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Zenobia Therapeutics, Inspired by Warrior Queen, Focuses on Developing Drug for Parkinson’s Disease

Xconomy San Diego — 

Zenobia Therapeutics is an example of San Diego’s great biotech circle of life.

Vicki Nienaber, Zenobia’s founding president and chief scientist, started the specialized drug discovery company almost 11 months ago, after learning that Japan’s Rigaku Americas Corp. was closing ActiveSight, its San Diego-based biotech research division.

Nienaber found lab space at the La Jolla Cove Research Center, which was the original site of the Scripps Research Institute and other San Diego biotechs, such as SIBIA Neurosciences. She also has hired a few employees who were previously at San Diego-based SGX Pharmaceuticals, the oncology-focused biotech that was acquired by Eli Lilly for $64 million last August.

Nienaber also worked at SGX. She told me they recruited her from Abbott Laboratories, where she was lead inventor of a breakthrough in technology that fractures large molecules, such as proteins, into fragments, and screens the fragments for potential drug candidates after determining their molecular structure using X-ray crystallography. Nienaber joined SGX to head a similar program at the San Diego biotech, and led its strategic biology alliances with Novartis and Lilly. She says she left SGX because of the San Diego biotech’s increasing focus on developing oncology drug candidates.

Vicki Nienaber

Vicki Nienaber

“Almost all other fragment screening companies are focused on oncology,” Nienaber said. “I wanted to focus on areas that weren’t being served,” which mostly involved early stage research in diseases of the central nervous system—primarily Parkinson’s, Huntington’s and Alzheimer’s.

In recent years, Nienaber’s research has focused in particular on a protein known as “lark2,” for its LRRK2 scientific designation, which is believed to be overactive in Parkinson’s Disease patients. LRRK2 has been linked to a genetic mutation that occurs in Parkinson’s patients that causes neurons to die. One theory being advanced is that LRRK2 triggers apoptosis, a “programmed” cell death that causes cells to whither and die the way leaves fall from a tree in autumn.

Just weeks before Rigaku announced its decision to shut down ActiveSite, Nienaber learned she had been awarded a multi-phase grant for therapeutics development from the Michael J. Fox Foundation for Parkinson’s Research. ActiveSite’s closure prompted Nienaber to start her own biotech, with much of the funding coming from the grant, which was initially $350,000, because the Fox Foundation had designated the grant specifically for Nienaber. She is collaborating with Christopher Ross of Johns Hopkins University in identifying molecules that could be used to block the activity of the LRRK2 protein.

Nienaber said the biotech’s name was her husband’s idea. “He was looking at a list of the top 10 bad ass female warriors of all time,” she told me. Zenobia was a 3rd Century Syrian queen who conquered Egypt and expelled its Roman prefect. She was later defeated by Aurelian, who returned her to Rome in a military parade, as a captive in golden chains. (Zenobia was later freed and became a prominent philosopher and Roman matron.)

The biotech currently has eight employees, including part-time workers. “One of the things that’s kind of unique about us is that we have a lot of women here, although it wasn’t by design,” Nienaber said.

At this time, Nienaber said, “Venture capital money is pretty much out of the equation” for Zenobia, chiefly because the biotech is at such an early stage in its research. “So our funding is primarily from grants, contract work, collaborations and such…Foundations are realizing there’s a gap between where companies (like Zenobia) are doing research, and where they actually are getting venture capital funding.”

The company also has been generating some revenues by providing contract services. Last week, for example, Zenobia said it had successfully completed the first phase of a contract services agreement with Syntonix Pharmaceuticals, a Biogen Idec subsidiary, in which Zenobia analyzed and mapped three unique protein structures for Syntonix. “Having access to these new experimental crystal structures will allow us to more efficiently design and develop our internal drug candidates,” Syntonix’ director of chemistry Adam Mezo said in a statement last week.

Zenobia also has developed a kit to help scientists learn fragment screening techniques for use in their laboratory research. “We’ve sold seven kits, and we just announced they were available in March,” Nienaber said. “We’re just trying to bring in money, and when we get some, it just goes right into our research.”