Some Future Day

Marc Beckman: Dr.Shoshana Shendelman thank joining some future day. welcome.
Shoshana Shendelman: Thank you for having Marc
Marc Beckman: This is great. This is great. So background is so interesting. Your family is from
Iran and at some point it was really your father and your mother who um, I after the revolution ended up coming to the States, but your father was already working in the States.
Shoshana Shendelman: Correct. So my family is Persian. We're Persian Jews. And, um, some of my family had already come to the United States before the revolution. The rest of my family essentially escaped just before or during the revolution. But my father was already here, um, because he was. Part of a small group of Iranian scientists that were being trained at MIT in nuclear engineering and nuclear physics, because now we think of Iran as a hostile government and, and not a friend of the us.
But at that time, the s Shah was in power and it was a very different situation. It was a friendly country to the U.S. Um, and so the United States was training nuclear scientists. Here at MIT, um, to eventually return to Iran. And during that time, the revolution happened. Um, my father was lucky enough to be able to just stay here in the United States.
Um, and so my sisters and I grew up here. I was actually the first person in my family born in the us, um, as compared to Iran.
Marc Beckman: So at the the revolution, was your father in Boston at that point in time?
Yes.
And with the rest of your family, your mother and your siblings.
Shoshana Shendelman: We were, I was born in 1978. Mm-hmm. Which is the year of the revolution. And I was born in Boston, Massachusetts. Um, I'm the oldest, so all of my sisters came after me. Um, but I do have family members. Um, one of my cousins in particular, um, was actually at PA V University when the revolution happened and.
Watched her classmates literally be, uh, gunned down by machine guns. Um, she's written a book about it, but there were some very scary experiences, but we were fortunate enough to already be in the US at the time.
Marc Beckman: That's interesting. So your father's career path is relevant to what's happening in today's politics. It's interesting because President Trump right now is allegedly negotiating directly with Iran regarding their nuclear capabilities.
Uh, what do you think about it?
Shoshana Shendelman: So my father now works for the US government, so he's not allowed to talk about any of the work that he does, which
actually, we don want him to funny way. Right? We don't want, right. But that, that's a funny way to grow up, right? Like not necessarily,
um, you know, knowing, um, you know, where he was or what he's working on. But obviously, I mean, it's a level of security and confidentiality that goes beyond anything that, you know, we can imagine in, in normal life.
Marc Beckman: Um. do you think the Trump administration. Would be willing would be willing to provide Iran with nuclear capabilities for energy purposes.
Shoshana Shendelman: I don't know enough about this topic. I am a scientist, but I'm not a nuclear scientist. But my understanding, just based on
level of physics that I've taken, is
that it's not a very big leap between, um,
um,
Nuclear facilities for power purposes as compared to transitioning to nuclear facilities for weapons purposes.
Marc Beckman: Yeah, think the news that's coming out today and yesterday with regards to Trump being flexible on that front is nonsense.
I personally believe that the American and Israeli governments are leaking it to give Iran the feel. they have some sort of an advantage or some sort of flexibility, but I can't imagine that America and Israel will permit any nuclear energy because of the issue that you raised. Go into Iran. furthermore, remember before October 7th, Saudi was about to join the Abraham Accords.
And one of the key issues as it related to the Abraham Accords was that Saudi had nuclear energy capabilities, right? So all of a sudden Iran will, but Saudi won't. no way that's way that's happening in that region.
Shoshana Shendelman: You're probably right. But it is way beyond
my area of expertise. I'm
one of those people
that
I know what I know,
and I'm also very aware of what I don't know. So
I can talk to
you forever about science and medicine and healthcare, but I'm probably not the best person to. Answer political
Marc Beckman: So let's get into
it. Let's talk about it. So your career path is super compelling, very interesting, but it's complicated, right? People think that all of a sudden you become a massive CEO and so qualified to find cures for diseases, underserved diseases, and rare diseases, but it's not easy.
So you're raised in the Boston area, your father is working at MIT. How did you pick this career path? How did you get into medical school? What happened?
Shoshana Shendelman: It's
I do
think that there's. A bit of a genetic component behind whether you are, um, interested and good at science. Um, and in my family there's definitely a thread there. We have a lot of doctors and scientists in our family. Um, I was always drawn to science. Number one, I found that to be the most interesting subject always in school, but I also recognized that I was pretty good at science as well.
So from a young age I was drawn to science. Um, I knew that I would do something in that field, um,
I didn't know exactly what, um, I remember being
asked when I was 10 or 11 years old in school, um, what I wanted to, to be when I grew up. Um, and my answer was a marine biologist
I had just read a book called The
Arm of the Starfish, which is actually about,
um, regeneration of
nerves.
Um, and I didn't fall too far from that. I actually studied neurobiology, um, as part of my
So
I always knew I wanted to do something
science. Um, and then of course
that evolved over time. You don't just decide when you're a child that you're gonna be a biotech, CEOI.
Marc Beckman: So where did you go to? medical School.
Shoshana Shendelman: So I did my PhD in cellular and molecular and biophysical studies at Columbia Medical School. Um, so Columbia actually has a PhD program that is within the medical campus itself that focuses more on aspects of science that are directly translational to diseases and understanding molecular mechanisms of disease.
