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Strong momentum in Project Nanoenzalutamide

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Company Announcement

Nanoform Finland Plc

February 29, 2024

08:10 a.m. Finnish time / 07:10 a.m. Swedish time

Strong momentum in Project Nanoenzalutamide

Project Nanoenzalutamide sees strong momentum. The clinical results in the pilot study were very promising. Now we are preparing for the manufacture of nanoformed material for the registration batches and the EU/US pivotal studies that are planned to start during 2024. In parallel, Nanoform and the members of the ONConcept® Consortium have initiated discussions with many potential partners around this exciting project. The interest in the project is broad and we expect to sign one or several license/commercial supply agreements during 2024. While we signed a record number of new non-GMP projects in 2023, revenues were hampered by low signings in 2H22 and the gross margin by costs related to project Nanoenzalutamide, but the free cash flow continued to improve. The balance sheet remains solid with EUR 47.5m in cash and no debt. The previously communicated target of signing one or several license/commercial supply agreements during 2024 is reiterated, while we add the following 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”.

10-12/2023 key financials
  • Revenue was impacted by slow signings during 2H22 and decreased to EUR 0.4 million, compared with EUR 1.0 million in 10-12/2022.
  • The gross profit decreased to EUR 0.3 million, with a gross margin of 74 % (EUR 0.8 million, 82 % in 10-12/2022) due to GMP QC costs related to the Project Nanoenzalutamide.
  • Total operating costs* remained unchanged at EUR 5.8 million (EUR 5.8 million).
  • The number of employees grew to 165 (150) compared with one year ago.
  • EBITDA came in at EUR -5.4 million (EUR -4.8 million).
  • The operating free cash flow improved to EUR -5.9 million (-6.8 million).
  • Basic EPS was EUR -0.07 (EUR -0.07).
  • Cash position** was EUR 47.5 million on December 31, 2023 (EUR 68.7 million).
1-12/2023 key financials
  • Revenue came in at EUR 2.6 million, stemming from 33 different customer projects (EUR 3.5m, 35 projects in 1-12/2022).
  • The gross profit decreased to EUR 1.7 million, with a gross margin of 67 % (EUR 3.1 million, 90 %) due to GMP QC costs related to the Project Nanoenzalutamide.
  • The number of employees increased to 165 (150).
  • Total operating costs* decreased by 1 % to EUR 22.2 million (EUR 22.5 million).
  • EBITDA came in at EUR -19.6 million (EUR -19.0 million).
  • The operating loss was EUR -22.5 million (EUR -21.4 million).
  • The operating free cash flow improved to EUR -23.1 million (-28.0 million).
  • Basic EPS was EUR -0.26 (EUR -0.29).
(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 bond

CEO’s review

What a start to the new year! After years of hard work by many dedicated and talented people both within and outside of Nanoform, it was extremely rewarding to get such a powerful set of promising data from our relative bioavailability study of nanoenzalutamide vs Xtandi®. Not only does the data in the clinical study provide a strong indication that our technology can be an improvement for medicines based on the current state-of-the-art and widely accepted industry approach of ASDs (amorphous solid dispersions), but it also gives us and our partners the reason to move forward towards pivotal studies with the aim to launch an improved patient-centric version of this blockbuster medicine in the coming years. From a broader point-of-view, it also shows the potential of what nanoforming potentially can do to many other ASDs both on the market and in the global pharmaceutical pipeline. We are further strengthened in our conviction that nanoforming can become a powerful and green new technology for the entire global pharma industry in their quest to help both patients with new and better medicines, but also help the planet by introducing environmentally friendly manufacturing technologies.We expect nanoenzalutamide not only to be the first first nanoformed medicine to reach the market - with a planned launch in 2027 in the US and 2028 in the EU - but also to be a significant revenue driver for Nanoform already in the upcoming years. We target 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 expect 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). We are now actively pursuing 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 launch of the product before the entry into the market of 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 strategy 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 are working on continuous improvement of the CESS® technology to further recycle CO2 used by the process to become a carbon sink.  This will be an attractive proposition for the pharma industry to achieve its ambitious net zero goals. There are already concerns in the industry that those industrial approaches that have a heavy carbon footprint, such as spray drying, may even 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 circa 100 kg of nanoformed GMP material for the registration batches and the pivotal bioequivalence studies in the EU and the US that are expected to commence late in 2024, with the read-outs in 2025. 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. A few words on the performance outside the nanoenzalutamide project: On the business development side we are making solid progress among large pharma, with a growing interest also in our biologics technology. While the biotech sector is still held back by tough funding conditions, there are clear signs of increased activity levels. While our revenue was hampered in 2H23 by low project signings in 2H22, the higher amount of signings in 2023 vs 2022 and the significant interest in Nanoform we have seen after the announcement of the nanoenzalutamide results make me confident in expecting more projects signed and improved cash flow in 2024 compared with 2023. We also have clearly more - by number and depth - strategic discussions with large and mid sized pharma compared to a year ago. For Nanoform the last three years since the IPO has been about making large investments and building a capable organization. The coming three to five years 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 Q4 & FY 2023 report can be found at: https://nanoform.com/en/financial-reports-and-presentations/

