Roche Scraps Mid-Phase Programs in Hepatitis B, Geographic Atrophy

 In its third-quarter report Tuesday, Roche announced it has cut two Phase II programs, one for hepatitis B and the other for geographic atrophy, a form of eye disease. 

RG7907 is a core protein allosteric modulator that was developed for hepatitis B. Early data suggested the drug disrupted hepatitis B viral replication, and a Phase I trial in 2019 led the company to believe it could be part of a combination of drugs that could cure the disease.

A Phase I trial was completed in July, and a Phase II trial of the drug in combination with other therapies was ongoing as of August.

Roche Diagnostics has several assays to detect hepatitis B and several pipeline drugs, including RG6084, RG6346 and RG7854.

RG6147 (galegenimab), an anti-HTRA1 anti-binding fragment, was being developed for geographic atrophy.

The Phase II trial launched in June 2019 and as of September, its status was “active, not recruiting.”

Roche also has a very active program for ophthalmological conditions, including macular degeneration and geographic atrophy. 

In a statement, CEO Severin Schwan specifically cited Vabysmo. The drug is the first and only FDA-approved treatment that blocks both VEGF and Ang-2 in wet age-related macular degeneration and diabetic macular edema. It was approved in Europe in the third quarter of this year. 

Roche's subsidiary Chugai Pharmaceutical removed a Phase II program of a CHU Oncolytic Type 5 adenovirus for esophageal cancer. These types of viruses are designed to selectively grow in tumor cells, killing them while minimizing disruptions to normal cells.

Other Business Updates

Roche reported 2% sales growth, despite what Schwan in a press conference called “the expected sharp decline in COVID-19-related products in both divisions in the third quarter.

"The demand for our newer medicines for multiple sclerosis, hemophilia, spinal muscular atrophy and cancer remains high," he said. 

COVID-19 drug sales dropped by $1 billion (U.S.) in the first nine months of the year. Those included Ronapreve, an antibody treatment, and Actemra, originally developed for arthritis. 

Roche Diagnostics also reported a 40% decrease in COVID-19-related tests year on year to $604,000.

“In spite of increasing incidence rates for COVID-19, we actually don’t see an increase in the demand for COVID-19-related products. It has nothing to do with inventories … there is simply much smaller demand than we have seen in the previous year,"  Schwan said. 

Clinical trial data for Roche's Alzheimer’s drug gantenerumab is expected to be published by the end of November, the company noted.

Since the announcement of positive Phase III data from Eisai and Biogen’s lecanemab for Alzheimer’s disease in late September, analysts and investors have been turning their focus to gantenerumab in a suddenly hopeful market.

If anything, Schwan worked to keep expectations low, saying, “It is very dangerous to cross-read on trials. We have to wait for the readout, and then we can take it from there.”

Jefferies analysts were pessimistic, writing in a note to investors that they expect the study to fail.

https://www.biospace.com/article/roche-scraps-mid-phase-programs-in-hepatitis-b-eye-disease/

Salarius Pauses Enrollment in Phase 1/2 Trial of Seclidemstat in Ewing Sarcoma

  Salarius Pharmaceuticals, Inc. (NASDAQ: SLRX), a clinical-stage biopharmaceutical company using protein inhibition and protein degradation to develop cancer therapies for patients in need of new treatment options, today announced that, as per protocol design, it is voluntarily pausing new patient enrollment in the company’s Phase 1/2 trial of seclidemstat as a treatment for Ewing sarcoma and FET-rearranged sarcomas.

The pause in new patient enrollment is due to a metastatic FET-rearranged sarcoma patient death that was classified as a suspected unexpected serious adverse reaction (SUSAR). Upon review of the SUSAR and available information by the company’s independent Safety Review Committee for the clinical trial, patients currently receiving seclidemstat treatment may continue treatment after consulting with their physician.

Salarius communicated the details of the SUSAR to the U.S. Food and Drug Administration (FDA) and intends to further analyze the available data with the goal of understanding how best to proceed and restart enrollment as soon as possible. Salarius continues to plan to release interim sarcoma clinical trial results later this year.

https://finance.yahoo.com/news/salarius-pharmaceuticals-announces-pause-patient-123000980.html

Decibel OKd to Proceed with Hearing Loss Gene Therapy Product Candidate

 The IND for DB-OTO provides clearance for the Company to initiate a pediatric Phase 1/2 clinical trial in the U.S. in children and infants, and is part of an international regulatory strategy for clinical development

One-time administration of DB-OTO has resulted in production of otoferlin protein and durable auditory brainstem responses to sound in a congenitally deaf rodent disease model

DB-OTO is Decibel’s second hearing therapeutic candidate to enter clinical investigation