And then. Also how to potentially treat those diseases.
Marc Beckman: Columbia still best in class when it comes to that category of education?
Shoshana Shendelman: I think so that's why I chose to go there over 20 years ago, and I think they still are. Um, I have two daughters who are actually undergraduates at Columbia and
I did
encourage them to consider Columbia.
Um, they both are
pre-med and plan to go to medical school.
I'll also
encourage them to think about Columbia Medical School because I do believe that Columbia is really at the top.
Marc Beckman: So you mentioned that this scientific piece runs almost genetically or inherently within your family. Is it just your daughters? Is it just your daughters and your father? Like how wide is medicine within, within your family?
Shoshana Shendelman: It's pretty wide. Um, one of my other sisters was
also a science
also a science major in college. She ended up going off the rails and studying law afterwards.
Um, but but quite a few,
Um, of my,
um, cousins
are physicians and my youngest sister, because I have four younger sisters. So one studied science and then went into law.
The other one studied science and one straight through to medical school, and she's a surgeon.
Marc Beckman: Amazing. So your parents have to be so proud. I mean, you guys are the best family ever, right?
Shoshana Shendelman: I'm pretty proud.
Marc Beckman: I'm pretty proud. That's amazing. That's really great. So, okay, so you finished medical school Mm-hmm. and then what happens?
You go into like big companies, you go big in corporate or. So
Shoshana Shendelman: I had an interesting experience.
Mm-hmm. Um, I
did my PhD in
the 2001 to 2005 timeframe. Um, and that was just when we were figuring out how to differentiate stem cells into other things. Um, now it's very well understood and we could almost do it on my kitchen counter, but at the time it was really a frontier of science in an evolving field.
Um, and so my PhD research was focused on Parkinson's disease and understanding the genetic causes of Parkinson's disease. A big piece of what I worked on was differentiating. Cells into dopamine neurons, which are affected in Parkinson's. Um,
and figuring
out how to go from stem cells to neurons at that time was a big jump in technology.
Um, and so when I finished my PhD.
I
went into consulting specifically because that knowledge base was not very widespread. Um, and that was really relevant for drugs and for technologies that were being brought into the biotech and pharmaceutical industry. I.
So
I went into consulting and started working on drug development in the pharmaceutical setting.
Um, I eventually started my own biotech company, um, and now I work with several biotech companies getting drugs
Marc Beckman: Just going back to Parkinson's, like I know so many families still are just in, you know, terrible situations because of So you're saying that leap with regards to stem cells, um, was significant.
But why is Parkinson's just, it seems like, honestly, like I feel like I'm, I'm meeting more and more people who have family, relatives, friends, whoever it might be with Parkinson's now, is it becoming more and more prevalent? Is it, uh, are, are, are we attacking it the right way?
Shoshana Shendelman: Well, I'll say a couple of interesting
things on this topic. Um, on the one
hand,
it is becoming more prevalent, which is partly because people are living longer because the incidence of Parkinson's disease from non-genetic. Causes increases as you age. So the longer people live, the more of them you'll have at later stages of their life that actually live long enough to develop Parkinson's disease.
But
there are also genetic causes, um, and the genetic causes of Parkinson's result in a much earlier onset form, um, which is, for example, in their forties or fifties as compared to over the age of 65. But we also now understand that there are genetic factors. That can influence or cause Parkinson's disease.
Um,
the most
commonly known as a pesticide that was used, um, over a few decades and that use of that pesticide or exposure to that pesticide in the agricultural industry was shown to result in Parkinson's disease in a number of
Marc Beckman: Wow. Interesting. I feel like so many people that I know are like just on a regular basis popping up and saying, oh my God, my uncle has Parkinson's, or.
If it's a genetically oriented disease, I know you're saying it's both genetic and non-genetic, but if genetically oriented and we have all of these advances in technology, like for example, ai, um, and and research of um, DNA, are doctors able to identify the causes at an earlier point of view and are they trying to use medication now to suppress it somehow?
Shoshana Shendelman: There are some Parkinson's to DR. Drugs
in clinical
trials now. Um, I think.
I think. It is a place
where we have an opportunity to really take leaps and bounds forward with new technology. Um, what I worked on for my PhD, which took me about four years, I'm pretty sure AI could do in a number of months right now.
Um, so that really accelerates our ability to advance, um, towards drugs. I'll tell you a funny side story. Um, I was at one of these dinners for, uh, for Columbia Medical School, and I was seated with a group of people that I didn't know previously, and we all started talking a group of scientists and physicians.
Um, and at some point the, the host of the dinner, um, sort of came over and said, so have you guys solved Parkinson's disease yet? And it turned out that it wasn't an accident that we were seated together. We were deliberately put there because we had all worked on Parkinson disease at some point in our careers.
And it is little bit unbelievable that there's still no effective drugs to treat such a terrible disease that affects such a. Broad number of patients. And at
first it seems like a. Off the cuff statement. So have you guys solved Parkinson's yet? Um, but then we all started thinking about it and, and why have
we not solved this yet?
