July 21, 2025 • 31 mins
Last week’s public disclosure that a gene therapy from Sarepta had caused a third death led FDA to ask the company to stop distributing its DMD gene therapy Elevidys, a move the biotech has resisted. The deaths, and disputes between FDA and Sarepta, raise questions about the future of AAV gene therapies, as well as the future of FDA’s platform technology designation. On the latest BioCentury This Week podcast, BioCentury’s analysts unpack the events surrounding Sarepta’s gene therapies and discuss how FDA, industry and patient groups should come together to learn the lessons from the tragic, avoidable deaths.
BioCentury’s analysts also assess Monday’s appointment of Stanford professor and biotech executive George Tidmarsh to lead FDA’s Center for Drug Evaluation and Research, and check in on the latest trends in venture financings. This episode of BioCentury This Week is sponsored by IQVIA Biotech.
View full story: https://www.biocentury.com/article/656537
#biotech #biopharma #pharma #lifescience #GeneTherapy #AAVTherapy #Sarepta #Elevidys
00:01 - Sponsor Message: IQVIA Biotech
02:03 - Gene Therapy
17:59 - Leading CDER
27:00 - Venture Report
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Episode Transcript
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(00:00):
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Alanna Farro (00:02):
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(00:45):
Last week public disclosurethat a sarepta gene therapy
had caused a third deathled FDA to ask the company
to stop distributingits DMD gene therapy.
A move the biotechcompany has resisted
the deaths and disputesbetween FDA and Sarepta.
(01:07):
Raise questions about thefuture of a a V gene therapies.
Jeff Cranmer (01:12):
The therapy Elevidys was the first and only
gene therapy approved for thefatal neuromuscular disease
on the latest BioCentury.
This week podcast BioCenturyanalysts take a look at
what the death means for thefuture of gene therapy plus.
At long last FDA hasselected a new director for
(01:36):
CDR, what do we know aboutStanford's George Tid Marsh?
We'll also take a look at somenumbers out of the world of
venture financing for biotech.
I'm Jeff Cranmer, and joiningme today on the BioCentury
Podcast are my colleagues.
Simone Fishburn (01:55):
Simon Fishburn, editor in chief.
Steve Usdin (01:57):
Steve Den Washington editor.
Selina Koch (01:59):
And Selena Koch, executive editor.
Jeff Cranmer (02:02):
Okay, Steve, I, I wanna jump right to you.
A lot has happened in thepast few days between FDA and
Sarepta regarding Elevidys,since the disclosure of the
third death of a patient.
in a Sarepta gene therapy trial,can you bring us up to speed?
Steve Usdin (02:22):
So, yeah.
So, okay.
So on, July 16,that would be like.
Last Wednesday, Sareptaannounced a major reorganization
laying off 500 employees,about 36% of its and
shifting its focus towardearly stage siRNA projects.
It also updated V and addedadding a black box warning.
(02:43):
That's a similar to labelsthat are on other a a V
gene vectors warning aboutpotential, liver toxicity
and, fatal liver, adverseeffects on Thursday evening.
BioCentury, I reported thatthere had been a third death
in a Sarepta trial that hasnever been disclosed previously.
(03:04):
It was a 52-year-old manwho was in a trial of a limb
girdle muscular dystrophy,gene therapy, from Sarepta.
He uses the same vectoras, Sarepta's DMD therapy.
The next day, otherreporters picked up on
that, um, expanded it, and,investors, picked up on it.
Also, there was alot of controversy.
(03:25):
Sarepta, issued astatement, had analyst q
and a in the statement.
And the q and a. They defendedtheir decision to not, disclose
the third death, previously, Soon Friday, there were several
actions that came out of FDA.
They, uh, placed a hold onall of Sarepta's limb girdle
muscular dystrophy trials.
(03:46):
They asked Sarepta tostop, distributing,
Elevidys, to any patients.
the company, which had announcedearlier in the week that it
was going to stop shippingelevators to non-ambulatory
patients because there hadbeen two previous deaths of
non-ambulatory DMD patientsRefused to stop shipping.
(04:06):
Elevidys this on a voluntarybasis to ambulatory
Duchenne muscular dystrophy.
Patients.
FDA also revoked the platformdesignation for the vector
that's used in, sarepta's limb,girdle muscular dystrophy, gene
therapies, and in Elevidys.
So that's where we are today.
Simone Fishburn (04:26):
Where to start with all of this?
Um, I think in a minuteI'm gonna bring in Selena.
Let's talk a little bit aboutthe implications for gene
therapies and a a v vectors.
I don't think you've saidthat to an a, a V vector, but
first of all, just clarify.
Um, for those who haven'tspent the weekend on Twitter
or wherever they're, orX or wherever they're
(04:47):
getting their information.
What is the reason thatit hadn't been disclosed?
I understand that Sarepta saidit's not materially relevant.
How has this come to light now?
And it seems in a way thatthis third, very, very
unfortunate death has actuallyjust catapulted the whole
company into a, a new phase.
(05:09):
I mean, it was clearly,as you say, it was having
layoffs and things.
It was clearly in sometrouble, but it, it just
seems to have compounded it.
Can you talk about that?
Steve Usdin (05:17):
Okay, so, so how's it come out now?
It came out now because I foundout about it and reported it.
if I hadn't found out aboutit and reported it, probably
somebody else would've,and it would've come out.
but certainly not atthe time that it did.
The company had nointention to make it public.
The companies reported it toFDA, so Sarepta told FDA that
(05:37):
there was an acute liver failurecase that was life-threatening.
They reported that, on June20th, 2025, that was from
their phase one trial of alimb girdle muscular dystrophy
therapy, and they followedup and notified FDA of the
death on July 3rd, 2025.
on Thursday morning, Sareptainformed patient advocates
(06:02):
of that death, but theydidn't announce it publicly.
when I found out about itand asked the company about
it, they said that they hadno intention or no timing
anyway, of making, a publicdisclosure of that death.
In their calls with analysts,they said that, they hadn't
disclosed it previouslyand that it wasn't material
because they had already.
(06:23):
Announced their intention todiscontinue that gene therapy.
I should also note that DougIngram, the CEO of Sarepta
said on that call that thefatal events associated
with their gene therapieswere very, very rare.
rare.
And in my conversations with,patient advocates, with mothers
of children who've had, DMD.
(06:44):
They were very concernedby that comment.
Been about 140 non-ambulatorypatients who've received
Elevidys, and, twoof them have died.
The limb girdle therapy,that caused a death that
was in a phase one trial.
There were four patientsin that trial and one died.
That doesn't reallymeet people's definition
(07:04):
of very, very rare.
Simone Fishburn (07:06):
So I think something that you wrote about,
in your story, which I thoughtgot to the heart of it is what?
Parents of the patients who'vebeen on these therapies are
going through right now, givenwhat you've talked about are
not incredibly long odds.
I wouldn't call them short odds,but they are quite serious odds.