Decibel Therapeutics (Nasdaq: DBTX), a clinical-stage biotechnology company dedicated to discovering and developing transformative treatments to restore and improve hearing and balance, today announced that it has received clearance from the U.S. Food and Drug Administration (FDA) for its Investigational New Drug (IND) application to initiate a Phase 1/2 clinical trial in pediatric patients of DB-OTO, its lead gene therapy product candidate. DB-OTO is designed to provide durable hearing in individuals born with profound congenital hearing loss due to an otoferlin deficiency.

https://finance.yahoo.com/news/decibel-therapeutics-receives-fda-clearance-110000094.html

Kiora Publishes Phase 1 Trial Results for Treatment for Ocular Inflammatory Disease

  Kiora Pharmaceuticals, Inc. (NASDAQ: KPRX), ("Kiora" or the "Company") announced that results from a Phase 1 study of KIO-100 (formerly PP-001) demonstrate its potential as a treatment for non-infectious uveitis, an ophthalmic inflammatory disease. The results were published today in a paper titled "A new small molecule DHODH-inhibitor [KIO-100 (PP-001)] targeting activated T cells for intraocular treatment of uveitis - a phase I clinical trial," in the journal Frontiers in Medicine.

This first-in-man, open-label, phase-1 clinical trial investigated the use of KIO-100 for treating uveitis, a T cell-mediated, intraocular inflammatory disease. Results showed that a single intravitreal injection of KIO-100 decreased intraocular inflammation in a dose dependent fashion, and improved visual acuity significantly during the duration of the study. The drug was well tolerated, with no serious side effects on intraocular tissues or other adverse events observed.

KIO-100 is a dihydroorotate dehydrogenase (DHODH) inhibitor, a validated drug target in the treatment of systemic autoimmune conditions. As a third generation DHODH inhibitor with an intravitreal formulation and a high potency for suppressing T and B cells, KIO-100 is a promising non-steroidal anti-inflammatory agent.

https://finance.yahoo.com/news/kiora-pharmaceuticals-announces-publication-phase-120000476.html

Roche exec says Biogen Alzheimer's data was reassuring

 A Roche executive said on Tuesday that positive trial data on an Alzheimer's disease drug candidate by rival Biogen was encouraging for companies such as Roche also pursing amyloid beta proteins as a drug target.

"Before that readout there was quite a raging debate, as to whether targeting a amyloid beta does anything whatsoever to clinical signs and symptoms of Alzheimer's. After that data that debate has to take on a different character and it reassures us in the validity of the pathway," the head of Roche's pharma unit Bill Anderson said in an analyst call. Roche earlier repeated that keenly awaited trial data on experimental Alzheimer's drug gantenerumab was due to be published at the end of November.

Eisai and its partner Biogen last month reported positive results on their lecanemab drug candidate

https://finance.yahoo.com/news/roche-exec-says-biogen-alzheimers-130155566.html

Quoin to Initiate 2nd Clinical Trial in Netherton Syndrome

 Quoin Pharmaceuticals Ltd. (NASDAQ: QNRX) (the “Company” or “Quoin”), a specialty pharmaceutical company focused on rare and orphan diseases, announces that it is initiating a second clinical trial in Netherton Syndrome patients.

This multicenter, open label study will be being conducted under Quoin’s currently open U.S. Investigational New Drug (IND) Application and will assess QRX003 topical lotion in Netherton patients who are currently receiving treatment including systemic therapy for symptomatic relief. QRX003 will be applied once daily over a twelve-week period, to pre-designated areas of the patient’s body. A number of different clinical endpoints will be assessed in the study.

The active ingredient in QRX003 is a broad-spectrum serine protease inhibitor, whose mechanism of action is intended to down-regulate the hyperactivity of skin kallikreins, leading to a more normalized rate of skin shedding. If proven to be safe and effective, long term daily application of QRX003 could lead to the development of a more normally functioning skin barrier and a significant improvement in the quality of life of Netherton patients.

https://finance.yahoo.com/news/quoin-pharmaceuticals-announces-plans-initiate-123000258.html

'Addiction drug shows promise lifting long COVID brain fog, fatigue'

 Lauren Nichols, a 34-year-old logistics expert with the U.S. Department of Transportation in Boston, said she has had difficulty thinking and concentrating, fatigue, seizures, headaches, and fatigue since contracting the novel coronavirus. Spring 2020 in pain.

Last June, her doctor suggested a lower dose of naltrexone, a generic drug commonly used to treat alcohol and opioid addiction.

After living in “thick clouds of fog” for more than two years, she said, “I can actually think clearly.