And by putting together a group of physicians and scientists that have different skillsets, different ways thinking, and then if you layer in some of the technological advancements like ai, this should be a solvable problem.
Marc Beckman: So if you take AI and unlock it, then on. All different types of medically related issues.
What do you ultimately think will happen? Where do you think AI as it relates to, um, finding cures to diseases, whether it's rare underserved, or even diseases that have, you know, some available medication today? Like where do you think AI is gonna start hitting, um, the most in the short term?
Shoshana Shendelman: I think we're going to see. Targets identified much more quickly than we did before. Um, so again, drawing this back to what did I work on that took me four years? Sure. You know, accelerating that, um, it takes scientists years in most cases to. Understand what the molecular pathways of a certain disease are, to understand what's happening here that's being derailed or that's sort of going away that it shouldn't, that's causing this disease.
And only once we understand those molecular targets then can we try to drug them. Um, and one the things that I've seen, AI just. Advance at a speed that I never could have imagined is target identification because there's so much information about available on a genetic level and a molecular level, biomarkers, blood samples, um, things that would take a person years and years and years to try to draw lines and connect the dots.
But AI can do that very quickly. And the first step is identifying what's the target that we need to hit. The second place is then drugging those targets. So what we're able to
do now, um, with AI is actually once we have a tar, a potential target, we know we want to drug it to look at what does that look like as compared to other known proteins or enzymes or receptors, and try to figure out. way quicker than we ever could in the past what kinds of drugs or even sometimes drugs that are already out there for other things could be used to hit that target.
Marc Beckman: So how, like in the short term, how do you think we'll see AI change the way we are as it relates to the medical community?
Shoshana Shendelman: I think we're
going to see, I. A huge acceleration of drug development, and
we need to
ensure
that the rest of what
happens after that keeps up with that pace. Because in the pharmaceutical industry, identifying a possible drug is just the first step, right?
we know the target, now we have a drug for it.
What do you do next? Testing, um, animal testing, in vitro testing and a test tube, and then. Testing in people, which is clinical trials, and that's a place where the procedures and policies right now for testing drugs. Are
the same that they
were 30 years ago. It's not moved forward at
speed
that our technology and things like AI are moving forward.
So I think the next place that we really need to see change, if we're going to keep up with our technological advancement, is on what we call the regulatory front. What do you do with those drugs to get them from a possible drug into an approved drug for patients?
Marc Beckman: So you're talking right now about the FDA
Shoshana Shendelman: FDA is a big piece of it. Is there,
Marc Beckman: Is there a state level of regulation that also needs to be updated?
Shoshana Shendelman: um,
I think that there's, there's very different levels. So having access to,
to drugs. Eventually the commercial market comes from the FDA,
But before
that there's access to clinical trials. There's a lot of regulations around who can be in a clinical trial and under what circumstances. Um, there, there are some more sort of localized aspects of driving that process forward.
But the big deciding factor is the FDA here and in Europe, the EMA.
Marc Beckman: So I'm seeing like a lot of issues popping up as it relates to artificial intelligence.
Creating advancements in different business sectors, but then for some reason or another, legislation can't keep up. Law enforcement in some cases can't keep up. So are you saying that the FDA is not in a position right now to. Basically keep up, like it's policies and procedures are so archaic that it won't be able to keep up with all of those incoming advancements in medicine.
Shoshana Shendelman: I think that's true. I think that we probably should have started this process years ago and sort of foreseen some of these changes that needed to be made. Just fact wise, the basic regulations at FDA for what you need to do to bring a drug through the development and approval process haven't been updated in 30 years.
I think we'd all argue that technology has advanced in 30 years.
Marc Beckman: Yeah, amazing. You know, world over since the
Shoshana Shendelman: we're
Marc Beckman: 1990s.
It's incredible.
Shoshana Shendelman: Yeah, we're overdue for an update, but I do think that there's a potential to move quickly now and catch up, and that's really where we need to look to the future.
It's easy to say, here's
all the things that are terrible.
Here are all the things people are doing wrong, but the important thing is how are we gonna fix it? And I think we have an opportunity in front of us to catch up quickly and change regulations to line up with where we are from a science and technology
Marc Beckman: But
you're saying it's too, it takes too much time.
Is that the key issue? Because is isn't that time, um, important as it relates to safeguarding, uh, innovation with regards to drugs and making sure that the users, patients are safe? Like if, we accelerate take, isn't it it dangerous?
Shoshana Shendelman: There are obviously some things that could be dangerous if they were accelerated and those should not be up for discussion. Um, I'll, I'll give you a real life example. So we recently heard from President Trump that monoclonal antibodies, um, or biologic treatments that are antibodies as drugs are no longer going to be required to be tested in certain animals.
Um. we have to see that enforced. We need to see that regulation change. We need to see that move through the FDA, but that's a good example of where a policy. Didn't keep up with technology. So antibodies are what our immune system, you know, develops against a target. We make antibodies as drugs and there are a lot of drugs right now which are monoclonal antibodies.
