On May 6, 2021 Aprea Therapeutics, Inc. (Nasdaq: APRE), a biopharmaceutical company focused on developing and commercializing novel cancer therapeutics that reactivate the mutant tumor suppressor protein, p53, reported financial results for the three months ended March 31, 2021 and provided a business update (Press release, Aprea, MAY 6, 2021, View Source [SID1234579431]).

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"During our recent R&D Day presentation, we reported an analysis of available data from its Phase 3 MDS trial. We identified an imbalance in dose modifications in the experimental arm which we believe negatively impacted efficacy, particularly the primary CR endpoint," said Christian S. Schade, Chairman and Chief Executive Officer of Aprea. "We remain confident that eprenetapopt and our next-generation oral agent, APR-548, represent important potential therapeutic options for cancer patients and are encouraged by emerging data from our ongoing clinical trials. We look forward to sharing data updates from these clinical trials as well as our continued progress in expanding the opportunity for our therapies in new indications."

Business Operations Update:

The Company is conducting, supporting, and planning multiple clinical trials of eprenetapopt (APR-246) and APR-548:

Phase 3 Frontline MDS Trial — In June 2020, the Company completed full enrollment of 154 patients in a pivotal Phase 3 trial of eprenetapopt with azacitidine for frontline treatment of patients with TP53 mutant MDS. The pivotal Phase 3 trial is supported by data from two Phase 1b/2 investigator-initiated trials, one in the U.S. and one in France, testing eprenetapopt with azacitidine as frontline treatment in TP53 mutant MDS and AML patients. The data from the U.S. and French Phase 1b/2 trials were published in The Journal of Clinical Oncology in January 2021 and February 2021, respectively. In December 2020, the Company announced that its pivotal Phase 3 trial failed to meet its predefined primary endpoint of complete remission (CR) rate. Analysis of the primary endpoint at this data cut demonstrated a higher CR rate (53% more patients achieving a CR) in the experimental arm receiving eprenetapopt with azacitidine versus the control arm receiving azacitidine alone but did not reach statistical significance. Based on a thorough analysis of the current Phase 3 trial data and comparisons to the U.S. and French Phase 1b/2 trials the Company believes that despite similar types and frequency of adverse events observed in the Phase 3 experimental arm and the Phase 1b/2 trials, patients in the Phase 3 experimental arm experienced substantially more study treatment dose modifications compared to the experience in the U.S. and French Phase 1b/2 trials. The Company believes that dose modifications of eprenetapopt and azacitidine led to undertreatment in the Phase 3 experimental arm that negatively impacted efficacy, particularly the primary endpoint of CR rate. The Company continues to follow patients who remain on-study and anticipates discussing with FDA the Phase 3 data and future possible regulatory pathways in the second half of 2021.
Phase 2 MDS/AML Post-Transplant Trial – The Company has completed enrollment of 33 patients in a single-arm, open-label Phase 2 clinical trial evaluating eprenetapopt with azacitidine as post-transplant maintenance therapy in TP53 mutant MDS and AML patients who have received an allogeneic stem cell transplant. The primary endpoint of the trial is the rate of relapse-free survival (RFS) at 12 months, with a published benchmark of ~30%. An interim analysis in April 2021 showed a 62% rate of RFS at 12 months, with a median RFS of 462 days. An interim analysis of overall survival (OS) showed a 77% OS at 1 year, with a median number of events not yet reached. The Company anticipates initial results from the primary endpoint of RFS at 12 months in the second quarter of 2021.
Phase 1/2 AML Trial – The Company is currently enrolling a Phase 1/2 clinical trial evaluating the safety, tolerability, and preliminary efficacy of eprenetapopt therapy in TP53 mutant AML patients. The lead-in portion of the trial evaluated the tolerability of eprenetapopt with venetoclax, with or without azacitidine, and no dose-limiting toxicities were observed in 12 patients receiving either regimen. Based on these results, the Company has expanded the trial to treat 33 additional frontline TP53 mutant AML patients with the combination of eprenetapopt, venetoclax and azacitidine. In the 19 frontline AML patients who are evaluable for efficacy with the triplet regimen, the Company has observed a 63% CR + CRi composite response rate and a 31% CR rate. The Company anticipates completion of enrollment in the triplet regimen expansion cohort during the second quarter of 2021 and availability of preliminary response rate data from the cohort also in the second quarter of 2021.
Phase 1 NHL Trial – The Company is currently enrolling a Phase 1 clinical trial in relapsed/refractory TP53 mutant chronic lymphoid leukemia (CLL) assessing eprenetapopt with venetoclax and rituximab and eprenetapopt with ibrutinib in order to further assess eprenetapopt in hematological malignancies. The first patient was enrolled in the first quarter of 2021. The Company is also planning to evaluate the combination of eprenetapopt with venetoclax in relapsed/refractory mantle cell lymphoma.
Phase 1/2 Solid Tumor Trial – The Company is currently enrolling a Phase 1/2 clinical trial in relapsed/refractory gastric, bladder and non-small cell lung cancers assessing eprenetapopt with anti-PD-1 therapy. The dose-escalation phase of the trial enrolled 6 patients with advanced solid tumors and no dose-limiting toxicities were observed. Based on these results, the Company is enrolling expansion cohorts for patients with advanced gastric, bladder and non-small cell lung cancers and has currently enrolled 15 patients across these expansion arms. A poster presentation for this trial has been accepted for presentation at the 2021 ASCO (Free ASCO Whitepaper) Annual Meeting (abstract TPS3161).
APR-548 Phase 1 Trial — The Company’s second product candidate, APR-548, is a next-generation p53 reactivator that is being developed in an oral dosage form. The Company has planned a Phase 1 dose-escalation clinical trial evaluating the safety, tolerability, and preliminary efficacy of APR-548 with azacitidine in frontline and relapsed/refractory MDS patients. The Company anticipates the first patient to be enrolled in the second quarter of 2021.
First Quarter Financial Results

