コグニション・セラピューティクス（CGTX）2026年第3四半期決算説明会：DLB第3相計画が進展

要点

• コグニション・セラピューティクス（Cognition Therapeutics）は、軽度から中等度のレビー小体型認知症（DLB）を対象としたゼルビメシン（zervimesine、開発コード「CT1812」）の承認申請用第3相臨床試験デザインについて、米食品医薬品局（FDA）と合意したと発表した。
• 計画中の試験では、ゼルビメシン100mgの1日1回経口投与とプラセボを9カ月間にわたり比較する。経営陣は、治験実施計画書（プロトコル）の策定完了および十分な資金確保を条件として、2027年半ばの治験開始を目指している。
• FDAとの協議を経て、同社はこの承認申請用試験に必要な被験者数を、当初のデザインで想定されていた約750名の半数未満に抑えられる見込みであるとしている。当初のデザインでは約1億5,000万ドルの費用が見積もられていた。
• 2026年9月30日時点の現金、現金同等物および使途制限付き現金同等物の合計は約3,150万ドルであった。経営陣は、現在の資金水準で2027年第4四半期までの事業運営および設備投資を賄うことができると見込んでいる。
• 第3四半期の純損失は410万ドル（基本的および希薄化後1株当たり0.04ドル）となり、前年同期の490万ドル（同0.06ドル）から縮小した。
• 軽度認知障害（MCI）および初期アルツハイマー病を対象とした被験者545名規模の第2相START試験は登録を完了しており、米国立加齢研究所（NIA）からの非希薄化資金（株式の希薄化を伴わない助成金）で賄われている。経営陣は2027年後半に主要結果（トップラインデータ）が得られると見込んでいる。

主要財務データ

事業および運営の業績

ゼルビメシンは血液脳関門を通過してシグマ2受容体に結合し、毒性タンパク質から神経細胞を保護することを目的として設計された低分子化合物である。経営陣によると、同薬はプラークに結合したり除去したりしないため、アミロイド関連画像異常（ARIA）のリスクを伴うとは予想されないという。

計画されているDLB対象第3相試験では、軽度から中等度の患者を登録し、ベースライン（試験開始時）で幻覚や妄想を呈する患者の割合を高める濃縮（エンリッチメント）登録を行う。主要評価項目は、神経精神症状評価尺度（NPI-12）の幻覚および妄想項目に焦点を当てる。また、主要な副次評価項目として日常生活動作尺度「ADCS-ADL」を予定している。

このデザインは、安全性と忍容性に関する主要評価項目を達成した第2相SHIMMER試験に基づいている。経営陣によると、ゼルビメシンは運動症状、認知機能の変動、神経精神症状全般において良好な改善傾向を示した。SHIMMER試験では精神病症状の進行が89%抑制されたと同社は述べている。

コグニション・セラピューティクスは本プログラムを単一のピボタル試験（検証的試験）として設計している。FDAはこのアプローチを検討することに同意しているものの、最終的な判断は完了した臨床試験データの審査結果次第となる。

同社はまた、軽度認知障害および初期アルツハイマー病患者545名を対象とした第2相START試験も進めている。同試験は被験者登録を完了しており、NIAからの助成金支援を受けている。最終被験者の登録は2025年11月に完了した。

経営陣はさらに、ゼルビメシンの新規結晶製剤を対象として最近成立した米国特許により、2045年までの保護が確保されると言及した。

経営陣の見通し

経営陣はDLB対象第3相試験の実施計画書を最終決定し、審査のためFDAに提出する予定である。並行して、同社は開発業務受託機関（CRO）や初期の治験実施施設の選定、ならびに投資家、金融機関、潜在的な戦略的提携先を含む資金調達の選択肢を検討している。

同社の目標は、2027年半ばにDLB対象第3相試験を開始することである。現在の支出動向を踏まえ、経営陣は手元資金により2027年第4四半期までの事業運営および設備投資を賄うことができると試算している。

