Showing posts with label in. Show all posts
Showing posts with label in. Show all posts

Thursday, October 1, 2026

Teriparatide + Zoledronic Acid Does Not Reduce Fracture Risk in Osteogenesis Imperfecta

In continuation of my update on Teriparatide and  Zoledronic Acid



Among adults with osteogenesis imperfecta, teriparatide plus zoledronic acid does not reduce fracture risk compared with standard care, according to a study published online May 14 in the Journal of the American Medical Association.

Jannie Dahl Hald, Ph.D., from Aarhus University Hospital in Denmark, and colleagues examined whether the parathyroid hormone analogue teriparatide followed by zoledronic acid reduces the risk for fractures in adults with osteogenesis imperfecta in a multicenter randomized clinical trial. Participants were randomly assigned to receive 20 μg of teriparatide daily by subcutaneous injection for two years, followed by 5 mg zoledronic acid infusion (176 participants), or to standard care, in which bisphosphonates and other bone-targeted medicines could be used (174 participants).

Most of the participants had type I osteogenesis imperfecta caused by pathogenic variants in the type 1 collagen genes. The researchers found that 36.9 and 36.4 percent of patients in the teriparatide plus zoledronic acid group and the standard care group, respectively, had incident fractures. There was significantly more of an increase in lumbar spine and total hip bone mineral density with teriparatide plus zoledronic acid than standard care. Teriparatide plus zoledronic acid was favored in several quality-of-life measures. The groups reported similar adverse events.

"We have been using drugs to increase bone density for decades in the hope that they might prevent fractures but the TOPaZ trial clearly shows that these medicines simply do not work," lead author Stuart Ralston, M.D., from the University of Edinburgh Institute of Genetics and Cancer in the United Kingdom, said in a statement.

Ref : https://jamanetwork.com/journals/jama/fullarticle/2849063
https://en.wikipedia.org/wiki/Zoledronic_acid
https://en.wikipedia.org/wiki/Teriparatide

Teriparatide + Zoledronic Acid Does Not Reduce Fracture Risk in Osteogenesis Imperfecta - Drugs.com MedNews

Monday, September 28, 2026

Breakthrough drug reverses aging in skin and dramatically speeds healing


A drug designed to eliminate worn out, aging cells may help older skin recover from injury much faster, according to research published in Aging (Aging-US). The work, titled "Topical ABT-263 treatment reduces aged skin senescence and improves subsequent wound healing," suggests that targeting "zombie cells" in the skin could one day improve healing after surgery, injury, or chronic wounds in older adults.



The research team included Maria Shvedova, Rex Jeya Rajkumar Samdavid Thanapaul, Joy Ha, Jannat Dhillon, Grace H. Shin, Jack Crouch, Adam C. Gower, Sami Gritli, and Daniel S. Roh from Boston University Aram V. Chobanian and Edward Avedisian School of Medicine.

Clearing Out Aging Cells

As skin gets older, damaged cells can accumulate instead of dying off. These cells, known as senescent cells, no longer work normally, but they remain active enough to interfere with nearby tissue. Over time, they can release inflammatory signals and other molecules that weaken the skin's ability to repair itself.

The researchers tested whether ABT-263, a senolytic drug, could reduce this burden when applied directly to aged skin. Senolytic drugs are designed to selectively remove senescent cells, which have been linked to aging, inflammation, and slower tissue repair.

In the study, aged mice received ABT-263 on their skin for five days. After treatment, the skin showed fewer signs of cellular aging. When the researchers then created small wounds, the treated mice healed more quickly than untreated mice.

By day 24, 80% of the mice treated with ABT-263 had fully healed wounds, compared with 56% of untreated mice.

A Surprising Healing Boost

One of the more unexpected findings was that ABT-263 briefly increased inflammation in the skin. In many cases, inflammation is seen as harmful, especially when it becomes chronic. But in this case, the short burst appeared to help prepare the skin for repair.

The treatment seemed to wake up healing pathways that are normally sluggish in older tissue. Gene activity increased in areas tied to wound repair, including collagen production, blood vessel growth, tissue remodeling, and other processes needed to close and strengthen damaged skin.

This matters because aging skin does not just wrinkle or thin. It also becomes less responsive after injury. That slower response can increase the risk of prolonged recovery after surgery, delayed closure of wounds, and complications in people with chronic skin injuries.

