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 therapy appears to improve risk levels and functional classes in certain patients, and stabilize other patients.

Cereno Scientific says its histone deacetylase (HDAC) inhibitor, CS1, led to stabilization or improvement in the majority of patients who participated in their phase IIa study.

“Key results from the study demonstrate both that the primary endpoint was met with good safety and tolerability of CS1 in patients with PAH, and, in addition, we also show a positive impact already after 12 weeks on clinical parameters meaningful to patients and regulatory authorities,” said Sten R. Sörensen, the company’s chief executive, in a news  release.

PAH is driven by epigenetic mechanisms that lead to disease progression and patient deterioration. Remodeling of pulmonary small arteries leads to poor lung function, right heart failure, and even death, the company noted. The epigenetic mutations behind PAH have become a popular therapeutic target as drugmakers aim to improve the standard of care for the disease.

In this case, CS1 is designed to work through epigenetic modification by inhibiting HDAC. In doing so, Cereno hopes the therapy can reverse vascular remodeling of the small lung arteries.

As a phase 2a study with a primary goal of assessing safety and tolerability, the study had a small patient population. Investigators recruited 25 patients at 10 clinical sites in the US; 21 participants completed the study and were included in the analysis.

In terms of safety, Cereno said no patients had serious adverse events, none were hospitalized, and none died. They also found no clinically significant changes in drug-related platelets or bleeding, and no changes in participant’s liver lab values.

However, they also found that nine of the participants had an improved score on the REVEAL risk assessment after 12 weeks, and another six  patients had a stable risk score. In addition, seven patients had an improved functional class after therapy, and 11 more were stable. Two-thirds of patients had sustained reductions in mean pulmonary arterial pressure.

“Their improvements in health and well-being are encouraging and reflect the potential effectiveness of the therapy,” said study investigator Jason Guichard, M.D., Ph.D., of the University of South Carolina School of Medicine, in the news release.

HDAC inhibitors have largely been developed as cancer therapies, primarily for hematologic malignancies. However, in recent years, drug developers have explored their potential in a range of other diseases, including 

Cereno said it will now begin discussions with regulatory authorities in order to prepare a pivotal trial. They also said they have received approval from the FDA for an expanded access program for patients that have completed the phase IIa trial and saw positive results.

“The judgment here by physicians in the trial and by patients [who were] willing to participate is that they have seen some benefits already in this trial and don’t want to wait to get hold of the drug,” said Sörensen, in a webcast announcing the results. “We feel that that’s a very strong signal that there is something positive going on with the drug in these patients.”

The company says it hopes to get approval from the FDA for its pivotal trial within the first half of next year. One key question will be how the therapy will perform over a longer period than the initial 12 weeks of the phase 2a study. Rahul Agarwal, M.D., M.B.A, the company’s chief medical officer, said during the webcast that he is optimistic.

“If one keeps in mind that this is a progressive, continuous disease, we hope to see, of course, better and even more pronounced results over a longer treatment period,” he said.

The results come from a phase IIa safety and tolerability study. The drug’s developers say they are working with regulatory authorities in hopes of launching a pivotal trial.

Clinical

HDAC Inhibitor Lowers Risk, Improves Functional Class in Some Patients with PAH

Bayer announced today that the FDA has accepted their new drug application (NDA) for menopause drug, elinzanetant, to treat moderate to severe menopausal vasomotor symptoms, according to details published in a 

. Elinzanetant is the first dual neurokinin-1 and 3 (NK-1 and 3) receptor antagonist in late development for menopause relief. The Prescription Drug User Fee Act (PDUFA) date is July 26, 2025, according to a Bayer representative. 

Elinzanetant is a once daily 120 mg pill formulated to treat vasomotor symptoms, also known as hot flashes. During menopause, decreased estrogen levels cause the body’s “thermostat,” or hypothalamus to become extra sensitive. When the hypothalamus thinks the body is too warm, it starts a chain reaction in the form of a hot flash to cool the body down. Elinzanetant decreases hot flashes by modulating estrogen sensitive neurons in the hypothalamus.

The NDA acceptance is based on positive data from three phase 3 OASIS studies, which demonstrated efficacy. Hot flash frequency and severity was measured using an electronic hot flash diary.

OASIS 1 and 2 studied the effects of elinzanetant in nearly 400 women ages 40 to 65 over 26 weeks across 15 countries. By week 4, 62.8% of participants in OASIS 1 and 62.2% of participants in OASIS 2 reported a decrease in hot flash severity. Full 

OASIS 3 extended the treatment window to 52 weeks. Details were presented at The Menopause Society Annual Meeting last month.

OASIS 4 is an expansion of the phase three program and will investigate the efficacy of elinzanetant for women experiencing hot flashes because of endocrine therapy or breast cancer prevention, not menopause.

