UPDATED: Fledging biotech Syntimmune gains $10M tranche from its Series A nest egg

New York biotech Syntimmune has hit its targets in preclinical testing and been given a $10 million gift for its efforts.

The autoimmune disease specialist that works under a virtual model achieved preclinical proof of concept and hit its preclinical safety testing targets, triggering a second $10 million tranche from its $26 million Series A financing pot.

The NY-based firm, which launched only two years ago, is seeking to develop novel treatments for IgG-mediated autoimmune diseases via its first-in-class lead candidate SYNT001.

The Series A financing has been co-led by Apple Tree Partners and Baxalta Ventures, the corporate venture capital arm of soon-to-be-acquired Baxalta ($BXLT), with participation by the Partners Innovation Fund and additional investors.

The $10 million will be used to complete a Phase 1a clinical trial in healthy volunteers of SYNT001. Syntimmune said in a statement that it intends to submit an IND application to the FDA for the drug in the second half of the year so it can start human testing in 2016.

Laurence Blumberg, founder, MD and COO of Syntimmune, told FierceBiotech it's achieved a lot in just a few years: "We're a bit further along than a nascent startup, so it's a bit misleading as we look very young as we've finally just now shared something about the company--but we've made remarkable progress since 2013.

"We've raised a lot of money and we were lucky in some ways as the funding environment back in 2013/14 [during the Series A funding round] was a lot more conducive than it is today, and we were right to take advantage of that healthy financing market, because a lot of early-stage biotechs are suffering."

But why autoimmune disease? Blumberg explained: "This type of biology we've thought about for 20 years and now we've found a new and important way to use these targets. And autoimmune disease is a big, unmet need and what we're focusing on--IgG--becomes pathologic and affects people across a major range of diseases, from orphan diseases to more common conditions. The treatments out there for these diseases are suboptimal. Most are treated with plasma exchange which is a really invasive therapy that involves having blood removed and then put back into a patient.

"We know a lot of people are thinking about this target and it's at the point where it's well validated and ready for trials. We have the team to push this forward better than anyone else as we house the international thought leaders on this target." This includes the scientific founder of the firm, Laurence's brother Richard Blumberg, who is co-director of the Harvard Digestive Diseases Center and Director of the Brigham Research Institute.

But what about the future--will Blumberg seek a Big Pharma buyout? "You don't build these companies to sell them," he said. "The challenge for us however is that this is not something we can fund ourselves indefinitely. We have a strong investor base but there is a lot you can do with a drug like this--and we think it will be a major blockbuster--from ultra-orphan indications to orphan indications. And after a while it will become too big for even the best capitalized biotech to continue on independently. So we may indeed look for a partner one day if there is the opportunity and the need but right now, it's not on the cards as we have the money we need for this stage of development." He says there may also be a float in the future, but that the "breadth of the opportunity from our platform will dictate what we do."

The firm said it also has two additional, earlier-stage therapeutic programs targeting other unique aspects of FcRn biology.

FcRn is a central mediator of IgG-related immunity and part of an important pathway that enables abnormal IgG responses in a large number of clinical settings, including autoimmune disease.

There are currently no commercially available therapies designed to block IgG-FcRn interactions, which underlie diseases that affect multiple organ systems and for which there are continuing substantial medical needs, such as inflammatory bowel disease, lupus erythematosus, dermatomyositis and others.

- check out the Syntimmune release