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Acadia Pharmaceuticals, the brain-focused drug developer, surprised some on Wall Street last week with a pair of predictions.The first: the companys two products, which brought in close to $1 billion in 2024, should eventually generate between $1.5 billion and $2 billion in combined yearly sales. The second: five experimental medicines in Acadias research pipeline could, if they come to market, collectively peak at $12 billion in annual sales.The road to such commercial success will be long and fraught, as neuroscience is a famously difficult, setback-ridden area of development. But Catherine Owen Adams, the industry veteran who took over as Acadias CEO last fall, maintains the company is up to the challenge.I didn't come into this job to putter along at a $3 billion market cap, she said in an interview. I believe this company can get to the next level of mid-cap. That's how I'm leading this business to get there.To reach that goal, Owen Adams said a top priority for her team is further building out Acadias research, through a much more assertive business development strategy. That may include deals for drugs in the later stages of testing the kind of assets that can serve as the value inflection point analysts have sought.The hope, according to the chief executive, is Acadias pipeline will grow at least 25% to 30% from external innovation in the coming years, and that $12 billion figure bumps up to $15 billion or $20 billion as a result.Owen Adams spoke to BioPharma Dive about why Acadia offered up such bold sales forecasts and where the company believes it can play to win in brain research.The following conversation has been edited and condensed for clarity.BIOPHARMA DIVE: Neuroscience is a wide area of research, and your pipeline reflects that. Acadia is going after two rare diseases, Alzheimers psychosis, depression, epilepsy. Why did the company decide to target those indications?CATHERINE OWEN ADAMS: When I came to Acadia nine months ago, part of the attraction was the pipeline. A lot in there was super interesting. I spent the last six to seven months with [research and development head] Liz Thompson trying to understand what we're trying to achieve. What's our focus?The brand we are developing is underserved neurological and rare diseases. Underserved is actually quite an important descriptor of those two areas, because we're not going to move into Alzheimer's degeneration. It's just too big for us.However, the areas around that, where there are maybe less players, less interest sometimes because it might be smaller populations, more niche populations we believe we can play to win.If you look at our pipeline, most of those fit. I would say [ACP-211] probably is on the bubble. Treatment-resistant depression is a fairly large space, but we argue its underserved, and we believe we have the opportunity to really make a difference there with something that has less monitoring.Forecasting Acadia's pipeline potentialDrugStageIndicationPeak sales potentialACP-101Ph. 3Prader-Willi Syndrome~$1-2BACP-204Ph. 2Alzheimers psychosis, Lewy body dementia psychosis>$2BACP-211Ph. 2Major depressive disorder>$2BACP-271Ph. 1-readyHuntingtons disease, tardive dyskinesia~$1-2BACP-711Ph. 2Essential tremor>$2BSOURCE: Table and estimates from research by Mizuho Securities analyst Uy Ear.How do you strike a balance between drugs seen as higher risk-reward, and those with maybe a more straightforward shot at getting through testing and onto the market?OWEN ADAMS: The balance for us comes with the ability to drive precision medicine into neurological diseases. The risks inherent within neuropsych, particularly, are subjective endpoints, large studies, high placebo effects. We all know it's hard. What we're trying to build is a movement into that space that's anchored in a bit more than just symptomatic endpoints.If you look at how we're running the Alzheimer's disease psychosis trial and the Lewy body dementia psychosis trial, what we unveiled at our R&D Day was an additional level of rigor around using biomarkers within those patients to try and understand the science.In each of those trials, while we have the more subjective endpoints of the various scales, we are also looking at augmenting that and making the populations more homogeneous using additional biomarkers.How important is having promising biomarkers when deciding whether or not to pursue a disease or subset of research? Are biomarkers part of go, no-go decisions?OWEN ADAMS: In some areas it's go, no-go. In the areas that we're in, the biomarker science is not necessarily concrete yet.My belief, having spent 25, 30 years in pharma and more recently, in oncology, where biomarker [reliance] goes without saying, is that as a society we need to get more precise about where we use medicines, what patients we use them, how we use them. Biomarkers are our current way of doing that, and we need to advance that within neuropsych. It's been sort of a backwater, in terms of advancing the science as fast as other areas. It's harder.My vision for Acadia becoming a biotech powerhouse in this space is built on precision medicine and data innovation. I'm really looking to push the company to advance the science, as well as, obviously, build a clinical trial we believe is the right one.If we look globally at