Compared to its relative prevalence, Celiac disease is the most common disease that has zero treatments on the market. To make matters worse, only four companies are developing drugs for treatment of Celiac, and until a few days ago, this entire field has been flying under the radar.
This changed on May 15, when AbbVie (ABBV) announced it will pay $70 million to secure rights to an early-stage therapy for celiac disease developed by Alvine Pharmaceuticals. Under the terms of the agreement, AbbVie holds an exclusive option to either acquire the drug (named ALV003) or equity in Alvine upon successful completion of a Phase 2b study that will be conducted by the latter. Should AbbVie exercise its option, Alvine would receive additional undisclosed payments.
Celiac disease is an autoimmune disorder that affects the small intestine of people with a genetic predisposition. The autoimmune reaction is triggered by gluten proteins (gliadins) that are found in common grains, such as wheat, barley, and rye. If untreated, Celiac-related inflammation can result in severe damage of the digestive tract tissue and can lead to anemia, infertility, osteoporosis, neurological dysfunction, and cancer.
Today, treatment means only one thing - a life-long gluten-free diet. However, the 6 million celiac patients in the U.S. and Europe are having a hard time maintaining a 100% gluten-free diet, as accidental gluten cross-contamination cannot be completely avoided. This results in a significant unmet need for non-dietary therapies to treat Celiac, and consequently, a big and untapped market - according to a GlobalData report, Celiac disease therapeutics in the seven major markets would generate revenues of more than $650 million by 2019.
Alvine's AVL003 contains two recombinant enzymes that break down gluten in the intestines. Following a six-week phase IIa study, Alvine reported that AVL003 reduced the gliadin-related intestinal inflammation among Celiac patients that consumed gluten. AbbVie's deal makes it the first big pharma player to join the Celiac therapeutics field, and according to the sum it is willing to risk on a drug that completed a single efficacy clinical study, it looks like it means business.
Besides Alvine, three other companies are developing celiac disease therapies. Alba Therapeutics, with a drug that inhibits the gut's permeability to gluten, and ImmusanT, which develops a vaccine for induction of gluten tolerance, are both private companies. The third company is BioLine Rx (BLRX), whose Celiac targeting drug, BL-7010, is expected to commence clinical development later this year.
BL-7010 is a non-absorbable, high molecular weight polymer that binds gliadins in the gastrointestinal tract and neutralizes their harmful effect in Celiac patients. In-vivo data for this drug includes high specificity to gliadins, prevention of pathological damage and reduction of intestinal inflammation, with no toxic effects. According to BLRX, results from a pilot clinical study with BL-7010 are expected mid-2014. Considering the current low price of BioLine's stock, the very short clinical trials with Celiac drugs and the very few competitors in the Celiac therapeutics field, BL-7010 could very well be BioLine's joker card.
As of now, the research and development of Celiac therapeutics is being performed solely by small biotechs. AbbVie's recent deal, that included a considerable upfront payment for a drug that completed only one clinical efficacy study, implies that big partnering opportunities are available for the other few companies in the race for a Celiac drug.