I don't know exactly thetime course, from when a
(07:27):
a, a patient is given thegene therapy that this kind
of toxicity might arise.
But maybe you can just channelwhat you've hearing from the
patient community in terms ofwhat they're going through.
Steve Usdin (07:37):
Well, one of the, parents told me that living
with a child who's received.
This gene therapy isa 24 7 panic attack.
the timing is severalweeks to months after,
receiving the therapy.
We don't know how manynon-ambulatory patients have
received Elevidys a month,two months, three months out.
(07:57):
but some of themcer certainly have.
Um, Roche and June saidthat worldwide there have
been about 140 to 150 boyswho are non-ambulatory
who'd received the therapy.
Since its approval in the UnitedStates, since its accelerated
approval for the non-ambulatorypopulation, that's how many
non-ambulatory patients havereceived it, given the fact that
there's always a lag between anapproval and when gene therapies
(08:21):
start to get distributed.
It's quite likely thata substantial portion of
those would've been inthe last year, certainly.
and that's got to be causing,and it is causing, that's
what advocates told me.
It is causing a greatdeal of anxiety, among the
families of the, of theboys who have received it.
Simone Fishburn (08:38):
Selena, maybe you wanna jump in now.
Perhaps you can extend thisconversation to the implications
for a a V gene therapies morebroadly and that technology.
Selina Koch (08:50):
Well, liver toxicity from a a v
gene therapies that'sa known risk, right?
This is, not new.
and it really has nothingto do with whether a patient
is ambulatory or whetherthey can no longer walk.
Right?
there's not a biologicalconnection to that distinction.
It's like some.
False shorthand.
So what we know from genetherapies of the past and from
(09:13):
this gene therapy is that it'sabout dose and it's about the
state of somebody's liver beforethey receive the gene therapy.
So older patients who havegrown more and are way more tend
to be non-ambulatory, right?
And they need higher doses.
I don't know if anybodyremembers, but four or five
years ago, Astellas reportedfour patient deaths in a
(09:36):
gene therapy trial for adifferent severe rare disease
that causes progressivemuscle weakening, right?
That was X-linkedmyotubular myopathy.
And those were veryyoung patients, but that
disease, you know, overtime it involves a liver.
So what they realized thereis, okay, well if you're having
some kind of liver dysfunctionbefore, it's probably not
(09:59):
a great therapy for you.
But now in that case, theytracked the, the 20 patients
who lived for a coupleyears and they found out
it was highly effective.
You had, all of those patientswere, none of them could sit
unassisted before that trial.
All of them were on ventilators.
Something like three quartersof them came off ventilators.
All of them could sit upand some of them could
(10:20):
even walk unassisted.
in a scenario like that,you take all of that in
and you're like, are therisks worth the benefits?
you know, maybe, butElevidys doesn't have the
luxury of really strongefficacy data like that.
Um, but when it comes tofuture gene therapies,
what does it mean?
It means we need to takestock of all of these
gene therapies, right?
Not just this one.
And think about what havewe learned across the board
(10:43):
about are there thresholdsfor preexisting liver talk?
are there thresholds for weightand dose that can guide who
can successfully, you know whois at lowest risk and are the
best candidates for receivingbenefit from gene therapies.
Steve Usdin (11:00):
the thing about this one that's so disheartening
is that not only were thesedeaths predictable, but
they were predicted, right?
it was Anticipated thatthis could happen when, when
Elevidys was approved for thenon-ambulatory population,
which as you say that again,it's not about the fact that
they're non-ambulatory, it'sthe fact that they're older,
they've had the diseaselonger, they're larger and
(11:22):
they need larger doses.
But it was predicted that thiswas a, a major safety risk and
they, they went ahead with it.
Anyway.
FDA went ahead with it.
The company went ahead with it.
And also it has to bepointed out that there
was little of any efficacydata in the non-ambulatory
population to support arisk benefit calculation.
Simone Fishburn (11:43):
Steve, I wanted to ask you, so we know
today from, the SEC filing ofSarepta that FDA has withdrawn
its platform designation fora a v that it had granted
not that long ago actually.
perhaps you can talk a littlebit about what you would've
wanted to see from an FDA onthis whole front related to
(12:08):
this particular finding, andthen what you would like to see
from them going forward on this.
Steve Usdin (12:12):
Well first I think they need to have
a lot more transparency.
This administration talksa lot about transparency.
They need to have a lotmore transparency around
their decision making.
Here in the statement thatthey put out they being FDA
on Friday, they referencednew safety information that
it caused them to take theactions that they took.
Well.
there doesn't seem to be a goodcase for saying it was new.
(12:33):
They knew aboutthis some time ago.
The only thing that seemsto have been new is the fact
that the rest of us found outabout it and there was a lot
of concern that was raised.
So I think thereneeds to be a lot more
transparency around that.
And then I think there alsoneeds to be transparency around
the approval of the platformdesignation for Sarepta, for
this gene therapy vector.
(12:54):
Why did FDA do it?
What was the process that theywent through in thinking about
it and, how are they gonnathink about this, um, platform
designation going forward?
What are the kind ofcriteria that they're gonna
apply for determining whatproducts should get it,
what products shouldn't get?
And finally I'd say, you know,speaking to what, um, Selena
(13:14):
said, you know, the, the otherreally big concern is, is, is
there gonna be an overreactionhere, by FDA, by regulators, and
almost certainly by investors.
and does that, is that goingto jeopardize the whole field
of gene therapy, in ways that,arguably aren't necessary
and that in some ways, youknow, mimic what happened.
(13:35):
in the past when we had a tragicdeath, Jesse Gelsinger death,
that really, set the field backfor a decade, there was really
a, like a decade where there wasimpossible to do, r and d or to
get any investment in the field.
I think that that would be.
Just as tragic as havingthese, deaths that have
happened that were avoidable.
Selina Koch (13:57):
Whole host of companies who are trying very
hard to solve the problem ofgene therapies, you know, just
getting stuck in the liver.
they're trying to bypass theliver and really concentrate
gene therapies in the targetorgan they're interested in.
It would be a shame if thatresearch we're unfundable, cause
that's the solution we need.
For this.
I just wonder if there were someinvestigation into all of these
(14:20):
gene therapies so far that wascomprehensive and got into the
nitty gritty if it might guideus a little bit and, how much
retargeting of liver would be asufficient amount to avoid most
of the toxicity, you know, canwe get any insight into that?
Steve Usdin (14:35):
And I think this is a case where really
the industry, academics, thepatient community, right?
And not just the United States,but around the world should
come together and ask thatquestion and other questions,
and then plot a strategy foranswering those questions.
Selina Koch (14:51):
Yeah.
And then on the platformthing, I don't know.
It's possible that theplatform designation just.
Isn't gonna apply anywhere.
I mean, Peter Marks had saidbefore he left, he didn't think
AAVs were a good use case.
Even though you can apply thatsame vector to other therapies.
