Ionis' metabolic disorder drug lowers high levels of a type of fat in study

 Ionis Pharmaceuticals said on Tuesday its experimental drug met the main goal of reducing abnormally high levels of the most common type of body fat in a late-stage trial in patients with a metabolic disorder.

The rare genetic disorder, known as familial chylomicronemia syndrome (FCS), prevents the body from breaking down fats and is characterized by extremely high levels of triglyceride.

FCS is caused by the deficiency or impaired function of lipoprotein lipase enzyme, responsible for breaking down triglycerides, which when accumulated in the blood leads to inflammation of the pancreas.

The National Pancreas Foundation estimates FCS to occur in 1 in 1-2 million people globally.

The drug candidate, olezarsen, showed statistically significant reduction in levels of triglyceride compared to placebo, as well as showed a 100% reduction in inflammation of the pancreas, Ionis said.

The company said it plans to file a marketing application with the U.S. Food and Drug Administration for olezarsen early next year.

Shares of the California-based drug developer rose 3% to $44 in premarket trading.

If approved, olezarsen will be the first available treatment for FCS in the U.S., Ionis said.

https://finance.yahoo.com/news/1-ionis-pharmas-genetic-disorder-112807957.html

Bullfrog: Positive Data from Preclinical Study for Treatment of Glioblastoma

  BullFrog AI Holdings, Inc. (NASDAQ: BFRG; BFRGW) ("BullFrog AI" or the "Company"), a technology-enabled drug development company using artificial intelligence (AI) and machine learning to enable the successful development of pharmaceuticals and biologics, today announced positive data in a preclinical study investigating the anti-cancer activity of a novel prodrug of mebendazole for the treatment of glioblastoma. The study assessed the relative efficacy of BF-222, a novel formulation of mebendazole that has been evaluated in clinical trials, and BF-223, a novel prodrug of mebendazole with improved solubility and bioavailability relative to BF-222, compared with placebo in mice that had been implanted with tumor cells as a model for human glioblastoma.

Animals treated with BF-223 had an average survival time of 27.9 days compared with 27.3 days for mice treated with BF-222 and 23.4 days for mice given placebo. Mice treated with BF-223 were administered 80% of the dose that mice treated with BF-222 received, and improved outcomes for both treatment groups were statistically significant compared to placebo. In addition, animals treated with equivalent doses of BF-222 and BF-223 showed comparable and significant reduction in tumor growth compared to control animals during the study.

https://finance.yahoo.com/news/bullfrog-ai-announces-positive-data-120000742.html

Italy's Alfasigma to buy US liver disease drugmaker Intercept for nearly $800 mln

 Italy's Alfasigma S.p.A agreed on Tuesday to buy drugmaker Intercept Pharmaceuticals for $794 million as it aims to expand its treatment portfolio in liver diseases and digestive system disorders, and bolster presence in the U.S.

The proposed all-cash acquisition would add Intercept's Ocaliva, a treatment for liver disease primary biliary cholangitis, to Alfasigma's portfolio.

Morristown, New Jersey-based Intercept forecasts sales of $320 million to $340 million from the drug this year.

Alfasigma's acquisition of Intercept and its drug Ocaliva makes "best economic cost sense" given that a larger company can market and commercialize the drug with better margins and scale than a smaller biotech, Jefferies analyst Michael Yee said.

The deal comes after Intercept dropped out of the race to develop the first treatment for patients with non-alcoholic steatohepatitis (NASH), a liver disease that affects 5% of U.S. adults, after the U.S. health regulator declined to approve its drug in June.

Following this, Intercept discontinued all NASH-related investments, reduced about one-third of its workforce and restructured its operations to strengthen focus on rare and serious liver diseases.

Alfasigma will pay $19 per Intercept share, which represents a premium of 82% to its last closing price. Intercept's stock surged 77.54% to $18.53 in morning trading on Tuesday, but has fallen 15.6% this year alone. Its shares are down significantly from their peak of roughly $445 in 2014.

The deal is expected to close by the end of the year.

Alfasigma is among Italy's biggest pharmaceutical groups, with its portfolio ranging from prescription drugs to nutraceuticals. Its over-the-counter products include Biochetase, Neo-Borocillin, Dicloreum and Yovis. 

https://finance.yahoo.com/news/1-italys-alfasigma-buy-drugmaker-122728481.html

Soleno: Significant Top-line Results from Study C602 of DCCR for Prader-Willi

 Study Met Primary Endpoint; Highly Statistically Significant Difference in Change from Baseline in HQ-CT Total Score for DCCR Compared to Placebo (p=0.0022)

Soleno Intends to Submit a New Drug Application for DCCR in PWS Mid-Year 2024

Company to Host Conference Call and Webcast Today at 9:00 AM ET

Conference Call and Webcast Details
Soleno will host a conference call and webcast to discuss these results today, September 26, 2023 at 9:00 AM ET. Details can be found below:

Title:	Randomized Withdrawal Period of Study C602 Top-line Results
Date:	Tuesday, September 26, 2023
Time:	9:00 AM ET
Conference Call Details:	Toll-free: 1-877-423-9813 International: 1-201-689-8573 Conference ID: 13741535
Call me™ Feature (avoid waiting for operator):	Click Here
Webcast:	Webcast Link – Click here

A replay of the call will be available following the call on the Investors section of the Soleno website.

https://finance.yahoo.com/news/soleno-therapeutics-announces-positive-statistically-113000169.html

Pakistan probes distributors of Roche cancer drug after patients go blind

 Pakistan said on Monday it was investigating two local distributors of Swiss pharmaceutical company Roche's Avastin cancer drug after 12 diabetic patients injected with the drug went blind.

