Nanoform's biologics technology showcased by large pharma at drug delivery conference; Nanoenzalutamide and nanoapalutamide partnering discussions progress as planned

HELSINKI, May 30, 2024 /PRNewswire/ -- Manufacturing of GMP material in Project Nanoenzalutamide commenced. EU/US pivotal studies are planned to start in 4Q24, with the read-out in 1Q25. One or several license/commercial supply agreements are expected to be signed during 2H24. Fimea's two-day inspection of our new GMP QC laboratory and new GMP lines is set for June 11-12. Takeda presented results from their nanoformed Biologics PoC study at DDF in Berlin, a strategic partnership was announced with CBC of Japan, a Business Finland grant of EUR 4m was won and EUR 15m was raised in a new share issue to build GMP capability to produce solid oral dosage forms for clinical trials and to create up to a dozen new product kernels like nanoenzalutamide and nanoapalutamide to be licensed out in the coming years. The previously communicated business targets for 2024: "Increased number of non-GMP and GMP projects in 2024 vs 2023" and "Improved operating free cash flow in 2024 vs 2023" are reiterated.

1-3/2024 key financials

• Revenue came in at EUR 0.6 million, stemming from 22 different customer projects (EUR 0.7m, 24 projects in 1-3/2023).
• The number of employees increased to 166 (152).
• Total operating costs* grew by 25 % to EUR 6.5 million (EUR 5.2 million) due to options related non-cash costs, investments in spare parts & building an internal maintenance function in addition to costs from the nanoenzalutamide project.
• EBITDA came in at EUR -5.7 million (EUR -4.5 million).
• The operating loss was EUR -6.5 million (EUR -5.1 million).
• The operating free cash flow improved to EUR -6.0 million (EUR -6.2 million)
• Basic EPS was EUR -0.09 (EUR -0.06)
• Cash position was EUR 41.3 million on March 31, 2024 (EUR 63.0 million).*

(Numbers in Brackets Refer to the Corresponding Last Year Reporting Period, Unless Otherwise Mentioned.)

^{* Defined as materials & services expenses, employee benefit expenses, and other operating expenses.
** Including Treasury bills. Part of the cash has been invested in short-term government bonds.
***Cash position after the new share issue was EUR 54.7 m on April 30, 2024.}

CEO's review

"Nanoform progresses on many fronts. Since our last report we have entered Japan by making a strategic partnership with CBC, Takeda presented results from their nanoformed biologics PoC study at DDF in Berlin, we won a Business Finland grant of EUR 4m to research and develop nanoparticle-enabled formulation platforms for oral, inhaled, long-acting injectable, and high-concentration subcutaneous injectable drug delivery and we raised EUR 15m in a new share issue to build GMP capability to produce solid oral dosage forms for clinical trials and to create up to a dozen new product kernels like nanoenzalutamide and nanoapalutamide to be licensed out in the coming years. Last, but certainly not least, in project Nanoenzalutamide, we have now started clinical manufacture related to the upcoming pivotal EU/US studies. The clinical trials are expected to commence in 4Q24 and the results are expected in 1Q25.

We expect nanoenzalutamide to be the first nanoformed medicine to reach the market - with a planned launch in 2027/28 in the US/EU - and to be a revenue driver for Nanoform already in the upcoming years. We expect to execute one or several licensing/commercial supply agreements in 2024 and expect these to include customary payments already at signing and later when meeting developmental- and regulatory milestones. Long-term we expect to receive royalty payments based on sales when the product is on the market.

Nanoenzalutamide is expected to progress via the ANDA^*/Hybrid generic pathway and as such will need to show bioequivalence versus the originator product, Xtandi^®. In the eyes of the regulators, bioequivalence typically means 80% - 125% of the Cmax and AUC in a large cohort study in fed and fasted states with a 90% confidence interval."