There's so much bespokedesign that happens in the
whole thing, like the comcombination of the cargo
(15:12):
and the vector and whatnot.
and so, he had pointedto, gene editing therapies
delivered in LMPs as.
Possibly the one placewhere this designation
could shine, but, you know,we'll have to wait and see.
It's not, not looking like,there's gonna be a, a large
number of relevant use cases.
Jeff Cranmer (15:32):
Steve, final thoughts here.
Steve Usdin (15:34):
I think that this is a case where we're,
we're really gonna have tosee whether the patient,
community, FDA, and industry,whether everybody can,
resist the urge to, to simplypoint fingers at everyone.
Else and plot a, agood path forward.
And, and there's a lot at stake.
And, and you can't forgetthat it's, it's right in
(15:55):
front of our face, right?
the patients, the boyswho have DMD still need.
Safe, effective therapies,it's still a relentless
and terrible disease.
that needs to betreated with urgently.
There needs to be urgent stepsto, to, develop, products
that are gonna help them.
and there's so many otherdiseases that potentially
could benefit from genetherapy that what's really,
(16:17):
really needed now is this kindof, overarching, attempt.
to plot a, um, a safe andscientifically responsible
path forward, to be able to,to use what's arguably could
be, you know, tremendouslyimportant therapy and, and,
and in other conditions.
Of course, gene therapy hasbeen tremendously effective and
safe and, and has, saved lives.
Jeff Cranmer (16:39):
Okay, and that course is the subject of
Steve's editor's commentary,that we published on Friday.
You can find that atBioCentury podcast.com.
We're gonna take a quickbreak, and then we're going
to turn to FDA and learn.
About the new CDR director,
Alanna Farro (17:08):
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Jeff Cranmer (17:46):
We're back on the BioCentury This Week podcast.
Appreciate you tuning inand if you like what you're
hearing, please subscribeand leave us a comment or
a question and we'll try torespond on an upcoming episode.
Okay, On Monday, FDACommissioner Marty Macari named
Stanford Cancer biologist GeorgeTid Marsh as the new head of
(18:09):
the agency's Center for drugevaluation and Research T Marsh
completed training at Stanfordin pediatric oncology and Neo.
Natology and his adjunctfaculty of pediatrics and
neonatology is foundingco-director of Stanford's
(18:30):
Master of Translational Researchand Applied Medicine Program.
He's also founded in binCEO of multiple Biopharmas,
including Horizon, whichAmgen acquired for 28 billion
a couple of years back.
Tid Marsh Succeeds.
Jacqueline Corrigan Curry.
The now former actingdirector of CDR, PO.
(18:52):
She held since Januarywhen, Patricia Cone stepped
down from the position.
Steve, of course.
Uh, as everyone knows, youfollow FDA quite closely.
What are you hearingabout Dr. Tidmarsh?
Steve Usdin (19:07):
it's really interesting.
First, the name cameout of the blue.
It really surpriseda lot of people.
It surprised people, seniorpeople at FDA and people
who watch FDA closely.
He has, as you mentioned,he has deep experience
developing drugs and industry.
He's trained as apediatric oncologist.
The other thing that you have toknow about him is that he's been
a very, very strong and adamantsupporter of anti-establishment
(19:31):
perspectives on COVID-19.
and really Dr. McCarey,with, Robert F. Kennedy Jr.
The HHS secretary, everythingseems to come back to COVID-19
in one way or another,but more on his resume.
Okay.
So you mentioned that, he has.
In biopharma, he was,president and CEO of La
(19:51):
Jolla Pharmaceutical.
When he was there, he helped,discover and, develop an
angiotensin two, drug forthe treatment of shock.
That was approved.
he was founder andCEO of Horizon Pharma.
As you mentioned, horizonwas, sold to Amgen for
28, billion dollars.
He also founded a company calledThreshold Pharmaceuticals.
(20:12):
And he is held senior positionsat Coulter Pharmaceuticals,
which was acquired by GlaxoSmithKlein Ceq, and other companies.
he led clinical developmentof Bexar and Doxyl, to
FDA, anti-cancer drugs.
he is also known as I said,for his, anti-establishment,
views on, COVID.
He's very, close personallywith Dr. McCarey, with, Vinad,
(20:35):
the CBER, director and withJay Charya, the NIH director.
he's praised, Robert F. KennedyJr. He wrote something, just
a month ago, where he attackedformer Seber director, Peter
Marks, and said that, Mackeryand, um, Kennedy were right to
have, forced, Marx to resign.
(20:56):
He attacked him in particularon his, policies on, COVID
and also on what we werejust talking about on, uh,
Marx's policies on, Elevidys,more recently in April.
He published a, a paper callingfor a reevaluation of the use
of talc in pharmaceuticalsand in other products.
That paper was publishedin a journal that Bachar
(21:18):
and McCarey founded.
interestingly, the paperwas submitted on April nine,
was published on April 10,which doesn't suggest that
there was, a traditionalkind of, peer review.
and he's, he's been veryactive in the last couple of
years, on the Stanford campus.
he, put up the money topay for a conference last
year, about, COVID issues.
(21:39):
and he moderated oneof the panels there.
Ery, Scott Atlas, who was avery controversial figure in,
um, COVID, Prasad and others,uh, spoke at that conference.
One of the things that I foundthat was really interesting,
in Ted Marsh's comments ofthe conference is that it
seemed to suggest, that hehas a, has views about the
(21:59):
off-label promotion of drugs.
He didn't actually come out andsay that, but in some of his
questions, it seemed to me that.
he was suggesting that hebelieves that FDA, has been
too restrictive, in, um,regulating off-label, comments
that, drug companies have made.
you know, which is interestingbecause all of the COVID,
um, issues really aren't.
(22:19):
Terribly relevant, um, toc you know, vaccines, come
under C but if you thinkabout how has his experience,
in this, anti establishmentCOVID world, how is that
going to, color or affectthe decisions that he makes?
At c You could imagine thatit might, might happen around,
speech issues in general.
(22:40):
Promotion of off-label ideas,uh, off-label truthful.
a non misleadingoff-label information
could be interesting.
Just wrapping up, one otherthing that he said that I
found interesting when hewas kind of talking about his
philosophy or his, the waythat he looks at the world,
he talked a a lot about thefact that some, uh, medicines
started out as fringe ideas.
Things that people, um,laughed at and didn't
(23:02):
think were reasonableand then they ended up.
Being approved and, and,and, and proved, right?
In some cases people got,Nobel prizes for them.
So, it leads me, leads me tothink that he's going to bring
that kind of thinking Cedar.
Simone Fishburn (23:16):
It's interesting, Steve, because
as you point out, somuch of it seems to just
always come back to COVID.
And I say that becausethere's also a, a thread
in some of the HHS, new,officials that is quite
industry adverse or industrysuspicious, but that doesn't
(23:38):
seem to be the case here.
with Dr.
