アキュラクス・ファーマシューティカルズ（ACXP）2026年第2四半期決算説明会：FDA承認への道筋と1,070万ドルの手元資金

要点

• アキュラクス・ファーマシューティカルズ（NASDAQ：ACXP）の2026年第2四半期末の現金残高は1,070万ドルとなり、2025年12月31日時点の760万ドルから増加しました。
• 2026年第2四半期の純損失は230万ドル（希薄化後1株当たり0.53ドル）となり、2025年第2四半期の220万ドル（同1.89ドル）から拡大しました。
• FDAは、単一の第III相IBZ-ASPIRE試験およびその他の完了済み研究終了後のNDA前相談において、特に有効性の結果が堅調である場合、イベザポルスタットに関する全体的なエビデンスを評価することに前向きな姿勢を示しました。
• 全額の資金が手当てされている、主に再発性クロストリジウム・ディフィシル感染症（CDI）患者20名を対象としたPATHFINDER試験は、2026年第4四半期に被験者登録を開始する見込みです。
• 経営陣は、4月の募集による資金調達およびコミットメント型株式調達枠の残額により、PATHFINDER試験および同社の事業運営資金を少なくとも1年間は賄えるとしています。ASPIRE試験の開始には引き続き追加資金が必要です。
• 2026年8月、FDAはイベザポルスタットの商標名として「Syfbezi」を条件付きで承認し、米国特許商標庁（USPTO）は商標登録査定を出しました。

主要財務データ

2026年上半期の研究開発費は、前年同期の110万ドルから140万ドルに増加しました。一般管理費は330万ドルから260万ドルに減少しました。純損失は前年同期の440万ドル（希薄化後1株当たり4.01ドル）から390万ドル（同1.13ドル）に縮小しました。

事業および業績の動向

2026年7月に行われたAcurxとFDAとの協議では、単一の第III相急性CDI試験で新薬承認申請（NDA）を裏付けられるかどうかが焦点となりました。FDAは、ASPIRE試験、PATHFINDER試験、およびその他の完了した臨床試験の終了後に行われるNDA前相談において、さらなる議論に応じる姿勢を維持しました。経営陣は、単一の第III相試験に基づく申請の可能性は、堅調な有効性と全体的なエビデンスパッケージにかかっていると強調しました。

PATHFINDER試験は、主に再発性CDIを対象とした20名のオープンラベル試験です。経営陣は、同試験の資金は全額手当てされており、治療および再発予防の双方に関する補助的エビデンスを提供できる可能性があると述べています。同社は立ち上げ活動を完了しており、2026年第4四半期に被験者登録が開始される見込みです。

ASPIRE試験は、国際的な第III相非劣性試験として計画されています。西欧や東欧などの地域が検討対象に含まれていますが、患者のスクリーニングはまだ開始されていません。同社は、PATHFINDER試験に必要な原薬（API）および製剤を十分に確保しており、ASPIRE試験に向けて適切な有効期限を持つ治験薬を十分に製造できる体制が整っていると述べています。

Acurxは、DNAポリメラーゼIII C阻害剤を研究するため、ライデン大学メディカルセンターとのパートナーシップも継続しました。この研究には、メチシリン耐性黄色ブドウ球菌（MRSA）由来のPol CとAcurxの阻害剤との複合体の初となる3次元構造の解明が含まれています。

同社は、イベザポルスタットおよびACX-375Cプログラムを保護する米国特許6件と国際特許10件を報告しました。その他の国別出願は現在審査中です。

経営陣の見通し

経営陣は、PATHFINDERの被験者登録が2026年第4四半期に開始されると見込んでいます。また、既存の財務資源により同試験を支援し、少なくとも1年間の事業運営資金を賄えるとしています。

ASPIRE試験の開始は、公的資金、民間資金、またはパートナーシップによる適切な資金調達の確保が条件となっています。Acurxは複数の資金調達に向けた取り組みが進行中であると述べましたが、具体的な完了のスケジュールは示していません。

経営陣はまた、PATHFINDER試験後の代替開発ルートの可能性についても説明しました。この探索的試験が成功裏に完了した場合、Acurxは抗菌薬および抗真菌薬の限定患者群ルート（LPAD）に基づく適用可能性についてFDAと協議する予定です。経営陣は、これにより単一の第III相試験に支えられた再発性CDI治療薬の申請が可能となり、コストをASPIRE試験の約半分に抑えられる可能性があると述べました。ただし、このルートの適用は引き続きFDAの審査にかかっています。

