Alan Mendelsohn, MD, Shares Insights Into Congenital Ichthyosis Patient Care and the Development of TMB-001

“We've had some children who've said this is the first time in their life that they can put on sneakers and run outside. We've had patients who started to sweat. I never have even thought about sweating as something that's important,” said Alan Mendelsohn, MD, while discussing the impact of congenital ichthyosis on patients. Mendelsohn, the chief medical officer of Timber Pharmaceuticals, a recently acquired LEO Pharma company, spoke to Dermatology Times after the “Think Like an Expert About Ichthyosis” session at the 2024 American Academy of Dermatology Annual Meeting in San Diego, California.

Timber Pharmaceuticals and LEO Pharma are currently developing TMB-001, a topical isotretinoin formulation for the treatment of ichthyosis. TMB-001 was granted FDA breakthrough therapy designation in May 2022 and is currently in phase 3 development in the ASCEND trial.

“I really appreciate Dermatology Times’ attention to rare diseases and their interest in trying to get the message out for patients who have rare diseases. There are companies out there that are hearing them that are really trying to be receptive and bring them the help that they are requesting,” concluded Mendelsohn.

Transcript

Alan Mendelsohn, MD: Hi, my name is Alan Mendelsohn. I'm the medical officer at Timber, a newly acquired LEO pharma company. And we'revery, very happy to be working with and working for LEO, who's really shown a major interest in developing drugs in the rare disease space.

Dermatology Times: What was the most impactful to you from the congenital ichthyosis and TMB-001 session at AAD?

Mendelsohn: I think probably the most important thing is that the AAD is really recognizing more and more the importance of rare disease, the importance of making sure the membership is aware of looking about and thinking about rare disease, and thinking about new treatment options for these diseases. I think, overall, the program was exceptional, particularly because it brings together the FIRST organization which is a patient representative organization. I think those are the most important things. I think that the cases are reflective of what not necessarily the average dermatologist sees, but certainly diseases that should raise their awareness to think about dry skin being potentially ichthyosis.

Dermatology Times: Part of the AAD session on ichthyosis was a patient's perspective. What are Timber Pharmaceuticals and LEO Pharma doing to help better serve patients with ichthyosis?

Mendelsohn: What is fascinating to me, having worked now in this space for about three years and talking to patients, and again, we're still blinded, we don't know what the results of the trial are. But things for example, that I without at ichthyosis, don't even think about twice, they're so incredible to hear patients talk about. So for example, we've had some children who've said, this is the first time in their life they can put on sneakers and run outside. We've had patients who started to sweat. I never have even thought about sweating as something that's important. But remember, people with ichthyosis, lack the, in many cases lack the ability to sweat. They're at a higher risk of heatstroke. And so just the ability to be able to sweat, to be able to go out and not have people make fun of them. To be able to have a normal life with your husband or wife is just, it's heartening to me. It's one of the most important projects I personally have ever been involved with in terms of the impact it has on the patient.

Dermatology Times: What does the future hold with the potential approval of TMB-001 for ichthyosis?

Mendelsohn: We're going to find out. The trial is ongoing. We will hopefully have the results later this year. We have a few presentations at this meeting, which will hopefully get the AAD, the membership more excited about treatment opportunities and rare disease in this particular case, ichthyosis. And then, as a company, I think we will move forward into lots more investment in thinking about how we can better affect patients with rare dermatological diseases. I think that's going to be a fascinating and very important thing for the dermatology community, both patients and practitioners alike. Again, as always, I really appreciate Dermatology Times' attention to rare disease, their interest in trying to get the message out for patients who have rare diseases, that there are companies out there that are hearing them that are really trying to be receptive and bring them the help that they are requesting. There's never a good time to have a rare disease, but I think now is the time for people with rare diseases to have some more hope. And we're going to move forward as quickly and as efficaciously as possible.

[Transcript lightly edited for space and clarity.]