Tma, she's as, as Jeffpointed out, he's been
a CEO several times.
He's been quitein this interface.
And so So it's gonna beinteresting to see how that
sort of tension plays out.
Clearly, as you say, he's afavor of what you might call
the serendipity or the onesthat everybody didn't believe,
(24:00):
and they're the ones who,bring the goods in the end.
and being more open tothat, which I think a lot
of people might think thatFDA could be more open.
There's also this slightgrievance oriented.
We were treated badly in COVID.
Nobody listened tous in COVID angle.
That seems to be coloringand maybe dominant among
the, the new, new heads.
(24:22):
how do you think about that?
Steve Usdin (24:24):
it's interesting, you know, there, related to
that, there's this, there'sa lot of tensions right
in the way that, the Trumpadministration thinks about.
FDA and publichealth in general.
And one of the, the tensionsis between, you know, praising
the biopharmaceutical industry,wanting to promote it.
saying that FDA, commissionerEry has said several many
(24:45):
times that he thinks that FDAshould do, should get out of
the way of industry that it,has been too, um, restrictive.
And on the other hand, heand HHS Secretary Kennedy,
Really vilify industry.
And they say that, um, uh, thebig pharmas, corrupted FDA,
that they've corrupted medicaljournals, that they've done all
kinds of things that Kennedyand, ery think, are nefarious.
(25:08):
and, Kennedy said that FDA inthe past has been a sock puppet.
For industry.
So it's really interestingthat ery reached out to someone
who has got more experience,I think, at a higher level in
industry, certainly than anyoneelse who's ever run CDER.
And I actually would be hardpressed to find anybody who's
(25:29):
ever worked at, FDA, who'shad the kind of, um, senior
positions and been involvedin the development of so
many drugs, as Tid Marsh.
and it's interesting alsobecause among RFK junior
supporters, the, the MAHAmovement, there's already a kind
of a backlash of if you lookat their websites and listen
(25:50):
to their podcasts, some of themare highly critical, for example
of in a Prasad and say that,uh, he's not adhering to the
precepts of the Maha movement.
and they've, they've urged,ery to get rid of him, which
I think is extraordinarilyunlikely to happen.
Ery seems to be, reallydelighted with Prasad.
and the other thing to thinkabout, I think, two things.
One is this appointmentand Prasad's appointment.
(26:13):
They, they represent, a kindof politicization of the
leadership of these centers.
There's always been a politicalelement to it, but I think
that this makes it much moreexplicitly so and it makes
me wonder how long theirtenures are going to be.
If there's a new commissionerwho has a different
political agenda and viewsthe world differently,
(26:35):
is that commissionergoing to change out?
The, center directorstraditionally, that hasn't
happened, but we've seen alot of tradition go right
out the window recently.
So, it'll be veryinteresting to see whether
that's the case here.
Jeff Cranmer (26:49):
Okay, thanks for that.
Steve and Steve's story,you'll be able to find at
BioCentury podcast.com.
And we will be, uh,closely watching, how
things, shake out at Cedar.
Let's do a little finance.
Bio.
Steven Hanson recentlycompleted his look at the third
(27:10):
quarter public markets, hispreview of the third quarter
public markets for biotech.
You can catch up on thatby tuning into last week's
BioCentury this week podcast.
And on the heels of that,uh, last week our director
of research, Meredith DurkinWolf, assessed how venture
(27:31):
financing has been in thefirst half of the year.
Selena, you, uh, edited thatand so much more while I
was, off traipsing around,uh, Spain eating pincho and
tapas and drinking kava and,uh, reluctantly coming back.
what did you learn?
Selina Koch (27:49):
Yeah.
Well these were, um, wepublished a couple of
what we called data bytes.
These are just smallbytes, one chart each, a
little bit of dialogue.
and the first oneI looked at just.
Total venture, you know, anyseries, any disclosed venture
in the first half of the yearcompared to the first half
of 20, 24 or 23, et cetera.
Going back to 2016.
And I think the big thingthat stood out to was
(28:12):
that the number of venturerounds, was the lowest
in that 10 year period.
So there was a slowdownin the, Raising of rounds.
it was not the smallestfirst half in that time
period for the amount raised.
2016 and 2017 were lower.
(28:33):
but that's because it seems theaverage round size was bigger.
which, may not be surprisingif you've been following
our coverage for a while.
it's, you know, the IPN windowremains pretty much closed.
Companies have to stayprivate for, for longer.
And investors, yeah, arekind of sometimes forced to
pick winners and, and to keepfunding those and keep those
(28:54):
companies moving along private.
so that could be one factorbehind that, observation.
Jeff Cranmer (29:00):
interesting.
Selena, uh, I'm, I know, uh,Meredith also took a look at
seed and Series A financings andcurious What we learned there.
Selina Koch (29:12):
Yeah, similar but different.
so if you look at, again,the number of seed and series
A rounds in the first halfversus other first halfs.
It's this been going downand down and down since
2021 with 20 five's.
First half being thesmallest in the 10 years.
Okay, so that part is the same.
But the amount raised inthe first half actually
(29:33):
was more than in 2024.
Um, it was pretty,pretty healthy.
and then when she looked, youknow, into the individual rounds
and figured, you know, tryingto figure this out, it seems
that 25% of the total raisedcame from four companies.
So.
Mega rounds.
These continue to be a trend.
We, we keep seeing them.
(29:55):
Uh, so we recorded inBioCentury 35 Cedar Series
a rounds of at least 200million in the past decade.
Four of them occurred inthe first half of 2025.
so that's, that was tyingthe first half of 23 for
the most in any half year.
and it was the largest total.
(30:15):
From those big mega rounds.
Jeff Cranmer (30:17):
And what's a mega round?
Are we,
Selina Koch (30:20):
In this case.
Jeff Cranmer (30:20):
million or where,
Selina Koch (30:21):
In this case, it was two 200 million or
Jeff Cranmer (30:23):
we're doing 200 million now.
Yeah.
Back in the day itwas a hundred million.
Selina Koch (30:27):
yeah.
Right.
Mega gets getting bigger.
Jeff Cranmer (30:30):
So, yeah, even, even, it's interesting, even in
these, you know, obviously verydifficult times for financing.
It's, uh, good to see that,the early stage companies
still getting funded
Selina Koch (30:41):
Yeah, well I think the take home is fewer
of them are getting funded,but the ones who are getting
funded are fund getting fundedfor a longer runway just
because that's what it takes.
Jeff Cranmer (30:50):
Interesting.
Okay, well, those data bytes,uh, up on BioCentury podcast.com
or BioCentury dot com, ifyou're a. Subscriber, you can
go there and, uh, access thatand appreciate you tuning in.
will be back next week, asusual, and, uh, later this week.
Don't forget to turninto our sister podcast.
(31:11):
later this week we will havepresident and CEO, John Lapore
of profound Therapeuticsin conversation with editor
in chief Simone Fishburn.
