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Aug 27, 2019
Zogenix, a pharmaceutical company focused on advancing rare disease therapies, has collaborated with the Modis therapeutics. Modis Therapeutics is a company aiming to advance in mitochondrial biology to develop disease-modifying therapies for rare genetic diseases. Modi’s lead drug MT1621 is a deoxynucleoside therapy which is in late-stage development for the treatment of inherited mitochondrial disorder including thymidine kinase 2 deficiency (TK2d). Tk2d is a form of mitochondrial DNA depletion syndrome. Clinical studies suggest that drug MT1621 has the potential to change the treatment landscape of disorders due to TK2 deficiency.
Under the terms of the transaction, Modis will receive an upfront payment of USD 250 million. The deal comprises USD 175 Million in cash and USD 75 Million in Zogenix common stock. Modis will also receive an additional amount of USD 100 Million after the FDA approval for it’s of MT1621in the U.S. and an amount of USD 50 Million upon European Medicines Agency approval. On future sales of the drug, Zogenix has agreed to pay a royalty of 5%.
A rare disease is defined as a condition that affects fewer than 200,000 people, in the U.S. There are an estimated 7,000 rare diseases. And the total number of Americans living with a rare disease is estimated at between 25-30 million. Several Pharma companies are working to advance the therapy methodologies of rare disease. Moreover, several government organizations are supporting the research by providing funds and favourable policies.
Amgen has entered into a definitive agreement with Celgene Corporation for the possession of its psoriasis and arthritis drug Otezla (apremilast) for USD 13.4 billion. Otezla is the only oral, non-biologic treatment for psoriasis and psoriatic arthritis.
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The Psoriasis therapeutic market is primarily driven by topical therapies, phototherapy, systemic medications, and biologics. However, these classic therapies have not completely met patients’ needs, especially in the most severe cases. In the current scenario, after biologics development, more effective treatments are available today than ever before. And yet most people living with psoriasis are either dissatisfied with their treatment or not using treatments appropriate to their level of disease severity, as observed by DelveInsight.
According to the National Psoriasis Foundation, approximately 8 million Americans are affected by psoriasis, resulting in a prevalence of 2– 4% in the United States, according to population-based estimates. Many Pharma and Biotech companies such as AnaptysBio, Boehringer Ingelheim, Bristol-Myers Squibb, Can-Fite BioPharma, Celgene, Eli Lilly, Dermavant Sciences, Immunic therapeutics, Pfizer, UCB Pharma, Allianz Pharma and others are developing Psoriasis potential therapies at the global level.
Breast cancer is the most common invasive cancer in women and the second leading cause of cancer death in women after lung cancer.
Recently, the Boston Children’s team conducted the first successful use of targeted CRISPR gene editing to cease the growth of a TNBC tumour in vivo in tumour-bearing mice. The new system is non-toxic and utilizes antibodies to selectively recognize cancer cells while sparing normal tissues.
The experiments showed that the CRISPR gene editing successfully managed to target the gene behind the Triple Negative Breast Cancer, Lipocalin 2. The new approach efficiently edited the 81% of the mutated genes in the tumour tissues. It helped in diminishing the growth of the tumour in mice by 77% with no toxic effects to neighbour normal tissues.
Triple-Negative Breast Cancer (TNBC) is defined as the heterogeneous breast cancer phenotype where the estrogen and progesterone receptor are negative, as assessed by immunohistochemistry (IHC), and there is a lack of overexpression of HER2, as assessed by immunohistochemistry (IHC), or the absence of its gene amplification, as assessed by fluorescence in situ hybridization technique.
The total Triple Negative Breast Cancer incident cases in the US were found to be 30,285 in 2016. As per the country analysis, China has the highest number of cases, followed by the US and other European EU- 5 countries. DelveInsight Triple-negative Breast Cancer forecasts say that the TNBC Therapeutic market in G8 Countries is expected to increase by 2027, at a CAGR of 18.9% for the study period 2016-2027. The United States accounted for 33% of the total sales in 2016 and is expected to occupy 37% of the total market by 2027.
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