Rare diseases are called Erythropoietic porphyria (EP) appear in the blood, but the patient can feel them most on the skin. Light sensitivity is the main symptom of the deficiency of these enzymes that affect red blood cells. Sunlight can cause a tingling sensation, or worse, a burning sensation.The only treatment approved by the FDA is Subcutaneous implant This will increase the production of skin protective pigments.
EP stems from the lack of a key enzyme that produces heme, which is an iron-containing molecule and a part of hemoglobin in the blood. Without this enzyme, metabolites will accumulate in the body, leading to increased light sensitivity. Chief Medical Officer Will Savage explained that Disc Medicine aims to treat this rare disease by inhibiting glycine, which is an amino acid and an important part of heme. This method aims to inhibit the accumulation of toxic metabolites.
“If all this works, this will be the first disease modification therapy that really touches the root cause of the disease,” Savage said.
Disc Medicine is looking ahead to Phase 2 testing of EP drugs (called bitopertin) and another drug that is being developed for different blood diseases.In order to implement these plans, a biotech company based in Cambridge, Massachusetts has Raised $90 millionThe Series B financing announced on Thursday was led by OrbiMed.
Bitopertin is the newest member of the Disc Medicine pipeline, Licensed From Roche in May. The pharmaceutical giant studied the molecule as a way to control glycine in the brain for potential applications in the treatment of neurological diseases. Disc Medicine CEO John Quisel said that the study was unsuccessful, but clinical data did show evidence that the drug inhibits heme synthesis. This effect is particularly pronounced in newly formed red blood cells. The potential for applying this molecule to EP led to a licensing agreement in May where Disc Medicine paid an undisclosed amount in advance and promised to pay more than $200 million in milestone payments related to the progress of the project.
Having completed the first phase of testing at Roche, bitopertin has become the most advanced program measured by Disc Medicine in its development phase. The company’s other clinical phase project, DISC-0974, began phase one testing in July as a potential treatment for chronic anemia caused by inflammation. Quisel said this anemia is caused by elevated levels of hepcidin, a protein that regulates iron and causes the body to increase iron reserves.
DISC-0974, one of a series of antibodies that Disc Medicine added to its product line in 2019 Dealing with AbbVie, Designed to target hemojuvenin, a protein that plays a role in iron absorption. Quisel says that by targeting and down-regulating hemojuvenin, the drug aims to release iron from storage in the body and restore red blood cell production. The main indication is severe anemia associated with myelofibrosis. But Savage pointed out that Disc Medicine for anemia is designed to treat primary diseases that are secondary to inflammation. The drug may have potential applications in the treatment of anemia caused by other diseases, such as chronic kidney disease, autoimmune disease and chronic heart failure.
Current treatments for anemia include blood transfusions and erythropoietin stimulators, which are injections of synthetic hormones to stimulate red blood cell production. Savage said these options are not ideal for patients.
“0974 is using the body’s own mechanisms to correct this problem [anemia], This is a particularly elegant way to solve the disease,” he said.
Data from the first phase of the DISC-0974 study is expected to be available in 2022. Quisel said that after demonstrating safety and determining the optimal dose, the plan will quickly begin testing antibodies in other indications. The second phase of bitopertin research is expected to begin in the first half of next year. Myelofibrosis and porphyria are the main disease targets of DISC-0974 and bitopertin, respectively. Quisel said that if these drugs are approved, Disc Medicine’s goal is to continue to develop these drugs and commercialize them for these indications. For larger indications, the company may consider establishing development partnerships with larger companies. Quisel estimates that the new cash will support the company by the end of 2023. At that time, Quisel expects that the company will have proof-of-concept data for these two molecules.
The third program, matriptase-2 inhibitors, aims to increase hepcidin and reduce iron in the blood. This method can be applied to diseases where the bone marrow produces too many red blood cells. This internally developed program is in the pre-clinical development stage.
Joining OrbiMed in the Series B financing are Arix Bioscience, Janus Henderson Investors, 5AM Ventures, Rock Springs Capital, Nantahala Capital Management, Willett Advisors and Alexandria Venture Investments. Earlier investors also participated, including Atlas Venture, Novo Holdings and Access Biotechnology.
CD medical photos