Kendall Square Orchestraprovides the music for about
a century This week, thegroup connects science and
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And other members of theGreater Boston community to
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AI-generated transcript.
Alanna Farro (00:02):
BioCentury This Week is brought to
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every phase of drug development.
(00:45):
Last week public disclosurethat a sarepta gene therapy
had caused a third deathled FDA to ask the company
to stop distributingits DMD gene therapy.
A move the biotechcompany has resisted
the deaths and disputesbetween FDA and Sarepta.
(01:07):
Raise questions about thefuture of a a V gene therapies.
Jeff Cranmer (01:12):
The therapy Elevidys was the first and only
gene therapy approved for thefatal neuromuscular disease
on the latest BioCentury.
This week podcast BioCenturyanalysts take a look at
what the death means for thefuture of gene therapy plus.
At long last FDA hasselected a new director for
(01:36):
CDR, what do we know aboutStanford's George Tid Marsh?
We'll also take a look at somenumbers out of the world of
venture financing for biotech.
I'm Jeff Cranmer, and joiningme today on the BioCentury
Podcast are my colleagues.
Simone Fishburn (01:55):
Simon Fishburn, editor in chief.
Steve Usdin (01:57):
Steve Den Washington editor.
Selina Koch (01:59):
And Selena Koch, executive editor.
Jeff Cranmer (02:02):
Okay, Steve, I, I wanna jump right to you.
A lot has happened in thepast few days between FDA and
Sarepta regarding Elevidys,since the disclosure of the
third death of a patient.
in a Sarepta gene therapy trial,can you bring us up to speed?
Steve Usdin (02:22):
So, yeah.
So, okay.
So on, July 16,that would be like.
Last Wednesday, Sareptaannounced a major reorganization
laying off 500 employees,about 36% of its and
shifting its focus towardearly stage siRNA projects.
It also updated V and addedadding a black box warning.
(02:43):
That's a similar to labelsthat are on other a a V
gene vectors warning aboutpotential, liver toxicity
and, fatal liver, adverseeffects on Thursday evening.
BioCentury, I reported thatthere had been a third death
in a Sarepta trial that hasnever been disclosed previously.
(03:04):
It was a 52-year-old manwho was in a trial of a limb
girdle muscular dystrophy,gene therapy, from Sarepta.
He uses the same vectoras, Sarepta's DMD therapy.
The next day, otherreporters picked up on
that, um, expanded it, and,investors, picked up on it.
Also, there was alot of controversy.
(03:25):
Sarepta, issued astatement, had analyst q
and a in the statement.
And the q and a. They defendedtheir decision to not, disclose
the third death, previously, Soon Friday, there were several
actions that came out of FDA.
They, uh, placed a hold onall of Sarepta's limb girdle
muscular dystrophy trials.
(03:46):
They asked Sarepta tostop, distributing,
Elevidys, to any patients.
the company, which had announcedearlier in the week that it
was going to stop shippingelevators to non-ambulatory
patients because there hadbeen two previous deaths of
non-ambulatory DMD patientsRefused to stop shipping.
(04:06):
Elevidys this on a voluntarybasis to ambulatory
Duchenne muscular dystrophy.
Patients.
FDA also revoked the platformdesignation for the vector
that's used in, sarepta's limb,girdle muscular dystrophy, gene
therapies, and in Elevidys.
So that's where we are today.
Simone Fishburn (04:26):
Where to start with all of this?
Um, I think in a minuteI'm gonna bring in Selena.
Let's talk a little bit aboutthe implications for gene
therapies and a a v vectors.
I don't think you've saidthat to an a, a V vector, but
first of all, just clarify.
Um, for those who haven'tspent the weekend on Twitter
or wherever they're, orX or wherever they're
(04:47):
getting their information.
What is the reason thatit hadn't been disclosed?
I understand that Sarepta saidit's not materially relevant.
How has this come to light now?
And it seems in a way thatthis third, very, very
unfortunate death has actuallyjust catapulted the whole
company into a, a new phase.
(05:09):
I mean, it was clearly,as you say, it was having
layoffs and things.
It was clearly in sometrouble, but it, it just
seems to have compounded it.
Can you talk about that?
Steve Usdin (05:17):
Okay, so, so how's it come out now?
It came out now because I foundout about it and reported it.
if I hadn't found out aboutit and reported it, probably
somebody else would've,and it would've come out.
but certainly not atthe time that it did.
The company had nointention to make it public.
The companies reported it toFDA, so Sarepta told FDA that
(05:37):
there was an acute liver failurecase that was life-threatening.
They reported that, on June20th, 2025, that was from
their phase one trial of alimb girdle muscular dystrophy
therapy, and they followedup and notified FDA of the
death on July 3rd, 2025.
on Thursday morning, Sareptainformed patient advocates
(06:02):
of that death, but theydidn't announce it publicly.
when I found out about itand asked the company about
it, they said that they hadno intention or no timing
anyway, of making, a publicdisclosure of that death.
In their calls with analysts,they said that, they hadn't
disclosed it previouslyand that it wasn't material
because they had already.
(06:23):
Announced their intention todiscontinue that gene therapy.
I should also note that DougIngram, the CEO of Sarepta
said on that call that thefatal events associated
with their gene therapieswere very, very rare.
rare.
And in my conversations with,patient advocates, with mothers
of children who've had, DMD.
(06:44):
They were very concernedby that comment.
Been about 140 non-ambulatorypatients who've received
Elevidys, and, twoof them have died.
The limb girdle therapy,that caused a death that
was in a phase one trial.
There were four patientsin that trial and one died.
That doesn't reallymeet people's definition
(07:04):
of very, very rare.
Simone Fishburn (07:06):
So I think something that you wrote about,
in your story, which I thoughtgot to the heart of it is what?
Parents of the patients who'vebeen on these therapies are
going through right now, givenwhat you've talked about are
not incredibly long odds.
I wouldn't call them short odds,but they are quite serious odds.
I don't know exactly thetime course, from when a
(07:27):
a, a patient is given thegene therapy that this kind
of toxicity might arise.
But maybe you can just channelwhat you've hearing from the
patient community in terms ofwhat they're going through.
Steve Usdin (07:37):
Well, one of the, parents told me that living
with a child who's received.
This gene therapy isa 24 7 panic attack.
the timing is severalweeks to months after,
receiving the therapy.
We don't know how manynon-ambulatory patients have
received Elevidys a month,two months, three months out.
(07:57):
but some of themcer certainly have.
Um, Roche and June saidthat worldwide there have
been about 140 to 150 boyswho are non-ambulatory
who'd received the therapy.
Since its approval in the UnitedStates, since its accelerated
approval for the non-ambulatorypopulation, that's how many
non-ambulatory patients havereceived it, given the fact that
there's always a lag between anapproval and when gene therapies
(08:21):
start to get distributed.
It's quite likely thata substantial portion of
those would've been inthe last year, certainly.
and that's got to be causing,and it is causing, that's
what advocates told me.