リスクと注視点

• 公的資金、民間資金、またはパートナーシップからの追加資金が得られなければ、ASPIRE試験を開始することはできません。
• FDAは新薬承認申請（NDA）において単一の第III相試験を受け入れることを確約していません。その評価は、臨床エビデンスの全体性と堅調さに依存します。
• PATHFINDER試験は患者数20名の探索的オープンラベル試験であり、その実施状況とデータ品質が今後の規制当局およびパートナーシップに関する協議において重要となります。
• 国際的なASPIRE試験の計画は依然として初期段階にあり、対象国の評価が続いており、スクリーニングはまだ開始されていません。
• 臨床開発費用の増加に伴い、Acurxは純損失の計上を続けています。

アナリストQ&Aの要点

経営陣は、利用可能な原薬（API）および製剤化されたイベザポルスタットはPATHFINDER試験にとって十分であると述べました。また、同社はASPIRE試験の開始に必要な供給分を製造できる体制を整えています。

治験の堅調さについて、経営陣は高品質なデータ、極めて少ない治験実施計画書（プロトコル）違反や欠損情報、主要評価項目および治験実施施設間での一貫した有効性、ならびに米国の臨床実態に関連性の高い患者集団を強調しました。追跡調査は治療後8週間まで計画されています。

ASPIRE試験の急性期治療における評価項目は、優越性ではなくバンコマイシンに対する非劣性を検証します。経営陣によると、統計的フレームワークでは信頼区間下限に対して10%の非劣性マージンが使用されています。

経営陣は、PATHFINDER試験のデータが今後のパートナーシップ交渉や規制戦略の計画にとって重要であると考えています。良好な結果が得られれば、より広範なASPIRE試験のエビデンスパッケージを裏付けるか、あるいは再発性CDIに対するLPADルートに関するFDAとの協議を支える可能性があります。

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

Greetings. Welcome to Acurx Pharmaceuticals to discuss Second Quarter 2026 Financial Results on August 14, 2026 Conference Cal l and provide business update. [Operator Instructions] Please note, this conference is being recorded.

I'll now turn the conference over to Rob Shawah, Chief Financial Officer. Thank you. You may begin.

Robert Shawah

Thank you, Rob. Good morning, and welcome to our call. This morning, we issued a press release providing financial results and company highlights for the second quarter of 2026, which is available on our website at acurxpharma.com. Joining me today is Dave Luci, President and CEO of Acurx, who will start by providing a corporate update and outlook. Following that, I'll provide some highlights of the financials from the second quarter ended June 30 and then turn the call back over to Dave for his closing remarks.

As a reminder, during today's call, we'll be making certain forward-looking statements, which are based on current information, assumptions, estimates and projections about future events that are subject to change and involve a number of risks and uncertainties that may cause actual results to differ materially from those contained in the forward-looking statements.

Investors should consider these risks and other information described in our filings with the Securities and Exchange Commission, including our quarterly report on Form 10-Q, which we filed yesterday, Thursday, August 13, 2026. You are cautioned not to place undue reliance on these forward-looking statements, and Acurx disclaims any obligation to update such statements at any time in the future. This conference call contains time-sensitive information that's accurate only as of the date of this live broadcast today, August 14.

I'll now turn the call over to Dave Luci. Dave?

David Luci

Thanks, Rob. Good morning, everyone, and thank you so much for joining us to review our financial results for the second quarter and also to hear some recent updates on our continuing progress. Then we'd be pleased to take any questions.

Our Executive Chairman, Bob DeLuccia and our Medical Director, Dr. Michael Silverman, have joined us today and will be available to answer questions about our recent FDA meeting and our clinical development plan for ibezapolstat, both in recurrent and C. diff infection and an acute CDI.

First, I'd like to briefly summarize just a few of our key activities for the second quarter of 2026 or in some cases, shortly thereafter. Last month, in July 2026, the company's research and development team met with the FDA for the purpose of seeking their guidance on our plan to conduct a single Phase III study in acute CDI and its acceptability as a pivotal trial for filing a new drug application, the outcome of which we provided in more detail in our August 3 press release.

Briefly, the FDA stated that it is open to further discussion on the totality of evidence from the ibezapolstat clinical development program at a pre-NDA meeting after completion of a single Phase III trial called IBZ-ASPIRE and any other clinical trials conducted prior to the pre-NDA meeting, which will include the PATHFINDER study, 20-patient open-label and recurrent CDI, particularly if the clinical efficacy results are robust.

As you may recall from our previous announcements, we've begun start-up activities to conduct the 20-patient groundbreaking PATHFINDER study in mostly recurrent CDI with enrollment anticipated to start in the fourth quarter. This trial was acknowledged by FDA as being significant and along with robust results from our ASPIRE trial will form the basis of a potential approval for the treatment of both CDI and for the treatment of CDI and prevention of recurrent CDI.