countries where we want to have our medicines available, I think having biomarker-led patient population identification is going to help, particularly in single-payer systems. We have seen an unwillingness to pay [when] you just use a medicine on everybody and see what happens.Analysts claim that, during turbulent markets, companies will double down in areas they feel most experienced. You have two products: Nuplazid for Parkinsons disease psychosis and Daybue for Rett syndrome. How much have those two products influenced your pipeline decisions?OWEN ADAMS: I think [they do], but it's not necessarily based on the last six months of market volatility.With most pharma companies, you see a tendency to understand an area, have a lot of learning and then build the next product to be better. You can see many examples. In the case of Rett syndrome, Daybue has GI side effects, and we are looking to improve upon that. We look at 2591 with the hope that maybe we can evolve the side effect profile and still have the same efficacy.Similarly, Nuplazid is a great drug. But there are areas where it could do better. We were limited because it had a QT signal, so we've designed ACP- 204 to eliminate the QT signal and to act faster. Those things, for me, make sense, because you're really derisking, even though the analysts haven't given us much credit.If you look at essential tremor with ACP-711, which we just licensed in from Saniona in December, its a very different space; high unmet need, a bit of a graveyard. We think we found some really interesting science.211 is in major depressive disorder, another very different space. But I was at Janssen when we launched Spravato, so I know the space and understand what it takes.I don't want us to be so focused that we miss opportunities. It's about balancing where we know and then new areas where we think we can learn.What were the biggest lessons you learned from the Spravato story?Spravato became a blockbuster product over seven years. It was not a blockbuster product out the door. The efficacy was always really strong, but the infrastructure that the U.S. healthcare system had in place when we launched Spravato was very underdeveloped.Catherine Owen AdamsPermission granted by Acadia PharmaceuticalsJ&J had to spend a lot of time developing that infrastructure and support systems to allow doctors to be able to use it for the right patients. Every six months to a year, we'd learn more, and they'd put different things in place to ensure it continued to evolve. It was a huge investment to get Spravato to where it is now.We are now the beneficiaries of a much more defined system where doctors get paid for monitoring. When we launched Spravato, there wasn't even a code that doctors could use to get paid. There's always a downside to being the first, but it was a long, long road. Longer than they thought anyway.What are Acadias BD priorities right now?OWEN ADAMS: We've defined our focus: underserved neurological and rare diseases. That's where we want to play to win. Within that space, we're fairly open about the science we want. Science that is hopefully first in class or best in class. Everybody says that, but I mean it. I don't want to be the second, third or fourth to market, unless we've got something we believe is truly differentiating, which I think is where 211 fits.Acadia is not afraid to make scientific risk decisions, regulatory risk decisions. Where I won't make a risk decision is financially. We are in a very strong financial position, and I believe for the future of our company, we need to hold true to minimizing financial risk.With that in mind, I'd also like to look at some slightly later-stage products. We've got a lot of earlier-stage products. I would love to see us focusing a bit more on Phase 2 Phase 3 if it's possible but Phase 2-stage products, where we can get something to market in the 2030 timeframe.The upside of this market being a little bit difficult is that there are quite a few earlier-stage companies who maybe are more financially fragile than they were six months ago. So there's opportunity to go in and make a deal.You gave two big sales predictions during the R&D day. One was on Nuplazid and Daybue. Why did you release that figure?OWEN ADAMS: I've come up through the commercial line, and I believe analysts need to understand where I think we can get to. I'm strongly commercial, and I don't put numbers out there that I don't believe we can achieve. Those numbers are based on strong, objective data that I stand behind, and I think the analyst community are underestimating and have been overly cautious about both Daybue and Nuplazid.One of the big things in the last two months is that we got clarity on our patent for Nuplazid, now out to 2038, so that gives us a lot more runway. Nuplazid will, at some point if the Inflation Reduction Act exists as it does today, which is a big caveat run into a pricing situation with the IRA, as every other drug will. But Daybue has the potential to continue to grow and to go global. I think people are underestimating ... our ability to stabilize the current patient population.I'd rather have a conversation about: are we going to hit $1.5 billion to $2 billion or not, than say Im not willing to go there. As a CEO with a commercial background, I should be willing to go there. I can't hide behind, Well, I don't