It is causing a greatdeal of anxiety, among the
families of the, of theboys who have received it.
Simone Fishburn (08:38):
Selena, maybe you wanna jump in now.
Perhaps you can extend thisconversation to the implications
for a a V gene therapies morebroadly and that technology.
Selina Koch (08:50):
Well, liver toxicity from a a v
gene therapies that'sa known risk, right?
This is, not new.
and it really has nothingto do with whether a patient
is ambulatory or whetherthey can no longer walk.
Right?
there's not a biologicalconnection to that distinction.
It's like some.
False shorthand.
So what we know from genetherapies of the past and from
(09:13):
this gene therapy is that it'sabout dose and it's about the
state of somebody's liver beforethey receive the gene therapy.
So older patients who havegrown more and are way more tend
to be non-ambulatory, right?
And they need higher doses.
I don't know if anybodyremembers, but four or five
years ago, Astellas reportedfour patient deaths in a
(09:36):
gene therapy trial for adifferent severe rare disease
that causes progressivemuscle weakening, right?
That was X-linkedmyotubular myopathy.
And those were veryyoung patients, but that
disease, you know, overtime it involves a liver.
So what they realized thereis, okay, well if you're having
some kind of liver dysfunctionbefore, it's probably not
(09:59):
a great therapy for you.
But now in that case, theytracked the, the 20 patients
who lived for a coupleyears and they found out
it was highly effective.
You had, all of those patientswere, none of them could sit
unassisted before that trial.
All of them were on ventilators.
Something like three quartersof them came off ventilators.
All of them could sit upand some of them could
(10:20):
even walk unassisted.
in a scenario like that,you take all of that in
and you're like, are therisks worth the benefits?
you know, maybe, butElevidys doesn't have the
luxury of really strongefficacy data like that.
Um, but when it comes tofuture gene therapies,
what does it mean?
It means we need to takestock of all of these
gene therapies, right?
Not just this one.
And think about what havewe learned across the board
(10:43):
about are there thresholdsfor preexisting liver talk?
are there thresholds for weightand dose that can guide who
can successfully, you know whois at lowest risk and are the
best candidates for receivingbenefit from gene therapies.
Steve Usdin (11:00):
the thing about this one that's so disheartening
is that not only were thesedeaths predictable, but
they were predicted, right?
it was Anticipated thatthis could happen when, when
Elevidys was approved for thenon-ambulatory population,
which as you say that again,it's not about the fact that
they're non-ambulatory, it'sthe fact that they're older,
they've had the diseaselonger, they're larger and
(11:22):
they need larger doses.
But it was predicted that thiswas a, a major safety risk and
they, they went ahead with it.
Anyway.
FDA went ahead with it.
The company went ahead with it.
And also it has to bepointed out that there
was little of any efficacydata in the non-ambulatory
population to support arisk benefit calculation.
Simone Fishburn (11:43):
Steve, I wanted to ask you, so we know
today from, the SEC filing ofSarepta that FDA has withdrawn
its platform designation fora a v that it had granted
not that long ago actually.
perhaps you can talk a littlebit about what you would've
wanted to see from an FDA onthis whole front related to
(12:08):
this particular finding, andthen what you would like to see
from them going forward on this.
Steve Usdin (12:12):
Well first I think they need to have
a lot more transparency.
This administration talksa lot about transparency.
They need to have a lotmore transparency around
their decision making.
Here in the statement thatthey put out they being FDA
on Friday, they referencednew safety information that
it caused them to take theactions that they took.
Well.
there doesn't seem to be a goodcase for saying it was new.
(12:33):
They knew aboutthis some time ago.
The only thing that seemsto have been new is the fact
that the rest of us found outabout it and there was a lot
of concern that was raised.
So I think thereneeds to be a lot more
transparency around that.
And then I think there alsoneeds to be transparency around
the approval of the platformdesignation for Sarepta, for
this gene therapy vector.
(12:54):
Why did FDA do it?
What was the process that theywent through in thinking about
it and, how are they gonnathink about this, um, platform
designation going forward?
What are the kind ofcriteria that they're gonna
apply for determining whatproducts should get it,
what products shouldn't get?
And finally I'd say, you know,speaking to what, um, Selena
(13:14):
said, you know, the, the otherreally big concern is, is, is
there gonna be an overreactionhere, by FDA, by regulators, and
almost certainly by investors.
and does that, is that goingto jeopardize the whole field
of gene therapy, in ways that,arguably aren't necessary
and that in some ways, youknow, mimic what happened.
(13:35):
in the past when we had a tragicdeath, Jesse Gelsinger death,
that really, set the field backfor a decade, there was really
a, like a decade where there wasimpossible to do, r and d or to
get any investment in the field.
I think that that would be.
Just as tragic as havingthese, deaths that have
happened that were avoidable.
Selina Koch (13:57):
Whole host of companies who are trying very
hard to solve the problem ofgene therapies, you know, just
getting stuck in the liver.
they're trying to bypass theliver and really concentrate
gene therapies in the targetorgan they're interested in.
It would be a shame if thatresearch we're unfundable, cause
that's the solution we need.
For this.
I just wonder if there were someinvestigation into all of these
(14:20):
gene therapies so far that wascomprehensive and got into the
nitty gritty if it might guideus a little bit and, how much
retargeting of liver would be asufficient amount to avoid most
of the toxicity, you know, canwe get any insight into that?
Steve Usdin (14:35):
And I think this is a case where really
the industry, academics, thepatient community, right?
And not just the United States,but around the world should
come together and ask thatquestion and other questions,
and then plot a strategy foranswering those questions.
Selina Koch (14:51):
Yeah.
And then on the platformthing, I don't know.
It's possible that theplatform designation just.
Isn't gonna apply anywhere.
I mean, Peter Marks had saidbefore he left, he didn't think
AAVs were a good use case.
Even though you can apply thatsame vector to other therapies.
There's so much bespokedesign that happens in the
whole thing, like the comcombination of the cargo
(15:12):
and the vector and whatnot.
and so, he had pointedto, gene editing therapies
delivered in LMPs as.
Possibly the one placewhere this designation
could shine, but, you know,we'll have to wait and see.
It's not, not looking like,there's gonna be a, a large
number of relevant use cases.
Jeff Cranmer (15:32):
Steve, final thoughts here.
Steve Usdin (15:34):
I think that this is a case where we're,
we're really gonna have tosee whether the patient,
community, FDA, and industry,whether everybody can,
resist the urge to, to simplypoint fingers at everyone.
Else and plot a, agood path forward.
And, and there's a lot at stake.
And, and you can't forgetthat it's, it's right in
(15:55):
front of our face, right?
the patients, the boyswho have DMD still need.
Safe, effective therapies,it's still a relentless
and terrible disease.
that needs to betreated with urgently.