^{*ANDA=Abbreviated New Drug Application} 

"The global annual sales of Xtandi^® is presently USD 6bn and growing. We plan for nanoenzalutamide to take a meaningful share of this market through its highly patient centric product differentiation (1 tablet vs 4 tablets) and unique IP position (different technology, crystalline product, different excipients), while not forgetting its green attributes. We now actively pursue commercial licensing and marketing partners for the product together with our partners in the ONConcept^® consortium. We see the program to be attractive to value added medicine companies as a uniquely differentiated and high value supergeneric product that can enable a product launch before market entry by other generic products based on the ASD formulation, for which the originator currently holds patents in both Europe and the US (with expiry dates in 2033). For the originator company we believe the nanocrystalline single tablet product offers a patient centric life cycle extension opportunity with compelling sustainability advantages that would be difficult for generic competitors to match. Avoiding the inherent stability challenges associated with amorphous materials is also a clear benefit for any company considering alternative formulation approaches.

Xtandi-tablets are formulated using a solubility-enhancement spray-drying process to create an amorphous solid dispersion. The major challenge with spray drying is that the process often requires large amounts of undesirable and toxic organic hydrocarbon solvents.  Nanoform's CESS^® process uses CO₂ of recycled origin, and is organic hydrocarbon solvent-free, offering a greener alternative to medicine developers that seek to be both patient- and planet-centric. Nanoform continuously improves the CESS^® technology, e.g. by planning to further recycle the CO₂ used by the process to become a carbon sink.  This is an attractive proposition for the pharma industry to achieve its ambitious net zero goals. There are already concerns in the industry that industrial approaches with a heavy carbon footprint, e.g. spray drying, may lose their relevance in the future because of their environmental burden.

The timelines for the commercial launch of nanoenzalutamide are demanding, but achievable. In 2024 we need to manufacture nanoformed GMP material for the registration batches and the pivotal bioequivalence studies. When positive, the submissions of the dossiers should be in 2025-26, with the aimed product launch after the expiry of the enzalutamide substance patent in the respective territories. For the United States this patent expiry is expected in 2027, and in Europe in 2028.

We now have an inspection date from Fimea. They will do a two-day inspection of our new GMP QC laboratory and our new GMP lines 2&3 on June 11-12. A successful inspection will expand our GMP capacity by more than 3x and will help our gross margin to return to the targeted 90%+.

On the business development side we have made solid progress among large pharma, with a growing interest also in our biologics technology. The US biotech sector shows clear signs of increased activity levels. While our revenue development during the last year was unsatisfactory, I am confident - based on the clearly increased - by number and depth - discussions with large-, mid-sized pharma and biotech companies, that we will reach our 2024 targets of more projects signed and improved cash flow in 2024 compared with 2023. 

For Nanoform the last three years has been about making large investments and building a capable organization. The coming three will be about preparing to launch nanoformed products together with partners onto the global markets. We are ready for the challenge. I look forward with confidence and excitement to the coming years. None of this can be done without our amazing employees and great partners. My sincere THANK YOU to you all for your continued dedication to Nanoform and for the inspiring and innovative work for which we're known."

Best Regards, 
Prof. Edward Hæggström, CEO Nanoform
Project Nanoenzalutamide and ASDs (amorphous solid dispersions)

Nanoenzalutamide is a great opportunity for us to show that small is a powerful ingredient in formulation. Due to the inherent poor solubility of the API, the current formulation of this blockbuster medicine - for treatment of prostate cancer - has been an amorphous solid dispersion ("ASD"). Amorphous API materials are notoriously unstable, and therefore require high amounts of polymers to stabilize the API - leading to a low drug load in the product and therefore, in the case of oral solid products, often to a high number of large tablets that need to be taken by the patient. This is a known problem, in particular for patient populations with challenges to swallow. The nanocrystalline formulation developed by Nanoform offers an attractive alternative with a substantially higher drug load in the final drug product and consequently a reduced tablet burden for the patient. We are encouraged by the broad interest shown in this patient centric reformulation and we look forward to signing license/supply agreements around this product opportunity in 2024. In addition to the patient benefit, we can with our proprietary technology offer opportunities to extend IP protection for the reformulated and improved product, expecting that in many cases our innovative formulations will be patentable. Importantly, current ASD based medicines are often protected by secondary patents that claim aspects of the ASD formulation. These secondary patents, such as in the case of the product in Project Nanoenzalutamide, often extend by several years the expiration of the primary patent claiming the API. In the case of Project Nanoenzalutamide, we believe that our nanocrystalline formulation is not in the scope of the patents claiming the ASD formulation. This should potentially enable entry earlier into the market, in the jurisdictions where the ASD formulation patents remain active, compared to ASD based generic formulations. ASDs remain a leading formulation strategy for poorly soluble APIs, particularly for oral solid dosage forms. There are currently some 50 marketed medicines that are ASDs and these sell in aggregate for some USD 50bn annually in the world. We are currently looking at several other marketed ASD opportunities to replicate our early successes with Project Nanoenzalutamide in addition to those ASDs still in the global drug development pipeline. According to STARMAP®, almost 80 per cent of the 46 ASDs we so far have starmapped may be well suited to be nanoformed by CESS^®.

