Exploring the Clinical Translation of a Parkinson’s Regenerative Therapy: Interview with Amit Rakhit

Welcome back to another interview for Oxford Global.

Today I'm joined by Amit Rakhit, who is chief development and medical officer at BlueRock Therapeutics.

The interesting work that BlueRock is doing in cell and gene therapy will be showcased at Cell 2025 under our advanced therapy development track.

And I'm delighted to speak to Amit about the advancements BlueRock are making in the field.

Amit, thanks very much for joining me today.

Great.

Thanks for having me.

Great.

Fantastic.

So Amit, you've had a fascinating career across pharma and biotech. What drew you to BlueRock Therapeutics in this next chapter of regenerative medicine?

That's a great question.

So I've been very fortunate to be part of multiple organisations starting in large pharma with Bristol Myers Squibb.

I was at Biogen for a while and you know, had some shorter stints at smaller companies like Ovid Therapeutics.

And what's always driven me in terms of opportunities has been the opportunity to make a difference in transforming medical care for people in need.

So patients who have high unmet medical need.

And I was really drawn to BlueRock because of its bold vision to transform patients’ lives with the opportunities in cell therapies.

And cell therapies is really at a turning point.

And to be part of a team that's pioneering this type of regenerative medicine, it's really an opportunity to bring first in class therapies to patients and really address an unmet medical need.

And that's what really draw me to BlueRock.

Thanks so much.

You've worked in oncology, neurology, rare disease and many more. How did those experiences shape your approach to development in BlueRock, and especially in the field as complex as cell therapy?

Each of those areas are unique.

They have their unique kind of needs and characteristics of diseases.

I think it's really about balancing rigorous science with the unmet need that's there, the unmet therapeutic need for people who are looking for new treatment options.

And at BlueRock, it really allows us, our team here, to pull an approach together for cell therapy, develop it in a way that is both scientifically rigorous and really patient centred in its mindset.

And I think the experiences that I've kind of had across those areas of neurology, rare diseases, I learned to think more broadly, but always stay anchored in the patient experience.

And that perspective really helps how we develop cell therapies at BlueRock.

Thank you.

Great.

And I wanted to get a, your opinions on clinical translation and your lead candidate thereof. So Bemdaneprocel is your lead programme targeting Parkinson's disease. What sets this approach apart from traditional dopamine replacement therapies?

Sure.

So Parkinson's is a devastating illness and there is a high unmet need for new treatment options effects millions of people around the globe.

We have started our exPDite-2 clinical trial that's currently ongoing in the US, Australia and Canada.

And what's really different is that, yeah, there have not been any new treatment advances in the Parkinson's therapeutic space for the last 60 years, except for more, I would think, dopamine replacement therapies.

This is not a dopamine replacement therapy.

Cell therapy with Bemdaneprocel is designed to actually replace the cells that are lost in Parkinson's.

So the hope is that instead of treating symptoms, we're actually aiming at part of the root cause.

Now, I think Parkinson is multifactorial.

So there is more to do and understand and this is still an investigational therapy and so we'll see when this clinical trial comes up.

But we are encouraged by the results that we've seen with our Part 1, the phase one study, and really look forward to the results after recruitment is complete in our Phase 3 programme.

And hopefully we'll bring this forward as a meaningful treatment option for people who are having moderate, you know, Parkinson's disease, who have outgrown the dopamine replacement therapies, which are no longer working for them.

That's fantastic, Thank you.

And then Bemdaneprocel is now in clinical development, as you mentioned. What are some of the key outcomes you're looking for in these trials, not just safety, but also efficacy and also patient function?

Sure.

So our phase one programme looks really at safety, and we found a favourable benefit risk profile with our Phase 1 programme.

And so the phase three programme really aims to continue to assess safety but also looking at measures that are meaningful measures for efficacy.

So that includes motor function, which is a hallmark of Parkinson's, which is the change in motor function and the loss of motor function.

So we're looking at motor function through various scales that have been validated and used in assessing people with Parkinson's.

We're looking at the durability of effect, how long this lasts, and we're also looking at improvements in overall patient quality of life.

And so it's multi prong that we're looking at multiple aspects to have a holistic view of how Bemdaneprocel can potentially be a meaningful treatment option for patients in the future.

Great.

And then in terms of the platform and the science behind it, BlueRock has built this proprietary platform to manufacture pluripotent stem cells at scale. What are some of the unique technical or regulatory challenges involved with scaling PSC based therapies?

You know, this is a growing field, scaling pluripotent stem cells.

Really challenging, but also kind of a thrilling environment to be in.

We're not just making molecules; we're actually making growing living cells.

But that means there's consistency that's needed quality, your regulatory alignment at every step.

And it's mastering this consistency and quality and regulatory compliance, which is unlike other therapies, unlike traditional biologic, you know, when you're working with living cells, it adds complexity in both the manufacturing and regulatory review.

And our, I believe our team, you know, is building leadership in this space.

And we're very proud of the work that we've been doing.

And an example is bringing forward a programme like Bemdaneprocel into a phase three programme, bringing in our ophthalmology programme into a phase one clinical trial.

So we're excited about our abilities to really provide that and shaping and kind of building this new therapeutic area.

Thank you.

And this technology has applications far beyond neurology. Where do you see the next frontier? Is it cardiology, immunology or elsewhere?

That's a great question.

I just mentioned our ophthalmology programmes, which we're very excited about.

Those are moving forward.

We have a phase one study called the CLARICO study that's opened in the US.

There are applications for cell therapy in other areas, as you mentioned in cardiology, in immunology, other areas within neurology such as dementia.

And these cell therapies could have, could potentially repair tissue, reset immune function.

And these are natural extensions of the work that we're doing already.

More to come.

But this is a fascinating growing field.

There are many people who are building their expertise.

I think BlueRock is at the forefront of that.

I'm very excited to see this area evolve into the future as the, you know, future of medicine is and transform clinical practise.

Great.

And then finally, just wanted to ask what excites you the most about the future of regenerative medicine and particularly also what BlueRock can bring to that future and what its role in it will be?

Yeah.

You know, I've been in medicine now 30 plus years.

What was science fiction at the time is now becoming true science fact and regenerative medicine is part of that.

That's exciting that the team here at BlueRock, the teams that are working in cell therapy and the regenerative medicine space, I would say gene therapy is part of that as well, really are creating the vision for the future that will transform clinical practise.

And that's what really excites me is the possibility of truly meaningfully changing lives of people who are dealing with serious illnesses and what a difference that makes for themselves as well as their families and loved ones.

So that's what's really drives me and drives the team at BlueRock and really be I'm really proud of that.

That's great.

Thank you so much.

And if you're interested in any of the topics that we covered today, be it regenerative medicine, cell therapy, or anything else, please do come along to Cell 2025 where BlueRock will be represented.

And the last thing to say is thank you so much, Amit, for join joining me today and best of luck in the future.

Thank you very much and great seeing you and looking forward to meeting you and the team at the conference.

Thanks.