July 2, 2025 • 15 mins
This week on BioTech Nation, Robert Blum, CEO of CytoKinetics, joins me to talk about the company’s decades-long mission to keep hearts beating stronger and longer. With one heart drug under FDA review and more in the pipeline, CytoKinetics is working to address the full spectrum of heart failure, from weak hearts to overactive ones, and even exploring muscle health beyond the heart to support healthy aging.
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Episode Transcript
Available transcripts are automatically generated. Complete accuracy is not guaranteed.
Dr. Moira Gunn (00:11):
If you're alive, your heart is beating, and
there's no doubt you have tokeep it that way and for a very
long time. One South SanFrancisco company, Cytokinetics,
is focused on many aspects ofdoing exactly that. Robert Blum
is its CEO. Robert, welcome toBiotech Nation.
Robert Blum (00:34):
Thank you. Nice to be here. Thank you.
Dr. Moira Gunn (00:37):
Now essential equipment for all human life is
that we have hearts, and thesehearts have to keep beating. And
as with everything, there arethe genetic cards we are dealt
as important as the life we havelived and how it affects it. Now
cytokinesis is focused onconditions of the heart and to
(00:58):
frame this I'd like just to makean early note for the listeners
that you have drug candidates inthe last phase, that's phase
three, one with a successfulreadout as we say, a successful
data outcome and you're waitingfor the FDA's final say in
December as of this recordingand those on the inside would
(01:21):
call this a PDUFA date, butDecember is the go no go date
from the FDA like bring it tomarket or we might want
something else. Very excitingtime for you. But cytokines also
has a drug candidate in phasetwo and another one in phase one
and even more preclinical andresearch.
(01:41):
So there's a lot of shots at alot of goals here at this point
all having to do with themedical conditions of the heart.
So let's start remembering theheart is a muscle which pumps
blood through veins, keepingblood circulating throughout our
bodies. Let's start withconditions of the heart, and
first off, I'd like to hearabout heart failure. What is
(02:05):
heart failure?
Robert Blum (02:06):
Heart failure is the number one reason why people
over the age of 65 in thiscountry are hospitalized. It's
the number one driver ofMedicare costs, and our company
has been focused in this areafor many, many years.
Cytokinetics is celebrating itstwenty seventh year since we
(02:26):
formed this company back a whileago, and our scientists are
pioneers and leaders inunderstanding the mechanics of
how heart muscle pumps blood forthe benefit of patients, and how
we can address syndromes ofheart failure by enhancing the
(02:49):
pumping function or suppressingthe pumping function depending
on what may be the underlyingproblem. There are forms of
heart failure where your heartis impaired and can't pump
enough blood. There are forms ofheart failure where your heart
is hyperactive and contractingto a too great an extent, and
(03:13):
the therapeutic objective is tosuppress the hyper
contractility.
So our scientists are experts inthe mechanics of heart muscle
pumping function, and we'vedeveloped potential medicines
that either activate or inhibitthe protein that drives the
(03:36):
force production of your heart.And this has been the benefit of
quite a long story, long historyof research and development, but
we're very excited. The firstamongst these potential
medicines is sitting in front ofthe FDA awaiting a potential
approval later this year for aform of this disease called
(04:00):
hypertrophic cardiomyopathy, andAlready as you've mentioned,
we've seen clinical trialresults and evidence that
suggest that a cardiac myosininhibitor, a cardiac muscle
inhibitor, can increase exercisestamina and relieve the burden,
(04:24):
the symptom burden for thesepatients. What's happening with
this disease, absent a treatmentsuch as this, is patients are
short of breath. They're havingdifficulty climbing stairs,
walking to the end of thedriveway.
These are patients that livewith the looming threat of
(04:44):
sudden cardiac failure, so thiscreates not only a cardiac
syndrome, but a psychologicalone, if you will, and the goal
of treatment is to relieve thestress, relieve the burden,
increase exercise capacity andstamina, all things that can be
measured in clinical trials aswe have done over the course of
(05:07):
many years.
Dr. Moira Gunn (05:08):
Is this the thickening of the heart muscle,
or is that different?
Robert Blum (05:12):
That's right, it is a thickening. So hypertrophic
cardiomyopathy is a disease thathas a hereditary or genetic
linkage. There's a familial linkto this disease, and patients
who have hypertrophiccardiomyopathy can develop an
obstruction in their heart,which creates a high pressure
(05:36):
gradient, and that's becausethere is a thickening of the
heart muscle because it's in aconstant hyperactive state. So
one has a goal with a potentialnew medicine to inhibit the
contractility, to relax thatmuscle, to lessen the
thickening, relieve the pressuregradient, and be enabling of
(06:00):
these patients to live moreactive functional lives.
