Brickell Plans to Submit New Drug Application to FDA for gel

Brickell Biotech will request the FDA in mid-2022 to approve its gel for excessive underarm sweating after decreasing sweat production and disease severity in two pivotal trials. 

The gel achieved statistical significance in lessening the severity of hyperhidrosis and the amount of sweat produced and enhanced secondary outcomes in the two-phase three trials conducted in the U.S, called Cardigan 1 and 2, as said by Brickell.

A total of 701 patients were either given the gel or placebo daily for six weeks across the two late-stage trials. The gel, known as sofpironium bromide gel, 15%, was given to patients aged nine years or older with primary axillary hyperhidrosis.

This is the first indication Brickell is targeting the gel. The underarm is the most common for hyperhidrosis, impacting about 65% of the 15 million people in the U.S. who have excessive sweating. 

At a 5% version, the gel was approved in Japan last September and brought to market by Brickell and local partner Kaken Pharmaceutical last November. 

Robert Brown, Brickell’s CEO, said in the statement that these data are highly encouraging and further reinforce their belief that sofpironium bromide gel, 15%, has the potential to become a best-in-class treatment option for the millions of patients who have primary axillary hyperhidrosis.

Nearly 50% of patients treated with the gel in the Cardigan 1 study and almost 64% of patients in the Cardigan 2 trial achieved at least a two-point improvement from baseline in a study measuring the severity of the excessive sweating disease. That is versus nearly 30% and 47% of patients on placebo. The gel decreased the amount of sweat by almost 130 mg and 146 mg in the two trials, whereas the placebo reduced it by 99 mg and 131.7 mg.  

Twin Health secures USD 140 Million funding to scale its Whole Body Digital Twin service

Twin Health has raised USD 140 Million in Series C financing round to scale its ‘Whole Body Digital Twin’ service for chronic metabolic disease.

Investors comprising ICONIQ Growth, Sequoia Capital India, Perceptive Advisors, Corner Ventures, LTS Investments, Helena, sweat amounts, and Sofina have participated in the funding round.

Twin Health is a precision health platform, which comprises IoT sensors, machine learning, and medical science to enhance human metabolic health.

Its Whole Body Digital Twin is a digital predictive tool proffering personalized nutrition, sleep, activity, and breathing guidance through an easy-to-use app.

ICONIQ founding partner Divesh Makan said that they are happy to collaborate with the visionary Jahangir Mohammed and his extraordinary team as they scale Twin Health and its innovative technology to fulfill one of the biggest healthcare challenges of their time.

Twin Health has the chance to transform and enhance the quality of life for so many people, and they are incredibly proud to support the company’s mission. As per the company, it also facilitates the reversal and prevention of chronic metabolic diseases while improving energy and physical health.

The digital tool was developed using many data points garnered through non-invasive wearable sensors and self-reported preferences and is supported by artificial intelligence (AI).

Bristol Myers Squibb provides update on Deucravacitinib in Moderate to Severe Ulcerative Colitis patients  

Bristol Myers Squibb declared the Phase 2 LATTICE-UC study assessing deucravacitinib, a first-in-class, oral, selective tyrosine kinase 2 (TYK2) inhibitor, compared to placebo in moderate to severe ulcerative colitis (UC) flunk the primary efficacy endpoint of clinical remission at Week 12, nor secondary efficacy endpoints. The safety profile of deucravacitinib was consistent with previously reported studies in psoriasis and psoriatic arthritis, and no new safety signals were seen.

Samit Hirawat, M.D., chief medical officer, Bristol Myers Squibb, said that Deucravacitinib was a highly effective, first-in-class, oral, selective TYK2 inhibitor in psoriasis, and they have ongoing Phase 3 trials exploring the potential of deucravacitinib in psoriatic arthritis. While they did not achieve proof of concept in this study, they are committed to advancing their deucravacitinib clinical program in inflammatory bowel disease, including ulcerative colitis and Crohn’s disease, and psoriatic arthritis, lupus, and other immune-mediated diseases. 

The company will finish a complete review of the data from LATTICE-UC, and the potential of deucravacitinib in UC continues to be assessed in IM011-127. This second Phase 2 trial also comprises a higher dose.

Bristol Myers Squibb continues to anticipate a more significant than USD 4 billion non-risk adjusted revenue target for deucravacitinib in 2029.

Yale spinout shows targeting cancer cells’ acidic environment might improve immunotherapy

Antibody-drug conjugates such as AstraZeneca and Daiichi Sankyo’s Enhertu have shown unparalleled efficacy in particular biomarker-driven tumor types. But those therapies are not as effective at attacking cancer cells, which lack the antigen they are designed to aim for. Yale University spinout Cybrexa Therapeutics reports promising early evidence indicating it may have a solution to that shortcoming.

Cybrexa has developed a peptide-drug conjugate (PDC) which, instead of focussing on a specific antigen, targets the acidic environment of cancer cells to proffer a toxic payload. The drug, coded CBX-12, enhanced the efficacy of immune checkpoint inhibitors in mice with colorectal cancer, Cybrexa revealed at the AACR-NCI-EORTC virtual International Conference on Molecular Targets and Cancer Therapeutics.