Nanoform's Q1 2024 report can be found at: https://nanoform.com/en/financial-reports-and-presentations/

Nanoform online presentation and conference call May 30, 2024, at 3:00 p.m. Helsinki time / 2:00 p.m. Stockholm time

Nanoform will publish its Q1 2024 report on May 30, 2024, at 8.10 a.m. Finnish time / 7.10 a.m. Swedish time. The company will hold an online presentation and conference call the same day at 3.00 p.m. Finnish time / 2.00 p.m. Swedish time. Nanoform will be represented by CEO Edward Hæggström, CFO Albert Hæggström and CCO Christian Jones. The presentation will be delivered in English.

The presentation will be broadcast live as a webcast available at:

https://ir.financialhearings.com/nanoform-q1-report-2024

Teleconference dial-in numbers:

Dial-in number to the teleconference will be received by registering via the link below. After the registration you will be provided phone numbers and a conference ID to access the conference. Questions can be presented by this dial-in function.

https://conference.financialhearings.com/teleconference/?id=50049781

 Significant events during 1-3/2024

• On January 5, 2024, Nanoform announced it had completed the First Subject First Visit (FSFV) in a trial to evaluate the relative bioavailability of its nanocrystalline enabled alternative to an amorphous solid dispersion (ASD); formulation of nanoenzalutamide and Xtandi^®[1], the number one prescribed androgen receptor inhibitor first approved by the FDA in 2012 to treat prostate cancer. The single-dose, randomized, comparative bioavailability study, performed by a contract research organization in North America, compared enzalutamide 160 mg film-coated tablets (Bluepharma Farmaceutica S.A.) and Xtandi 4x40 mg film-coated tablets (Astellas Pharma Europe B.V.).
• On January 10, 2024, The Board of Directors of Nanoform Finland Plc decided on the issue of stock options under an option program open to all employees. The total number of option rights to be issued is at most 1,240,412. The stock options are entitled to subscribe for at most 1,240,412 shares in Nanoform. Each stock option entitles to subscribe for one new share. The subscription price for shares subscribed with stock options is EUR 1.70 per share. The total subscription price of the shares shall be paid to the company's fund for invested own free equity.
• On January 26, 2024, Nanoform announced that one of its leading nanoformulation drug products had received promising clinical results. These were from a relative bioavailability study of nanocrystalline-enabled enzalutamide (nanoenzalutamide) tablet formulation, an alternative to the amorphous solid dispersion (ASD) used in Xtandi^®[1], the number one prescribed androgen receptor inhibitor^[2] first approved by the FDA in 2012, and by the EMA in 2013 to treat prostate cancer. The nanoenzalutamide tablet formulation was developed in a partnership with the ONConcept^® Consortium (Bluepharma, Helm, and Welding) whereby Nanoform´s proprietary controlled expansion of supercritical solutions (CESS^®) technology provides the opportunity for an improved and differentiated finished product. Tablet-burden and dysphagia are well-documented challenges for prostate cancer patients, and the development of a 160mg, single tablet per day regimen may be preferable for patients in need of reducing their total number of daily pills. A patent application for the nanoenzalutamide formulation has already been jointly filed by Helm and Nanoform. We aim for product launch after the expiry of the enzalutamide substance patent in the respective territories. For the United States this patent expiry is expected in 2027, and in Europe in 2028. This unique IP position may allow the nanoenzalutamide product to enter the market prior to other generic competition based on the ASD formulation, which is currently patent protected in the US and Europe until 2033. [1] Xtandi is a registered trademark of Astellas Pharma Inc. [2] Source: xtandi.com
• On February 15, 2024, Nanoform announced that it has won a grant of up to 4.3 million euros from Business Finland, the Finnish government organization for innovation funding and trade. The grant represents 50% of the costs associated with Nanoform's research and development project for nanoparticle-enabled formulation platforms for oral, inhaled, long-acting injectable, and high-concentration subcutaneous injectable drug delivery technologies for next generation medicines. The work is expected to take place during 2024 and 2025.
• On February 29, 2024, Nanoform announced it had received positive results from its own pre-clinical, in vivo study of a nanocrystalline-enabled apalutamide oral formulation, which shows potential to enable a much smaller tablet than Erleada®^[3], a nonsteroidal antiandrogen (NSAA) blockbuster amorphous solid dispersion (ASD) medicine used to treat prostate cancer. [3] Erleada is a registered trademark for Apalutamide owned by Johnson & Johnson / Janssen Biotech, Inc.
• On March 12, 2024, The Board of Directors approved share subscriptions based on stock option programs 2/2019 and 1/2020. A total of 11,200 Nanoform Finland Plc new shares were subscribed and the entire subscription price for subscriptions made with the stock options of EUR 13 thousand was entered in the Company's reserve for invested unrestricted equity.
• On March 26, 2024, The Board of Directors of Nanoform Finland Plc decided on the issue of stock options to key individuals. The total number of option rights to be issued is at most 200,000. The stock options entitle to subscribe for at most 200,000 shares in Nanoform. Each stock option entitles to subscribe for one new share. The subscription price for shares subscribed with stock options is EUR 3.00 per share, which is approximately 25 percent more than the closing price on 25 March 2024. The total subscription price of the shares shall be paid to the company's fund for invested own free equity.