Cash and cash equivalents: As of March 31, 2021, the Company had $77.6 million of cash and cash equivalents compared to $89.0 million of cash and cash equivalents as of December 31, 2020. The Company expects cash burn for the full year 2021 to be between $30.0 million $35.0 million. The Company believes its cash and cash equivalents as of March 31, 2021 will be sufficient to meet its current projected operating requirements into 2023.
Research and Development (R&D) expenses: R&D expenses were $6.8 million for the quarter ended March 31, 2021, compared to $9.1 million for the comparable period in 2020. The decrease in R&D expenses was primarily due to decreases in clinical trial costs for (i) our pivotal Phase 3 clinical trial of eprenetapopt with azacitidine for the frontline treatment of TP53 mutant MDS which completed enrollment in Q2 2020 and (ii) our Phase 2 post-transplant MDS/AML clinical trial. These decreases were partially offset by increases in clinical trial costs for our Phase 1/2 clinical trial for the treatment of TP53 mutant AML with venetoclax and azacitidine, our Phase 1/2 clinical trial in relapsed/refractory gastric, bladder and non-small cell lung cancers assessing eprenetapopt with anti-PD-1 therapy, and our Phase 1 clinical trial in relapsed/refractory TP53 mutant chronic lymphoid leukemia (CLL) assessing eprenetapopt with venetoclax and rituximab, and eprenetapopt with ibrutinib.
General and Administrative (G&A) expenses: G&A expenses were $3.4 million for the quarter ended March 31, 2021, compared to $2.8 million for the comparable period in 2020. The increase in G&A expenses was primarily due to increases in non-cash stock-based compensation and insurance expense.
Net loss: Net loss was $9.7 million, or $0.46 per share for the quarter ended March 31, 2021, compared to a net loss of $9.4 million, or $0.45 per share for the quarter ended March 31, 2020. The Company had 21,186,827 shares of common stock outstanding as of March 31, 2021.

On May 6, 2021 Aldeyra Therapeutics, Inc. (Nasdaq: ALDX) (Aldeyra), a clinical-stage biotechnology company focused on the development of novel therapies with the potential to improve the lives of patients with immune-mediated diseases, reported financial results for the quarter ended March 31, 2021 and provided recent business highlights (Press release, Aldeyra Therapeutics, MAY 6, 2021, View Source [SID1234579428]).

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"We expect 2021 to be a catalyst-rich year for Aldeyra as we continue to advance reproxalap, our lead program, toward potential commercialization in anterior ocular inflammatory disease," stated President and CEO Todd C. Brady, M.D., Ph.D. "We recently completed the Phase 3 INVIGORATE Trial of reproxalap, achieving statistically significant superiority over vehicle across all assessed signs and symptoms of allergic conjunctivitis, including ocular itching and redness. We look forward to meeting with the U.S. Food and Drug Administration in the second half of this year to discuss the INVIGORATE results and the potential submission of a New Drug Application (NDA). In addition, we remain on track to report top-line results in the second half of this year from the Phase 3 TRANQUILITY and TRANQUILITY-2 clinical trials of reproxalap in dry eye disease.

