Metabolic disorder

• SILVER SPRING, Md., April 24, 2024 /PRNewswire/ -- Today, the U.S. Food and Drug Administration approved Pivya (pivmecillinam) tablets for the treatment of female adults with uncomplicated urinary tract infections (UTIs) caused by susceptible isolates of Escherichia coli, Proteus mirabilis and Staphylococcus saprophyticus.
• "The FDA is committed to fostering new antibiotic availability when they prove to be safe and effective, and Pivya will provide an additional treatment option for uncomplicated UTIs."
• Uncomplicated UTIs are bacterial infections of the bladder in females with no structural abnormalities of their urinary tract.
• Approximately one-half of all women experience at least one UTI in their lifetime.

• You may have heard of Ozempic, the “miracle drug” for weight loss, but did you know that it was actually designed as a new treatment to manage diabetes?
• In Canada, diabetes affects approximately 10 per cent of the general population.

Locks and keys

• Every cell in the body needs sugar as an energy source, but too much sugar can be toxic to cells.
• This equilibrium needs to be tightly controlled and is regulated by a lock and key system.
• Cells cover themselves with locks that respond perfectly to insulin keys to facilitate the entry of sugar into cells.
• The body can encounter difficulties producing an adequate number of insulin keys, and/or the locks can become stubborn and unresponsive to insulin.

Severe insulin-deficient diabetes: We’re missing keys!

• In the severe insulin-deficient diabetes (SIDD) subtype, the key factories — the beta cells — are on strike.
• Why the beta cells go on strike remains largely unknown, but since there is an insulin deficiency, treatment often involves insulin injections.

Severe insulin-resistant diabetes: But it’s always locked!

• In the severe insulin-resistant diabetes (SIRD) subtype, the locks are overstimulated and start ignoring the keys.
• There are many treatment avenues for these patients but no consensus about the optimal approach; patients often require high doses of insulin.

Mild obesity-related diabetes: The locks are sticky!

• Mild obesity-related (MOD) diabetes represents a nuanced aspect of Type 2 diabetes, often observed in individuals with higher body weight.
• The locks are “sticky,” so it is challenging for the key to click in place and open the lock.

Mild age-related diabetes: I’m tired of controlling blood sugar!

Mild age-related diabetes (MARD) happens more often in older people and typically starts later in life. With time, the key factory is not as productive, and the locks become stubborn. People with MARD find it tricky to manage their blood sugar, but it usually doesn’t lead to severe complications. Among the different subtypes of diabetes, MARD is the most common.

Unique locks, varied keys

• In Canada, unique cases of Type 2 diabetes were identified in Indigenous children from Northern Manitoba and Northwestern Ontario by Dr. Heather Dean and colleagues in the 1980s and 90s.
• Read more:
  Indigenous community research partnerships can help address health inequities

  Childhood-onset Type 2 diabetes is on the rise across Canada, but disproportionately affects Indigenous youth.

• Acknowledging this distinct subtype of Type 2 diabetes in First Nations communities has led to the implementation of a community-based health action plan aimed at addressing the unique challenges faced by Indigenous Peoples.

A mosaic of conditions

• Type 2 diabetes is not uniform; it’s a mosaic of conditions, each with its own characteristics.
• Since diabetes presents so uniquely in every patient, even categorizing into subtypes does not guarantee how the disease will evolve.

Lili Grieco-St-Pierre receives funding from Fonds de recherche du Québec - Santé (FRQS). Jennifer Bruin receives funding from the Canadian Institutes of Health Research (CIHR), Natural Sciences and Engineering Research Council of Canada (NSERC), JDRF, Diabetes Canada.

• Medicines & Healthcare products Regulatory Agency (MHRA) for the company’s Clinical Trial Authorization (CTA) application to expand the Phase 1/2 OTC-HOPE study evaluating ECUR-506 into the U.K.
• The OTC-HOPE study is investigating ECUR-506, incorporating an ARCUS nuclease, for the treatment of Ornithine Transcarbamylase (OTC) deficiency in infants.
• In 2021, Precision licensed to iECURE an ARCUS nuclease that inserts a functional copy of the OTC gene for treatment of OTC deficiency.
• “Congratulations to iECURE for bringing the first gene editing approach into clinical investigation for patients with OTC deficiency.

• - Discovered and developed with Argo's advanced RNA interference (RNAi) technology, these programs will reinforce and expand the Novartis RNAi portfolio on Cardiovascular and Metabolic diseases (CVM).
• SHANGHAI, Jan. 7, 2024 /PRNewswire/ -- Shanghai Argo Biopharmaceutical Co., Ltd. ("Argo"), a biotechnology company focused on the discovery and development of next-generation RNAi therapeutics, today announced that it has entered into two exclusive license and collaboration agreements with Novartis PHARMA AG ("Novartis").
• Under the first agreement, Argo has granted Novartis exclusive global licenses to develop and commercialize a Phase 1 stage program.
• Under the second agreement, Argo granted Novartis an exclusive ex-Greater China license to develop and commercialize a Phase 1/2a clinical-stage program for cardiovascular disease treatment.