Significant events after 1-3/2024

• On April 8, 2024, Nanoform announced that effective April 10, 2024, onwards, the Certified Adviser to Nanoform Finland Plc is Carnegie Investment Bank Ab (publ). The Certified Adviser to Nanoform Finland Plc until April 9, 2024, was Danske Bank A/S, Finland Branch.
• On April 9, 2024, Nanoform announced a collaboration with PlusVitech, a biotechnology company developing treatments for cancer, to use Nanoform's state-of-the-art nanomedicine technology to repurpose the anti-nausea medicine aprepitant as a treatment for lung cancer. The development program will include nanoforming the current active ingredient into crystalline nanoparticles and formulating a simplified dose regimen with fewer and smaller pills. The partnership is expected to include API supply for late-stage clinical programs and eventual product launch. Following PlusVitech's positive first time in human studies, a Phase 2 study with 24 patients has commenced, investigating the efficacy of high dose aprepitant in a non-small cell lung cancer (NSCLC) population that is refractory to standard treatment. The current formulation carries a high pill burden of potentially dozens of capsules per day, with a complicated regimen for patients that are most often frail and have trouble swallowing (dysphagia). The new formulation by Nanoform is designed to deliver substantially higher drug load with better bioavailability, which simplifies the dose regimen and improves patient convenience and compliance. Repurposing an existing drug offers a potentially faster, risk-reduced and cost-effective development path to provide new treatments for high medical need indications like NSCLC.
• On April 11, 2024, Nanoform announced a strategic partnership whereby CBC Co., Ltd. ("CBC"), will utilize its extensive experience in the Japanese pharmaceutical industry to identify opportunities for Nanoform's cutting-edge nanomedicine engineering technologies. The partnership commenced immediately, and the companies have planned several joint activities to introduce their collaboration, including the held representation of Nanoform by CBC at CPHI Japan, which took place at Tokyo Big Sight, Tokyo, from April 17-19, 2024.
• Nanoform's Annual General Meeting (the "AGM") was held on April 16, 2024. 46 shareholders representing more than 60 per cent of all outstanding shares and votes were represented at the meeting. The Annual General Meeting supported all the Board of Directors' proposals. The AGM approved the financial statements and discharged the Board of Directors and the CEO of the Company from liability for the financial year 2023. The AGM further resolved the number of members of the Board of Directors to be four and the AGM re-elected Miguel Calado (Chairperson), Mads Laustsen, Albert Hæggström and Jeanne Thoma as ordinary members of the Board of Directors for the next term of office.
• On April 24, 2024, Nanoform announced that it had successfully completed a new share issue raising EUR 15.4m by issuing 7m new shares (8.9% dilution) at EUR 2.20 (SEK 25.60) per share in order to invest in the commercialization of nanoparticle enabled formulations for next generation medicines. The placing attracted a considerable number of leading Nordic and institutional investors. The proceeds will be used to build a GMP level formulation facility to produce solid oral dosage forms for clinical trials, to development of up to a dozen pre-clinical nanocrystalline alternatives to ASD medicines ready for partnering in clinical trials and to co-fund several projects to be taken by Nanoform and its partners into clinical trials in the EU and the US, and ultimately through to the market. The total number of issued shares in the company after the placing will be 85,445,164.
• On May 22, 2024, at the 15th Global Drug Delivery & Formulation Summit in Berlin, Andreas Liebminger, Ph.D., Global Head of Plasma-derived Therapies Pharmaceutical Sciences, Takeda, presented data obtained in a proof of concept study, conducted in collaboration with Nanoform. Controlling the viscosity and aggregation of protein-based solutions is important for pharmaceutical formulators. Because injection volume is limited by the device, therapeutic protein formulations which are to be delivered via intramuscular or intravenous injection need to be highly concentrated. At protein concentrations greater than 200 mg*mL-1 however, viscosity increases to significantly higher than 20 cP (centipoise) to quickly exceed the maximum 40 cP viscosity deemed acceptable for a conventional subcutaneous injection. The data support the potential of Nanoform's patented biologics platform to achieve high protein concentrations in suspension formulations that are suitable for subcutaneous injection, as shown by results of syringeability and injectability studies.