“If approved, elinzanetant will offer a new non-hormonal option to women seeking treatment for moderate to severe vasomotor symptoms and we look forward to the review by the agency," Christine Roth, executive vice president of Global Product Strategy and Commercialization and member of the Pharmaceutical Leadership Team at Bayer, said in the news release.

In addition to the OASIS trials, Bayer is conducting NIRVANA, a phase 2, double blind study exploring the use of elinzanetant to treat sleep disturbances associated with menopause. Approximately 69% of women experience sleep disturbances during menopause. Difficulty falling or staying asleep can be attributed to hot flashes or changes in mood, specifically depression.

About 47 million women enter menopause every year. Up to 80% of menopausal women report having hot flashes which are one of the top medical concerns for women during this period. Severe symptoms can last up to 10 years after a woman’s last menstrual cycle.

If approved, elinzanetant would be another non-hormonal option for menopausal women seeking hot flash relief. 

Formulary Watch

FDA Accepts NDA for Menopause Drug, Elinzanetant

Greenwalt speaks frequently at conferences and is one of the leading analysts of market conditions for pharmaceuticals. He spoke with Peter Wehrwein, managing editor of Managed Healthcare Executive.

This transcript has been edited for clarity and length.

I took a look at your LinkedIn. I’d guess you’ve been in the industry for about 20 years.

I started at J&J [Johnson & Johnson] 25 years ago. The first third of my career was in sales. So I was a rep, going in and out of the office, carried a bag, and was an area manager for a few years. And then I went inside, at Allergan at the time, and joined the marketing team. So I launched a couple of products, managed some inline products, managed portfolios toward a loss of exclusivity. And then about 10 years ago, I came over to IQVIA to the market access team. So over my career, I’ve spent about a third in sales, a third in marketing and a third in market access.

If there’s a hat that you can have on when you’re commercializing a product, I have had it on at one point or another.

So you seem to be a numbers guy. Were you one of these kids who played with numbers, read the box scores?

I’ve been an avid baseball fan, yes. Probably no surprise. I’ve always been attracted to statistics and trying to understand how and why things work. I was fortunate enough when I got into pharmaceuticals that without knowing it, there’s a huge analytic undercurrent that fuels this business. So being able to make sense of all this information — it’s complex, it’s difficult — that analytic background and the nerd in me likes this stuff.

I am a Chicago Cubs fan. I grew up in the Midwest, a diehard Cubs fan. We watched through all the good years and all the bad years, and I very thoroughly enjoyed the World Series when they won it a few years ago.

What exactly is your job at IQVIA? I’ve heard you give presentations. But if somebody asked me exactly what is in your portfolio, I’m not sure whether I could describe it.

I lead IQVIA’s market access team. We work broadly across the industry. Last year, we worked with over 200 manufacturers. Basically, if they have a product in the U.S. market or coming to the U.S. market, we are engaged with them. Internationally, we’re working with companies from all over the world.

Part of the challenges of bringing a product into the U.S. is that there are a lot of different gateways that you have to work through.

The first one is access. If you don’t have access, you’re not going to be able to find success in the U.S. market, and that’s becoming really elusive. So our teams, broadly, are helping clients understand the U.S. market, what’s the path for a product to come into the market from a reimbursement perspective, and we’ll look at all kinds of analytics to help us understand that.

And then we also have teams that manage contract operations and, kind of behind the scenes, the revenue management side of the business. So for anything that has to deal with clients’ P&L [profit and loss] or gross to net, we have teams that are deeply versed in the means to manage these products and how those interplay with brand success and some of the challenges that come along with access.

We do more, but that’s, in a nutshell, kind of what we do.

What trends are you seeing in access? You’ve spoken about access becoming more difficult, and the window for return on investment getting smaller.

Launch access today is harder to attain than it’s ever been before. We see more products launching, and it’s taking longer to gain any access, if they’re able to gain access at all.

Add to that the challenges of margin compression through growing rebates and growing discounts, things like the 340B [drug pricing] program or patient service programs, which are increasingly expensive … [they are] lowering the ceiling a little bit of a brand’s potential in the market.

The third piece of this is the Inflation Reduction Act, adding in maximum fair price negotiations for Medicare at nine years for a small molecule and 13 years for a large molecule drug.

Those three pieces together essentially shrink the economic lifespan of a product. That’s challenging. It changes a lot of the math and decision-making about bringing a product to market.

But if we know that, and we know that this is how this is going to play out over the next decade, it also allows us to think about the strategies that are going to be needed as a pharmaceutical manufacturer launching a product and finding success over the next decade.