Why Topical Treatment Matters

ABT-263 has drawn interest because it can target senescent cells, but oral senolytic drugs may cause side effects because they circulate through the body. Applying the drug directly to the skin could offer a more focused approach.

In this study, topical ABT-263 reduced signs of senescence in aged mice, but it did not appear to have the same effect in young mice. That suggests the treatment may be most active in older tissue, where senescent cells have built up.

The researchers believe this targeted approach could be especially useful before surgery or in people at risk for poor wound healing. Instead of waiting for a wound to struggle, a treatment might one day help prepare older skin in advance.

"Our study underscores the potential of topical senolytic treatments to enhance wound healing in aging skin, presenting a potentially promising strategy for preoperative care."

Newer Research Points in the Same Direction

Since this 2024 work, the broader field has continued moving toward localized senolytic strategies for skin repair. A 2025 review in Ageing Research Reviews described cellular senescence as a key contributor to skin aging and skin disease, while noting that senolytics and related therapies could become useful tools for targeting harmful senescent cells in the skin.

A 2026 study took the idea further in diabetic wound healing, a major medical challenge often marked by chronic inflammation, poor blood vessel growth, and cellular senescence. Researchers developed a localized wound dressing carrying ABT-263 and reported that it reduced senescent cell burden, improved healing in diabetic mice, and showed no detectable systemic toxicity in that model.

At the same time, scientists are careful not to portray senescent cells as purely bad. A 2024 Frontiers in Immunology review emphasized that senescence can play a helpful role during normal wound repair, but persistent senescent cells may contribute to chronic wounds, fibrosis, and abnormal healing. The challenge is timing and precision: removing the harmful lingering cells without disrupting the useful early repair signals.

Promise With Important Caution

The findings are exciting, but they are still early. The ABT-263 skin study was done in mice, and more work is needed before scientists know whether the treatment is safe or effective in people.

Researchers will also need to answer key questions about dosing, timing, long term safety, and whether the benefits apply to human skin, surgical recovery, diabetic wounds, or other slow healing conditions.

Still, the idea is powerful. By clearing away cells that hold aging skin back, topical senolytic treatments could someday help the body repair itself more quickly and more effectively. For older adults facing surgery or chronic wounds, that could make healing less difficult, less risky, and far faster.


https://en.wikipedia.org/wiki/Navitoclax
https://www.aging-us.com/article/206165/text
Breakthrough drug reverses aging in skin and dramatically speeds healing |

Friday, September 18, 2026

Daily orforglipron treatment reduces weight and blood sugar in seniors


In continuation of my update on orforglipron




A new analysis to be presented at this year's European Congress on Obesity (ECO 2026, Istanbul, Turkey, 12-15 May) examined daily oral orforglipron treatment for the treatment of obesity, with or without diabetes, in users aged 65 years and over, with results and a safety profile similar to that seen in the ATTAIN clinical trial programme population. Lead author for this post-hoc analysis is Dr Deborah Horn, Director of the Center for Obesity Medicine and Metabolic Performance at McGovern Medical School at UTHealth Houston, Houston, Texas, USA, and colleagues.

Limited clinical data exist on the use of incretin-based obesity management medications in older adults with or without type 2 diabetes (T2D), and a lack of specific analyses related to older users could be a factor that could make providers or patients in this age group hesitant when considering therapy choices.

Orforglipron is a novel small-molecule, non-peptide, oral glucagon-like peptide-1 receptor agonist (GLP-1 RA) developed by Eli-Lilly and Company (Lilly), who sponsored these analyses. The drug was approved by the US Food and Drug Administration for chronic weight management on April 1, 2026.

Orforglipron demonstrated significant weight reduction vs. placebo in the phase 3, randomised, double-blind, multinational ATTAIN-1 and ATTAIN-2 clinical trials in participants with obesity or obesity and T2D, respectively. In this new analysis of those trials, the authors evaluated efficacy and safety of orforglipron versus placebo in a sub-group of participants aged 65 years and older from ATTAIN-1 and ATTAIN-2.

The ATTAIN-1 and ATTAIN-2 global clinical trials evaluated once-daily orforglipron 6 mg, 12 mg, or 36 mg vs. placebo as an adjunct to healthy diet and physical activity in participants from 9 and 10 countries, respectively. In this sub-group analysis, efficacy outcomes were analysed separately for each trial, and safety data were pooled. The primary endpoint was percent change in body weight from baseline to week 72.