There needs to be urgent stepsto, to, develop, products
that are gonna help them.
and there's so many otherdiseases that potentially
could benefit from genetherapy that what's really,
(16:17):
really needed now is this kindof, overarching, attempt.
to plot a, um, a safe andscientifically responsible
path forward, to be able to,to use what's arguably could
be, you know, tremendouslyimportant therapy and, and,
and in other conditions.
Of course, gene therapy hasbeen tremendously effective and
safe and, and has, saved lives.
Jeff Cranmer (16:39):
Okay, and that course is the subject of
Steve's editor's commentary,that we published on Friday.
You can find that atBioCentury podcast.com.
We're gonna take a quickbreak, and then we're going
to turn to FDA and learn.
About the new CDR director,
Alanna Farro (17:08):
Grand rounds.
Bio's r and d conference willmake its European debut in 2025.
Join us to debate keytranslational bottlenecks and
unlock scientific breakthroughswith commercial potential.
Connect with VCs, academicinnovators, and biopharma r
and d and BD leaders to explorecutting edge advancements
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(17:31):
therapeutic modalities,and enabling technologies.
Join us for the inauguralBioCentury Grand Grounds
Europe in Cambridge.
U.K. the September 17th to 19th.
Register and learn moreat BioCentury grand
grounds europe.com.
Jeff Cranmer (17:46):
We're back on the BioCentury This Week podcast.
Appreciate you tuning inand if you like what you're
hearing, please subscribeand leave us a comment or
a question and we'll try torespond on an upcoming episode.
Okay, On Monday, FDACommissioner Marty Macari named
Stanford Cancer biologist GeorgeTid Marsh as the new head of
(18:09):
the agency's Center for drugevaluation and Research T Marsh
completed training at Stanfordin pediatric oncology and Neo.
Natology and his adjunctfaculty of pediatrics and
neonatology is foundingco-director of Stanford's
(18:30):
Master of Translational Researchand Applied Medicine Program.
He's also founded in binCEO of multiple Biopharmas,
including Horizon, whichAmgen acquired for 28 billion
a couple of years back.
Tid Marsh Succeeds.
Jacqueline Corrigan Curry.
The now former actingdirector of CDR, PO.
(18:52):
She held since Januarywhen, Patricia Cone stepped
down from the position.
Steve, of course.
Uh, as everyone knows, youfollow FDA quite closely.
What are you hearingabout Dr. Tidmarsh?
Steve Usdin (19:07):
it's really interesting.
First, the name cameout of the blue.
It really surpriseda lot of people.
It surprised people, seniorpeople at FDA and people
who watch FDA closely.
He has, as you mentioned,he has deep experience
developing drugs and industry.
He's trained as apediatric oncologist.
The other thing that you have toknow about him is that he's been
a very, very strong and adamantsupporter of anti-establishment
(19:31):
perspectives on COVID-19.
and really Dr. McCarey,with, Robert F. Kennedy Jr.
The HHS secretary, everythingseems to come back to COVID-19
in one way or another,but more on his resume.
Okay.
So you mentioned that, he has.
In biopharma, he was,president and CEO of La
(19:51):
Jolla Pharmaceutical.
When he was there, he helped,discover and, develop an
angiotensin two, drug forthe treatment of shock.
That was approved.
he was founder andCEO of Horizon Pharma.
As you mentioned, horizonwas, sold to Amgen for
28, billion dollars.
He also founded a company calledThreshold Pharmaceuticals.
(20:12):
And he is held senior positionsat Coulter Pharmaceuticals,
which was acquired by GlaxoSmithKlein Ceq, and other companies.
he led clinical developmentof Bexar and Doxyl, to
FDA, anti-cancer drugs.
he is also known as I said,for his, anti-establishment,
views on, COVID.
He's very, close personallywith Dr. McCarey, with, Vinad,
(20:35):
the CBER, director and withJay Charya, the NIH director.
he's praised, Robert F. KennedyJr. He wrote something, just
a month ago, where he attackedformer Seber director, Peter
Marks, and said that, Mackeryand, um, Kennedy were right to
have, forced, Marx to resign.
(20:56):
He attacked him in particularon his, policies on, COVID
and also on what we werejust talking about on, uh,
Marx's policies on, Elevidys,more recently in April.
He published a, a paper callingfor a reevaluation of the use
of talc in pharmaceuticalsand in other products.
That paper was publishedin a journal that Bachar
(21:18):
and McCarey founded.
interestingly, the paperwas submitted on April nine,
was published on April 10,which doesn't suggest that
there was, a traditionalkind of, peer review.
and he's, he's been veryactive in the last couple of
years, on the Stanford campus.
he, put up the money topay for a conference last
year, about, COVID issues.
(21:39):
and he moderated oneof the panels there.
Ery, Scott Atlas, who was avery controversial figure in,
um, COVID, Prasad and others,uh, spoke at that conference.
One of the things that I foundthat was really interesting,
in Ted Marsh's comments ofthe conference is that it
seemed to suggest, that hehas a, has views about the
(21:59):
off-label promotion of drugs.
He didn't actually come out andsay that, but in some of his
questions, it seemed to me that.
he was suggesting that hebelieves that FDA, has been
too restrictive, in, um,regulating off-label, comments
that, drug companies have made.
you know, which is interestingbecause all of the COVID,
um, issues really aren't.
(22:19):
Terribly relevant, um, toc you know, vaccines, come
under C but if you thinkabout how has his experience,
in this, anti establishmentCOVID world, how is that
going to, color or affectthe decisions that he makes?
At c You could imagine thatit might, might happen around,
speech issues in general.
(22:40):
Promotion of off-label ideas,uh, off-label truthful.
a non misleadingoff-label information
could be interesting.
Just wrapping up, one otherthing that he said that I
found interesting when hewas kind of talking about his
philosophy or his, the waythat he looks at the world,
he talked a a lot about thefact that some, uh, medicines
started out as fringe ideas.
Things that people, um,laughed at and didn't
(23:02):
think were reasonableand then they ended up.
Being approved and, and,and, and proved, right?
In some cases people got,Nobel prizes for them.
So, it leads me, leads me tothink that he's going to bring
that kind of thinking Cedar.
Simone Fishburn (23:16):
It's interesting, Steve, because
as you point out, somuch of it seems to just
always come back to COVID.
And I say that becausethere's also a, a thread
in some of the HHS, new,officials that is quite
industry adverse or industrysuspicious, but that doesn't
(23:38):
seem to be the case here.
with Dr.
Tma, she's as, as Jeffpointed out, he's been
a CEO several times.
He's been quitein this interface.
And so So it's gonna beinteresting to see how that
sort of tension plays out.
Clearly, as you say, he's afavor of what you might call
the serendipity or the onesthat everybody didn't believe,
(24:00):
and they're the ones who,bring the goods in the end.
and being more open tothat, which I think a lot
of people might think thatFDA could be more open.
There's also this slightgrievance oriented.
We were treated badly in COVID.