"We believe we continue to operate from a position of financial strength," Dr. Brady stated. "With the recent follow-on public offering, we expect to have sufficient capital to prepare reproxalap for NDA submission and a potential commercial launch, if approved, while investing in the clinical development of ADX-629, ADX-2191, and other product candidates in retinal and systemic immunological diseases with unmet medical need."

Recent Highlights and Program Updates

Primary, Key Secondary, and All Secondary Endpoints Met in Phase 3 INVIGORATE Allergic Conjunctivitis Clinical Trial: In the first-ever Phase 3 clinical trial of a novel investigational product in an allergen chamber, 0.25% reproxalap ophthalmic solution (reproxalap) demonstrated statistically significant improvement over vehicle for the primary endpoint of ocular itching (p<0.0001), the key secondary endpoint of ocular redness (p<0.0001), and the secondary endpoints of ocular tearing and total ocular severity score (each p<0.0001). The results of INVIGORATE, the second positive Phase 3 trial for reproxalap in allergic conjunctivitis, indicate potential clinical utility before and during exposure to moderate to high levels of pollen.
Phase 3 TRANQUILITY and TRANQUILITY-2 Dry Eye Disease Trial Results Expected in Second Half of 2021: Patient enrollment has begun in the dry eye chamber Phase 3 TRANQUILITY Trial of reproxalap. The primary endpoint of the trial is ocular redness, which was statistically lower (p=0.03) for reproxalap relative to vehicle in the TRANQUILITY run-in cohort results announced in January 2021. Tear RASP (reactive aldehyde species) levels will also be assessed. Approximately 150 dry eye disease patients are expected to be enrolled per arm. Reproxalap will be administered four times the day prior to entry into the dry eye chamber, just before entry into the chamber, and 45 minutes after chamber entry. Enrollment in TRANQUILITY is ongoing, and enrollment in the confirmatory TRANQUILITY-2 Trial is expected to begin in the second quarter of 2021. Aldeyra plans to report top-line results from both trials in the second half of this year.
Phase 2 Clinical Trial Results from ADX-629, an Orally Available RASP Inhibitor, Expected Second Half of 2021: Initial Phase 2 clinical results from ADX-629, a novel orally available RASP inhibitor currently undergoing testing in asthma, psoriasis, and COVID-19, are expected in the second half of 2021. ADX-629 represents a first-in-class systems-based therapeutic approach for an orally administered RASP inhibitor, the potential applicability of which could extend to a myriad of immune-mediated diseases that today are treated with single-target drugs that can lead to serious toxicity.
Public Offering Completed: Aldeyra sold 10,000,000 shares of its common stock at a public offering price of $12.50 per share in an underwritten public offering. The offering generated gross proceeds of $125.0 million and net proceeds of $117.3 million after deducting underwriting discounts, commissions, and estimated offering expenses.
First-Quarter 2021 Financial Summary

Cash and cash equivalents as of March 31, 2021 were $138.4 million. Based on Aldeyra’s current operating plan, the company believes that existing cash and cash equivalents, as of March 31, 2021, together with the net proceeds from the sale of common stock in the underwritten public offering in May 2021, will be sufficient to fund currently projected operating expenses through the end of 2023, including potential NDA submission for reproxalap; initial commercialization of reproxalap, if approved; and continued early and late-stage development of the company’s product candidates in ocular and systemic immune-mediated diseases.

For the quarter ended March 31, 2021, Aldeyra reported a net loss of $11.3 million, compared with a net loss of $9.9 million for the quarter ended March 31, 2020. Net loss per share was $0.25 for the quarter ended March 31, 2021, compared with $0.34 for the same period in 2020. Losses have resulted from the costs of Aldeyra’s clinical trials and research and development programs, as well as from general and administrative expenses.

Research and development (R&D) expenses were $7.7 million for the quarter ended March 31, 2021, compared with $6.6 million for the same period in 2020. The increase of $1.1 million is primarily related to clinical development and manufacturing costs, partially offset by lower personnel related costs and a decrease in preclinical costs.

General and administrative expenses were $3.1 million for the quarter ended March 31, 2021, compared with $3.0 million for the quarter ended March 31, 2020.

For the quarter ended March 31, 2021, total operating expenses were $10.8 million, compared with total operating expenses of $9.6 million for the same period in 2020.

Conference Call & Webcast Information

Aldeyra will host a conference call at 8:00 a.m. ET today to discuss its first-quarter 2021 financial results and recent highlights. The dial-in numbers are (866) 211-4098 for domestic callers and (647) 689-6613 for international callers. The Conference ID number is 6779202. Due to the expected high demand on our conference call provider, please plan to dial in to the call at least 15 minutes prior to the start time.