Um, testing a human monoclonal antibody in an animal doesn't make a lot of sense because the animals won't respond to it the same way that people would. Um, it does make sense when there's what we call small molecule drugs or traditional drugs compounds. That's what that regulatory pathway was built for originally.
And then when we had monoclonal antibodies or any antibodies actually as drugs. The regulations weren't so we were still doing this testing in animals that made no sense conceptually because animals wouldn't respond the same way as people to antibodies. So that's sort of a, a real life example of where we, we need change and we are changing, but there's a lot more behind that.
There's a lot more that needs to
Marc Beckman: I mean, it just seems totally ridiculous, honestly, just to put it bluntly. I mean, it's insane. and. Um, I think it's a good example where once again, you have a governmental entity that is not as sophisticated as the private sector and as a result things are a little bit sluggish, um, and, and hurt society.
So if you're gonna break down the FDA beyond this, how else do you think the FDA can be updated? What could we do to improve the process from start to finish for, you know, good drugs, to be approved, to help patients, to help people?
Shoshana Shendelman: I think AI is one place where we need to take a close look and quickly, because we now have AI approaches, um, and ways of looking at data that are actually not only faster, but more accurate than some of the traditional lab-based testing that we have been used to doing and, and are required to do through the current regulatory protocols.
Um, so really looking at where is AI actually a better assessment of patient safety, um, and potentially efficacy versus versus the traditional in the lab, you know, or in an animal setting is an easy place to start. I think also a recognition of. The importance of of bringing drugs more quickly to patients and the urgency to treat, I think for a long time, and maybe to your point, because it's a government entity and sometimes there is this way of thinking in government entities, um, that it's very risk averse.
Um, patient safety is of course the most important thing when you're developing drugs. A lot of patients though, are facing fatal diseases. So when you talk about patient safety in the context of somebody who's dying, it's a very different conversation. There has to be. A risk benefit balance, and I think that in some cases, on the risk benefit assessment, we've been worried about the risk without the context of what's the benefit if it takes 10 years to develop a drug and bring it through the regulatory process.
A lot of those patients have unfortunately already died.
Marc Beckman: you gimme an example of drug? Any drug recently that, um, the regulatory process slowed it down and it should not have been slowed down. It should have been pushed through. Um, but for the red tape, but for the bureaucracy of the FDAI
Shoshana Shendelman: had a personal experience with this just year.
Marc Beckman: Uhhuh.
Shoshana Shendelman: Um, so, uh. We were developing a drug at my former company for a pediatric rare disease called Galactosemia. Um, this is a terrible, rare disease that affects children. It causes permanent neurological damage and there's no treatments available, so there's literally no options.
Um, there's also, there was nothing else in clinical trials, so our drug was the one opportunity. and I believe that we showed a very strong safety and efficacy profile. I met a lot of the children who actually had received the drug in our clinical trials after the
Mm-hmm. Um, to able to
tie that benefit to actual children and actual families, it's really different than, you know, analyzing things on paper and sort of looking at numbers.
Um. In this case, there was FDA bureaucracy about how data was transferred, you know, through an electronic system handled in the clinical trial, which ultimately resulted in the drug not being approved. And there was a huge backlash from the patient community and the families because to hear that a drug is not available to your child because of a bureaucracy element.
Without there being any patient safety issues or any patient safety concerns is really heartbreaking.
Marc Beckman: So, just so I understand, so the drug was not approved because of an electronic data mistake. Is that it?
I'm trying to,
keep, I'm trying, to simplify it for the audience. Like it wasn't the drug. It wasn't, it wasn't, it wasn't, um, rejected based on the merits of the drug, based on the efficacy of the drug. It was disapproved based on the process. It was a procedural mistake?
Shoshana Shendelman: Based on the data processing and handling procedures that, you know, resulted in that efficacy data. But yes, very bureaucratic.
Marc Beckman: But did you have the data to back it up? Was the data there to back it up?
Shoshana Shendelman: Yes.
Yes,
Marc Beckman: So, But
Shoshana Shendelman: there is a
certain way that these things need to be handled from an FDA perspective. These regulations are hundreds and hundreds of pages and they haven't necessarily kept up with the technology that we use in clinical trials.
Um, so in this case, we were using some specific electronic behavioral, um, tests that hadn't been used in clinical trials before. So we worked with. An expert firm to develop the process of how that electronic data should be captured and moved through in an FDA compliant manner. Um, again, hundreds and hundreds of pages of regulation.
At the end of the day, it wasn't the way that the FDA wanted us to do it. Um, and so the data that was generated in that way from the clinical trial was discarded.
So
Marc Beckman: So So do you think the drug will ultimately be approved?
Shoshana Shendelman: I do, I really believe in the drug and the science. Um. I've seen the effect that it's had on these children, um, and it's safe and well tolerated. And so I do think that eventually this will win out and hopefully be available to patients and families. Um, but I understand the urgency on their side and, you know, every year that goes by with children, it's more permanent neurological damage happening and that can't be recovered.
Marc Beckman: right now, those patients, those families have no hope.
Shoshana Shendelman: If they weren't in our clinical trials, they have no hope. We were able to continue providing the drug to any patients who are in our clinical trials. It's a very small number. It was about 50 patients out of 3000 in the us. So if you're not one of those 50 patients, you have no hope right now. So.