Dr. Moira Gunn (06:03):
I think it's a little difficult to separate out
some of the earlier or drivingconditions from the the overall
heart condition. I mean, manypeople say, oh, I have high
blood pressure. Well, I'm afraidI'm get I'm approaching high
blood pressure. I'm taking pillsfor it. That ends up creating a
(06:27):
heart failure condition or aheart condition, or is it which
one's the chicken and whichone's the egg?
Robert Blum (06:34):
Well, that's the interesting thing is it's a
difficult thing because there'sa circular feedback system here
that patients oftentimes willhave comorbidities. They may
have high blood pressure, theymay have problems with the
electrical rhythms of theirheart. All these things combine
(06:55):
together, oftentimes with othersyndromes such as diabetes or
metabolic disease, putting moreand more pressure on the heart.
So patients with heart failureoftentimes have complications
associated with otherconcomitant conditions. What we
(07:16):
unfortunately don't have in ourarmamentarium of medicines are a
lot of drugs that work directlyon the mechanical function of
the heart, and that's whereCytokinetics comes in.
Over twenty five years ofresearch has led us to a place
where we're developing potentialnew medicines that act directly
(07:38):
on the machinery of the heart toeither enhance or suppress its
function as would be beneficialto patients. So as a result of
that, we've developed a pipelineof medicines that we're
developing in different stagesof clinical trials. We hope to
have multiple new medicines forthose cardiologists who treat
(08:02):
conditions underlying heartfunction?
Dr. Moira Gunn (08:06):
Well I have to say as I study your clinical
trials, you begin to see thefine points of your heart. For
example, in one case, have aweakening of the heart, and so
you have a muscle activator. Inanother case, it's hyperactive.
(08:26):
So your heart's actingdifferently, and the hyperactive
one then needs an inhibitor.Take us through this.
It's almost like the same drugor the same idea for a drug.
Robert Blum (08:38):
We've developed three potential medicines all
directed to the same protein,the same enzyme called cardiac
myosin that is driving the forceproduction of heart cells. So
one of these is an activator,two of them are inhibitors, and
we can develop all three of themfor different conditions that
(09:01):
relate to heart musclemechanics. And in that way bring
forward a potential pipeline orportfolio of new medicines for
cardiologists that treat theseunderlying conditions.
Dr. Moira Gunn (09:16):
Now, one of your phase three trials is in the
pediatric space. Are heartconditions common in the
pediatric space, and is itdifferent other than the age of
the patient?
Robert Blum (09:29):
Unfortunately it is common, and initially we studied
adults with this condition ofhypertrophic cardiomyopathy, but
it is a genetic predisposition,and it can emerge in adolescents
or children. So now that we'veseen evidence to support the use
(09:49):
of our cardiac myosin inhibitorin adults, it's now incumbent
upon us to evaluate itspotential in adolescents, and
ultimately in pediatrics,because ultimately we hope all
of these patients may benefitfrom this innovation.
Dr. Moira Gunn (10:07):
Now in phase one, that's the first when you
go into humans, you have what'scalled a fast skeletal muscle
tropopin activator. I may nothave that pronounced correctly.
What is skeletal muscle, andwhat has that got to do with the
heart?
Robert Blum (10:25):
So very interesting, you should ask
that. So you have cardiacmuscle, you have skeletal
muscle, you have smooth muscle,and your various muscle types
have evolved alternative formsof these same proteins that
drive muscle contractilityoutside of the heart as well. So
we're developing an activator offast twitch skeletal troponin,
(10:52):
which is a regulator of skeletalmuscle contractility. And here
we've seen evidence thatactivating skeletal troponin can
produce more skeletal muscleforce and power, and also alter
time to muscle fatigue. So theseare not your cardiac drugs,
(11:15):
these are potential newmedicines for diseases of
neuromuscular pathophysiology.
Dr. Moira Gunn (11:22):
So you started with the heart, but that's just
where you started.
Robert Blum (11:26):
We started with the heart. We're extending this know
how or these innovations toskeletal muscle, and quite
frankly, this illuminates for uswhat we believe to be a pathway
to healthy aging over time. Ouraging demographics call for
(11:46):
potential new medicines that canbe enabling of us to live more
active and more functionallives. So how we approach active
daily living is rooted, webelieve, in muscle biology and
cytokines hopes to be both apioneer and a leader in areas of
(12:06):
healthy aging over time as well.