In August 2026, the company received FDA conditional acceptance and USPTO Trademark Allowance of its proprietary name or brand name for ibezapolstat which I'll share with you now is Syfbezi. These initial milestones will form the basis for the commercial identity ibezapolstat as the company prepares to advance it towards its international Phase III registration program and ultimate commercialization.

Also in July, we signed and announced last week a continuation of our scientific partnership with Leiden University Medical Center to advance development of our DNA pol III C inhibitors. This new partnership builds on the previously reported successful results from the innovative research grant received from the Dutch government in November 2025. This new partnership will enable further mechanistic research into DNA pol III C inhibition to accelerate the development of novel new antibiotics that are systemically active against a wide range of Gram-positive pathogens resistant to currently available antibiotics.

This new research also aims to generate the first-ever 3D structure of pol C from methicillin-resistant Staphylococcus aureus in complex with an Acurx inhibitor to advance discovery of new compounds to treat this high-priority clinical pathogen as designated by the CDC and the FDA.

In the same month in July, a presentation of scientific data by Dr. Kevin Garey and his team from his laboratory at the University of Houston College of Pharmacy demonstrated that following treatment in a state-of-the-art laboratory model with ibezapolstat, the beneficial microorganisms in the gut will have the opportunity to repopulate the microbiome in a beneficial way that prevents recurrence.

In addition, IBZ and fidaxomicin were superior in biofilm experimental models with IBZ significantly more effective at killing C. diff than vancomycin and fidaxomicin. Acurx prepared to commence its Phase III clinical trial program with the ASPIRE trial for the treatment of both CDI and reduction of recurrence pending appropriate funding from public or private sources or partnerships. Our recent progress and efforts to secure this funding are ongoing.

I'd also point out that our PATHFINDER trial is fully funded and if successful, will elevate the product profile of IBZ as well as provide significant supportive data for FDA's evaluation. In April, the company announced the closing of a registered direct offering of up to $7.1 million issuing 825,085 shares of our common stock or prefunded warrants at a purchase price of $3.03 per share, priced at the market under NASDAQ rules.

In addition, in a concurrent private placement, the company issued unregistered short-term warrants to purchase up to 1.65 million shares of common stock. The short-term warrants have an exercise price of $2.78 per share and are immediately exercisable upon issuance and will expire 24 months following the effective date of the registration statement -- registering a resale of the shares of common stock underlying the short-term warrants. This additional funding when coupled with the remaining availability under our Equity Line of Credit, ensures that the company has a financial resource to conduct the PATHFINDER clinical trial in recurrent C. difficile and fund operations for at least 1 year.

Also in April, a scientific poster showing that our new DNA pol III C systemically absorbed antibiotics in preclinical development to treat other Gram-positive infections, achieve potentially therapeutic plasma levels and reduce MRSA tissue burden while maintaining a higher gut microbial diversity similar to baseline and distinct from linezolid. These data were presented at the 35th Congress of ESCMID, held in Munich, Germany, Dr. Khurshida Begum, Research Scientist in the laboratory of Dr. Kevin Garey at University of Houston presented the poster entitled Preclinical microbiome evaluation of novel Pol C inhibitor compounds.

Using microbiome profiling, metagenomics, the authors concluded that DNA pol III C antibiotic compounds represent a targeted strategy to treat resistant Gram-positive infections while preserving microbiome structure, minimizing downstream complications associated with antibotic-induced dysbiosis.

Commenting on the significance of this data Dr. Garey from the University of Houston stated, discovering highly selective antibacterial agents that specifically target systemic bacterial pathogens while avoiding the eradication of trillions of health-promoting bacteria in our gut microbiome, is the clinical holy grail of antibiotic development.

Initial works at the University of Houston with Acurx, novel pol III C inhibitors has demonstrated favorable gut microbiome [indiscernible] sparing effects. The novel findings presented at ESCMID demonstrate these positive microbiome results via class effect of DNA pol III C inhibitors potentially positioning them as unique additions to the anti-gram positive therapeutic armamentarium. So this work, coupled with our recently announced scientific partnership with Leiden University Medical Center will pave the way for further rational design of novel DNA pol III C inhibitors to expand our opportunities for lead optimization and our portfolio of groundbreaking anti-infective therapeutics.