Nanoform online presentation and conference call February 29, 2024, at 3:00 p.m. Helsinki time / 2:00 p.m. Stockholm time

Nanoform will publish its Q4 & FY 2023 report on February 29, 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-q4-report-2023

Teleconference dial-in numbers:

Dial-in number to the teleconference will be received by registering on the link below. After the registration you will be provided phone numbers and a conference ID to access the conference.

https://conference.financialhearings.com/teleconference/?id=50046525

Significant events during 1-12/2023
  • As of January 1, 2023, Antonio da Silva was appointed CBO and a member of the management team.
  • On January 3, 2023, Nanoform established a new subsidiary in the UK, Nanoform U.K. Ltd.
  • On January 10, 2023, the Board of Directors approved share subscriptions based on stock option programs 3/2019, 5/2019 and 1/2020. A total of 29,000 Nanoform Finland Plc new shares were subscribed and the entire subscription price for subscriptions made with the stock options of EUR 34 thousand was entered in the Company’s reserve for invested unrestricted equity.
  • On February 28, Nanoform announced two new near-term business targets for 2023: “Increased number of non-GMP and GMP projects signed in 2023 vs 2022” and “Improved operating free cash flow in 2023 vs 2022”.
  • Nanoform’s Annual General Meeting (the “AGM”) was held on April 12, 2023. The AGM approved the financial statements and discharged the Board of Directors and the CEO of the Company from liability for the financial year 2022. The Meeting decided that no dividend will be paid for the financial year that ended on December 31, 2022. 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 12, 2023, the Board of Directors approved share subscriptions based on stock option programs 2-3/2019 and 1/2020. A total of 37,000 Nanoform Finland Plc new shares were subscribed and the entire subscription price for subscriptions made with the stock options of EUR 41 thousand was entered in the Company’s reserve for invested unrestricted equity.
  • In April, Nanoform won a new grant from the Bill & Melinda Gates Foundation to work on several of the foundation’s drug development projects.
  • In May Nanoform’s Manufacturer’s Authorization and GMP Certificate were updated to include nanoforming of multiple APIs in the GMP facility.
  • In June, Nanoform submitted a notification to the Finnish Medicines Agency to update our Manufacturer’s Authorization (MIA). The objective of this notification was to include the following in our MIA: Our new production facilities and equipment (GMP2&3), our new Quality Control laboratory (GMP QC) and Nanoforming of APIs to be used in products with a Marketing Authorization. Due to this notification, a GMP inspection is expected to take place during 1H24.
  • Nanoform previously disclosed on November 15, 2021, that it has signed an agreement to manufacture nanoformed GMP material for a European headquartered international company. Following 12 months of preclinical development work, two privately held European pharmaceutical development and manufacturing organizations decided to join Nanoform and the European headquartered international company in funding the development and commercialization of this more patient centric version of a current blockbuster drug. For this purpose, the parties entered into a collaboration agreement on November 17, 2022. Under the terms of the agreement, Nanoform and the three other parties will fund in equal shares the completion of this development program. In the event that the commercialization is successful, Nanoform expects to retain a 25 % share of the net-income received by the parties. In May 2023, after Fimea renewed Nanoform’s GMP Certificate, Nanoform commenced the clinical manufacture related to this project.
  • In June, Nanoform and Celanese Corporation, a global specialty materials company, provided an update on their collaboration to evaluate the synergies between their respective technologies in the field of nanoparticle-enabled drug delivery. The result, presented at the Biotech Outsourcing Strategies Conference in Basel on July 3, 2023, demonstrated significant reduction in the initial burst effect seen commonly in high drug load implants by combining Nanoform’s CESS® particles with Celanese’s Celanese VitalDose® EVA copolymer delivery technology for drug-eluting implants. Notably they also demonstrated that nanoformed particles can enable longer sustained release properties for long-acting drug products and smaller implants. This opens up many possibilities for drug developers.
  • During the third quarter, the clinical manufacture related to Project Nanoenzalutamide was successfully completed and the produced nanomaterial was released and shipped for manufacture of the final drug product. It was announced that clinical trials are expected to commence in 4Q23, that results are expected in 1Q24 and that if the results are positive, the targeted timeline for one or several license/commercial supply agreements is during 2024.
  • Chief Quality Officer Johanna Kause became a member of Nanoform’s management team as of September 1st, 2023. Johanna Kause, who is responsible for all matters related to quality, has been with the company since January 2021.
  • We received notice of allowance from the United States Patent and Trademark Office (USPTO) for our US patent application (US17947490) directed at the process we have developed to nanoform biological molecules. We are encouraged by this positive response that reflects our innovative work also in the field of large molecules. We have filed several patent applications directed at the biologics nanoforming technology in other jurisdictions that are currently pending. Following granted patents in the United States, Japan, and Canada, we also received notification from the European Patent Office (EPO) of their intention to grant our corresponding European patent application (EP15793857.2) directed at the CESS® technology for manufacture of our small molecule nanoparticles.
  • We conducted promising initial in vitro trials with two major pharma companies looking at monoclonal antibodies (mAb’s). These results further strengthen our proposition that nanoparticles are relevant for improved product development and more patient centric commercial products in the field of mAb’s and we look forward to advancing these developments with our pharma clients.
  • In October, Nanoform announced that its customer TargTex S.A. had been granted Orphan Drug Designation by the FDA for its nanoformed drug candidate TTX101 to be used in patients with malignant gliomas.  The orphan drug designation follows the generation of a preclinical rodent data package in which a survival advantage was shown for this nanoform-enabled medicine candidate. The hydrogel nanoformulation developed by Nanoform enabled a 200-fold increase in drug load compared to bulk and a 5-fold increase in drug load compared to nanomilling. Hence Nanoform’s proprietary technology and nanoformulation expertise will enable TargTex’s drug candidate TTX101 to move towards clinic. TargTex is currently raising funds to take this innovative treatment to clinic and is planning a phase 1/2a clinical trial in recurrent glioblastoma (GBM) patients across the US and EU, in which nanoformed TTX101 is applied as adjunct to surgery after tumour excision.
  • In October, Nanoform announced that it had granted AstraZeneca Plc a global online STARMAP® license. STARMAP® is a digital AI version of the CESS® technology that enables in silico experiments to determine which molecules should be nanoformed. The license will enable AstraZeneca to screen molecules from drug discovery through to lifecycle management. As part of this licensing agreement, Nanoform will receive access to compound libraries and large data sets to undertake STARMAP® screening and propose innovative product development concepts and strategies in collaboration with AstraZeneca. This comes after several years of early-stage collaboration between Nanoform and AstraZeneca and a successfully completed technology evaluation partnership including STARMAP® which has resulted in clinical candidate feasibility studies. STARMAP® is well aligned with AstraZeneca’s ambitious sustainability goals.