Dr. Moira Gunn (12:08):
Now I want to return to the current clinical
trials because you are enrollingin a number of them and all over
the world, mostly in The UnitedStates, but all over the world.
And what is it like to begenerally in one of these
clinical trials?
Robert Blum (12:28):
There are a number of visits, a number of
assessments that get performed,and patients are asked to
participate actively in measuresof muscle function and also
quality of life over thelongitudinal period of their
study, and our goal with theseclinical trials is to conduct an
(12:51):
experiment. Does the addition ofone of our experimental
medicines, when added tostandard of care, provide even
more therapeutic gain.
Dr. Moira Gunn (13:03):
I keep coming back to December. It may be
thumbs up, thumbs down. I keepthinking about this. It's twenty
seven years to be an overnightsuccess.
Robert Blum (13:17):
It's funny you should say that.
Dr. Moira Gunn (13:19):
Must be pretty daunting.
Robert Blum (13:21):
We oftentimes joke that nobody will ever accuse us
of being an overnight success. Istarted this company twenty
seven years ago with leadingacademic scientists, and we
industrialized the work that wasoccurring in their academic labs
at Stanford and UCSF and UCSD,and it's taken us this many
(13:43):
years to do all of thepioneering work that we've done,
both in our laboratories and inclinical studies around the
world, and it may come to afocal point, transformative as
we hope it can be, in December.This is a very exciting time.
We're a larger company now. Wehave a footprint all over the
world, but we're still a companyrooted in science.
(14:07):
Science is in our soul, and wedo believe that our patient
centricity has been the guidinglight for us all of these many
years. So being on the cusp ofpotentially enabling of one of
our medicines to be in the handsof physicians and patients, it's
an incredibly gratifying andfulfilling thing to think about.
Dr. Moira Gunn (14:28):
Well, we're pulling for you here at Biotech
Nation.
Robert Blum (14:33):
Thank you.
Dr. Moira Gunn (14:34):
Thank you.
Robert Blum (14:35):
Thank you very much.
Dr. Moira Gunn (14:35):
Thank you for coming in. Please come back,
give us an update.
Robert Blum (14:39):
I look forward to that and hopefully the next time
we have an opportunity to speakwe can be talking about FDA
approval for one of ourmedicines. I should also
mention, we're seeking approvalall over the world, in China,
also throughout Europe. Our goalis to ensure that our medicines
are available with equitableaccess regardless of geographic
(15:02):
borders.
Dr. Moira Gunn (15:03):
Robert Blum is the CEO of Cytokinetics. More
information is available on theweb at cytokinetics.com. That's
cyto,kinetics,cytokinetics.com.
If you're alive, your heart is beating, and
there's no doubt you have tokeep it that way and for a very
long time. One South SanFrancisco company, Cytokinetics,
is focused on many aspects ofdoing exactly that. Robert Blum
is its CEO. Robert, welcome toBiotech Nation.
Robert Blum (00:34):
Thank you. Nice to be here. Thank you.
Dr. Moira Gunn (00:37):
Now essential equipment for all human life is
that we have hearts, and thesehearts have to keep beating. And
as with everything, there arethe genetic cards we are dealt
as important as the life we havelived and how it affects it. Now
cytokinesis is focused onconditions of the heart and to
(00:58):
frame this I'd like just to makean early note for the listeners
that you have drug candidates inthe last phase, that's phase
three, one with a successfulreadout as we say, a successful
data outcome and you're waitingfor the FDA's final say in
December as of this recordingand those on the inside would
(01:21):
call this a PDUFA date, butDecember is the go no go date
from the FDA like bring it tomarket or we might want
something else. Very excitingtime for you. But cytokines also
has a drug candidate in phasetwo and another one in phase one
and even more preclinical andresearch.
(01:41):
So there's a lot of shots at alot of goals here at this point
all having to do with themedical conditions of the heart.
So let's start remembering theheart is a muscle which pumps
blood through veins, keepingblood circulating throughout our
bodies. Let's start withconditions of the heart, and
first off, I'd like to hearabout heart failure. What is
(02:05):
heart failure?
Robert Blum (02:06):
Heart failure is the number one reason why people
over the age of 65 in thiscountry are hospitalized. It's
the number one driver ofMedicare costs, and our company
has been focused in this areafor many, many years.
Cytokinetics is celebrating itstwenty seventh year since we
(02:26):
formed this company back a whileago, and our scientists are
pioneers and leaders inunderstanding the mechanics of
how heart muscle pumps blood forthe benefit of patients, and how
we can address syndromes ofheart failure by enhancing the
(02:49):
pumping function or suppressingthe pumping function depending
on what may be the underlyingproblem. There are forms of
heart failure where your heartis impaired and can't pump
enough blood. There are forms ofheart failure where your heart
is hyperactive and contractingto a too great an extent, and
(03:13):
the therapeutic objective is tosuppress the hyper
contractility.
