Regulus's Polycystic Kidney Disease Candidate Shows Benefit At Interim Analysis

 

• Regulus Therapeutics Inc (NASDAQ: RGLS) has announced an incremental update from the first cohort of patients from its ongoing Phase 1b trial evaluating RGLS4326 in Autosomal Dominant Polycystic Kidney Disease (ADPKD).

• ADPKD is a genetic disorder characterized by the growth of numerous cysts in the kidneys.

• In the first cohort, nine patients were enrolled and received 1 mg/kg of RGLS4326 subcutaneously every other week for four doses.

• The mean increase in polycystins 1 and 2 (lower protein levels associated with ADPKD) at the end of the study compared to baseline levels for all nine patients in the first cohort was 58% (p=.0004) 38%, respectively.

• As disclosed earlier, treatment with RGLS4326 was generally well-tolerated with no serious adverse events reported. All reported adverse events were mild and generally transient.

• These data will be presented at the Oppenheimer Rare & Orphan Disease Summit today.

• Additional data from this first cohort will be presented at PKD Connect in June 2021, and an abstract will be submitted to the American Society of Nephrology annual meeting in November.

• Data from the second cohort is expected in the third quarter.

https://finance.yahoo.com/news/reguluss-polycystic-kidney-disease-candidate-144746091.html

Seres Therapeutics Presents Data Across Broad Microbiome Pipeline

 Data validates strength of Seres’ investigational microbiome therapeutics pipeline in potentially addressing unmet treatment needs in recurrent C. difficile infection and ulcerative colitis –

– In Phase 3 ECOSPOR III study, SER-109 significantly reduced recurrence rates of C. difficile infection at 24 weeks with an observed safety profile comparable to placebo –

– Preclinical data support the potential of SER-301 as a therapeutic modality for mild-to-moderate ulcerative colitis through microbiome restoration –

https://finance.yahoo.com/news/seres-therapeutics-presents-data-across-161500780.html

Gracell Bio update conference call June 4

 Gracell Biotechnologies Inc. (NASDAQ: GRCL) (“Gracell”), a global clinical-stage biopharmaceutical company dedicated to discovering and developing highly efficacious and affordable cell therapies for the treatment of cancer, today announced that it will host a conference call and webcast to present an update on the results of a Phase 1 first-in-human multicenter investigator-initiated study of the FasTCAR-enabled BCMA/CD19 dual-targeting CAR-T cell therapy GC012F in relapsed/refractory multiple myeloma. This data was accepted as poster presentation at the 2021 American Society of Clinical Oncology Annual Meeting.

Conference call and webcast details:
Friday, June 4, 2021 @ 11:00 am ET
Investor domestic dial-in: 877-407-0784
Investor international dial-in: +1 201-689-8560
Conference ID: 13719675
Live webcast link: https://ir.gracellbio.com/news-events/events-and-presentations
A replay of the webcast will be available on ir.gracellbio.com shortly after the conclusion of the event for 90 days.

https://finance.yahoo.com/news/gracell-biotechnologies-host-clinical-conference-120000091.html

Precision Bio to Present Data from First Gen Allogeneic CAR T Program in Non-Hodgkin Lmphoma at ASCO

 Precision BioSciences, Inc. (Nasdaq: DTIL), a clinical stage biotechnology company developing allogeneic CAR T and in vivo gene correction therapies, today announced that additional data from its Phase 1/2a study of PBCAR0191, the Company’s first generation, off-the-shelf allogeneic CAR T candidate targeting CD19 will be presented at the 2021 American Society of Clinical Oncology (ASCO) Meeting being held virtually June 4-8, 2021.

Abstracts are available and can be viewed on the ASCO web site at www.asco.org. Additional data including updated response rates, safety, and durability will be presented at the ASCO meeting.

Title: Preliminary safety and efficacy of PBCAR0191, an allogeneic, off-the-shelf CD19-targeting CAR T product, in relapsed/refractory (r/r) CD19+ NHL
Poster Session: Hematologic Malignancies—Lymphoma and Chronic Lymphocytic Leukemia, Abstract 7516
Date/Time: Friday, June 4, 2021 at 9:00 a.m. ET
Presenting Author: Bijal Shah, M.D., Moffitt Cancer Center

https://www.businesswire.com/news/home/20210519005904/en/Precision-BioSciences-to-Present-Data-from-its-First-Generation-CD19-Allogeneic-CAR-T-Program-in-RelapsedRefractory-Non-Hodgkin-Lymphoma-at-the-2021-American-Society-of-Clinical-Oncology-Meeting

ResMed upped to Overweight from Neutral by JPMorgan

 Target to $219 from $206

https://finviz.com/quote.ashx?t=RMD

Bristol embraces AI drug discovery with $1.2bn Exscientia alliance

 Exscientia has signed up another big pharma partner for its artificial intelligence (AI) based drug discovery platform, this time attracting a $50 million upfront buy-in from Bristol-Myers Squibb. 

BMS wants to harness Exscientia’s tech to discover small-molecule drugs across a range of therapeutic categories – including immunology and oncology – and is promising another $125 million in near- and mid-term milestones to the project if all goes well.

All told, the value of the deal could be as much as $1.2 billion, one of the largest ever signed by a company in the AI dug discovery category. It follows a smaller collaboration with Celgene – acquired by BMS last year – that dates back to 2019.

