Discovering a novel class of drug and getting a product from inception to market could be viewed as a life’s work to be rewarded with a gentle-paced retirement.
But for biochemist Kunwar Shailubhai, it is the start point to build a new company and wrestle more therapies from the laboratory through the energy-sapping regulatory phase to the patient.
At 58 years old, he is still shuttling between the US and the UK as the London-based company, Tiziana Life Sciences, where he is an executive board member, gathers momentum with a promising pipeline across multiple programmes for oncology and immunology.
Pioneering research
He rose to acclaim in the late 1990s when he unpicked gut disorder pathways, leading to the discovery of a master regulator of gut physiology which opened the door to treatments for colon cancer and GI inflammation. His pioneering research culminated in the drug, Trulance, which has received FDA approval and is being used to treat irritable bowel syndrome and chronic constipation.
That success is underscored by 22 issued patents, a slew of pending patent applications and 40 research publications while also serving as chief scientific officer of Synergy Pharmaceuticals. He has also served as a group leader at Monsanto Co, a senior staff fellow at the National Institutes of Health and as an assistant professor at the University of Maryland.
“Now that our first drug has FDA approval, maybe the pace has slowed a bit,” he says. “But I still have 10 to 15 years of a productive career so I am continuing with renewed motivation. I have 30 years’ experience as a biochemist and this is my passion. I don’t want to stop now.
“I’m very excited about what Tiziana can achieve. I’ve done my homework and know what I’m doing. It is motivating and keeps me focused.”
Novel drug candidates
Tiziana, a clinical stage biotech company with offices in London and New York and a research staff of seven at a facility in Philadelphia, focuses on the discovery and development of novel drug candidates. Its fully human engineered CD3 antibody Foralumab has reached phase II with potential for a wide range of autoimmune and inflammatory diseases such as non-alcoholic steatohepatitis (NASH), primary biliary cholangitis, ulcerative colitis, type 1 diabetes and inflammatory bowel disease.
If the science is intriguing, the company’s set-up is also drawing attention.
It is a spin-off collaboration of proven biotech winners. Its executive triumvirate has an impressive track record. Shailubhai is joined by entrepreneur Gabriele Cerrone and Gary Jacob who were influential in building the the NASDAQ-quoted Synergy Pharmaceuticals. Dr Jacob remains Synergy’s chairman and Shailubhai its chief executive officer but Tiziana is now generating its own heat.
Cerrone, who has a track record of seeding new enterprises, co-founded Synergy and also orchestrated the sale of Inhibitex to Bristol-Myers Squibb for $2.5bn.
“What Gabrielle can do, I can’t. And what I can do, he can’t,” adds Shailubhai, who was a scientific adviser on the Inhibitex sale. “We have known each other a long time and it is a collaboration of talents. He is brilliant at identifying technologies, cutting business deals and raising money. I come from the science part with experience on which technologies to target to build a company.
“Because we have known each other so long, we have built up that trust and close connection and friendship that is very important to success. There are millions of biotech companies out there but, sadly, the majority of them fail. The main reason, I believe, is because they are not focused enough; they try to do everything under the sun and lose sight of their principal aim. They become diluted. We take calculated risks, you have to, but there is no point in trying to do everything.
“I have one piece of advice for biotech entrepreneurs and that is focus, focus, focus.”
Template for success
The big question is whether Tiziana is building a ‘lean and keen’ template for success that could provide lessons for an industry on laser alert for new business models. The answer may not come until it has proven that its first burst of activity can be sustained or repeated.
For the moment, the mood music is bright. Research into its lead compound Milciclib, a small molecule inhibitor of cyclin dependent kinases (CDKs), has just been published in the journal Cancer Chemotherapy and Pharmacology, which concluded its treatment regimen showed ‘encouraging clinical benefit’.
That promise needs to be backed by financial muscle and, with a burn rate of £200,000 a month and a £10m fund needed for the next set of clinical trials, 45-year-old Cerrone’s investment skills will be tested.
Clinical trials
The company is moving cautiously and set its clinical trials in Israel, Italy, Turkey and Greece. “We decided on trials in Europe because they are easier to organise and are cost-effective. After that we can then move to the US,” says Shailubhai. “Big pharma has deep pockets and can afford to consider a number of approaches and try them to see if they work. Tiziana cannot do that as we don’t have such deep pockets. We have to think through all of the possibilities and come up with an approach which is the best to move forward.
“The first trial was with 16 cancer patients who were refractory to the treatment with existing drugs. The fact these patients responded well and the treatment was safe gave me a lot of comfort that we have something with a lot of potential and we could see benefits in a lot of cancers as well. We are also building a strong intellectual property estate by filing patents to cover other oncology indications.
“This fits well with our mission to tackle unmet needs. Autoimmune conditions are increasing and diabetes is at epidemic levels.
A lot of NASH patients also have these conditions so we want to look at and understand the underlying causes to see what else we can come up with there.”
Database of diseases
The company is also shrewdly operating in areas predicted for huge growth. The NASH market is expected to be worth $20bm by 2025 and financial brokers recently valued Tiziana as worth above $500m.
Tiziana may be lean but has its own R&D centre so that its laboratory discoveries remain under tight control and its IP can be protected.
But the company did hit controversy last year when it paid £217,000 for a biobank of blood samples from 13,000 residents living in a province of Sardinia with a high proportion of centenarians – almost 50 times greater than the UK rate.
Tiziana says the database could hold the keys to new drugs and diagnostic tests but local politicians and campaigners say the purchase was unethical because the people did not consent to their data, collected by local physicians, being used by a for-profit firm.
“If we can isolate the DNA from these samples and do the sequencing, then we would have a database which would be a gold mine to dig into different diseases and it could be very useful,” says Shailubhai. “The scientific rationale is very powerful.”
He shows no sign of slowing down or losing his enthusiasm, adding: “I tell people that the biotech industry is a roller coaster ride. If you are able to go through it, there can be huge rewards. But you have to hold on for the whole ride.”