Cybrexa advanced CBX-12 into a phase 1/2 study in May. The phase 1 portion of the trial will evaluate the safety and tolerability of the PDC at several dosing schedules in patients with solid tumors to determine the best dose for phase 2 testing.

Antibody-drug conjugates link antibodies specific to surface antigens on cancer cells with a toxic payload. Instead of antibodies, CBX-12 uses a peptide called pHLIP, developed by scientists at Yale University and the University of Rhode Island. The peptide aims at the acidic environment, which is unique to tumor tissues.

Cybrexa’s proprietary alphalex technology lets the peptide form a corkscrew like helix when it comes in contact with acidic or low pH conditions. It then penetrates the cancer cell membrane and releases a potent cytotoxic agent; in CBX-12’s case, that anti-cancer warhead is a compound called exatecan.

FDA clearance to Landos Biopharma’s LABP-104 for treating Systemic Lupus Erythematosus (SLE)

The late clinical stage biopharmaceutical company – Landos Biopharma Inc, announced that they got much anticipated FDA clearance for the company’s Investigational New Drug (IND) application for LABP-104. Landos Biopharma is utilizing it’s LANCE Advanced A.I. platform in discovering and producing novel and emerging therapies for patients with autoimmune disorders. LABP-104 is a clinical trial drug which is to be administered orally in patients experiencing Systemic Lupus Erythematosus (SLE). It is a systemically distributed LANCL2 antagonist which activates the LANCL2 pathway in order to restore the immune system activity to homeostasis by enhancing the regulatory T cell function and increasing the mitochondrial metabolism.

The Phase I clinical trial for  LABP-104 will be a double-blind, randomized, placebo-controlled, ascending dose, multi-cohort study to evaluate the efficacy, safety, tolerability and pharmacokinetics of LABP-104 in healthy volunteers. The number of expected participants is 56 and they will be enrolled in two parts – SAD – a single ascending dose study and then MAD – a multiple ascending dose study. A single oral dose of LABP-104 or placebo will be administered in the SAD trial where the participants shall be randomized to five cohorts. Whereas three oral doses of LABP-104 or placebo once daily for 7 days shall be administered in the MAD trial where the participants will be randomized into three cohorts. Here, the primary endpoint will be indicative of the safety and tolerability of LABP-104 and the secondary endpoint will be representing the pharmacokinetics of the once-daily oral therapeutic.

Amydis Receives an NIH grant of USD 3 million to explore ASYN in the retina of non-human primate models of Parkinson’s Disease and Multiple System Atrophy 

Amydis is one of the leading ocular tracer pharmaceutical companies with a broad spectrum of drugs (retinal tracers) in neurodegenerative disorders. It recently got awarded with the prestigious NIH Grant of USD 3 million to explore for the same cause. Amydis announced that the NIH Grant was awarded by NINDS (National Institute of Neurological Disorders and Stroke) to investigate the presence of ASYN (Alpha Synuclein) in the retina of non-human primate models of Parkinson’s Disease and Multiple System Atrophy. The NIH Grant is a 2 year long duration award which will fund the Investigational New Drug (IND) application related studies of the new therapy. And then it will finally aid in filing the IND application to the USFDA.

Amydis conducted a pre-IND meeting with the USFDA in order to gain agreement on designing the first-in-human Phase 1/2a clinical trials in patients and healthy volunteers. These clinical trials will mark the efficacy for small-molecule retinal tracers targeting the biomarker ASYN for the diagnosis of synucleinopathies. This treatment as directed by Amydis is a pathway to detect ASYN in the retina with its proprietary compounds. Its purpose is to be used additionally with already commercially available retinal imaging instrumentation that is readily available in the market and is also routinely used in ophthalmology practice. 

Conclusively, Amydis is aiming at producing an uncomplicated, widely accepted as well as affordable measure to identify ASYN in patients. 

FDA Fast Track Designation Grant to GenSight Biologics’ Optogenetic Therapy GS030 for treating Retinitis Pigmentosa

GenSight Biologics’ – a biopharma concentrated at developing, manufacturing and commercializing new and innovative gene therapies for retinal neurodegenerative and central nervous system disorders, declared that USFDA approved a Fast Track Designation to GS030, their Optogenetic Therapy as an aid to treat people experiencing Retinitis Pigmentosa. GS030 is administered in addition to AAV2-based gene therapy with optogenetics to treat the condition.

“These are truly groundbreaking findings that move the promise of optogenetics another step from therapeutic concept to clinical use,” commented Bernard Gilly, Co-Founder and Chief Executive Officer of GenSight. “These could not have occurred without the close collaboration we enjoyed with our partners at the Institut de la Vision, the Institute of Ophthalmology Basel and Streetlab. We are especially grateful to the patients who are participating in our trial, whose experiences and input will help us design the next stage of GS030’s clinical development. We will now accelerate the GS030 program to make it our second product to reach the market after LUMEVOQ.” The clinical study named PIONEER, which is a Phase I/II first-in-human, multi-centre, open-label dose-escalation study is being held across 3 centres in the United Kingdom, France, and in the United States. It will be indicative of the safety and tolerability of GS030 in patients with late-stage Retinitis Pigmentosa.