経営陣によると、アルツハイマー病を対象としたSTART試験の主要結果は2027年後半に公表される見込みである。

リスク要因と注目点

• 計画中のDLB対象第3相試験の実施には追加の資金が必要であり、同社は現在も資金調達および戦略的選択肢の検討を続けている。
• FDAは単一のピボタル試験による方針を検討する予定だが、その受け入れは完了した試験データの審査結果次第である。
• DLBには、病期や治療効果を測定するための確立された画像バイオマーカーや血液バイオマーカーが存在しない。そのため、第3相試験のデザインでは精神病症状の変化を基礎疾患の進行状況を示す代替指標（プロキシ）として使用している。
• DLBにおける認知機能検査は、注意力や認知機能が分単位および日単位で変動するため、評価が複雑化する可能性がある。
• 治験の開始は、実施計画書の策定完了、FDAによる審査、委託業者の選定、治験施設の準備、および資金調達の進捗に依然として左右される。

アナリスト質疑応答の要点

経営陣によると、以前のDLB試験構想では約750名の被験者が想定され、約1億5,000万ドルの費用が見込まれていた。しかし、FDAとの協議および評価対象を精神病症状へ絞り込んだことを受け、同社はその半数未満の被験者数でも統計学的検出力の高い承認申請用試験を実施できると考えている。

START試験について、経営陣は以前のSHINE試験との比較を目的としたベースラインデータの公表や分析は行っていないと述べた。同社は、近く開催されるアルツハイマー病臨床試験会議（CTAD）において、バイオマーカーの活用を含む被験者募集方法について説明する予定である。

精神病症状の臨床的妥当性について、経営陣は幻覚や妄想がDLB患者の80%近くに見られ、一般に疾患の進行に伴い悪化すると指摘した。今回採用された評価尺度は特定の種類の幻覚や妄想に限定されず、被験者全体に広く適用可能である。

経営陣はまた、現在DLBの症状管理に使用されている抗精神病薬との潜在的な差別化要因として、安全性を挙げた。同社は、ゼルビメシンが抗精神病薬ではなく、単なる急性症状の緩和にとどまらず、基礎疾患自体の進行を遅らせることを目指して開発されている点を強調した。

決算説明会トランスクリプト全文

決算説明会の完全なトランスクリプト

経営陣による説明

Operator

My name is Mark, and I will be your conference operator today. This call is being recorded. I would like to turn the presentation over to your host for today's call, Mike Mauger, Financing Director for LifeSciAdvisors. proceed, Mr. Moyer.

Mike Moyer

Thank you, operator, and good morning, everyone. Welcome to Cognition Therapeutics' Third Quarter 2026 Conference Call. With me today are Lisa Ricciardi, President and Chief Executive Officer; John Doyle, Chief Financial Officer; and Dr. Tony Caggiano, Chief Medical Officer. This morning, the company issued a press release detailing its financial results for the quarter.

We encourage everyone to read this morning's press release as well as cognitions Form 10-Q, which is now filed with the SEC and available on our website. In addition, this conference call is being webcast through the company's website and movie archived for 30 days. Please note that certain information discussed on the call today is covered under the safe harbor provisions of the Private Securities Litigation Reform Act.

We caution listeners that during this call, management will be making forward-looking statements. Actual results could differ materially from those stated or implied by these forward-looking statements due to the risks and uncertainties associated with the company's business. These forward-looking statements are qualified by the cautionary statements contained in cognitions press releases and SEC filings. This conference call contains time-sensitive information that is accurate only as of the date of this live broadcast.

Cognition undertakes no obligation to revise or update any forward-looking statements to reflect events or circumstances after the date of this conference call. With that, I would now like to turn the call over to Lisa Ricciardi.

Lisa Ricciardi

Mike, thank you, and good morning, everyone. As you can see on the agenda, first topic we'd like to cover on this call is the registrational plan for zervimesine , or CT1812 and the treatment of people with dementia with Lewy bodies, which we'll refer to in this call as DLB. Two weeks ago, we announced that we reached alignment with the FDA on a planned registrational trial for zervimesine and people living with DLB Investors who have been following Cognition are aware that this topic has been the subject of several meetings with the FDA.

We conducted meetings in January, May and October. And all along, we've encountered FDA staff at every step who are engaged and constructive. And as a result of our interactions, Cognition Therapeutics is the first company to align on a Phase III trial with the FDA for this class of drugs to treat DLB, to treat the condition of DLB. Other companies have designed Phase III trials for symptomatic treatments such as antipsychotics or medications for agitation in this population. Zervimesine is unique in that it is designed to slow the course of the disease, not just treat the symptoms. I am proud that we have tackled an unchartered development path together with the FDA.

