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This Purdue University grad aims to find drugs for rare genetic disorders

Sindhu Kashyap
27th Apr 2017
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Aten Porus is a biotechnology startup that focuses on drug discovery for rare and orphan diseases.

Aditya Kulkarni, a PhD in chemical biology and drug delivery systems from Purdue University, felt that there was a distinct need for a startup in the drug discovery space. In the course of his PhD, Aditya, who was working at an NPC patient foundation at Notre Dame, Indiana, close to Purdue University, was exposed to Niemann-Pick Type C disorder, an extremely rare disorder with approximately 2,500 patients globally.

As soon as he got back to India, he, along with his friend Arun B. Papaiah, started studying the rare disease landscape and met with patient foundations such as the Organisation for Rare Diseases India and Lysosomal Storage Disease Foundation, India.

“This was when we realised that there was a huge, unmet medical need in the area of rare diseases, and decided that it was an important issue that needed to be addressed so as to develop affordable medicines and make them accessible for patients with these diseases in India. We then decided to focus on this area, starting Aten Porus Lifesciences in Bengaluru,” says Aditya.

Founding team @ Aten Porus

Aten Porus is a biotechnology startup that focuses on drug discovery for rare and orphan diseases, which, Aditya adds, number close to 7,000. Orphan diseases are those that afflict fewer than 200,000 people globally, and there is often a lack of effective medication to address such diseases. Aditya’s background in drug delivery systems and gene therapy during his PhD and experience in polymer chemistry were useful in developing the platform technology.

By studying the current issues faced by existing treatments or treatments under development, Aditya and Arun realised that the background in drug delivery could be exploited for making superior or innovative drugs for life-threatening rare diseases. As a next step, they began approaching angel investors and VC firms to raise seed capital.

Setting up the startup and team

“Most traditional VCs in India declined investment considering drug discovery extremely risky. But we then met Srinivasan Namala (CEO, Porus Labs) and pitched our idea to him, and he agreed immediately to invest and come in as a partner, since he always wanted to be a part of drug discovery. The conversation about our plans for APL, including his decision to invest in APL, all happened in one meeting, over a cup of coffee,” says Aditya.

Now, the leadership team comprises of Arun, Aditya, and Srinivasan. The team consists of seven scientists working at APL from various backgrounds, such as Organic Chemistry, Analytical Chemistry, and Cell Biology. “For our first few hires, we returned to our Alma Mater, Christ University, and hired members of the core team from there,” says Aditya.

Excess accumulation of cholesterol and lipids in the body is implicated in various life-threatening rare genetic disorders, like Niemann-Pick Type C disorder, and Familial Hypercholesterolemia. APL has been working on a platform technology consisting of a family of drugs that can regulate storage of excess cholesterol and lipids in specific organs of interest.

India is host to an estimated 70 million patients suffering from rare genetic disorders, and there are close to 350 million such patients globally. Despite a significant unmet medical need, less than five percent of these diseases have a clinically approved treatment, and the available treatment options are prohibitively expensive, with treatment costs ranging between Rs 50 lakh and Rs 1.5 crore for patients with rare diseases.

The workings

One of the challenges faced by the team early on was the limited awareness of rare diseases in India, and hence the hesitation from both investors and funding agencies alike. The drugs being developed by APL can specifically enter the target organ and remove excess accumulated cholesterol. Diseases such as Niemann-Pick Type C have extremely high deposits of cholesterol that cannot be reduced by conventional therapeutic alternatives.

The cholesterol deposits result in severe damage to the brain, liver, and spleen, and are fatal to the patients, with most not surviving beyond 18 to 20 years of age. Using this underlying strategy, APL is applying its technology platform to other rare lipid storage or lipid-linked disorders too, such as Focal Segmental Glomerulosclerosis and Alport Syndrome, both rare kidney disorders.

There are very few companies focused on drug discovery for rare diseases globally, and amongst them, most employ a biological approach to targeting the diseases, such as gene therapy and protein/enzyme replacement therapy. Biotechnology, genetics, and so on are not areas about which one hears a lot in the startup space.

Be it Kiran Mazumdar Shaw's work or a one-off biotech company getting funded, the news pieces are few and far between. Among the few that have ventured into this space are Sequoia-backed Medgenome and Rajan Anandan’s MapMyGenome, another genetics startup that has received funding.

“We our using a materials chemistry/drug delivery approach to rare diseases. Usually, drug delivery and materials chemistry are commonly employed for oncology drugs, or even infectious diseases. However, we are using these strategies for lethal and rare genetic disorders,” says Aditya. ATL is a pure-play R&D company, and so far, all the investments have gone into developing and validating their technology platform.

The team claims to have identified promising drug candidates for two extremely rare diseases, both with a high unmet medical need and without a clinically approved treatment – Niemann-Pick Type C disorder and Focal Segmental Glomerulosclerosis.

“Our revenue will come from the licensing of our technology to pharma or biotech partners who would want to acquire our drug platform and carry out clinical development for launching in the market. We are currently in early talks with potential pharma partners for licensing,” says Aditya.

Grants and future

APL is supported by grants from the Biotechnology Industry Research Assistance Council (BIRAC), Government of India. Of this, approximately Rs 50 lakh is from BIG Grant and approximately another Rs 50 lakh is from SBIRI. Recently, APL received an additional round of funding of $1.4 million from a US-based investor to take the candidates through preclinical testing.

With the investment, the company has spun off its US arm, known as Oraxion Therapeutics and based out of New York, to solely focus on preclinical development of this technology platform. APL is also looking at applying for orphan drug designation for its drug for Niemann-Pick Type C disorder.

The revenue model is based on licensing or partnering of their drug platform to global pharma or biotech companies who would carry out further clinical development. The company would then make its revenue through upfront payments, milestone payments, and royalties, as is the norm in the biotech startup world.

APL is broadening its pipeline by testing these drugs in rare kidney disorders such as Alport Syndrome, again an orphan disease without a clinically approved treatment available.

“With close to 70 million patients in India, 30 million in the US, and 350 million globally, the global orphan drug market is expected to reach about $ 200 billion by 2020. The company has initiated conversations with global pharma and biotech companies for joint clinical development of the drug for Niemann-Pick as well as the extension of the platform to other rare lipid storage disorders,” says Aditya.

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