December 2, 2015
By Mark Terry, BioSpace.com Breaking News Staff
Semma Therapeutics focuses on diabetes research that uses stem cells in an effort to cure the disease. The company is built on research that came out of Doug Melton’s laboratory at the Harvard Stem Cell Institute, where Melton and his team, including Semma’s scientific co-founder Felicia Pagliuca, were able to generate beta cells in a petri dish. These stem cell-derived beta (Scbeta) cells were tested in vitro and in vivo to compare them to beta cells within human pancreatic islet cells, where insulin is produced. The Scbeta cells responded in a way very similar to the key functional feature of endogenous human beta cells, essentially mimicking the activity of normal human beta cells.
Early preclinical work has shown that when the human Scbeta cells are transplanted into mice, they show high levels of human insulin in their blood after a glucose challenge. In animal models, they have been shown to effectively control diabetes.
The company’s focus is on developing a delivery system and method of creating transplantable stem cells as a possible cure for Type 1 diabetes.
For the last twenty years or so, Melton’s research with stem cells has focused on Type 1 diabetes. The reason behind that, said Robert Millman, the company’s co-founder and chief executive officer, is because two of Melton’s children were diagnosed with Type 1 diabetes 18 years ago. “We felt that since Doug has focused his whole work on his two children, Sam and Emma, that the company should become Semma, Sam and Emma,” Millman said in an exclusive interview with BioSpace .
Company Leadership
Robert Millman—chief executive officer and co-founder of Semma Therapeutics. He has been involved in the formation and acted as the intellectual property (IP) counsel for several companies, including Epizyme , iPierian, and Verastem . Most recently Millman founded and led CoStim Pharmaceuticals as its first president.
Doug Melton—scientific founder and a board observer. He is the Xander University Professor at Harvard and a Howard Hughes Medical Institute investigator. He is also co-chair of the Department of Stem Cell and Regenerative Biology and the Harvard Stem Cell Institute.
Walter Blättler—vice president of manufacturing. Prior to joining Semma, Blättler was vice president of development at CoStim Pharmaceuticals. Before that he was executive vice president of Science and Technology at ImmunoGen .
Felicia Pagliuca—scientific co-founder and vice president of technology and corporate development. Pagliuca was a postdoctoral fellow in Doug Melton’s laboratory, where she managed collaborations and developed strategies for generating insulin-producing cells. She is an expert in stem cell biology and diabetes and one of the inventors of Semma Therapeutics’ key technologies.
Moses Goddard—chief medical officer. He is currently an associate professor of surgery at Brown University. He was most recently co-founder and chief executive officer of CytoSolv.
Christopher Thanos—vice president of delivery.
Jeff Imbaro—vice president of operations and alliances.
David Cordo—chief financial officer.
Company Financing
On March 18, 2015, Semma announced it had closed on a $44 million Series A financing round made up mostly of equity financing and strategic funding. The financing was led by MPM Capital, with Fidelity Biosciences, ARCH Venture Partners, and Medtronic participating. The company intends to use the money to advance its program through clinical proof-of-concept in humans.
Pipeline
At the moment, the company’s focus is on developing an encapsulating device that would allow for the functioning beta cells to be implanted while protecting them from the body’s own immune system. It’s looking at a couple different routes, both internal and external, and about one third of the Series A funding will go toward developing or buying an appropriate device.
Market Competition
A lot of big players in the diabetes market such as Sanofi and MannKind are undoubtedly keeping an eye on what Semma is doing. Other companies also working in this area include ViaCyte and BetaLogics.
ViaCyte, located in San Diego, is focused on the differentiation of stem cells into pancreatic beta cell precursors (PEC-01) and uses a subcutaneous implant in a retrievable and immune-protective encapsulation device, Encaptra. It is currently conducting a Phase I/II trial called STEP ONE.
BetaLogics is a division of Janssen Research & Development, a Johnson & Johnson company. Based in New Jersey, BetaLogics is also focused on transformational stem cell therapy for diabetes. And like ViaCyte and Semma, the focus is on building an implantable device that incorporates pancreatic precursor cells.
Dollars and Deals
In addition to its Series A financing round, Semma signed an undisclosed agreement with Novartis Pharmaceuticals . It also recently acquired Rhode Island company Cytosolv, which is a drug delivery technology company. The company’s headquarters in Boston focuses on the beta sell manufacturing and purification issues. Its Rhode Island location, formerly Cytosolv’s headquarters, now dubbed “Semma South,” focuses on the delivery system. “The founders of Cytosolv were trying to do similar things with porcine islets, so they have a lot of experience with cell delivery systems,” Millman said.
Millman also noted that the company’s relationship with Medtronic, “which provides great access,” he said, “to and new knowledge in the delivery area.”
Of its relationship with Novartis, much of it remains undisclosed, but Millman said, “They are a strategic partner. Semma looks to them to be one of the pharmaceutical companies that made a commitment to gene therapy, and therefore it was a very good partner of choice.”
Semma has also created relationships with the JDRF Diabetes Foundation and the Diabetes Research Institute at the University of Miami as a way of partnering with organizations that have a vested interest in the technology and access to delivery solutions.
What to Look For
The industry will be waiting for news on Semma’s encapsulation device, which is the key to utilizing its stem cells. It’s a fairly tough nut to crack, and there is some expectations that there will be potential strategic deals to move that along. Alternatively, the company is also working internally on encapsulation, so potential news about an innovative development might be pending as well.
“If all goes well,” said Millman, “you’ll see announcements of agreements with companies that have delivery systems. You’ll see fundraising activities…. We’re up to 27 people and we have two sites focused on separate aspects of the company. You’ll see announcements of some of our partnerships, increases in our staff a little bit more, and filling out our senior team, as well as our scientific advisory board.”
Millman said that they plan to test a lot of third-party devices for efficacy with the company’s cells, as well as continuing to work on developing their own. “Everyone who has a solution,” he said, “please talk to us.”
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