In ATTAIN-1 and ATTAIN-2, 616 randomised participants were ≥65 years of age (n=196 and 420, respectively). Of those, 613 received treatment (orforglipron 6 mg, n=118; 12 mg, n=135; 36 mg, n=146; placebo, n=214). And 79.1% of ATTAIN-1 and 86.2% of ATTAIN-2 participants had hypertension as a comorbidity.

At Week 72, the percent change in weight from baseline in users aged 65 and over in ATTAIN-1 (participants with obesity and without T2D) was –7.9%, –11.3%, and –13.0% with orforglipron 6 mg, 12 mg, and 36 mg, respectively, vs. –1.6% with placebo, all statistically significant findings. Similar results were found for those users aged 65 years and over with T2D and obesity in ATTAIN-2: orforglipron 6 mg: –7.5%; 12 mg: –8.3%; 36 mg: –12.2%; placebo: –2.3% again with all results statistically significant.

Participants with T2D experienced reductions from baseline in glycated haemoglobin (HbA1c – a measure of blood sugar control) of –1.5%, –1.6%, and –1.7% with 6 mg, 12 mg, and 36 mg orforglipron, respectively, vs. –0.1% with placebo. BMI, waist circumference, triglycerides, non-HDL cholesterol, and health-related quality of life also improved in orforglipron vs. placebo treatment groups in participants with or without T2D. 

https://en.wikipedia.org/wiki/Orforglipron

Thursday, September 17, 2026

Semaglutide shows sustained weight loss benefits in older adults

In continuation of my update on semaglutide



A new analysis of the STEP trails carried out by semaglutide manufacturer Novo Nordisk has analysed various trials to show the safety and efficacy of the obesity drug semaglutide in older adults (over 65 years), and found similar efficacy and safety as in the general trial populations . The study is by Prof Luca Busetto from the University of Padova in Italy and colleagues including from Novo Nordisk, who sponsor this new study.

Individuals of advanced age with obesity represent a vulnerable group, often presenting with comorbidities and frailty, and being at risk of adverse events (AEs). Information on the use of glucagon-like peptide-1 (GLP-1) receptor agonists including semaglutide in this population is limited; therefore, the authors decided assessed the efficacy and safety of once-weekly subcutaneous semaglutide 2.4 mg in individuals aged 65 years and over.

The analysis pooled together data from the STEP 1, 3, 4, 5, 8 and 9 trials (only in people with obesity or overweight, not diabetes, because weight loss in obesity drug trials is always lower in people with diabetes than without – thus results cannot be compared or mixed). It included participants aged ≥65 years with body mass index of at least 30 kg/m², or at least 27 kg/m² and at least 1 obesity-related complication (without diabetes) who were randomised to receive semaglutide 2.4 mg or placebo. All participants received lifestyle intervention and, in STEP 3, intensive behavioural therapy. Endpoints were assessed from baseline to week 68 and included percentage change in body weight; proportion of participants achieving categorical body weight reductions (≥10%, ≥15%, ≥20%); change in waist circumference; proportion of participants achieving waist to height ratio (WHtR) <0.53; shift in BMI category; and changes in cardiometabolic risk factors (glucose parameters, blood pressure, serum lipids and hs-CRP). Adverse events (AEs) were also assessed.

Of the total population in the selected trials (N=4523), 358 participants (8%) were aged 65 years or older and included in the analysis (semaglutide 2.4 mg, n=248; placebo, n=110), with most (90%) being aged 65–74 years (and the others 75 years and over). Across the pooled semaglutide and placebo groups at baseline, mean age was 69 years, bodyweight was 99.0 kg, BMI was 36.6 kg/m² and waist circumference was 115 cm; 72% were female.

At week 68, there was a mean −15.4% change in body weight for semaglutide 2.4 mg vs −5.1% for placebo, and a mean −14.3 cm vs −6.0 cm change in waist circumference, respectively. Proportions of participants achieving body weight reduction thresholds in the semaglutide 2.4 mg vs placebo groups were 66.5% vs 15.5% (at least 10%), 46.8% vs 6.4% (at least 15%) and 28.6% vs 2.7% (at least 20%), respectively.