Marc Beckman: the FDA approves that. So the FDA is approving that those patients can continue to use the drug that it didn't.
approve.
Shoshana Shendelman: Correct.
Marc Beckman: So how is that logical
Shoshana Shendelman: Approval is different from providing access is probably what they would say. So one of the. elements that can encourage patients to participate in clinical trials is saying after the trial is over, you can potentially have access to the drug in the period of time between the trial completion and approval as long as the drug is safe.
Marc Beckman: I gotcha. So, so let's go back. Let's talk about your company a little bit. I mean, you were an incredible force. You built up this company. Um, I learned in talking to you before today that it is really expensive to take a drug, take a concept, and bring it all the way through. Uh, to approval. So let's talk about this drug specifically.
You start, you, you realize at some point you have this company, you realize at some point that you need more money to, um, get these drugs into the pipeline so that they can eventually be approved by the FDA. So how much is it typically these days in America? Like how expensive is that process to get like one new drug approved?
Shoshana Shendelman: It's hundreds of millions of dollars. Um. drugs for larger indications that require larger clinical trials, which is the majority of drugs that we see these days, cost hundreds of millions of dollars, sometimes approaching a billion dollars to get the drug approved. Um, because there are a number of clinical trials required and for drugs that are not.
For rare diseases, those clinical trials often need to include thousands of patients, sometimes in the order of 2000 to 3000 patients per trial. And you need two phase three trials. So we're talking about thousands of patients. Those trials cost hundreds of millions of dollars to run
in case of rare diseases. It's somewhere in the ballpark of 50 to a hundred million dollars because they're smaller trials. There's less patients. In the case, we were just speaking about 3000 patients in the United States. So you're talking about smaller trials that cost a little less. I think a lot of people would still argue that $50 million is a lot of money, though.
money.
Sure.
It's a lot. So the typical path that companies take, and this is what I, um, I've done as well.
Is to develop the
earlier stages of a drug in a private company setting where you can raise tens of millions of dollars from private investors. Um, when you get to the point of needing to run phase three trials.
It's very difficult to do that in a private setting. I IPO'd my company for that reason. Um, because in the public markets you can raise a magnitude of capital that is beyond what you can raise in the private markets. And when you get to that stage of needing to run a $50 million trial or a hundred million dollar trial, um, the, the place to do that is usually in the public markets.
Marc Beckman: it's of interesting also because I would think that the big pharma companies don't want to play with these smaller drugs because it's not really scalable. ultimately. Right. It's a rare disease, but yet rare diseases are ubiquitous. I think you told me that there's 10% of the American population that are suffering from rare and underserved diseases today.
Shoshana Shendelman: That's correct. There are 30 million people in the United States with a rare disease. The problem is that there are 7,000 different rare diseases, so it's not rare to have a rare disease. 10% of our population is walking around with a a rare disease. Mm-hmm. But when you break it down to all of those 7,000 individual diseases, many have only a few thousand patients with that particular disease in this country. The technical definition is having less than one in every 200,000 patients with disease in order for it to be designated as rare. Um, in many cases though, it's an ultra rare disease.
Marc Beckman: Right. That's amazing actually. So for you, you were inspired to go into the rare disease sector because you wanted to help these individuals. Like what was the impetus? Like what clicked your, your emotions to, to get into that, into that field?
Shoshana Shendelman: I did, but my career wasn't always that way. So I think we were talking earlier about my PhD work was on Parkinson's disease. That's a very large disease state. Um, you know, I've worked in multiple sclerosis, rheumatoid arthritis. Those are much larger disease indications, not rare. Um, and as a scientist. I always heard about the numbers we just discussed. I, I knew there were 7,000 rare diseases. I knew that 95% of patients with a rare disease had no treatment options available. Um, but I didn't really internalize what that meant until we had a family experience. Um, and that really shaped my career and changed my perspective. So, um, my cousin, who was a physician himself, um, Dr. Fred Ban, was a, you're a prominent physician, right? Prominent neurosurgeon, um, head of neurosurgery at Lennox Hill Hospital, in fact, um, and someone that I was very close to, um, a very meaningful person in my life, um, developed leukemia and was treated effectively for leukemia.
But after that treatment with an immune system that wasn't working properly, he developed a multi-drug resistant infection.
We hear about these often, right? We hear about the threat of antimicrobial resistance, but there are bacterial strains that are resistant to every antibiotic It's horrifying. Um, and unfortunately he developed one of those infections and there was nothing we could do. My family has a lot of doctors and scientists.
We all tried everything we could, um, every treatment that we possibly get globally,
globally. Um. But unfortunately nothing worked. Nothing was available. Um, and ultimately he passed away. But it was that experience of being told there's nothing we can do. There are no drugs available for this disease.
Say
your goodbyes
It's horrifying
And it really changed my perspective on drug development in general, and it transformed it from just statistics numbers on a page. 95% of people with a rare disease have no treatment options available. To what is that actually like when that's your loved one that has the rare disease and there is nothing you can do?