The Drug Regulatory Authority of Pakistan (DRAP) said the health authorities in Punjab, the most populous province, had launched the investigation into local use of the drug Avastin, which is licensed for use in Pakistan.

"Incidents of loss of vision in diabetic patients have been reported following treatment with Altered/Dispensed/Diluted Avastin injection," the regulator said in a statement.

Javed Akram, the province's Minister for Specialised Health, said police were questioning two men they believe to be the drug's distributors in the state.

"A high level committee has been constituted to probe the issue. A case has been registered against the distributor and his aide," Akram said.

Following the incidents, the DRAP instructed the importer to recall the suspected batches of Avastin 100mg injection, which it said had been created illegally.

"The sale/distribution of registered Avastin injection has been put on halt till verification of its quality through sampling and laboratory testing to safeguard public health," it said in a statement on its website.

On its website, Roche said Avastin was approved in more than 130 countries, including the United States, to treat several types of cancer.

"Roche strongly condemns this criminal act of counterfeiting and is doing everything in its power to cooperate with the authorities to protect patients from counterfeits," said Roche in a statement to Reuters.

"In Pakistan, the vision loss from Avastin has been identified by the authorities as a case of contamination by a third party supplier," it added.

The regulator said in its statement that in the cases concerned Avastin had been used off-label, meaning outside its approved use, to treat diabetes-related eye conditions.

Cancer drug Avastin, when used at much lower doses, is similar to eye drug Lucentis and is used in many countries as a low-cost option to treat certain blindness-causing conditions.

In its statement, Roche said: "Avastin is not approved for any use in the eye. Counterfeit medicines pose a health risk to patients because their content may be ineffective and contain harmful ingredients."

Alam Sher, Punjab's deputy drug controller who filed the police complaint against the distributors, told Reuters that some companies buy Avastin and repackage it in smaller doses to make it more affordable for patients.

A sharp drop in the value of the local currency against the U.S. dollar has inflated the price of drugs in Pakistan, many of which are either imported or based on imported ingredients. Record high inflation has also diminished the purchasing power of many people.

https://news.yahoo.com/pakistan-bans-roche-cancer-drug-092010146.html

Ascletis to start phase 3 for Recurrent Glioblastoma

  Ascletis Pharma Inc. (HKEX: 1672, "Ascletis") today announces the completion of enrollment of 120 patients in the Phase III registration clinical trial of ASC40 combined with bevacizumab for treatment of recurrent glioblastoma (rGBM). ASC40 is an oral, selective small molecule inhibitor of fatty acid synthase (FASN), a key enzyme which regulates de novo lipogenesis (DNL). ASC40 inhibits energy supply and disturbs membrane phospholipid composition of tumor cells by blocking de novo lipogenesis ^[1].

The Phase III registration clinical trial (ClinicalTrials.gov Identifier: NCT05118776) is a randomized, double-blind, placebo-controlled, multi-center clinical trial in China to evaluate progression-free survival (PFS), overall survival (OS) and safety of patients with rGBM. Approximately 180 patients will be randomized at the ratio of 1:1 to Cohort 1 (oral ASC40 tablet, once daily + Bevacizumab) and Cohort 2 (matching placebo tablet, once daily + Bevacizumab). Based on prespecified interim analysis condition, 120 patients are likely to lead sufficient events for interim analysis of PFS. The interim analysis will be conducted after 93 PFS events are observed.

Glioblastoma (GBM) is the most aggressive diffuse glioma of astrocytic lineage and is considered a grade IV glioma based on the World Health Organization (WHO) classification ^[2]. Research shows that glioblastoma (GBM) accounts for 57% of gliomas and has an incidence rate of approximately 2.85 to 4.56 per 100,000 population in China per year, suggesting approximately 40,000 to 64,000 new cases of GBM per year ^[3]. In the U.S., GBM represents 56.6% of gliomas and has an incidence rate of approximately 3.21 per 100,000 population per year ^[4]. Over 90% GBM patients will relapse after surgery, radiation and chemotherapies. Effective treatments are extremely limited for patients with rGBM.

https://www.biospace.com/article/releases/ascletis-announces-completion-of-enrollment-of-120-patients-in-the-phase-iii-clinical-trial-of-fasn-inhibitor-asc40-combined-with-bevacizumab-for-treatment-of-recurrent-glioblastoma/

Generic Drugmakers Fall Short for Poorest Patients

• Analysis examined 50 medicines from five large manufacturers
• Some essential drugs still inaccessible despite price declines

 Efforts by major generic drugmakers to expand access to essential medicines have fallen short for many of the world’s poorest patients, a new study found.

Only one of out 50 products made by five large generic drug makers — Cipla Ltd., Hikma Pharmaceuticals Plc, Sun Pharmaceutical Industries Ltd., Teva Pharmaceutical Industries Ltd. and Viatris Inc. — had a pricing strategy that took into account a patient’s ability to pay in low-to-middle income nations, according the report from the Access to Medicine Foundation.

https://www.bloomberg.com/news/articles/2023-09-26/generic-drugmakers-fall-short-for-poorest-patients-report-says