In the semaglutide 2.4 mg group, 11.3% achieved a WHtR <0.53, compared with 4.5% with placebo. A greater proportion of semaglutide-treated participants improved their BMI category from baseline to week 68 compared with placebo (see Figure full abstract). A BMI of <27 kg/m² (so called healthy weight) was achieved by 27.0% of participants in the semaglutide group vs 5.5% in the placebo group; and the proportion of elderly patients in the overweight and obesity class I, II and III categories all fell in the semaglutide group at week 68 due to the increase in participants who had reached a healthy weight.

For participants achieving both a BMI of 27 or less and a WHtR of <0.53 these values were 10.5% vs 2.7%, respectively. Greater improvements in cardiometabolic risk factors were observed with semaglutide 2.4 mg vs placebo (see Table full abstract), including blood pressure, blood fats, cholesterol and glycated haemoglobin (HbA1c – a measure of blood sugar control used in diagnosis of diabetes).

Proportions of participants experiencing AEs and serious AEs in the semaglutide 2.4 mg vs placebo groups were similar for AEs overall (89.1% vs 84.5%), but higher for semaglutide re: serious AEs - 19.0% vs 12.7%, respectively. Constipation and dizziness rates (known side effects of this class of drug) were higher with semaglutide, while fractures and hypoglycaemia were comparable to placebo, both affecting less than 1% in each group.

https://en.wikipedia.org/wiki/Semaglutide

Thursday, September 3, 2026

Nuvalent Announces Submission of New Drug Application to FDA for Neladalkib in TKI Pre-treated Advanced ALK-Positive NSCLC


Nuvalent, Inc. (Nasdaq: NUVL), a clinical-stage biopharmaceutical company focused on creating precisely targeted therapies for clinically proven kinase targets in cancer, announced the submission to the U.S. Food and Drug Administration (FDA) of the Company's NDA for neladalkib, an investigational ALK-selective inhibitor, in TKI pre-treated advanced ALK-positive NSCLC.

New Drug Application (NDA) based on data in TKI pre-treated patients from the global ALKOVE-1 Phase 1/2 clinical trial
"The advancement of neladalkib from first clinical trial initiation to NDA submission in less than four years represents a remarkable pace in oncology drug development, underscoring the vigor and urgency our team brought to this program and our deep commitment to the ALK-positive NSCLC community," said Darlene Noci, A.L.M., Chief Development Officer at Nuvalent. "We would like to extend our sincere gratitude to the patients, families and investigators who have made this progress possible, and are committed to working closely with the FDA throughout the NDA review process toward our goal of bringing neladalkib to patients as quickly as possible."

The application is based on data in TKI pre-treated patients with advanced ALK-positive NSCLC treated with neladalkib in the global, registration-directed ALKOVE-1 Phase 1/2 clinical trial. In this population, neladalkib demonstrated encouraging overall activity, including intracranial responses, the ability to address key drivers of disease progression, and a generally well-tolerated safety profile consistent with its ALK-selective, TRK-sparing design. The company plans to share detailed results at a future medical meeting.

Neladalkib has received breakthrough therapy designation from the FDA for the treatment of patients with locally advanced or metastatic ALK-positive NSCLC who have been previously treated with 2 or more ALK tyrosine kinase inhibitors and orphan drug designation for ALK-positive NSCLC.

About Neladalkib
Neladalkib is an investigational, brain-penetrant, ALK-selective inhibitor created with the aim to overcome limitations observed with currently available ALK inhibitors. Neladalkib is designed to remain active in tumors that have developed resistance to first-, second-, and third-generation ALK inhibitors, including tumors with single or compound treatment-emergent ALK mutations such as G1202R. In addition, neladalkib is designed for central nervous system (CNS) penetrance to improve treatment options for patients with brain metastases, and to avoid inhibition of the structurally related tropomyosin receptor kinase (TRK) family. Together, these characteristics have the potential to avoid TRK-related CNS adverse events seen with dual TRK/ALK inhibitors and to drive deep, durable responses for patients across all lines of therapy. Neladalkib has received breakthrough therapy designation from the U.S. Food and Drug Administration (FDA) for the treatment of patients with locally advanced or metastatic ALK-positive non-small cell lung cancer (NSCLC) who have been previously treated with 2 or more ALK tyrosine kinase inhibitors and orphan drug designation for ALK-positive NSCLC.