“Exscientia’s application of AI technologies is proving capable of generating best-in-class molecules while also reducing discovery times,” said Rupert Vessey, president of research and early development at BMS.

Last year, Exscientia claimed a first by becoming the first company to take an AI-designed drug into the human testing stage – a candidate for obsessive-compulsive disorder partnered with Sumitomo Dainippon Pharma (DSP).

Since then, the company has started trials of two other drugs – Evotec-partnered cancer immunotherapy EXS-21546 and another DSP candidate for Alzheimer’ disease psychosis – and in April secured new financing of up to $525 million.

The Oxford, UK-based company has also signed pharma partnerships with Bayer, Sanofi, GlaxoSmithKline, and Roche, as well as co-development deals with smaller biotechs including Evotec, Blue Oak Pharma, Apeiron and Rallybio, but the latest with BMS is its largest to date.

It is also working on antiviral candidates for COVID-19 with the help of $4.2 million in backing from the Gates Foundation.

The appeal lies in Exscientia’s AI platform that can take projects from concept to clinical trials in a matter of months rather than years by narrowing down the hunt for suitable molecules so that far fewer have to be tested in the lab.

A report published by GlobalData earlier this year predicted that AI will transform the pharmaceutical industry in the coming years by delivering productivity improvements and efficiencies across multiple areas of the business, including drug discovery.

It identified almost 100 partnerships between AI vendors and large pharma companies for drug discovery since 2015, with numbers increasing over time and around 27 signed last year. Along with Exscientia, other players in the category include BenevolentAI, Atomwise, Recursion Pharma, and Insilico Medicine.

For BMS, the partnership is its second in the space of 24 hours, coming after it paid $200 million upfront to US biotech Agenus for exclusive rights to an anti-TIGIT antibody with potential as a cancer immunotherapy. That deal is also back-loaded with $1.46 billion in potential milestone payments.

https://pharmaphorum.com/news/bms-embraces-ai-drug-discovery-with-1-2bn-exscientia-alliance/

FDA OKs J&J 1st Targeted Therapy for Type of Non-Small Cell Lung Cancer

 Today, the U.S. Food and Drug Administration approved Rybrevant (amivantamab-vmjw) as the first treatment for adult patients with non-small cell lung cancer whose tumors have specific types of genetic mutations: epidermal growth factor receptor (EGFR) exon 20 insertion mutations.

The FDA also approved the Guardant360 CDx (Guardant Health Inc.) as a companion diagnostic for Rybrevant today.

“Advances in precision oncology continue to facilitate drug development, allowing diseases like lung cancer to be subset into biomarker-defined populations appropriate for targeted therapies,” said Julia Beaver, M.D., chief of medical oncology in the FDA’s Oncology Center of Excellence and acting deputy director of the Office of Oncologic Diseases in the FDA’s Center for Drug Evaluation and Research. “With today’s approval, for the first time, patients with non-small cell lung cancer with EGFR exon 20 insertion mutations will have a targeted treatment option.”

Lung cancer is the most common cancer type and the leading cause of cancer-related deaths worldwide, with non-small cell lung cancer accounting for 80% to 85% of all lung cancers, according to the American Cancer Society. Approximately 2% to 3% of patients with non-small cell lung cancer will have EGFR exon 20 insertion mutations, which are a group of mutations on a protein that causes rapid cell growth, and consequently, helps cancer spread. EGFR exon 20 insertion mutations are the third most common type of EGFR mutation.

Researchers evaluated Rybrevant’s efficacy in a study of 81 patients with non-small cell lung cancer and EGFR exon 20 insertion mutations whose disease had progressed on or after platinum-based chemotherapy. The main outcome measured was overall response rate (proportion of patients whose tumor is destroyed or reduced by a drug). In the trial population in which all patients received Rybrevant, the overall response rate was 40%. The median duration of response was 11.1 months, with 63% of patients having a duration of response of 6 months or more.

The most common side effects of Rybrevant include rash, infusion-related reactions, skin infections around the fingernails or toenails, muscle and joint pain, shortness of breath, nausea, fatigue, swelling in the lower legs or hands or face, sores in the mouth, cough, constipation, vomiting and changes in certain blood tests. Rybrevant should be withheld if patients develop symptoms of interstitial lung disease and permanently discontinued if interstitial lung disease is confirmed. Patients taking Rybrevant should limit sun exposure during and for two months after treatment. Rybrevant may cause problems with vision. Rybrevant can also cause fetal harm when administered to a pregnant woman; therefore, the pregnancy status of females of reproductive potential should be confirmed before treatment is started.

Rybrevant received Priority Review and Breakthrough Therapy designation for this indication. Priority Review directs overall attention and resources to the evaluation of applications for drugs that, if approved, would be significant improvements in the safety or effectiveness of the treatment, diagnosis or prevention of serious conditions when compared to standard applications. Breakthrough Therapy designation is designed to expedite the development and review of drugs that are intended to treat a serious condition and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over available therapy on a clinically significant endpoint(s).

The FDA granted approval of Rybrevant to Janssen Pharmaceutical Companies of Johnson & Johnson.

https://www.fda.gov/news-events/press-announcements/fda-approves-first-targeted-therapy-subset-non-small-cell-lung-cancer