Um, and so I shifted at that point and really decided
to dedicate
my career to developing drugs for rare and underserved diseases. So diseases that have no treatment options available, or no really viable treatment
Marc Beckman: So, I mean, that's like such heavy news, right? Especially for a family like yours that has so many resources, so many capabilities.
What do families do when they hear this news? Like, where do they turn? What, what, what happens next? I. Where do they go for help?
Shoshana Shendelman: We experienced it firsthand. Um, it's horrifying. There's not a lot of places to turn, is the unfortunate answer there. Um, and I think some recent statistics showed that, um, about 80% of patients and families that are faced with a rare diag, a rare disease diagnosis, are unable to find specialists with that expertise
they need.
Wide 4k: Mm-hmm.
Shoshana Shendelman: Putting aside,
95% of them have any drugs available
or any
treatment options. Um, but even just finding the right people to even, you know, diagnose or treat that illness is, is very difficult.
Marc Beckman: So you go, so you have this terrible experience. I'm so sorry. Like, I have to acknowledge it I mean, it's heartbreaking.
And then truly, I mean, it's like
Shoshana Shendelman: it was horrible.
Marc Beckman: It's like debilitating. I know. It impacts you still today, I would imagine your whole family of course. And then you, you start this company. And you're building, you're growing, and then all of a sudden you decide that you're gonna take it public. You're going to IPO Applied Therapeutics.
What made you think that you could do that? That's like, an all, it's amazing that you got to where you are as a woman, right? And then all of a sudden you're like, I'm gonna go further I'm going to Wall Street. What made you, what inspired you to do that? What made you think can take this company and go public?
Shoshana Shendelman: I think it was what needed to be done in order to get to the next stage. And I think, you know, that's a way that I've viewed my career and my life. And I think there are a lot of
especially
women in science, um, who have had to overcome a lot to, to feel like, well, there's a hurdle, but I'm gonna.
Get over that hurdle. And so obviously it's not easy to IPOA company, but that's what I needed to do in order to get to the next stage of development to be able to raise $50 million, a hundred million dollars for that phase three stage trial that we needed to do, um, for our lead drug and then a second drug after that.
Um, and it's, it's not easy to IPOA company, um, but it's what had to be done. So I just. Like everything else, and I think many of us are this way. Look at the problem. How are we going to solve it? And then just step by step, try to move through that problem.
Marc Beckman: So it's
so impressive, right? You single handedly took a company public. It's my understanding, the company went to a billion dollar market cap.
Huge success. You have two new drugs in the marketplace that reach the third phase of the process to be approved. How many, I mean, that's very rare too. That's an amazing success and amazing too. How many drugs ever make it to the third phase?
Shoshana Shendelman: Only 10% of drugs make it to phase three. So I think some of these numbers are really shocking to
Right? Right. To find out that it
hundreds of millions of dollars to get through phase three trials and only 10% of drugs even make it to
phase Correct. Um, but it is,
it's diseases, it's medicine. It's a place where we need to move forward and we need to advance.
And so, you know, it's, it's worth,
It's
worth the cost and it's worth the effort and it's worth the timeline because in many cases we're saving people's lives or we're transforming their lives. And that's why the biotech and pharmaceutical industry, I think is so
important.
Marc Beckman: So you're, you're deeply involved personally with regards to steering the progress of these two drugs.
You're deeply involved personally with regards to running this company that has a billion dollar market cap. and you
are a very hands-on mom, right? I mean, like you are like not missing any part of motherhood, right?
Shoshana Shendelman: correct. I'm very hands on. Um, I have two daughters. I think
it is
especially meaningful to me that I am the oldest of five girls in my family. So we're, I'm a girl's girl.
Yeah. I'm the oldest of five girls. I have
two daughters. They're also science majors in college. They both go to Columbia. They're both planning to go to medical school and I have been very hands-on with them from the start.
Um, obviously I love my children. I do think that there's a lot that is even outside of academics that we learn from in life. So just as an example, I coached their softball
team. Mm-hmm. Um, a girl
softball league starting when they were about seven years old. Um, straight through the league and
that time together, you know, not only building the
skillset training. I think it was also important for them to see me leading the softball team. So not just standing on the sidelines cheering for them, but coaching that team. Um, it, it made a difference. So Did you
Marc Beckman: win?
Shoshana Shendelman: We often won. There you go. So, and we often turn things around. So the number of times that I had to give a bunch of 10-year-old girls a speech of, we're down by 10 points, but we can turn this around.
Um, you know, it's probably more than you'd
imagine.
Marc Beckman: Did you ever like stop while you're building these three columns and be like, I, I'm a young person. I've worked my butt off to do all of this, but look how proud I can be. Like, did you ever stop and realize at that moment. You're really accomplishing so much like this is your life and look what you're doing.
you ever have a moment to like really enjoy it?
Shoshana Shendelman: I probably haven't, but that's
probably, that's probably a
fault. I think it's a lot of times easier to see that from the outside, whereas. People from the inside that are trying to drive forward and, and you know, be successful both from a career perspective and a parenting perspective. I think are oftentimes, and this was certainly the case for me, always thinking about what else can I do?