About the ALKOVE-1 Phase 1/2 Clinical Trial
The ALKOVE-1 trial (NCT05384626) is a first-in-human Phase 1/2 clinical trial for patients with advanced ALK-positive NSCLC and other solid tumors. The completed Phase 1 portion enrolled ALK-positive NSCLC patients who previously received at least one ALK TKI, or patients with other ALK-positive solid tumors who had been previously treated or for whom no satisfactory standard of care exists. The Phase 1 portion of the trial was designed to evaluate the overall safety and tolerability of neladalkib, with additional objectives including determination of the recommended Phase 2 dose (RP2D), characterization of the pharmacokinetic profile, and evaluation of preliminary anti-tumor activity. The global, single arm, open label Phase 2 portion is designed with registrational intent for TKI pre-treated patients with advanced ALK-positive NSCLC. Global enrollment in ALKOVE-1 remains ongoing for adult and adolescent patients with ALK-positive solid tumors outside of NSCLC, and adolescent patients with ALK-positive NSCLC.

Nuvalent Announces Submission of New Drug Application to FDA for Neladalkib in TKI Pre-treated Advanced ALK-Positive NSCLC

Wednesday, August 5, 2026

Nuvalent Announces Submission of New Drug Application to FDA for Neladalkib in TKI Pre-treated Advanced ALK-Positive NSCLC

Nuvalent, Inc. (Nasdaq: NUVL), a clinical-stage biopharmaceutical company focused on creating precisely targeted therapies for clinically proven kinase targets in cancer, today announced the submission to the U.S. Food and Drug Administration (FDA) of the Company's NDA for neladalkib, an investigational ALK-selective inhibitor, in TKI pre-treated advanced ALK-positive NSCLC.



New Drug Application (NDA) based on data in TKI pre-treated patients from the global ALKOVE-1 Phase 1/2 clinical trial
"The advancement of neladalkib from first clinical trial initiation to NDA submission in less than four years represents a remarkable pace in oncology drug development, underscoring the vigor and urgency our team brought to this program and our deep commitment to the ALK-positive NSCLC community," said Darlene Noci, A.L.M., Chief Development Officer at Nuvalent. "We would like to extend our sincere gratitude to the patients, families and investigators who have made this progress possible, and are committed to working closely with the FDA throughout the NDA review process toward our goal of bringing neladalkib to patients as quickly as possible."

The application is based on data in TKI pre-treated patients with advanced ALK-positive NSCLC treated with neladalkib in the global, registration-directed ALKOVE-1 Phase 1/2 clinical trial. In this population, neladalkib demonstrated encouraging overall activity, including intracranial responses, the ability to address key drivers of disease progression, and a generally well-tolerated safety profile consistent with its ALK-selective, TRK-sparing design. The company plans to share detailed results at a future medical meeting.

Neladalkib has received breakthrough therapy designation from the FDA for the treatment of patients with locally advanced or metastatic ALK-positive NSCLC who have been previously treated with 2 or more ALK tyrosine kinase inhibitors and orphan drug designation for ALK-positive NSCLC.

About Neladalkib
Neladalkib is an investigational, brain-penetrant, ALK-selective inhibitor created with the aim to overcome limitations observed with currently available ALK inhibitors. Neladalkib is designed to remain active in tumors that have developed resistance to first-, second-, and third-generation ALK inhibitors, including tumors with single or compound treatment-emergent ALK mutations such as G1202R. In addition, neladalkib is designed for central nervous system (CNS) penetrance to improve treatment options for patients with brain metastases, and to avoid inhibition of the structurally related tropomyosin receptor kinase (TRK) family. Together, these characteristics have the potential to avoid TRK-related CNS adverse events seen with dual TRK/ALK inhibitors and to drive deep, durable responses for patients across all lines of therapy. Neladalkib has received breakthrough therapy designation from the U.S. Food and Drug Administration (FDA) for the treatment of patients with locally advanced or metastatic ALK-positive non-small cell lung cancer (NSCLC) who have been previously treated with 2 or more ALK tyrosine kinase inhibitors and orphan drug designation for ALK-positive NSCLC.


https://go.drugbank.com/drugs/DB16688

Saturday, August 1, 2026

Cogent Biosciences Announces Submission of New Drug Application for Bezuclastinib in Gastrointestinal Stromal Tumors (GIST)


Cogent Biosciences, Inc. (Nasdaq: COGT), a biotechnology company focused on developing precision therapies for genetically defined diseases, announced the completion of the submission of its New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for bezuclastinib in patients with Gastrointestinal Stromal Tumors (GIST) who have received prior treatment with imatinib. Based on the positive results from the PEAK trial, the bezuclastinib NDA was submitted under the FDA’s Real-Time Oncology Review (RTOR) program, which is intended to enable a more streamlined review process. Bezuclastinib was also granted Breakthrough Therapy Designation as a treatment for GIST earlier this year.




