Orphazyme (ORPH) is a biopharmaceutical company, which engages in the development of treatment for patients living with life-threatening and debilitating rare diseases. It provides Arimoclomol program and molecular entities program.
Arimoclomol is an investigational drug candidate that amplifies the production of heat shock proteins (HSPs). HSPs can rescue defective misfolded proteins, clear protein aggregates, and improve the function of lysosomes. Arimoclomol is administered orally, and has now been studied in 10 phase 1, four phase 2 and three pivotal phase 2/3 trials. Arimoclomol is in clinical development for NPC and Gaucher disease. Arimoclomol has received orphan drug designation (ODD) for NPC in the US and EU. Arimoclomol has received fast-track designation (FTD) from the U.S. Food and Drug Administration (FDA) for NPC. In addition, arimoclomol has received breakthrough therapy designation (BTD) and rare-pediatric disease designation (RPDD) from the FDA for NPC. Arimoclomol is an investigational treatment and has not been approved by the FDA.
Amyotrophic lateral sclerosis (ALS) is a rare, rapidly progressive, and always fatal neurodegenerative disease. Protein misfolding and aggregation in motor neurons are hypothesized to be important contributors to the disease process, which ultimately leads to paralysis of skeletal muscles as well as the muscles that enable breathing. The patient population in Europe and the United States is estimated to be approximately 50,000 patients. Currently, there are only limited treatment options available. Arimoclomol has been granted Orphan Drug Designation (EU and US) for the treatment of ALS.
Some investors are hinting that it might be up 900% due to a short squeeze. The company has 34.95M outstanding shares with a float of 24.59M shares and only 65k volume in the last 3 months. A very low float can cause the stock to skyrocket.
Now, you’re asking what caused people to buy if there’s no news?
Well, one poster on the Yahoo Finance Board posted;
“In 2011, CytRx sold the rights to arimoclomol to Orphazyme A/S (formerly Orphazyme ApS) in exchange for a one-time, upfront payment of $150,000 (USD) and the right to receive up to a total of $120 million (USD) in milestone payments upon the achievement of certain pre-specified regulatory and business milestones, as well as royalty payments based on a specified percentage of any net sales of products derived from arimoclomol. Orphazyme, a public company trading on the Nasdaq Copenhagen exchange, is testing arimoclomol in three additional indications beyond ALS, including Niemann-Pick disease Type C (NPC), Gaucher disease and sporadic Inclusion Body Myositis (sIBM). Orphazyme anticipates announcing top-line results for their pivotal Phase 2/3 trial in NPC during 2018, and if positive, expects to submit a New Drug Application (NDA) to the U.S. Food and Drug Administration and a Marketing Authorization Application (MAA) to the Europeans Medicines Agency (EMA) during the second half of 2019”
Yahoo Finance Board User
Although its reported that, They failed their Phase 1 for the treatment of ALS, its reported that the PDUFA date for Niemann-Pick is June 17, 2023. They are pivoting towards Niemann-Pick.
In January 2019, the medication narrowly missed its first primary objective in a 50-person Phase II/III NPC research study. The severity of the condition was reduced by 74%, and the p-value was 0.0506. Disease development slowed in individuals over the age of 4 and in children who had been receiving an Actelion medicine, which is sometimes prescribed for the disease, according to two pre-specified subgroup analyses. However, the business did not reveal any data for this round of ALS trials, putting the public in the dark about how it performed in the experiment.
The company said in a statement that topline data will be provided during the virtual European Network to Cure ALS meeting from May 12-14, while detailed data will be released later in the year.