What can I do better and what can I do next? Um, and sometimes that holds you back from sort of looking at things and saying, wow, everything I've already done is an amazing accomplishment. And so that's something that I have a lot of self recognition of. I, I do need to stop a little bit more.
If you and I are talking about this right now, the fact that both of my daughters are science majors at Columbia doing amazingly, they're straight A students. Hopefully they go on to medical school. I have a very close family. I'm extremely close with my sisters, my cousins, my aunts. I am really very fortunate in life, um, and also in my career to have founded a company, gone through an IPO, gotten two drugs through phase three.
Um, but you don't always stop
notice that the time. I,
Marc Beckman: I think it's a
common trait for people that are builders, right? I often talk about the fact that the photographer Hallsman mentioned there builder builders there are destroyers, and you're a builder, right?
Clearly you're a builder. But what happens is builders sometimes feel alone. Even while you're accomplishing, you're not there celebrating your accomplishments. You just keep going and pushing forward and creating. And you know, in your case you're trying to like save humanity. You're trying to raise a family.
You're trying to like build value for Wall Street. Did you ever have those moments where you're like, Ugh, I am all alone?
Shoshana Shendelman: I
think you're right. It's very isolating in a lot of ways. right? Um, you are often
the only person in
that exact situation. It's unrelatable to a lot of people. It's an extreme amount pressure.
Right.
Um, and you know, from, from, from both the families that you're developing drugs for and also investors, I'm sure most industries are like that, not just biotech, but.
for
When you're the CEO of a company, and especially when you founded that company, there's not a lot of separation between you and the company's success. So it's, it's a lot of pressure, but I do think, and it's sometimes isolating, um, but I do think that that's where, um, in my case, having.
a
very supportive, extended family and a lot of aunts, uncles, cousins, mostly women in my family.
There's a lot of really successful
women, um, that couldn't understand exactly what I was going through. Um, but provided a lot of generalized support that women can do amazing things and be successful on both the career and the family front, I think really helps to get me through.
So
Marc Beckman: So what happened
when things went a little south, like the drug, the two drugs at some point, because of this procedural issue that the FDA called out, they're not approved, and then all of a sudden there's like a hit piece this like rag magazine.
Um, as I see it, it's like a gossipy magazine. Um, and it's about you. You discover that there's this hit piece on you. You've been so committed your entire life, your entire career. You're trying to do the right thing for patients and families with rare diseases for Wall Street and investors. And of course, while you're taking care of your children and your family, and then boom, all of a sudden this story comes out.
What are you thinking?
Shoshana Shendelman: I'll be honest, Marc. It was a horrible experience. Um, I don't wish that on anyone. Um, in my case, basically. Aside from really, you know, pressing salt in the wound, we had recently found out that our drug was not getting approved by the FDA. We received a complete response letter or CRL, um, and a warning letter on top of that.
So it was really a, a low point, but ready to get back up there and sort of say, okay, let's fix the problems. Let's resubmit,
let's go talk
to the FDA, let's get this drug to patients, um, to sort of be taken down. Um. On a personal level as well. Um, so in my case, the article was extremely personal. Um, and it didn't just discuss me as a scientist or me as cEO, but me as a person, um, when I had dedicated my career, you know, for the last 10 years towards patients and families that have no other treatment options.
Um, and. It's, it's a horrible experience. It just really sort of threw me to the floor. Um, I was surprised that number one, somebody would do something like that. Um, but, you know, number two, that it could have such a big impact, um, and, and really sort of tear down a lot of what I had built.
Marc Beckman: Yeah. I understand that there's a, um, uh, routine with regards to that publication targeting other women CEOs in particular, Yeah. and then, um, shorting stocks.
And obviously what I'm saying is pure speculation, but we've done some research on our side and we've seen. That there's a correlation between hit pieces coming from that magazine specifically against female CEOs, stock prices being shorted and hedge funds making money off of it. So, you know, it's something also that's debilitating.
And I think that issue recur is a recurring issue in the biotech space, right? Where, where articles are, are planted and then, um, funds, hedge funds and, and other speculators are shorting stocks and they're making millions and billions of dollars and. You know, it's kind of lousy given the fact that here you are trying to literally save people's lives.
It's, it's a, it's a difficult thing, but yet you still, you know, you walked away with a ton of credibility, right? You moved on with still, you still have your position as a, one of the board of, you're on the board of trustees at Columbia. I mean, you're, you know, without a doubt, still a pillar within the medical community.
Shoshana Shendelman: It's true. I am really honored to be a trustee of Columbia University. I'm vice chair of the medical school board as well. Um, and I have moved on to work on other drugs, help other companies, lead other companies, um, through the drug development process. Um, but you're right. You know, it, it did, um, it did take a toll and it, it is.
Very unfortunate that, um, some people have chosen taking down female biotech CEOs as a business model. Um, and you know, I, I think that makes it even harder for women to advance in science and biotech in the
Marc Beckman: What would you say to women, um, whether they're in biotech or not, that need to overcome adversity? People like you who are builders, who are saving lives, um, but then all of a sudden get.
Hit and all of a sudden life comes to a screeching halt. Just for a second, how would you inspire women, um, in particular to realize that they could get through tough times?
