was associated with more than 935,000 deaths worldwide in 20204.

The regulatory pathway for LentinexHP, on the basis of positive results from the current clinical study, is expected to be relatively straightforward as LentinexHP is a dietary supplement, or potentially a medicinal food, rather than a pharmaceutical drug. The regulatory pathway will be confirmed once the results of the current trial have been received.

The Company’s strategy of advancing LentinexHP as a dietary supplement rather than as a prescription medicine is to accelerate the route to market of the product and to maximise access for patients.

Dr Stoil Boychev, Oncologist, Cancer Specialist at SHATO and Principal Investigator of the trial, said:

"I am hopeful that LentinexHP will improve the condition of our patients and will become a great anti-cancer therapy. SHATO has been a centre of oncology research and treatment in Bulgaria for more than 70 years and we are excited to explore new treatment options where limited options are currently available. This trial, which uses a dietary supplement with a benign side effect profile as an adjunct to chemotherapy, is potentially a very important step in improving the quality of life of patients with colorectal cancer."

Dr Bjørn Kristiansen, GlycaNova’s Founder and Chief Executive Officer, said:

"We are delighted that the first patient has been dosed in this pioneering clinical trial of LentinexHP, which is being given as a dietary supplement alongside chemotherapy in late-stage colorectal cancer. The start of the trial, which follows earlier clinical and pre-clinical work, moves us forward in our quest to provide an effective adjunct therapy in solid tumour cancers to meet a very significant unmet medical need.

"I would like to thank the SHATO hospital, and the Principal Investigator Dr Stoil Boychev, for their commitment to this novel trial, the final results from which are expected around the end of next year."

BridgeBio Pharma Presents Updated Encouraging Clinical and Biomarker Data from its BBP-812 Canavan Disease Gene Therapy Program at the 2023 American Society of Gene and Cell Therapy (ASGCT) Annual Meeting

On May 22, 2023 BridgeBio Pharma, Inc. (Nasdaq: BBIO) ("BridgeBio" or the "Company"), a commercial-stage biopharmaceutical company focused on genetic diseases and cancers, reported promising positive data from six participants dosed in CANaspire, its Phase 1/2 clinical trial of BBP-812, an investigational intravenous (IV) adeno-associated virus serotype 9 (AAV9) gene therapy for the treatment of Canavan disease (Press release, BridgeBio, MAY 22, 2023, View Source [SID1234631910]).

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"Canavan disease is a rapidly progressive, rare neurological disease that affects children from birth and has no treatment options beyond supportive care. The changes in key biomarkers, brain myelination and – critically – clinical function observed in children receiving BBP-812 in our study are compelling, and as we continue to advance our program we are hopeful that we will see outcomes that make a meaningful difference in the lives of children with Canavan disease and their families," said Genevieve Laforet, M.D., Ph.D., vice president of clinical development at Aspa Therapeutics, the BridgeBio affiliate that is developing BBP-812 for Canavan disease. "We are beyond grateful to the children and their families who are participating in CANaspire as well as the study investigators, who together are making it possible to explore the potential of BBP-812 as a therapy for Canavan disease. We intend to work with the FDA to move our program forward as rapidly as possible, including exploring available expedited programs such as accelerated approval."

As of March 23, 2023, findings from the trial include:

All participants showed a rapid and lasting decrease in levels of NAA, a key chemical marker elevated in children with Canavan disease, after dosing with BBP-812
The duration of individual participant follow-up ranged from 1 to 15 months post-treatment
The most recent data continue to show reductions in NAA in all participants and all compartments tested. Across individual participants, percent decreases in NAA from baseline ranged between 70% and 95% in cerebrospinal fluid (CSF), 29% and 88% in urine, and 8% and 75% in brain (by magnetic resonance spectroscopy)
All participants in the CANaspire trial had urine NAA levels consistent with typical Canavan disease prior to receiving BBP-812. After receiving BBP-812, urine NAA levels in all participants fell to what can be considered less severe Canavan disease as reported in the scientific literature and observed in the Company’s natural history study
MRI scans indicated the presence of improved myelination, which is essential for brain development, in the brainstem and cerebellum of all participants
These findings have been noted as early as 3 months after BBP-812 administration and were present at post-dosing Month 12 in the participant who has been followed the longest in the trial
In contrast to the natural history of typical Canavan patients in which already-limited functional abilities are lost over time, no trial participants have lost function and there have been incremental improvements in achievement of developmental milestones
Improvement in the GMFM-88 sitting dimension (measuring upright head control and sitting ability) has been shown to varying degrees in all participants who have been evaluated post-treatment, including one participant who has developed the ability to walk with a mobility aid and has a score on the GMFM-88 sitting dimension consistent with that of unaffected children
BBP-812 has been shown to be generally well-tolerated to date, with a safety profile consistent with other systemically administered AAV9 gene therapies
The Data and Safety Monitoring Committee (DSMC), an independent panel of expert physicians and clinical trial experts, has conducted a thorough evaluation of the CANaspire trial safety data and have endorsed the Company’s plan for advancement to the next dosing level
Florian Eichler, M.D., director of the leukodystrophy service at Massachusetts General Hospital, Center for Rare Neurological Disease and principal investigator of the CANaspire gene therapy clinical trial for Canavan disease, reflects on the progress of the trial: "The reduction in NAA to levels consistent with less severe Canavan disease, MRIs indicating potential improvement of myelin in the brainstem, and the measurable functional gains observed among participants, together are unlike any findings I have seen in children with Canavan disease."

About CANaspire
CANaspire is a Phase 1/2 open-label study designed to evaluate the safety, tolerability, and pharmacodynamic activity of BridgeBio’s AAV9 gene therapy candidate, BBP-812, in pediatric patients with Canavan disease. Each eligible patient will receive a single intravenous (IV) infusion of BBP-812. The primary outcomes of the study are safety, as well as change from baseline of urine and central nervous system N-acetylaspartate (NAA) levels. Motor function and development will also be assessed.

For more information about the CANaspire trial, visit TreatCanavan.com or ClinicalTrials.gov (NCT04998396).

About BBP-812
BBP-812 is an investigational AAV9 gene therapy for Canavan disease. Using AAV gene therapy, BridgeBio seeks to deliver functional copies of the ASPA gene throughout the body and into the brain, potentially correcting the disease at its source. Preclinical proof-of-concept results have shown the approach restores survival and normal motor function in Canavan disease models. BBP-812 was granted Fast Track Designation, Rare Pediatric Drug Designation, and Orphan Drug Designation by the U.S. Food and Drug Administration. BBP-812 was also granted Orphan Drug Designation by the European Medicines Agency.