You mentioned being able to launch faster. It’s often a question of investment, focus, and being willing to generate the evidence that the payers need to see in order to add it to formulary. But those questions start even prelaunch as a product is still in development, so that’s early in the product’s lifespan.

Beyond that, there are things like indication stacking and indication sequencing. There’s life cycle management, there’s net present value calculations, and mergers and acquisitions.

All kinds of elements get wrapped up in this question of that shrinking economic window.

Pharmacy benefit managers [PBMs] have been in the news a lot lately. Is it their market power — particularly that of the big three, CVS Caremark, Express Scripts, and Optum Rx — that is shrinking this window of economic return and making access more difficult?

I don’t know if there is any one party that is at most at fault in something like this. There are real challenges broadly in the healthcare system. You talk about margin compression for the pharmaceutical manufacturers, but the PBMs operate on low single-digit margins as well.

Finding the ability to manage a complex portfolio of products, having an added cost center as new things like the GLP-1s [glucagon-like peptide 1] come on the market, and we see more rare and orphan disease products come in.

Can you blame the PBM for wanting to manage the cost with formulary restrictions? You really can’t. That’s probably that’s why they exist in the first place.

I take your point that the window is narrower, but the pharmaceutical industry just doesn’t seem to be a hurting industry.

It’s always a question of performance versus expectation. If the expectations are not aligned to market reality, you can have overinvestment. One of the issues that happens in pharmaceuticals is that you have forecasts that are not playing out as a product comes into the market. If you’re a manufacturer and you’re investing hundreds of millions of dollars just in the promotional spend, and you’re not getting that level of return back, then it is really a question of how to balance those pieces out. Some of the work that we do helps with that.

What I’ve been beginning to wonder about GLP-1s is whether they’ve been overhyped. Do you see any evidence of the predictions of wide adoption and then swamping payer budgets, that maybe this isn’t coming to pass? There are various studies showing that people come off them at a fairly high rate. Is there a bubble that is about to burst here?

I think we’re at the very beginning of really understanding what it means to have obesity treatments in the market that are truly effective. These products work. But there are questions with regard to on-label and off-label utilization, cosmetic utilization, disconnects between when weight is being lost versus the health benefit being experienced by the patient maybe a decade or more later. We’re just at the very early stages of working this out.

Long term, though, we know that patients with obesity over their lifetimes have more comorbidities, are more likely to have diabetes, are more likely have cardiovascular challenges, renal challenges, chronic pain, osteoarthritis, sleep apnea — the list goes on.

The need to treat this patient population, and treat it very seriously as a leading indicator and a leading cause for developing these comorbidities later in life, it’s really important. It’s a societal question. I don’t think it’s overhyped. I think, if anything, we’re just beginning to learn what it means to treat these diseases.

Add to that the deep, deep, deep pipeline of products getting ready to come into the market. At IQVIA, right now we’re tracking almost
180 different molecules for the treatment of obesity or obesity-related diseases that are in some form of phase 1 through 3 clinical development. Even if only a fraction of those is successful in coming into the market, we’re going from a couple of products today to 10, 12, 15 products in the near future. And that is also going to have an effect because net prices will come down, it will be easier for payers to afford, and you’re going to have more strategic decision-making by these different players as they’re trying to find niches in the market to find success. It’s got a long way to go to play out. We’re watching it very closely.

Exactly. Eli Lilly and Novo Nordisk, they’re in the lead right now, for sure. These products had a major impact on the market capitalization of both organizations. Lilly went on to be the number-one valued company in the life sciences industry, and Novo, number two, jumping past J&J.

The major trend that I see is that the biosimilar market seems to be actually maturing. There was a lot of angst about patient reluctance and physicians being wary. But biosimilars are supposed to have the effect on biologics that generics had on small molecule drugs. Look at what happened with statins. The generics drove down price and made them a cheap medicine, right? Do you see that happening with the biosimilars? Are the copycats working?

That is a complex question. It depends on how you measure success. If you’re in a biosimilar manufacturer, you’re going to measure success by volume moving to your product. If you’re the payer, you’re going to measure success by net prices coming down. Those two aren’t necessarily compatible. Look at what happened with Humira last year. There were 12 biosimilars that launched throughout the year; most of those happened right in the middle of the year. At the end of 2023, Humira, as the brand name, held 98% of the market share, despite 12 being in the market and some of these products having discounts of up to 85%. What did they [AbbVie, the manufacturer of Humira] do? They contracted. They gave deep discounts on Humira so the net prices came down, and they came down to the point where it didn’t make sense for the PBM or the payers to move patients away from the branded product to the biosimilar.

Luke Greenwalt, MBA, vice president and lead, IQVIA Market Access Center of Excellence, has joined the editorial advisory board of Managed Healthcare Executive.

John Figueroa

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