Now we're working to complete a protocol for zervimesine Phase III program. A complicating factor with DLB is the lack of biomarkers available to measure the progression of disease. Because of this, we and the FDA agreed on clinical endpoints that are aimed at showing changes in disease progression. And with that, let me introduce our Chief Medical Officer, Tony Caggiano, who can discuss these endpoints and the details of our trial. Tony?

Anthony Caggiano

Thanks, Lisa. Cognition's Founder, Dr. Susan Catalano, was strategically focused on small molecules that would protect neurons while efficiently crossing blood-brain barrier. As a small molecule, zervimesine has no need for brain-shuttle technology to access the CNS. It binds to the sigma-2 receptor and protects neurons from toxic proteins. It does not bind to or remove plaque so we would not expect ARIA to be at risk with zervimesine.

And finally, we have an extensive patent estate on zervimesine. The U.S. CTO recently issued a patent on a new crystal formulation of zervimesine, which provides protection through 2045. Here, you can see the proposed study schematic for our Phase III trial of zervimesine in mild-to-moderate DLB. Participants will be randomized to receive either 100 milligrams of zervimesine or placebo orally once a day for 9 months. Many of the endpoints will look familiar to you as they were employed in the Phase II SHIMMER study. The SHIMMER study met its primary goal of safety and tolerability. Zervimesine treatment in SHIMMER was shown to have a meaningful impact across the core features of DLB, including motor symptoms, fluctuations of cognition and neuropsychiatric symptoms. In brief, everything was moving in the right direction.

Neuropsychiatric symptoms in SHIMMER were evaluated using the NPI-12, a tool that measures 12 behavioral symptoms, including psychosis, which is defined as hallucinations and delusions. Other behaviors, including agitation, anxiety and apathy are also reported in the NPI-12. When we focus on just the psychosis systems, progression was slowed by 89% with zervimesine treatment. Many of the symptoms we measured at SHIMMER are core features required for diagnosis of DLB. The fact that zervimesine treatment impacted all of these symptom domains confirms that it is not asymptomatic drug. By protecting neurons, we believe zervimesine is slowing progression of the underlying disease or it is a disease-modifying agent. The measurement of disease progression DLB is challenging.

Alzheimer's disease has imaging and blood test designed to determine disease stage and treatment effects. None of these are available yet in DLB. During our meetings with the FDA, we discussed options for measuring changes in DLB disease progression. Both FDA divisions of neurology and psychiatry agreed that changes in psychosis may serve as a proxy for changes in the underlying disease. Today, clinicians rely on changes in key symptoms to monitor the patient's disease. However, not all symptoms are easily evaluated in DLB. For example, measuring cognition is complicated by the fluctuations that people and DLB experience. These fluctuations of attention and cognition can change for a minute to minute and day-to-day. So a cognitive test taken Tuesday at 3 p.m., for example, may have a very different result from the same test taken Thursday at 10 a.m. This makes it difficult to reliably test cognition and clinical trial participants.

Interestingly, psychosis affects up to 80% of people with DLB. It is often present in early stages before the other hallmark symptoms emerge, and it worsens in a predictable fashion as the disease progresses. Psychosis is easily measurable and maybe a proxy for changes in overall disease progression. The FDA agreed with its assessment, and we are designing a Phase III study with this in mind. The 2 end points we discussed with FDA are shown on this slide. Within the NPI-12, 2 of the 12 symptoms are hallucinations and delusions. This tool is called the NCI in measures the frequency and severity of 2 symptoms. The primary outcome measure in the Phase III trial is the NPI. This assessment tool is well known to investigators and has been used extensively in clinical trials. When we discussed the summer data with the FDA, we identified that hallucinations and delusions, the lutein dilution results were some of the strongest from the study. The NPI which analyze these data has some excellent signal-to-noise ratio, suggesting it will be a powerful tool treatment differences in our planned registrational trial.