Shoshana Shendelman: I think there are some cliche statements that actually really Revinate, like they might cliches. I thought you were gonna
Marc Beckman: say I'm being cliche.
Shoshana Shendelman: No,
Marc Beckman: No,
Shoshana Shendelman: you
know the, I
often think, um, especially my. Cousin Fairy that we were
about earlier Yeah. Really
inspired me to, um, to develop drugs for, for rare diseases, um, would often say things like, um, nothing worthwhile is ever easy, you know, and if you wanna do something important, it's going to be hard.
Um, and you just have to fight for it. And I think that there's a lot of cliches around that, but they're, they're true. Um. I, when I look at what I've been able to accomplish in my career, and when I look at the effect that that's had on my daughters and their friends and some of the girls that I used to coach in softball who are also now in college, I have been able to affect change.
It's not been easy, but there has been a positive result. Um, and so I hope that.
With, with
women or girls who are facing challenges, especially in male dominated industries, it will not be easy and we will have to work harder than anyone else, but we can overcome that and move everyone forward with us.
Marc Beckman: Well, it's kind of interesting because it's also who you are, right? You're not gonna all of a sudden change because some guy, some weirdos hiding behind a keyboard is writing a poor story about you. And then all of a sudden you get shift careers. You're gonna become a fashion designer or a filmmaker like you are.
are, There's nothing wrong with fashion designers. You are, but no, but you are a scientist. You are here to save lives, and you're never going to stop that. You're not going to pivot. And the fact that, you know, Columbia University has, you know, proven that they stand behind you like one of the most important institutions, not withstanding everything that's going on in that campus right now.
I mean, it says a lot, right? You're not changing. This is who you are.
Shoshana Shendelman: Yeah, it's funny you bring that up. Um, a few years ago there was a similar kind of attempt to target, you know, what they would refer to as quote my credibility. Um, and the attempt was, um, you know, where did I even come from and did I even really have a PhD from Columbia? Um, and, you know, tweeted out and, and posted, um.
And, you know, shortly afterwards I was announced as a trustee of Columbia, and you
pretty sure right, that Columbia knows if I went to school there for sure. And the dates of my degrees, of course. Um, and so they, they chose the
wrong element there to try to hit on credibility. But I think it's interesting that the attempt was made, um, and I, and.
and the, the number of
people that asked if they could see my degree and could I prove that I had a PhD from Columbia on its own was shocking because I can't imagine anyone asking that of a male CEO of a pharmaceutical company. Um, so I think just that,
you know, level of someone tweeting out that they didn't believe
that I had a PhD from Columbia, and then actual investors and others coming to me and saying,
can you prove to me that you have a PhD
from Columbia?
Marc Beckman: It's Absurd,
Shoshana Shendelman: Absurd. And then of course in that case being announced as a trustee of Columbia, put all of that to bed. But I think we need to look at things like that, you know, in a sim with a similar
of trying to take down women. Do
women.
Marc Beckman: Do you think the world is getting a little bit better on front? Like I know now you've been, you know, approached by investors, you've been approached by other companies to put you into serious position right away to lead publicly traded companies and beyond but some of the most important new medications as well.
Um, do you think people shifted or do you think it will always, that that issue of, um. I guess, uh, gender discrimination is always going to be there.
Shoshana Shendelman: I think we're getting better, um,
which is important, right? We just, we have to establish progress forward. I see with
my own daughters, um, that what they're looking at as science majors in college now,
um, in
labs, doing research, um, and you know, potentially going to medical school. It's really different from the way things were 25 ago Yeah. when I was in that setting.
Um, and so we have made progress and
while I wish that things were all the way there, you know, where we needed
them to be, where everything was, you know, equal. Um, and there was no gender bias on a professional level. I don't think we're there yet, but we're moving in that direction. So. An interesting example is that the number of women in STEM is increasing.
The number of female physicians, I think is just at about 50%
now. That's, um, that's progress. Those numbers were not so good, you know, 10
years ago. Um,
but where there's still a
gap is women in leadership positions. And so the number of female biotech and pharmaceutical CEOs is very low. The number of. Female
CEOs
of public
pharmaceutical or biotech companies is a
handful.
Um, a handful.
a handful. Um, and that's not okay. That's where we need to move in the next
So we're moving forward. Hopefully we'll find ways to
catalyze that,
accelerate it and move forward more quickly. Um, but at least
not moving backwards. That's a plus.
Marc Beckman: Shoshana. All of my guests end the show with me. They end the episode with me in the same way. I give a leading question and it incorporates the name of the show some future day, and then they finish.
Are you up for that with me today?
Shoshana Shendelman: Yes, I'm up for it
Marc Beckman: Okay. Okay. In some future day, American medicine will improve. Because the FDA will be radically changed by
Shoshana Shendelman: taking a
practical and forward looking view of
drug approvals that keeps up with the American advancement of science and technology.
Marc Beckman: Beautiful. Is there anything else that you'd like to add to our conversation?
Thank you for having me,
Shoshana Shendelman: Marc.
Marc Beckman: Thank you for being with me. I appreciate it.