With regard to our patents to date, Acurx has secured 6 U.S. patents and an additional 10 patents internationally, including Australia, Canada, Europe, Israel, India, Japan, Korea and Mexico. All of which protect key aspects of our company's ibezapolstat and the ACX-375C program, targeting DNA pol III C. Additional country-level patent applications remain under review. Also and significantly in the first quarter, a new patent was issued related to IBZ and it's used to treat CDI while reducing the recurrence of the infection as well as improving the health of the gut microbiome. Additional country-level patent applications remain under review.

We continue to identify and pursue funding opportunities for our Phase III ASPIRE trial for the treatment of both acute CDI and a reduction of recurrence. We have several initiatives underway to this end, and we'll report our progress on future updates. As we've continually reported, IBZ's clinical and nonclinical results continue to outperform in a serious and potentially life-threatening infectious disease caused by C. difficile bacteria that the CDC categorizes as an urgent threat and calls for new classes of antibiotics for initial treatment that also have a low incidence of recurrence.

Furthermore, IBZ has FDA QIDP and Fast Track designations for treatment of CDI as well as SME or small and medium enterprise status in the EU. All Acurx compounds and preclinical development are FDA and Fast Track eligible, not received yet, and target positive infectious disease classified as serious threat priorities by CDC and FDA.

We remain confident that while development of IBZ competitive profile continues to evolve and strengthen, we'll continue to successfully navigate through these challenging times in our industry sector. And now back over to Rob Shawah, our CFO, to guide you through the highlights of our financial results for the second quarter of 2026. Rob?

Robert Shawah

Thanks, Dave. Our financial results for the second quarter ended June 30, 2026, were included in a press release issued earlier this morning. The company ended the quarter with cash totaling $10.7 million, compared to $7.6 million as of December 31, 2025. During the quarter, the company raised a total of approximately $2.5 million of gross proceeds through a Registered Direct Offering, as well as $0.8 million under the Equity Line of Credit.

Research and development expenses for the 3 months ended June 30, 2026, were $1.1 million compared to $0.5 million for the 3 months ended June 30, 2025, an increase of $0.6 million. The increase was due primarily to an increase in manufacturing costs of $0.3 million, and an increase in consulting costs of $0.3 million as a result of costs associated with the new recurrent CDI trial program.

For the 6 months ended June 30, Research and development expenses were $1.4 million compared to $1.1 million for the 6 months ended June 30, 2025. The $0.3 million increase was due primarily to a $0.1 million increase in consulting fees and a $0.2 million increase in manufacturing costs as a result of costs associated with the new recurrent CDI trial program.

General and administrative expenses for the 3 months ended June 30 were $1.2 million compared to $1.7 million for the 3 months ended June 30, 2025, a decrease of $0.5 million. The decrease was primarily due to a $0.3 million decrease in professional fees, a $0.1 million decrease in legal costs and a $0.1 million decrease in share-based compensation expense.

For the 6 months ended June 30, general and administrative expenses were $2.6 million that was compared to $3.3 million for the 6 months ended June 30, 2025, a decrease of $0.7 million. The decrease was due primarily to a $0.3 million decrease in professional fees, a $0.2 million decrease in legal costs, and a $0.2 million decrease in share-based compensation expense.

The company reported a net loss of $2.3 million or $0.53 per diluted share for the 3 months ended June 30, 2026 that was compared to a net loss of $2.2 million or $1.89 per diluted share for the 3 months ended June 30, 2025. For the 6 months ended June 30, the company reported a net loss of $3.9 million or $1.13 per diluted share. That was compared to a net loss of $4.4 million or $4.01 per diluted share for the 6 months ended June 30, 2025, all for the reasons previously mentioned. The company had 4,683,253 shares outstanding as of June 30, 2026.

With that, I'll turn the call back over to Dave.

David Luci

Thanks, Rob, and to all of you for joining us today. Before bringing our operator back to open the call for questions, I'm pleased to welcome to the call our Medical Director, Dr. Michael Silverman and our Executive Chairman, Bob DeLuccia to assist with Q&A regarding our recent FDA meeting and our ibezapolstat clinical development program.

And now back to the operator to open the call for questions. Operator?

Operator

[Operator Instructions]

We'll move on to our question will be from Matthew Keller of H.C. Wainwright.

質疑応答

Matthew Keller

So my first one related to manufacturing. I was wondering, do you have enough API for the planned upcoming trials? And I guess, where do you stand or where do you stand potentially on manufacturing ibezapolstat?

David Luci

Thank you, Matt. Bob, would you like to...

Robert DeLuccia

We stand on -- and we have plenty of API and also the formulated product is all ready to go to support the PATHFINDER trial, and we're poised to have enough API manufacturing with appropriate dating to start the ibezapolstat ASPIRE trial as well.