In addition, enrollment will be enriched with patients who have hallucinations and delusions at baseline. Together, these features give us confidence that the trial we have designed will be adequately powered and sufficiently controlled with a moderate sample size. In addition to the NKI2, we are planning to include the ADCS-ADL for Alzheimer's Disease Cooperative study activities of daily living scale as a key secondary endpoint. By using these 2 together, we will show how zervimesine treatment impact the progression of the disease. Before we move on, I want to make an important distinction. -- even machine is not an antipsychotic. Antipsychotics are effective on reducing acute symptoms of those inflammation or delusions. Antipsychotics are not, however, effective as slowing in the course of the disease. As they're anything as the disease-modifying agents, we had approached the FDA to use a composite endpoint to assess the diverse symptomatology of DLB.

In meeting with FDA, it became clear that there is no validated measure developed to assess its diverse symptomatology. FDA cognition agreed that cytosis could be used as an indicator of the overall disease progression. I'll turn the call back over to Lisa now, who can provide updates on the next steps for both DLB and Alzheimer's program.

Lisa Ricciardi

Thank you, Tony. Our immediate next step is finalization of the protocol, which will be submitted to the FDA for review. Consistent with our discussions with the FDA, our plan is to design a single pivotal study for zervemecine in DLB. We've had several conversations with the FDA, and they have agreed to consider a single pivotal trial. Their decision will be a matter of review upon receipt of our completed clinical trial data. Having just concluded our discussions with the FDA and having just received their minutes, we're now evaluating our options with investors, bankers and strategic to secure sufficient study funding to conduct the study.

And in parallel, we're evaluating vendors for CRO partners as well as the look at initially clinical sites for the trial. Our goal remains to initiate this study in mid-2027. Now in addition to our work on DLB, we're also approaching an important milestone in our START study. The START study an ongoing trial is a Phase II study in mild cognitive impairment and early Alzheimer's disease. This study has 545 patients in it. As a reminder, START is fully enrolled, fully funded with nondilutive capital from the NIA. The last participant in start was enrolled in November of 2025. thus, we're expecting top line results in the second half of 2017. With that, John Doyle will review our financial results and provide a bit more color around our cash position. John?

John Doyle

Thank you, Lisa. Cash, cash equivalents and restricted cash equivalents as of September 30, 2026, were approximately $31.5 million. Total obligated grant funds remaining from the NIA were $18.2 million. R&D expenses were $5.1 million for the third quarter of '26 compared to $3.8 million for the comparable period in '25. The increase in research and development expenses was driven by higher trial activities with contract research organizations and professional fees. General and administrative expenses were $2.3 million for the quarter compared to $2.6 million in 2025. The decrease in general administrative expenses was driven primarily by lower professional fees and compensation.

The company reported a net loss of $4.1 million or $0.04 per basic and diluted share for the third quarter compared to $4.9 million or $0.06 per basic and diluted share for the same period in 2025. Based on our current spend, we estimate that we have sufficient cash to fund operations and capital expenditures into the fourth quarter of 2027. Lisa?

Lisa Ricciardi

Thank you, John. Operator, please open the call to questions.

Operator

[Operator Instructions]

And your first question comes from the line of William Wood with B. Riley Securities.

質疑応答

William Wood

So 2 for us if we can with your current funds submission into first quarter 2027, could you provide -- or fourth quarter, sorry? Could you provide any extra details on the cost of your DLB trial, maybe you're thinking on advancing that forward where the funds might come from? And then also in terms of with the START trial moving quickly towards your readout in the third quarter, could you provide any color on the baseline there, how it may compare to Phase 2 shines load pause population analysis?

Unknown Executive

Yes. Thanks for the questions. Regarding the DLB study, we have been considering trials of about 750 participants when we initially planned the study and this was targeting symptoms that include cognition and fluctuation. Trials of this size would have been about $150 million. Given our discussions recently with the FDA and clear focus on DLB and their agreement for us to use we can come back to a well-powered registrational trial with less than half of this original number of participants. Regarding the START trial, we have not released or even analyzed the baseline data in order to compare it to the SHINE study. There will be a detention at the CTAD meeting coming up in 2 weeks.

Looking at how we recruited individuals in this study, which is slightly different than other studies had to the use of biomarkers and so forth, but [indiscernible] data will not be presented yet. Thank you very much.

Operator

Next question comes from the line of Ram Silverado with H.C.Wainwright.

Raghuram Selvaraju

Congratulations on all the recent progress, particularly on the FDA front. I was wondering if you could give us some additional qualitative color around the nature of hallucinations and delusions suffered typically by DLB patients and how these worsen, along with the kind of inevitable progression of the disease as well as what you anticipate would be considered a clinically meaningful impact on that specific arena of DLB symptomatology.