feel comfortable. Okay, then, why am I here?There was also that $12 billion peak annual sales forecast you put on the pipeline programs. Whats the biggest challenge you see getting to that number?OWEN ADAMS: Without wanting to be flippant, the biggest hurdle is the clinical outcome of the trials. The trial designs that we're looking at; the thinking that's gone into them; the additional biomarkers; the additional steps we're putting in place ... I think we're really focused on getting the best Phase 3 trial designs we can.Then, it becomes: can you commercialize with that outcome? Again, I stand behind the numbers. I don't think they're unreasonable. I've not pushed the team. I understand how all parts of a forecast are built.The biggest headwind, ultimately, will be the clinical data and the access environment that exists when we launch. Those two are outside of our control. We can design the clinical trials, we can prepare for the access environment, but that will make a difference. '

Acadia Pharmaceuticals has proposed that its pipeline of experimental medicines could, if eventually brought to market, peak at $12 billion in annual sales.The brain drug developer disclosed this estimate Thursday, during its first major research and development event. The company expects eight programs to be in human testing this year, and to have data from five mid- to late-stage trials between now and the end of 2027. Five of those programs could potentially reach blockbuster status, or $1 billion or more in yearly sales, according to analysts at the investment firm Mizuho Securities.In a note to clients, Mizuho analyst Uy Ear wrote that among those programs, the majority of value comes from a drug code-named ACP-204. Researchers are currently conducting a couple mid-stage clinical trials to evaluate this drug as a possible treatment for the psychosis tied to Alzheimers disease and a related condition known as Lewy body dementia. Data from the Alzheimers study are slated to come next year.Acadia said ACP-204 is designed to build upon one of the companys two marketed products, Nuplazid, which has a similar chemical structure and was first approved in the U.S. a decade ago to treat Parkinsons disease psychosis. Nuplazid accounted for nearly two-thirds of the $958 million in revenue Acadia recorded last year. But a key patent protecting the drug from generic competition is set to expire in 2030.On Wednesday, Acadia also predicted the combined peak sales of Nuplazid and its other commercial medication, Daybue, will fall in the range of $1.5 billion to $2 billion. Approved in 2023, Daybue is used for patients with Rett syndrome, a rare, genetic disorder that impairs brain function and development.Marc Goodman, an analyst at Leerink Partners, described that forecast as very bullish. Consensus on Wall Street, according to Ear, is that sales for the two products will crest around $1.3 billion.Ear added that the wide range offered by Acadia is likely due to the recent uncertainty surrounding how many patients will stop taking Daybue because of its side effects. The drugs label includes warnings for diarrhea and weight loss.Along with ACP-204, Acadias pipeline includes ACP-101, a drug for another rare, genetic, neurological disease called Prader-Willi Syndrome. High-level results from a Phase 3 experiment should arrive sometime between October and December. Theres also ACP-211, for major depression; ACP-711, for essential tremor; and ACP-2591, a next-generation approach to addressing Rett syndrome. Each of those three programs are in early-stage testing.Forecasting Acadia's pipeline potentialDrugStageIndicationPeak sales potentialACP-101Ph. 3Prader-Willi Syndrome~$1-2BACP-204Ph. 2Alzheimers psychosis, Lewy body dementia psychosis>$2BACP-211Ph. 2Major depressive disorder>$2BACP-271Ph. 1-readyHuntingtons disease, tardive dyskinesia~$1-2BACP-711Ph. 2Essential tremor>$2BSOURCE: Table and estimates from research by Mizuho Securities analyst Uy Ear.Additionally, Acadia has a preclinical therapy, ACP-271, that it plans to test in patients with Huntingtons disease or tardive dyskinesia, a condition hallmarked by uncontrolled movements and a side effect that can arise from the long-term use of some brain-stabilizing medications like antipsychotics.Analysts reacted well to Acadias updates. Ear left the R&D presentation encouraged, while Goodman wrote how his team is now incrementally more positive on the company. Acadias stock price has been weighed down over much of the last five years, as the company hit setbacks trying to expand the use of Nuplazid into more lucrative markets.In his note, Goodman acknowledged that investors will probably have a show me the data attitude regarding these new assets as well as a healthy skepticism around the potential of both ACP-204 and ACP-101. So it may take some time for pipeline value to creep into the companys valuation.Should Nuplazid and Daybue ultimately achieve Acadias new forecast, that would make the companys stock attractive even if the experimental programs dont pan out, Goodman wrote.However, the real upside is that Acadia hits on an asset or two to make the name a sustainable growth story, he added. We are certainly willing to be patient to see this story play out.Acadia shares traded around $22.60 apiece late Thursday morning, essentially flat from the prior days close. '