On July 27, 2021 Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) and AstraZeneca reported that the companies have entered into a collaboration to research, develop and commercialize small molecule compounds directed against the GPR75 target with the potential to treat obesity and related co-morbidities (Press release, Regeneron, JUL 27, 2021, View Source [SID1234585231]). The collaboration builds on the recent discovery from the Regeneron Genetics Center of rare genetic mutations in the GPR75 gene associated with protection against obesity and on early joint research initiated soon after discovery of the target so that potential treatments can be developed as quickly as possible. The companies will evenly split research and development costs and share equally in any future potential profits.

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As published in Science, the new target was found by sequencing nearly 650,000 people and identifying individuals with rare protective mutations. Individuals with at least one inactive copy of the GPR75 gene had lower BMI and, on average, tended to weigh about 12 pounds less and faced a 54% lower risk of obesity than those without the mutation. Strong associations were also seen with improvements in diabetes parameters, including glucose lowering. Obesity and insulin resistance are key drivers in the development of type 2 diabetes and often lead to cardiorenal complications, as well as liver disease.

"The next era of medicine is being fueled by important genetics findings that direct drug developers on how to deploy our toolkit of biologics, small molecules and gene editing technologies in order to safely help patients in need," said George D. Yancopoulos, M.D., Ph.D., President and Chief Scientific Officer of Regeneron. "As experts on genetics and human biology, Regeneron is excited to join forces with the chemistry and small molecule leaders at AstraZeneca, as we seek to develop new medicines tackling the harmful and costly obesity epidemic."

"We are pleased to announce this important collaboration with Regeneron to identify small molecule modulators against GPR75, a newly identified target with genetic validation in metabolic disorders. Obesity and insulin resistance remain key drivers in the development of type 2 diabetes and areas of significant unmet medical need," said Mene Pangalos, Executive Vice President, BioPharmaceuticals R&D at AstraZeneca.

Obesity is associated with many serious health complications and drives organ dysfunction, including in the heart, liver, kidneys and pancreas. Worldwide the prevalence of obesity has more than tripled since 1975, and approximately 650 million adults are estimated to live with obesity today.

On July 27, 2021 iSpecimen Inc. (Nasdaq: ISPC) ("iSpecimen" or the "Company"), an online marketplace for human biospecimens, reported that it will report its financial results for the second quarter ended June 30, 2021, before the market open on Tuesday, August 10, 2021 (Press release, iSpecimen, JUL 27, 2021, View Source [SID1234585247]).

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The Company will host a conference call and audio webcast that morning at 8:30 a.m. Eastern Time featuring remarks by Christopher Ianelli, MD, PhD, CEO and President, Tracy Curley, CFO, and Jill Mullan, COO.

For interested individuals unable to join the conference call, a replay will be available through August 24, 2021, at +1-844-512-2921 (U.S. Toll Free) or +1-412-317-6671 (International). Participants must use the following code to access the replay of the call: 13721607. An archived version of the webcast will also be available on iSpecimen’s Investor Relations site: View Source

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 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 BridgeBio Pharma, Inc. (Nasdaq: BBIO) (BridgeBio), a commercial-stage biopharmaceutical company founded to discover, create, test and deliver meaningful medicines for patients with genetic diseases and cancers with clear genetic drivers, reported a non-exclusive, co-funded clinical collaboration with Bristol Myers Squibb to evaluate the combination of BBP-398, a potentially best-in-class SHP2 inhibitor, with OPDIVO (nivolumab) in patients with advanced solid tumors with KRAS mutations with the hope of providing an effective new treatment option for patients with difficult-to-treat cancers (Press release, BridgeBio, JUL 27, 2021, View Source [SID1234585215]).

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The collaboration will also include the initiation of a Phase 1/2 study to evaluate the safety and preliminary efficacy of BBP-398 in combination with both OPDIVO as doublet therapy, and OPDIVO plus a KRASG12C inhibitor as triplet therapy in non-small cell lung cancer (NSCLC) with KRAS mutations, as first- and second-line treatment options. Under the terms of the non-exclusive collaboration, BridgeBio will sponsor the study and Bristol Myers Squibb will provide nivolumab. Both BridgeBio and Bristol Myers Squibb will share the cost of clinical development activities for the combination trial.

SHP2 is a protein-tyrosine phosphatase that links growth factor, cytokine and integrin signaling with the downstream RAS/ERK MAPK pathway to regulate cellular proliferation and survival. Overactivity of the SHP2 pathway, often driven by distinct genetic mutations, is a critical contributor to many forms of cancer, and is a mechanism of resistance to several targeted therapies.

"Cancers that are driven by hyperactive MAPK signaling, including certain RAS mutations such as KRASG12C, may be sensitive to SHP2 inhibition," said Frank McCormick, Ph.D., chairman of oncology at BridgeBio. "With this collaboration, we hope to better elucidate our SHP2 inhibitor’s ability to enhance immuno-oncology and other targeted therapies to potentially provide options for patients with difficult-to-treat cancers as quickly and safely as possible."

KRAS mutations occur in approximately 27% of NSCLC cases and approximately 17% of malignant solid tumors. Combination of anti-PD-1 treatment with BBP-398, and other targeted therapies, could be promising for patients with KRAS mutations.

"A priority of ours is to develop innovative medicines that target tumor intrinsic mechanisms including the MAPK pathway," said Emma Lees, senior vice president, oncology research at Bristol Myers Squibb. "We look forward to beginning the clinical exploration of the mechanistic rationale and therapeutic benefit from combining robust MAPK pathway inhibition and PD-1 blockade in KRAS mutant NSCLC."

"We are pleased to collaborate with Bristol Myers Squibb and advance BridgeBio’s larger strategy to fully interrogate BBP-398, a potentially best-in-class SHP2 inhibitor, as an ideal combination agent for certain cancer patients given its profile and potential for once daily dosing," said Eli Wallace, Ph.D., chief scientific officer of oncology at BridgeBio. "By partnering with one of the strongest oncology players in the industry, we hope to provide a new therapeutic approach to cancer patients in need."

BridgeBio is currently advancing its Phase 1 clinical trial in patients with solid tumors driven by mutations in the MAPK signaling pathway, including RAS and receptor tyrosine kinase genes.

OPDIVO is a trademark of Bristol-Myers Squibb Company.

About BBP-398

BBP-398 was developed through a collaboration with The University of Texas MD Anderson Cancer Center’s Therapeutics Discovery division. BridgeBio previously entered into a strategic collaboration with LianBio for clinical development and commercialization of BBP-398 in combination with various agents in solid tumors such as NSCLC, colorectal and pancreatic cancer, in mainland China and other major Asian markets.