So our scientists are experts inthe mechanics of heart muscle
pumping function, and we'vedeveloped potential medicines
that either activate or inhibitthe protein that drives the
(03:36):
force production of your heart.And this has been the benefit of
quite a long story, long historyof research and development, but
we're very excited. The firstamongst these potential
medicines is sitting in front ofthe FDA awaiting a potential
approval later this year for aform of this disease called
(04:00):
hypertrophic cardiomyopathy, andAlready as you've mentioned,
we've seen clinical trialresults and evidence that
suggest that a cardiac myosininhibitor, a cardiac muscle
inhibitor, can increase exercisestamina and relieve the burden,
(04:24):
the symptom burden for thesepatients. What's happening with
this disease, absent a treatmentsuch as this, is patients are
short of breath. They're havingdifficulty climbing stairs,
walking to the end of thedriveway.
These are patients that livewith the looming threat of
(04:44):
sudden cardiac failure, so thiscreates not only a cardiac
syndrome, but a psychologicalone, if you will, and the goal
of treatment is to relieve thestress, relieve the burden,
increase exercise capacity andstamina, all things that can be
measured in clinical trials aswe have done over the course of
(05:07):
many years.
Dr. Moira Gunn (05:08):
Is this the thickening of the heart muscle,
or is that different?
Robert Blum (05:12):
That's right, it is a thickening. So hypertrophic
cardiomyopathy is a disease thathas a hereditary or genetic
linkage. There's a familial linkto this disease, and patients
who have hypertrophiccardiomyopathy can develop an
obstruction in their heart,which creates a high pressure
(05:36):
gradient, and that's becausethere is a thickening of the
heart muscle because it's in aconstant hyperactive state. So
one has a goal with a potentialnew medicine to inhibit the
contractility, to relax thatmuscle, to lessen the
thickening, relieve the pressuregradient, and be enabling of
(06:00):
these patients to live moreactive functional lives.
Dr. Moira Gunn (06:03):
I think it's a little difficult to separate out
some of the earlier or drivingconditions from the the overall
heart condition. I mean, manypeople say, oh, I have high
blood pressure. Well, I'm afraidI'm get I'm approaching high
blood pressure. I'm taking pillsfor it. That ends up creating a
(06:27):
heart failure condition or aheart condition, or is it which
one's the chicken and whichone's the egg?
Robert Blum (06:34):
Well, that's the interesting thing is it's a
difficult thing because there'sa circular feedback system here
that patients oftentimes willhave comorbidities. They may
have high blood pressure, theymay have problems with the
electrical rhythms of theirheart. All these things combine
(06:55):
together, oftentimes with othersyndromes such as diabetes or
metabolic disease, putting moreand more pressure on the heart.
So patients with heart failureoftentimes have complications
associated with otherconcomitant conditions. What we
(07:16):
unfortunately don't have in ourarmamentarium of medicines are a
lot of drugs that work directlyon the mechanical function of
the heart, and that's whereCytokinetics comes in.
Over twenty five years ofresearch has led us to a place
where we're developing potentialnew medicines that act directly
(07:38):
on the machinery of the heart toeither enhance or suppress its
function as would be beneficialto patients. So as a result of
that, we've developed a pipelineof medicines that we're
developing in different stagesof clinical trials. We hope to
have multiple new medicines forthose cardiologists who treat
(08:02):
conditions underlying heartfunction?
Dr. Moira Gunn (08:06):
Well I have to say as I study your clinical
trials, you begin to see thefine points of your heart. For
example, in one case, have aweakening of the heart, and so
you have a muscle activator. Inanother case, it's hyperactive.
(08:26):
So your heart's actingdifferently, and the hyperactive
one then needs an inhibitor.Take us through this.
It's almost like the same drugor the same idea for a drug.
Robert Blum (08:38):
We've developed three potential medicines all
directed to the same protein,the same enzyme called cardiac
myosin that is driving the forceproduction of heart cells. So
one of these is an activator,two of them are inhibitors, and
we can develop all three of themfor different conditions that
(09:01):
relate to heart musclemechanics. And in that way bring
forward a potential pipeline orportfolio of new medicines for
cardiologists that treat theseunderlying conditions.
Dr. Moira Gunn (09:16):
Now, one of your phase three trials is in the
pediatric space. Are heartconditions common in the
pediatric space, and is itdifferent other than the age of
the patient?