Researchers seeking a long-term COVID cure say the drug could have similar effects on millions of people suffering from pain, fatigue and brain fog months after contracting the coronavirus. want to know if

The drug has been used with limited success to treat a similarly complex post-infectious syndrome characterized by cognitive impairment and overwhelming fatigue called myalgic encephalomyelitis/chronic fatigue syndrome (ME/CFS). I’m here.

According to a Reuters review of Clinicaltrials.gov and 12 interviews, at least four studies are available to test naltrexone in hundreds of long-term COVID patients, taking advantage of its use in ME/CFS and a small number of long-term COVID pilot studies. A clinical trial is planned. ME/CFS and long her COVID researcher.

It is also on a candidate list of treatments to be tested in the US National Institutes of Health’s billion-dollar RECOVER initiative, a clinical trial adviser told Reuters.

Unlike treatments aimed at addressing specific symptoms caused by COVID damage to organs such as the lungs, low-dose naltrexone (LDN) reverses some of the symptoms that cause the underlying medical condition. It is possible, they said.

Naltrexone has anti-inflammatory properties and has been used in low doses for years to treat conditions such as fibromyalgia, Crohn’s disease and multiple sclerosis, according to the University of Alabama Neuroinflammation, Pain and Fatigue Research. said Dr. Jared Younger, Director of the Institute. at Birmingham.

Naltrexone at 50 milligrams, ten times the lower dose, is approved for the treatment of opioid and alcohol dependence. Some generic manufacturers sell 50 mg pills, but low-dose naltrexone must be purchased from a pharmacy.

Younger, author of Scientific Review of Drugs as New Anti-Inflammatory Agents, submitted a grant application in September to study LDN for long COVID. It should,” he said.

Still, the drug is unlikely to help all long-term patients with COVID, a cluster of about 200 symptoms ranging from pain and heart palpitations to insomnia and cognitive impairment. In one ME/CFS study, 74% had improved sleep, reduced pain, and neurological deficits.

“It’s not a panacea,” says Jaime Seltzer, a Stanford University researcher and director of scientific outreach for the advocacy group MEAction. “These people were not cured, but they were helped.”

‘HUMAN AGAIN’ Dr. Jack Lambert, an infectious disease specialist at University College Dublin Medical School, used LDN to treat the pain and fatigue associated with chronic Lyme disease.

During the pandemic, Lambert recommended LDN to colleagues treating patients with lingering symptoms after a COVID attack.

It worked so well that he conducted a pilot study with 38 long-term COVID patients. After two months, they reported improvements in energy, pain, focus, insomnia, and overall recovery from COVID-19, according to findings published in July.

Lambert, who plans large-scale trials to confirm these results, believes that LDN may repair the damage of the disease rather than mask the symptoms of the disease.

Other planned LDN trials include a trial by the University of British Columbia in Vancouver and a pilot by Ann Arbor, Michigan-based startup AgelessRx. The study, which included 36 volunteers, should have results by the end of the year, said company co-founder Sajjad Zarzala.

Scientists are still working on explaining the mechanism of how LDN works.

Experiments by Dr. Sonya Marshall Gladysnik, Australia’s National Center for Neuroimmunology and Emerging Diseases, suggest ME/CFS and long-term COVID symptoms result from a drastic reduction in the function of the immune system’s natural killer cells doing. Laboratory experiments suggest that LDN may have helped restore normal function, but this theory has yet to be confirmed.

Others believe the infection triggers immune cells in the central nervous system called microglia to produce cytokines, inflammatory molecules, and cause fatigue and other symptoms associated with ME/CFS and long-term COVID.Younger believes that naltrexone calms down these hypersensitive immune cells.

Zack Porterfield, Ph.D., a virologist at the University of Kentucky and co-chair of the RECOVER task force looking at commonalities with other post-infectious syndromes, said he recommended including LDN in RECOVER treatment trials. I was.

Other treatments under consideration were antivirals, such as those from Pfizer, sources said. (PFE.N) Paxlobid, anticoagulants, steroids, dietary supplements. A RECOVER official said it has received dozens of proposals and cannot comment on which drugs it will test until the trial is complete.

Dr. Hector Bonilla, Co-Director of the Stanford Post-Acute COVID-19 Clinic and RECOVER Advisor, has used LDN in 500 ME/CFS patients, reporting about half the benefits.

He has studied LDN in 18 long-term COVID patients, 11 of whom have shown improvement, and believes that larger, more formal trials will determine whether LDN provides real benefits. says.

RECOVER patient advisor Nichols was “ecstatic” when he learned that LDN was being considered for a government-funded trial.

LDN didn’t solve all of her COVID-related problems, but it allowed Nichols to work all day without a break and have a social life from home.

“It made me feel human again.”

https://canadiantrends.ca/health/addiction-drug-shows-promise-lifting-long-covid-brain-fog-fatigue/