A live webcast of the conference call will also be available on the Investor Relations page of the company’s website at View Source After the live webcast, the event will remain archived on the Aldeyra Therapeutics website for 90 days.

On May 6, 2021 Jasper Therapeutics, Inc., a biotechnology company focused on hematopoietic cell transplant therapies, and Amplitude Healthcare Acquisition Corporation (Nasdaq: AMHCU), a special purpose acquisition company (SPAC) sponsored by affiliates of Avego Management, LLC and Metalmark Capital, reported they have entered into a definitive business combination agreement (Press release, Jasper Therapeutics, MAY 6, 2021, View Source [SID1234579421]). Upon closing of the transaction, anticipated to occur in the third quarter 2021, the combined company will be renamed Jasper Therapeutics, Inc., and its common stock is expected to be listed on Nasdaq under the ticker symbol "JSPR ."

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"We would like to thank our financial partners at Amplitude and our prestigious group of investors," said Bill Lis, executive chairman and CEO, Jasper Therapeutics. "This transaction provides significant capital to accelerate the development of our two innovative programs, Jasper’s first-in-class clinical stage anti-CD117 antibody transplant conditioning agent and in parallel our groundbreaking research stage Engineered Hematopoietic Stem Cell platform, both of which have the potential to transform the field and expand hematopoietic stem cell therapy cures to a far greater number of patients than is possible today."

"At Jasper we are focused on a mission to cure several life threatening diseases such as blood cancers, sickle cell disease, severe combined immunodeficiency, and severe autoimmune diseases that affect a large number of patients who have historically been underserved by industry research and development, including infants, women, minorities and the elderly."

In addition to the funds held in Amplitude’s trust account (approximately $100 million less any redemptions), the transaction also includes commitments for a $100 million private investment in public equity (PIPE) priced at $10.00 per share. Investors in the PIPE include lead investor Federated Hermes Kaufmann Funds and affiliates of the SPAC sponsor including Avego, Velan Capital and Metalmark, as well as Amgen, Christian Angermayer’s Apeiron Investment Group, Kingdon Capital Management, and Woodline Partners LP, in addition to existing Jasper investors Abingworth LLP, Qiming Venture Partners USA, Surveyor Capital (a Citadel company), Roche Venture Fund and Alexandria Venture Investments, LLC. Jasper Therapeutics is expected to have cash resources of approximately $180 million at the closing of the transaction (less any redemptions from the Amplitude trust account).

The boards of directors of both Jasper Therapeutics and Amplitude have unanimously approved the proposed transaction. Completion of the transaction, which is expected in the third quarter of 2021, is subject to approval of Amplitude’s stockholders and the satisfaction or waiver of certain other customary closing conditions.

"Jasper Therapeutics has a strong management team with deep scientific expertise in the field and a track record developing and commercializing novel drugs, along with a pipeline that could make it a formidable leader in hematopoietic stem cell transplantation for a broad range of indications," said Vishal Kapoor, President of Amplitude. "When Jasper Therapeutics emerges as a public company, it will be positioned as a well-funded leader in hematopoietic stem cell conditioning and engineering, an area that has seen far too little innovation."

Jasper Therapeutics expects to use the cash resources of the combined company following the merger and PIPE to support the clinical development of JSP191, a first-in-class humanized monoclonal antibody in clinical development as a conditioning agent that clears hematopoietic stem cells from bone marrow, creating an empty space for donor or gene-corrected transplanted stem cells to engraft. To date, JSP191 has been evaluated in more than 90 healthy volunteers and patients. It is currently enrolling in two clinical trials for acute myeloid leukemia (AML)/ myelodysplastic syndromes (MDS) and severe combined immunodeficiency (SCID) and is scheduled to begin enrollment in 3 additional studies in 2021 for severe autoimmune disease, sickle cell disease and Fanconi anemia patients undergoing hematopoietic cell transplantation.

Jasper Therapeutics also expects to use the cash resources of the combined company to continue to advance its preclinical Engineered Hematopoietic Stem Cell (eHSCs) platform, which is designed to overcome key limitations of allogeneic and autologous gene-edited stem cell grafts. By using mRNA or DNA editing, Jasper Therapeutics can give the donor or gene-edited stem cells a proliferative and survival advantage over the patient’s existing stem cells. Preclinical data have demonstrated that Jasper’s eHSCs grow faster and outcompete normal hematopoietic stem cells and that they can be engineered to become resistant to inhibition by JSP191, suggesting that they could be combinable as a conditioning and therapeutic pair.