Robert Blum (09:29):
Unfortunately it is common, and initially we studied
adults with this condition ofhypertrophic cardiomyopathy, but
it is a genetic predisposition,and it can emerge in adolescents
or children. So now that we'veseen evidence to support the use
(09:49):
of our cardiac myosin inhibitorin adults, it's now incumbent
upon us to evaluate itspotential in adolescents, and
ultimately in pediatrics,because ultimately we hope all
of these patients may benefitfrom this innovation.
Dr. Moira Gunn (10:07):
Now in phase one, that's the first when you
go into humans, you have what'scalled a fast skeletal muscle
tropopin activator. I may nothave that pronounced correctly.
What is skeletal muscle, andwhat has that got to do with the
heart?
Robert Blum (10:25):
So very interesting, you should ask
that. So you have cardiacmuscle, you have skeletal
muscle, you have smooth muscle,and your various muscle types
have evolved alternative formsof these same proteins that
drive muscle contractilityoutside of the heart as well. So
we're developing an activator offast twitch skeletal troponin,
(10:52):
which is a regulator of skeletalmuscle contractility. And here
we've seen evidence thatactivating skeletal troponin can
produce more skeletal muscleforce and power, and also alter
time to muscle fatigue. So theseare not your cardiac drugs,
(11:15):
these are potential newmedicines for diseases of
neuromuscular pathophysiology.
Dr. Moira Gunn (11:22):
So you started with the heart, but that's just
where you started.
Robert Blum (11:26):
We started with the heart. We're extending this know
how or these innovations toskeletal muscle, and quite
frankly, this illuminates for uswhat we believe to be a pathway
to healthy aging over time. Ouraging demographics call for
(11:46):
potential new medicines that canbe enabling of us to live more
active and more functionallives. So how we approach active
daily living is rooted, webelieve, in muscle biology and
cytokines hopes to be both apioneer and a leader in areas of
(12:06):
healthy aging over time as well.
Dr. Moira Gunn (12:08):
Now I want to return to the current clinical
trials because you are enrollingin a number of them and all over
the world, mostly in The UnitedStates, but all over the world.
And what is it like to begenerally in one of these
clinical trials?
Robert Blum (12:28):
There are a number of visits, a number of
assessments that get performed,and patients are asked to
participate actively in measuresof muscle function and also
quality of life over thelongitudinal period of their
study, and our goal with theseclinical trials is to conduct an
(12:51):
experiment. Does the addition ofone of our experimental
medicines, when added tostandard of care, provide even
more therapeutic gain.
Dr. Moira Gunn (13:03):
I keep coming back to December. It may be
thumbs up, thumbs down. I keepthinking about this. It's twenty
seven years to be an overnightsuccess.
Robert Blum (13:17):
It's funny you should say that.
Dr. Moira Gunn (13:19):
Must be pretty daunting.
Robert Blum (13:21):
We oftentimes joke that nobody will ever accuse us
of being an overnight success. Istarted this company twenty
seven years ago with leadingacademic scientists, and we
industrialized the work that wasoccurring in their academic labs
at Stanford and UCSF and UCSD,and it's taken us this many
(13:43):
years to do all of thepioneering work that we've done,
both in our laboratories and inclinical studies around the
world, and it may come to afocal point, transformative as
we hope it can be, in December.This is a very exciting time.
We're a larger company now. Wehave a footprint all over the
world, but we're still a companyrooted in science.
(14:07):
Science is in our soul, and wedo believe that our patient
centricity has been the guidinglight for us all of these many
years. So being on the cusp ofpotentially enabling of one of
our medicines to be in the handsof physicians and patients, it's
an incredibly gratifying andfulfilling thing to think about.
Dr. Moira Gunn (14:28):
Well, we're pulling for you here at Biotech
Nation.
Robert Blum (14:33):
Thank you.
Dr. Moira Gunn (14:34):
Thank you.
Robert Blum (14:35):
Thank you very much.
Dr. Moira Gunn (14:35):
Thank you for coming in. Please come back,
give us an update.
Robert Blum (14:39):
I look forward to that and hopefully the next time
we have an opportunity to speakwe can be talking about FDA
approval for one of ourmedicines. I should also
mention, we're seeking approvalall over the world, in China,
also throughout Europe. Our goalis to ensure that our medicines
are available with equitableaccess regardless of geographic
(15:02):
borders.
Dr. Moira Gunn (15:03):
Robert Blum is the CEO of Cytokinetics. More
information is available on theweb at cytokinetics.com. That's
cyto,kinetics,cytokinetics.com.
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