Bezuclastinib NDA submitted under the FDA’s RTOR program based on positive results from Phase 3 PEAK trial; bezuclastinib previously granted Breakthrough Therapy Designation in GIST
Bezuclastinib combination demonstrated 16.5 month mPFS and 46% ORR in imatinib-resistant GIST patients, dramatically improving upon the current standard of care
“We are excited to complete our PEAK NDA submission which marks a significant step toward bringing a new therapy to patients with second-line GIST,” said Andrew Robbins, President and Chief Executive Officer. “Based on the strength of the PEAK data, we believe the bezuclastinib combination has the potential to meaningfully change the treatment landscape for these patients. We are grateful to the patients, investigators, and study teams who made this possible.”

Pivotal data from PEAK, a global, randomized Phase 3 clinical trial evaluating bezuclastinib in combination with sunitinib vs. sunitinib monotherapy in patients with GIST who have received prior treatment with imatinib, were reported in November 2025. As disclosed in the top-line results, the bezuclastinib combination demonstrated a substantial and highly statistically significant clinical benefit on the primary endpoint of progression free survival (PFS), reducing risk of disease progression or death compared to the current standard of care by 50% (hazard ratio of 0.50, 95% CI: 0.39 – 0.65). mPFS, as assessed by blinded independent central review, was 16.5 months for the bezuclastinib combination vs. 9.2 months for sunitinib monotherapy. Additionally, the bezuclastinib combination demonstrated an unprecedented ORR in imatinib-resistant patients, with 46% of patients treated with the bezuclastinib combination achieving an objective response compared to 26% of patients treated with sunitinib. The bezuclastinib combination was generally well tolerated, and no unique risks were observed with the novel combination when compared to the known safety profile of sunitinib. Data for overall survival remains immature.

At the time of data cutoff, based on the number of ongoing patients receiving treatment on the bezuclastinib combination arm, the estimated mean duration of treatment for the bezuclastinib combination is projected to exceed 19 months.

Cogent plans to present full results from the PEAK trial at a major medical meeting during the first half of 2026. Additionally, Cogent is on track this quarter to initiate a Phase 2 trial investigating the benefit of the bezuclastinib plus sunitinib combination for first-line GIST patients with exon 9 mutations who are naive to, or recently initiated treatment with, imatinib. The NDA submission with bezuclastinib in Advanced Systemic Mastocytosis (AdvSM) also remains on track for the first half of 2026.

Bezuclastinib - Expanded Access Program
Working with the FDA, Cogent has established active Expanded Access Programs (EAPs) for U.S. patients with GIST or SM who meet disease-specific criteria and could benefit from treatment with bezuclastinib or the combination of bezuclastinib and sunitinib. A growing number of sites now offer access to the bezuclastinib EAPs. For more information please visit: https://www.cogentbio.com/bezuclastinib-program-development/#our-expanded-access-policy

Inducement Grants Under Nasdaq Listing Rule 5635(c)(4)
Cogent also announced today that, on March 30, 2026, the Compensation Committee of Cogent’s Board of Directors, made up entirely of independent directors, approved the grants of “inducement” equity awards to six new employees under the company’s 2020 Inducement Plan with a grant date of March 30, 2026. The awards were approved in accordance with Listing Rule 5635(c)(4) of the corporate governance rules of the Nasdaq Stock Market. The employees received, in the aggregate, (i) nonqualified options to purchase 21,100 shares of Cogent common stock and (ii) 15,700 restricted stock units (RSUs). Each option has a 10-year term, an exercise price equal to the closing price of Cogent’s common stock on the grant date, and a 4-year vesting schedule with 25% vesting on the 1-year anniversary of the grant date and the remainder vesting in equal monthly installments over the subsequent 36 months, provided such employee remains employed through each such vesting date. The RSUs vest annually in equal installments over 4 years from the grant date, provided such employee remains employed through each such vesting date.