Anthony Caggiano

Yes, thanks for those questions. So people at DLB have hostesses and dilution, then the interesting thing is that Nearly 80% of individuals have hallucinations and delusions. Now the type of hallucinations and delusions in this disease is a little different than other conditions. But in reality, that's not particularly important. We certain types of [indiscernible] in that in character are somewhat different than you might expect in schizophrenia or Alzheimer's disease. Now the nice thing with the tool that we're using it's not restricted based on a particular type of hallucination or dilution. It's general and allows it to be really crafted towards each and every participant.

So indeed, people with DLB, their hallucinations and delusions worsen throughout the course of the disease. And the important thing that we did over the last year here is to work with the FDA to basically agree that whether you're looking at times or fluctuations or motor center, in this case, the neuropsychiatric symptoms they all could be a reasonable proxy for how the disease is progressing. And because we look at hallucinations and dilutions over the last months, if that subject to the issues of fluctuations that these other measures are, so it's a very good clinical tool to use. So that's why we're using this as a general measure of disease progression.

Lisa Ricciardi

Ram, I can add that we have done a great deal of work with people who live with DLB. We don't refer to them as patients because we're not their doctors and what you hear from these individuals and hallucinations of several nature. One of the things they talk a lot is about smell. I'm selling bird coast. That seemed to be a common thing, surprisingly other hallucinations our oral AURA, why is the American -- the [indiscernible] banner playing all day long. There are visual hallucinations. I've heard repeatedly from physicians about patient people with DLB commenting on trains running through the back there are children coming to the home for things of this nature. Now they're largely not bothered by these things. but it does take them a while to come forward and talk about it.

On the dilution side, I think it's pretty straight forward what's most common [indiscernible] syndrome, so you're looking across the table at your husband or your wife and you're going, you are not my husband or my wife. Where is that person, you actually see correctly something is there, but the brain interprets it as something else. So at a fundamental level, these are sort of definitional characterizations of hallucinations and delusions from patients as well as from the people who treat them. It's pretty consistent.

Raghuram Selvaraju

And then just with respect to the risk-benefit profile that you expect to target with zervimesine . Lisa, you mentioned during your prepared remarks that atypical antipsychotics has historically been used in managing the symptoms of DLB patients. But of course, that group of drugs is well known to have well-documented chronic side effects that can be potentially very debilitating. These are not side effects that are associated with zervimesine or the class of drug towards urban belongs. So would you deem that to be potentially a future meaningful clinical and competitive edge for zervimisine were it to be approved in DLB?

Lisa Ricciardi

Excellent question. The answer is very much yes. We've done a fair amount of research with prescribing doctors who treat this population. And they say pretty much what you said, Rob, they go, "Look, I know I have a lousy hand of cards here, if you will. -- there are drugs that have warning black box warnings. There are drugs, let's say, there are fatalities in DLB patients. And so when they talk about how they treat these patients, they carefully weigh where they want to take the risk, but they're aware of it. And so we believe that coming forward with a drug that can treat this population and have a safety profile that's been demonstrated in this population as well as what Tony indicated earlier, a very unlikely incidence of any ARIA at all.

We feel that, that will be an important differentiator. There really are not drugs for this patient population. You open the medicine chest and you find things from other conditions and you apply them to the symptoms, and that's about as good as it gets. And the language I've articulated here is not different from the caregivers or the physicians to think about it in the same way. So I do think what you've expressed is a very important potential point of differentiation that we hope to demonstrate in our registrational trial.

Operator

Thank you. There's no further question at this time. And now I will turn the call back to Cognitions's CEO, for closing remarks. Ms. Ricciardi, please go ahead.

Lisa Ricciardi

Thank you. In closing, I want to reiterate how excited we are to be able to advance zervimesine into a Phase III study. We plan to evaluate the potential to slow the progression of this disease. The work we undertook with the FDA's office of Neuro Science concluded with the regulatory path for revenue. But you know what, we believe we're paving the way for future innovations in this disease area.

We want to thank the trial participants and their families as well as our advisers clinical trial investigators and our staff. Thank you for joining us. I'll turn the call back to the operator to conclude our call.

Operator

That concludes today's call. You may now disconnect.