Lab Rats series: A Pune biotech startup’s new approach to vitiligo cure

Mumbai: Every day, specialized cells in the skin produce melanin, the pigment that gives skin its colour. In people with vitiligo, a trigger can cause the cells to send out a distress signal. The immune system turns against these cells, destroying them and leaving behind patches of skin that lose their pigment.

Existing cures involve suppressing the immune system or even targeting the distress signal pathway.

The biggest piece of the puzzle is: what triggers the cell to release a distress signal in the first place?

Pune-based Ahammune Biosciences believes the answer could lie in intervening before the immune system launches its attack.

For chief executive Parul Ganju, an effective treatment must do more than halt the disease. It should stop the patches from spreading, restore pigmentation and ensure that the colour does not fade again.

After a decade of research, Ahammune has advanced its lead candidate, AB1001, to phase 2 clinical trials, with a team of just 10 and $8-9 million raised through grants and equity funding.

“That’s what has helped us in the last ten years to take the molecule into phase 2, at a fraction of the cost that any company outside India would spend,” Ganju said.

In drug development, phase 1 trials are focused primarily on establishing safety, tolerability and dose range in healthy volunteers, before they can progress to phase 2, tested in a larger group of patients to see how it works while still remaining safe.

While there has been a rising interest in Indian biotech funding, Ganju characterized it as investors “still testing the waters…Because there's still less understanding of how commercialization is going to take place in the long term.”

What does its drug do?

“We identified a pathway that leads to the trigger of this autoimmunity in the skin. And then we designed molecules to target this pathway,” said Ganju.

Its lead molecule, AB1001, is a first-in-class drug, meaning a completely new mechanism of action to treat a disease. It has already progressed further than most other Indian discovery-stage molecules and is currently in phase 2 clinical trials across the country.

In drug development, phase 1 trials are focused primarily on establishing safety, tolerability and dose range in healthy volunteers, before they can progress to phase 2, tested in a larger group of patients to see how it works while still remaining safe.

Existing treatments include topical corticosteroid creams, oral steroids, as well as a new class of drugs called JAK inhibitors, which target the cellular messaging pathways after the autoimmune response has been triggered.

Steroidal treatments suppress the immune system, arresting the spread of patches. In early stages, some creams can also help restore colour. However, they have significant side-effects.

JAK inhibitors like Ruxolitinab, which is the first US Food and Drug Administration-approved JAK treatment for non-segmental vitiligo, can also have severe side-effects, especially the oral drugs, including major adverse cardiovascular events, blood clots, serious infections and cancer.

“If you think of what attributes a good vitiligo treatment should have, there should ideally be three attributes: one, it should stop the spread of the patches…Second is also bringing back color to these patches so that they become even to your skin tone, and third is when they come back, they remain like that,” said Ganju.

The molecule that Ahammune has identified addresses all three.

Its drug candidate is not an immunosuppressant and has passed the safety check in its phase 1 trials, which is crucial in long-term treatments.

Currently, it is being developed as a topical cream, but an oral medicine is in early stages of development as well. It’s also working on two other programmes that target skin-related autoimmune disorders, which are in the pre-clinical stage.

The cornerstone of Ahammune’s strategy going forward is to keep the therapy affordable for Indian patients. “We want to take it all the way to commercialization on our own for the Indian market, while pursuing partnerships for the US and European markets, considering that we have patents in place,” said Ganju, emphasizing that accessibility remains a core focus.

Currently in India, for early stages, doctors prescribe generic versions of steroid creams like Elocon and Tenovate, which cost about ₹100-300 for a 10g tube.

On the other hand, the newer therapies like the JAK inhibitors can cost about ₹15,000 for a 15g tube, according to prices listed on online pharmacies.

Next round of funding

Ahammune is now looking at its next round of funding as it expands its team and deepens its strategy.

“The idea is to choose areas which have not had any sort of huge interest from the clinical fraternity to do research… The things that Ahammune is trying to do have a huge use case. There are many, many patients across the world, yet not enough clinical research has happened. So, we have taken that path,” said Uday Chatterjee, Ahammune board member and angel investor.

Success would open a lot of doors for large investments, both from national and international sources, Chatterjee added. “Which is definitely the lifeblood, because without a large infusion of capital, you cannot do this work. So what they're doing has in that sense a very vital importance to the future of this development,” he said.