https://en.wikipedia.org/wiki/Bezuclastinib

Monday, July 6, 2026

FDA Approves Lynavoy (linerixibat) for Cholestatic Pruritus in Patients with Primary Biliary Cholangitis

GSK  announced   the US Food and Drug Administration (FDA)  approval of  Lynavoy (linerixibat) for the treatment of cholestatic pruritus in adult patients with PBC. Lynavoy, an ileal bile acid transporter (IBAT) inhibitor that reduces multiple drivers of chronic itch, is the first medicine approved in the US for this indication.5
Lynavoy, an ileal bile acid transporter (IBAT) inhibitor, is the first medicine approved in the US for the treatment of cholestatic pruritus in patients with PBC
Up to 89% of people living with PBC experience cholestatic pruritus, an internal itch with a debilitating impact on quality of life1-4



Approval based on the positive GLISTEN phase III trial with regulatory reviews underway in the EU, UK, Canada and China

GSK previously announced on 9 March a licence agreement under which Alfasigma S.p.A. will acquire worldwide exclusive rights to develop, manufacture and commercialise linerixibat. This transaction is ongoing and is subject to customary conditions, including applicable regulatory agency clearances such as under the Hart-Scott-Rodino Act in the US.

Cholestatic pruritus is an internal itch experienced by up to 89% of people living with PBC, a rare autoimmune disease that can lead to liver failure.1-4 It is a serious condition that can be debilitating, with patients experiencing sleep disturbance, fatigue, impaired quality of life and even sometimes requiring liver transplantation in the absence of liver failure

https://en.wikipedia.org/wiki/Linerixibat

FDA Approves Lynavoy (linerixibat) for Cholestatic Pruritus in Patients with Primary Biliary Cholangitis

Tuesday, June 30, 2026

Praxis Precision Medicines Announces FDA Acceptance and Priority Review of New Drug Application for Relutrigine in Patients with SCN2A and SCN8A DEEs

Praxis Precision Medicines, Inc. (NASDAQ: PRAX), a fully integrated, leading central nervous system (CNS) precision neuroscience biopharmaceutical company, today announced that the U.S. Food and Drug Administration (FDA) has accepted for priority review its New Drug Application (NDA) for relutrigine, for the treatment of SCN2A and SCN8A developmental and epileptic encephalopathies (DEEs). The FDA has set a target action date under the Prescription Drug User Fee Act (PDUFA) of September 27, 2026.




  • FDA assigned PDUFA target action date of September 27, 2026

“Our first FDA acceptance of an NDA submission marks a significant milestone in our evolution to a commercial-stage company and an important step toward delivering innovative, precision neuroscience therapies to patients in need. SCN2A/8A DEEs have no currently approved targeted therapies and relutrigine, if approved, would be the first disease-modifying therapy for children suffering from these devastating and fatal conditions. We look forward to working closely with the FDA during the review process while continuing to advance our launch preparations,” said Marcio Souza, president and chief executive officer.

Relutrigine for treatment of SCN2A/8A DEEs
The NDA is supported by positive results from the EMBOLD study, which was stopped early for efficacy following a successful interim analysis and recommendation from the Data Monitoring Committee. Relutrigine has an Orphan Drug Designation, as well as a Rare Pediatric Disease Designation and a Breakthrough Therapy Designation. If granted approval, relutrigine will be the first FDA-approved therapy for SCN2A/8A DEE as well as be eligible for a Pediatric Review Voucher.

Relutrigine is also being investigated in broad DEEs through the EMERALD trial, which is expected to be completed by the end of 2026.

About Relutrigine
Relutrigine is a first-in-class small molecule in development for the treatment of developmental and epileptic encephalopathies (DEEs) as a preferential inhibitor of persistent sodium current, shown to be a key driver of seizure symptoms in severe DEEs. Relutrigine’s mechanism of precision sodium channel (NaV) modulation is consistent with superior selectivity for disease-state NaV channel hyperexcitability. In vivo studies of relutrigine have demonstrated dose-dependent inhibition of seizures up to complete control of seizure activity in SCN2A, SCN8A and other DEE mouse models. Relutrigine has been generally well-tolerated in three Phase 1 studies and has demonstrated biomarker changes indicative of NaV channel modulation. Data from cohort 1 of the Phase 2 EMBOLD study demonstrated a well-tolerated, robust, short- and long-term improvement in motor seizures in a heavily pre-treated population, alongside maintained seizure freedom in some patients with SCN2A- and SCN8A-DEE. Relutrigine has received Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation from the FDA for the treatment of SCN2A-DEE, SCN8A-DEE and Dravet syndrome; as well as Breakthrough Therapy Designation (BTD), and ODD from the European Medicines Agency for the treatment of SCN2A-DEE and SCN8A-DEE.