Nobody listened tous in COVID angle.
That seems to be coloringand maybe dominant among
the, the new, new heads.
(24:22):
how do you think about that?
Steve Usdin (24:24):
it's interesting, you know, there, related to
that, there's this, there'sa lot of tensions right
in the way that, the Trumpadministration thinks about.
FDA and publichealth in general.
And one of the, the tensionsis between, you know, praising
the biopharmaceutical industry,wanting to promote it.
saying that FDA, commissionerEry has said several many
(24:45):
times that he thinks that FDAshould do, should get out of
the way of industry that it,has been too, um, restrictive.
And on the other hand, heand HHS Secretary Kennedy,
Really vilify industry.
And they say that, um, uh, thebig pharmas, corrupted FDA,
that they've corrupted medicaljournals, that they've done all
kinds of things that Kennedyand, ery think, are nefarious.
(25:08):
and, Kennedy said that FDA inthe past has been a sock puppet.
For industry.
So it's really interestingthat ery reached out to someone
who has got more experience,I think, at a higher level in
industry, certainly than anyoneelse who's ever run CDER.
And I actually would be hardpressed to find anybody who's
(25:29):
ever worked at, FDA, who'shad the kind of, um, senior
positions and been involvedin the development of so
many drugs, as Tid Marsh.
and it's interesting alsobecause among RFK junior
supporters, the, the MAHAmovement, there's already a kind
of a backlash of if you lookat their websites and listen
(25:50):
to their podcasts, some of themare highly critical, for example
of in a Prasad and say that,uh, he's not adhering to the
precepts of the Maha movement.
and they've, they've urged,ery to get rid of him, which
I think is extraordinarilyunlikely to happen.
Ery seems to be, reallydelighted with Prasad.
and the other thing to thinkabout, I think, two things.
One is this appointmentand Prasad's appointment.
(26:13):
They, they represent, a kindof politicization of the
leadership of these centers.
There's always been a politicalelement to it, but I think
that this makes it much moreexplicitly so and it makes
me wonder how long theirtenures are going to be.
If there's a new commissionerwho has a different
political agenda and viewsthe world differently,
(26:35):
is that commissionergoing to change out?
The, center directorstraditionally, that hasn't
happened, but we've seen alot of tradition go right
out the window recently.
So, it'll be veryinteresting to see whether
that's the case here.
Jeff Cranmer (26:49):
Okay, thanks for that.
Steve and Steve's story,you'll be able to find at
BioCentury podcast.com.
And we will be, uh,closely watching, how
things, shake out at Cedar.
Let's do a little finance.
Bio.
Steven Hanson recentlycompleted his look at the third
(27:10):
quarter public markets, hispreview of the third quarter
public markets for biotech.
You can catch up on thatby tuning into last week's
BioCentury this week podcast.
And on the heels of that,uh, last week our director
of research, Meredith DurkinWolf, assessed how venture
(27:31):
financing has been in thefirst half of the year.
Selena, you, uh, edited thatand so much more while I
was, off traipsing around,uh, Spain eating pincho and
tapas and drinking kava and,uh, reluctantly coming back.
what did you learn?
Selina Koch (27:49):
Yeah.
Well these were, um, wepublished a couple of
what we called data bytes.
These are just smallbytes, one chart each, a
little bit of dialogue.
and the first oneI looked at just.
Total venture, you know, anyseries, any disclosed venture
in the first half of the yearcompared to the first half
of 20, 24 or 23, et cetera.
Going back to 2016.
And I think the big thingthat stood out to was
(28:12):
that the number of venturerounds, was the lowest
in that 10 year period.
So there was a slowdownin the, Raising of rounds.
it was not the smallestfirst half in that time
period for the amount raised.
2016 and 2017 were lower.
(28:33):
but that's because it seems theaverage round size was bigger.
which, may not be surprisingif you've been following
our coverage for a while.
it's, you know, the IPN windowremains pretty much closed.
Companies have to stayprivate for, for longer.
And investors, yeah, arekind of sometimes forced to
pick winners and, and to keepfunding those and keep those
(28:54):
companies moving along private.
so that could be one factorbehind that, observation.
Jeff Cranmer (29:00):
interesting.
Selena, uh, I'm, I know, uh,Meredith also took a look at
seed and Series A financings andcurious What we learned there.
Selina Koch (29:12):
Yeah, similar but different.
so if you look at, again,the number of seed and series
A rounds in the first halfversus other first halfs.
It's this been going downand down and down since
2021 with 20 five's.
First half being thesmallest in the 10 years.
Okay, so that part is the same.
But the amount raised inthe first half actually
(29:33):
was more than in 2024.
Um, it was pretty,pretty healthy.
and then when she looked, youknow, into the individual rounds
and figured, you know, tryingto figure this out, it seems
that 25% of the total raisedcame from four companies.
So.
Mega rounds.
These continue to be a trend.
We, we keep seeing them.
(29:55):
Uh, so we recorded inBioCentury 35 Cedar Series
a rounds of at least 200million in the past decade.
Four of them occurred inthe first half of 2025.
so that's, that was tyingthe first half of 23 for
the most in any half year.
and it was the largest total.
(30:15):
From those big mega rounds.
Jeff Cranmer (30:17):
And what's a mega round?
Are we,
Selina Koch (30:20):
In this case.
Jeff Cranmer (30:20):
million or where,
Selina Koch (30:21):
In this case, it was two 200 million or
Jeff Cranmer (30:23):
we're doing 200 million now.
Yeah.
Back in the day itwas a hundred million.
Selina Koch (30:27):
yeah.
Right.
Mega gets getting bigger.
Jeff Cranmer (30:30):
So, yeah, even, even, it's interesting, even in
these, you know, obviously verydifficult times for financing.
It's, uh, good to see that,the early stage companies
still getting funded
Selina Koch (30:41):
Yeah, well I think the take home is fewer
of them are getting funded,but the ones who are getting
funded are fund getting fundedfor a longer runway just
because that's what it takes.
Jeff Cranmer (30:50):
Interesting.
Okay, well, those data bytes,uh, up on BioCentury podcast.com
or BioCentury dot com, ifyou're a. Subscriber, you can
go there and, uh, access thatand appreciate you tuning in.
will be back next week, asusual, and, uh, later this week.
Don't forget to turninto our sister podcast.
(31:11):
later this week we will havepresident and CEO, John Lapore
of profound Therapeuticsin conversation with editor
in chief Simone Fishburn.
Kendall Square Orchestraprovides the music for about
a century This week, thegroup connects science and
technology professionals.
And other members of theGreater Boston community to
(31:33):
collaborate, innovate, andinspire through music while
supporting causes relatedto healthcare and education.
Alanna (31:41):
Would like to thank IQVIA Biotech for supporting
the bio this week podcast.
To learn more about how IQVIABiotech can help you turn your
vision into venture capital.
Go to IQIAbiotech.com/visionaries.
I.
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