Company near-term business targets for 2024

• Increased number of non-GMP and GMP projects signed in 2024 vs 2023
• Improved operating free cash flow in 2024 vs 2023
• To sign one or several license/commercial supply agreements during 2024

Company mid-term business targets 2025

• To nanoform at least 70 new Active Pharmaceutical Ingredients (API) annually
• To have in place 35 operating production lines of which 7 to 14 are expected to be GMP production lines
• Over 90 percent gross margin
• To have 200–250 employees
• To be cash flow positive

For further information, please contact:
Albert Hæggström, CFO
albert.haeggstrom@nanoform.com / +358 29 370 0150

For investor relations queries, please contact:
Henri von Haartman, Director of Investor Relations
hvh@nanoform.com / +46 7686 650 11

About Nanoform

Nanoform is the medicine performance-enhancing company that leverages best-in-class innovative nanoparticle engineering technologies, expert formulation, and scalable GMP nano-API manufacturing to enable superior medicines for patients. The company focuses on reducing clinical attrition and on enhancing drug molecules' performance through its nanoforming technologies and formulation services, from pre-clinical to commercial scale. Nanoform will help improve bioavailability and drug delivery profiles, drive differentiation, patient adherence and extend the lifecycle potential of products. Nanoform's shares are listed on the Premier-segment of Nasdaq First North Growth Market in Helsinki (ticker: NANOFH) and Stockholm (ticker: NANOFS). Certified Adviser: Danske Bank A/S, Finland Branch, +358 40 744 1900. For more information, please visit www.nanoform.com.

Forward-Looking Statements

This press release contains forward-looking statements, including, without limitation, statements regarding Nanoform's strategy, business plans and focus. The words "may", "will", "could", "would", "should", "expect", "plan", "anticipate", "intend", believe", "estimate", "predict", "project", "potential", "continue", "target" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements in this press release are based on management's current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release, including, without limitation, any related to Nanoform's business, operations, clinical trials, supply chain, strategy, goals and anticipated timelines, competition from other companies, and other risks described in the Report of the Board of Directors and Financial Statements for the year ended December 31, 2023 as well as our other past disclosures. Nanoform cautions you not to place undue reliance on any forward-looking statements, which speak only as of the date they are made. Nanoform disclaims any obligation to publicly update or revise any such statements to reflect any change in expectations or in events, conditions or circumstances on which any such statements may be based, or that may affect the likelihood that actual results will differ from those set forth in the forward-looking statements. Any forward-looking statements contained in this press release represent Nanoform's views only as of the date hereof and should not be relied upon as representing its views as of any subsequent date.

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