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Axcella Health Granted Broad Patent Covering EMM Compositions

26 Nov Axcella Health Granted Broad Patent Covering EMM Compositions

Axcella Health (NASDAQ: AXLA), a biotechnology Company focussed on leveraging the power of Endogenous Metabolic Modulators, announced the issuance of a third EMM-related patent covering an array of EMM compositions, including AXA1125 and AXA1957, the company’s product candidates for non-alcoholic steatohepatitis (NASH). The patent broadens the coverage for composition of matter claims, as per a Company statement.

The Company’s research is centred around Endogenous Metabolic Modulators (EMM) that has the potential to regulate human metabolism and have a profound impact on health. Axcella’s proprietary human‑focused AXA Development Platform, is a first-in-class integrated platform that has the ability to provide advanced analytics of metabolism regulation and dysregualtion, correlate data with its proprietary database, namely AxcellaDB and perform predictive combinatorial drug metabolism and pharmacokinetics analysis. In addition the Company’s proprietary primary human cell system assists in evaluating a design pathway from the data generated using the platform. The candidates thus generated aim at restoring the body’s homeostasis, critical for the optimal functioning and health of the human body.

Paul F. Fehlner, J.D., Ph.D., Senior Vice President and Chief Intellectual Property Officer of Axcella commented,

“This issuance follows our two prior patents related to specific EMM compositions and methods of treating liver dysfunction and liver disease. We believe our growing patent portfolio reinforces Axcella’s leadership position in utilizing EMMs to address metabolic dysregulation while also underscoring the pioneering nature of the company’s AXA Design Platform.”

AXA1125 and AXA1957 are two AXA candidates, developed using novel compositions of EMM’s ,which have the ability to regulate metabolic, inflammatory and fibrotic pathways implicated in liver health and the development of NASH and non-alcoholic fatty liver disease (NAFLD). The candidates are currently being evaluated in non–IND clinical study to study their safety tolerability and physiology in more than 100 adult subjects with NAFLD. The Company intends to file for an IND in the near future for these two candidates.

Axcella Health (NASDAQ: AXLA)

Market Cap: $79.28M; Current Share Price: 3.43 USDChart
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Nonalcoholic Steatohepatitis (NASH) is a form of Non-Alcoholic Fatty Liver Disease (NAFLD) and is characterized by the buildup of FAT in the liver. This condition is marked by hepatitis, inflammation, cell damage and fat deposits in the liver. These fat deposits can cause fibrosis of the liver, in turn leading to liver cancer or cirrhosis.

According to an estimate only 20 percent of the people suffering from NAFLD have NASH, while the rest only have a simple fatty liver. While nearly 40 percent in the U.S are afflicted with NAFLD, around 3 to 4 percent have NASH. The American Liver foundation estimates that over 100 million people suffer from NAFLD in the U.S alone.

Though the exact cause of the disease is still unknown, NASH often develops from underlying conditions such as hepatitis, inflammationand type 2 diabetes, and can affect people of any age. Individuals who have insulin resistance, high triglyceride levels or abnormal cholesterol levels, hypertension and uncontrolled blood glucose are at an increased risk of developing this condition. NASH also increases the chances of developing cardio vascular anomalies and can lead to death from liver-related causes.

The diagnosis usually involves blood tests, use of imaging techniques such as ultrasound, CT scans and MRI, and a liver biopsy. Losing weight through a healthy diet and exercise can help reduce the fat in the liver and is usually the recommended course of action. Currently there are no approved therapies for this condition, with treatment limited to alleviating the symptoms of the condition.

According to a report by Reports and Data, the Global NASH market will be worth over USD 13.38 Billion by 2026. The healthcare costs associated with this disease are likely to reach USD 18 billion by 2030 from USD 5 billion now, if the disease is left untreated.

The Company has developed a robust pipeline of candidates using its proprietary human focused AXA Development Platform, which are a novel composition of EMMs engineered in distinct ratios, and capable of regulating multiple metabolic functions. Axcella’s pipeline consists of candidates targeting liver, muscle, and blood disorders. The AXA candidates typically exhibit the following characteristics such as being multifactorial, setting a safety precedent, possessing combinability potential, orally administrable, faster designing and scalability to name a few.

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AXA1665, its lead candidate is indicated for the treatment of Overt Hepatic Encephalopathy (OHE), a form of metabolic encephalopathy, which is a common complication of cirrhosis. The candidate can tackle sarcopenia and support the ammonia detoxification process simultaneously, resulting in reduced mortality, improving outcomes, reduction in cirrhosis complications and maintaining remission of HE. The candidate can target multiple metabolic pathways related to key organ systems such as (liver, muscle and gut) and restore plasma amino acid balance, ammonia handling and muscle wasting. The Company is evaluating the candidate in a non–IND, IRB-Approved Clinical Study, the results of which are expected to be released in H1 of 2020.

Axcella is also pursuing the possibility of developing an AXA muscle candidate indicated for the treatment of disuse-related muscle atrophy, including total knee arthroplasty, total hip arthroplasty, hip fracture related myopenia, rotator cuff repairs or injury, and other musculoskeletal atrophy or injuries, which has completed two Non-IND IRB Approved Clinical Studies. In addition, the Company will be initiating a Non-IND, IRB-Approved Clinical Study for its AXA blood candidate in H2, 2019.

Risk Assessment  

  • The Company is targeting to become an industry leader in Endogenous Metabolic Modulators based therapeutics, and its innovative approach of using endogenous metabolic modulators to reprogram dysregulated signalling pathways, is marking a shift towards a more holistic approach to addressing disorders that have an underlying metabolic component including those related to cardiac as well as renal diseases.
  • Axcella recently entered into a strategic collaboration with CYTOO for informing the development of new AXA Candidates in skeletal muscle growth and function by leveraging CYTOO’s MyoScreenTM muscle-on-a-plate platform. CYTOO’s MyoScreen muscle-on-a-plate platform uses patients’ primary cells to establish culture conditions that mimic the morphology, contractile and metabolic functions of human muscle in vivo as per a company statement. Collaborations and partnerships are crucial for the survival of clinical stage companies as they not only help improve processes but also provide research, marketing and regulatory support, which is essential for developing a product.
  • Clinical trials are fraught with risk, the Company has so far evaluated its candidates only in non-IND, IRB-approved clinical studies and intends to initiate crucial phase 2b/3 trials in 2020 and 2021 in Overt Hepatic Encephalopathy (OHE) and NASH, along with possible candidates for acute atrophy and sickle cell disease. In addition it has also completed patient enrolment in AXA1125/AXA1957 by recruiting more than 100 adult subjects.
  • As per the Company’s Q3, 2019 financial results, it had $105.4 million in Cash and cash equivalents as of September 30, 2019, including proceeds from an IPO issued in May 2019. The Company believes that the funds will be sufficient to meet the company’s operating needs through the second quarter of 2021, including its ongoing clinical trials.
  • Disclosure: I/we have no positions in any stocks mentioned, and no plans to initiate any positions within the next 72 hours.

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