STARMAP® Online has been created as a direct request from Nanoform’s current and future partners who seek to maintain the level of confidence STARMAP® offers, while integrating it into their own in-house molecule-selection processes.  STARMAP® Online creates the opportunity for clients to perform large numbers of in silico CESS® experiments from their desktop.  This approach further supports Nanoform’s green ambition by ensuring that Nanoform progresses the molecules with the greatest probability of success. STARMAP® Online offers:

Security and safety – the interface has been developed in alignment with ISO27001:2017 standards.

Client submissions are confidential and seen only by clients (not by Nanoform), allowing molecules to be screened without sharing structures. Outputs are presented directly to the client via the system.

Scalability and agility: The ability to manage thousands of molecules in a single submission to support the selection of candidates from molecule libraries is possible.

Novel insights: STARMAP® Online holds a database of some 20,000 pre-analyzed, public-domain disclosed drugs and candidates. Clients can request thematic evaluations and understand the power of CESS® in different therapeutic areas, target classes, and disease areas.

  • On December 5, 2023, the Board of Directors approved share subscriptions based on stock option programs 2/2019 and 1/2020. A total of 4,000 Nanoform Finland Plc new shares were subscribed and the entire subscription price for subscriptions made with the stock options of EUR 6 thousand was entered in the Company’s reserve for invested unrestricted equity.
  • During 2023, 22 new non-GMP projects and one GMP project were signed, both with new and repeat customers, both US and Europe based. We also signed our first major pharma customer from Japan.
  • During 2023 one new non-GMP line was commissioned, taking the total number of lines to 19 non-GMP lines and one GMP line. GMP lines 2&3 will be commissioned after they are inspected and approved by Fimea, which is expected to happen during 1H 2024.
Significant events after 1-12/2023
  • 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, which is performed by a contract research organization in North America, compares 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. The single-dose, randomized, comparative bioavailability study, which was performed by a contract research organization in North America, compared enzalutamide 160mg filmcoated tablets (Bluepharma) and Xtandi® 4x40 mg film-coated tablets (Astellas Pharma Europe B.V.). 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.
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 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-formulation 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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