Matthew Keller

Perfect. And then a second question, if I may -- go ahead, sorry.

Robert DeLuccia

Yes. No, I want to make sure that answered your question.

Matthew Keller

Yes, yes. And the second question, I guess, if I may. Again, you guided that the ASPIRE trial will be international trial. I was wondering what geographies you guys are considering and if you've begun sort of activities in those regions for that trial?

Robert DeLuccia

I can answer that as well, too. Mike, are you on the line, you can join in just to give an idea of the scope of the trial internationally.

Michael Silverman

Well, the plans in international. I don't have my finger on the pulse of every country that we've considered, but certainly Western and Eastern Europe. Bob, please expand if you can.

Robert DeLuccia

Yes, we can follow up with the details of the countries, but we haven't begun screening for that yet, but it will be all inclusive of those countries that we know have generally high incidence of C. difficile infection obviously.

Operator

[Operator Instructions] Our next question is from the line of James Molloy of Alliance Global Partners.

James Molloy

On the -- one of the things you guys highlighted on the August 3, you touched on the FDA is also may -- if the data is robust enough, it may give you induction as well as maintenance of remission is can you walk through sort of what constitutes reduction of remission? What constitutes the robust enough data? I know the FDA won't guide to that exactly. But in your mind, what gives you guys coming out of the PATHFINDER trial and going into ASPIRE? What are you sort of -- what's your target to -- can talk about sort of the FDA's interactions regarding that, please?

Robert DeLuccia

This is Bob. I'll let Mike join in. At the meeting, we laid out the parameters as to what would constitute robust. And Mike, do you want to go over those.

Michael Silverman

Yes. Thanks for the question. As you say, it's not something that can be specifically prescribed. But as Bob said, we proposed a number of potential criteria based on good clinical practices and also various FDA guidances. I like to think about it, the support of robustness in 2 general categories. One is what are these items that we would naturally build into a clinical trial, good clinical practive, high-quality data, minimization of protocol violations, minimization of missing data, those sorts of things to ensure that the data are trustworthy.

The second bucket of activities -- your second bucket of criteria would be those things that are inherent in drug, consistency of efficacy data across all of our endpoints, across all of our trial sites, across all of our countries. And I think this goes back to the previous question. We also need to ensure that our patient population is representative of the kinds of patients we've seen in the United States. So we do an international trial. We have to have an eye on clinical practice and clinical guidelines that are applicable in the United States. Those are the sorts of things that we're building in this trial. I hope that helps.

Robert DeLuccia

Yes. Just to build on that a little bit. Thank you, Mike and Bob. One of the features of this new ASPIRE trial design is to measure the patients an extraordinary amount of time after the end of treatment, 8 weeks after the end of treatment, which we don't know that that's been done before, but I think that also speaks to the robustness of the data. So if you're seeing no reinfection 8 weeks after the end of treatment and patient population that's had 3 or more prior episodes in the past year, we think the FDA will find that to be persuasive.

James Molloy

I guess, what's sort of the bogey with vanco that you're trying to beat assuming you do have some, of course, but how much better than vanco do you think the FDA will say that's robust?

Michael Silverman

Yes. In terms of -- it's another good point in statistical significance of the results. This is not a superiority trial. This is a noninferiority trial. So we don't have to show superiority over vancomycin for the clinical care acute treatment endpoint. We need to show noninferiority within standard bonds, which is a statistical concept but the lower limit would be confidence interval within 10%. That's a non-inferiority approach.

James Molloy

Excellent. And then maybe a final question for me would be, I know that the PATHFINDER is first, you've guided to maybe a year, 1.5 years to enroll. How much is -- and before you go to the ASPIRE trial, the final potentially pivotal trial, how important is the PATHFINDER data for a potential partnership to help fund the Phase III ASPIRE trial down the road?

David Luci

We think that's quite important. And we're heartened by the FDA's enthusiasm with our being willing to conduct that trial. Now interestingly, whether or not the ASPIRE trial is eventually funded, there's a second pathway to FDA approval under the LPAD pathway for recurrent C diff. So if we finish the 20 patients exploratory trial, open label, that we call PATHFINDER, we will meet with the FDA, and we have the possibility to be considered an LPAD pathway program, which would make -- which will make us -- give us the ability to file for approval in recurrence C. difficile with just one Phase III trial, which may be somewhere in the neighborhood of half the price of one of the ASPIRE trials.

Operator

This now concludes our question-and-answer session. And ladies and gentlemen, this also concludes today's conference. We thank you for your participation. Have a wonderful day.

David Luci

Thank you, Rob.

Operator

Thank you.