Thursday, May 21, 2026

FDA Approves Lasix ONYU (furosemide) for Treatment of Edema in Heart Failure




SQ Innovation, Inc. today announced that the U.S. Food and Drug Administration (FDA) has approved its drug-device combination Lasix ONYU (furosemide injection) for the treatment of edema (due to fluid overload) in adult patients with chronic heart failure. Lasix ONYU was developed to enable subcutaneous infusion of furosemide outside the healthcare setting for selected patients, as prescribed by a clinician without the need for a healthcare professional to administer the drug.

About 6.7 million Americans suffer from heart failure, with the prevalence expected to rise to 8.7 million by 2030. Heart failure is a leading cause of hospitalizations for individuals aged 65 and older with approximately 1.2 million hospitalizations per year [1].

Lasix ONYU consists of a novel high-concentration formulation of furosemide combined with a state-of-the-art small Infusor for treatment at home. The innovative design includes a reusable unit that can be used for 48 treatments and a plastic sterile single-use unit that is discarded after treatment. The two-component design reduces manufacturing complexity and cost, allowing the product to be offered at a different, more favorable price point which is expected to reduce barriers to widespread adoption.

“Lasix ONYU has the potential to be transformative in the care of patients experiencing worsening heart failure due to fluid overload,” said Pieter Muntendam, MD, founder, President and CEO of SQ Innovation. “Treating selected patients at home offers important benefits to patients, health systems and payors. We look forward to launching Lasix ONYU with leading health systems in the 4th quarter of 2025.”

Bioavailability and diuretic response were determined in a clinical study in which Lasix ONYU demonstrated complete bioavailability (112%) resulting in similar diuresis (115%) and natriuresis (117%) when compared to the same dose given by IV bolus. The biphasic delivery of furosemide by the Infusor resulted in a tempered diuretic response while IV bolus administration led to a shorter period of more intense diuresis. The results of the study were published in a leading cardiovascular journal [2].

“Heart failure is the most common serious medical condition in the U.S. and affects about one in four Americans during their lifetime. The number of patients affected is expected to double over the next 20 years and we currently already often lack adequate resources to take care of the 6.7 million patients affected presently – there are not enough beds, clinicians and funds”, said Dr. Javed Butler, Professor of Medicine at University of Mississippi and President, Baylor Scott and White Research Institute. “The only two actionable solutions now are more widespread adoption of guideline directed medical therapy (GDMT) and treating more patients at home with products such as subcutaneous diuretics instead of hospitalization for intravenous diuretics.”

“Decongestion through use of IV diuretics has been the cornerstone of treatment for reducing edema and hypervolemia in heart failure patients for over five decades,” stated S. Craig Thomas, Immediate Past President of the American Association of Heart Failure Nurses (AAHFN), an organization dedicated to advancing nursing education, clinical practice, and research to improve outcomes for heart failure patients. “The availability of accessible, affordable, and novel options that do not require the presence of a healthcare professional allows for transformative new clinical care-delivery. This means patients who now would typically need to be hospitalized for several days of IV treatment can instead remain home, supported by periodic or remote monitoring. The significance of this shift away from inpatient care for patients, hospitals, and payers cannot be overstated.”

Starting this quarter, Lasix ONYU will be available from leading pharmaceutical distributors enabling timely availability at participating medical facilities and affiliated retail pharmacies.

SQ Innovation is hosting a Conference Call and Webcast on Thursday October 9, at 4:30pm ET to introduce the product and answer questions from the community. Participating in the conference call will be:

Pieter Muntendam, MD, President and CEO of SQ Innovation
Mustafa M. Ahmed, MD, Professor of Medicine, Section Chief, Heart Failure, University of Florida Health, Gainesville, FL
Craig Thomas, Nurse Practitioner, Advanced Heart Failure Center, University of Virginia Health System, Charlottesville, VA and Immediate Past President American Association of Heart Failure Nurses (AAHFN)

https://en.wikipedia.org/wiki/Furosemide

FDA Approves Lasix ONYU (furosemide) for Treatment of Edema in Heart Failure