Transaction Overview

Assuming a share price of $10.00 per share and no redemptions of Amplitude shares, Jasper Therapeutics is expected to have an initial market capitalization of approximately $490 million dollars. Upon the closing of the business combination, and assuming no redemptions of shares of Amplitude by its public stockholders, Jasper Therapeutics is expected to have cash resources of approximately $180 million at the closing of the transaction (less any redemptions). The proceeds will be funded through a combination of approximately $100 million cash in trust by Amplitude (less any redemptions from its trust account) and a $100 million concurrent PIPE of common stock issued at $10.00 per share, anchored by leading institutional investors. As part of the transaction, Jasper Therapeutics’ existing equity holders will roll 100% of their equity into the combined company.

The boards of directors of both Jasper Therapeutics and Amplitude have unanimously approved the proposed transaction, which is expected to be completed in the third quarter of 2021. The transaction is subject to, among other things, the approval of the stockholders of both Jasper Therapeutics and Amplitude, satisfaction or waiver of the conditions stated in the definitive business combination agreement.

Additional information about the transaction will be provided in a Current Report on Form 8-K to be filed by Amplitude with the SEC and will be available at the SEC’s website at www.sec.gov. In addition, Amplitude intends to file a registration statement on Form S-4 with the SEC, which will include a proxy statement/prospectus, and will file other documents regarding the proposed transaction with the SEC.

Advisors

Credit Suisse is acting as lead PIPE placement agent and capital markets advisor to Jasper Therapeutics, William Blair is acting as co-placement agent and financial advisor and Cantor Fitzgerald as co-placement agent. Paul Hastings LLP is serving as legal counsel to Jasper Therapeutics. BMO Capital Markets and Oppenheimer & Co. Inc. are acting as capital markets advisors to Amplitude. Wilmer Cutler Pickering Hale and Dorr LLP is serving as legal counsel to Amplitude.

Webcast

The management team of Jasper Therapeutics will host a webcast on Friday, May 7 at 10:00 am ET to provide a brief overview of Jasper and the proposed merger. The webcast can be accessed here: View Source

About Jasper Therapeutics

Jasper Therapeutics is a biotechnology company focused on the development of novel curative therapies based on the biology of the hematopoietic stem cell. The company’s lead compound, JSP191, is in clinical development as a conditioning antibody that clears hematopoietic stem cells from bone marrow in patients undergoing a hematopoietic cell transplantation. This first-in-class conditioning antibody is designed to enable safer and more effective curative hematopoietic cell transplants and gene therapies. Jasper Therapeutics is also advancing the development of a novel hematopoietic stem cell engineering platform.

On May 6, 2021 Aptorum Group Limited (Nasdaq: APM, Euronext Paris: APM) ("Aptorum Group" or "Aptorum"), a clinical-stage biopharmaceutical company, reported that it has entered into an agreement with Exeltis ("Exeltis") (a division of the global pharmaceutical group Insud Pharma) to develop, manufacture and commercialize a novel preclinical candidate from Aptorum in the following territories: the European Union and Latin America (with an option to expand the collaboration to the United States) (Press release, Aptorum, MAY 6, 2021, View Source [SID1234579420]). This novel candidate is intended to target woman’s health and gynecological conditions, such as endometriosis or related conditions. Under the Agreement, Aptorum Group will retain the development rights in other jurisdictions in the world, as well as the right to develop the novel candidate into a drug product. Commercialisation of the product is subject to relevant regulatory approvals in their respective jurisdictions.

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Dr. Clark Cheng, Chief Medical Officer and Executive Director of Aptorum Group, commented: "Endometriosis is a systemic gynecological condition that is often painful and chronic, estimated to affect approximately 10% of reproductive-age women1. Symptoms of endometriosis include, but are not limited to chronic pain and in extreme cases infertility, thereby placing significant physical, emotional and economical impact on the patients. Currently, the mainstay of treatment includes surgery and/or hormonal treatment with oral contraceptives, neither of which are ideal, especially for reproductive aged women suffering such disease2. We believe that the candidate’s non-hormonal approach has the potential to address such unmet needs and we are excited to partner with Exeltis to develop this product to provide patients with a viable alternative."

On May 6, 2021 Isofol Medical AB (publ) ("Isofol"), (Nasdaq First North Premier Growth Market: ISOFOL) reported its primary recruitment objective with the recruitment of 440 patients in the global phase III AGENT study (Press release, Isofol Medical, MAY 6, 2021, View Source [SID1234579419]). Today the company announces that it has completed the recruitment of Japanese patients in accordance with the regulatory requirements by the PMDA (the Japanese Medicines Agency) to reach market approval in Japan. As previously communicated, Isofol expects the top line results for the AGENT study to be available during H1 2022.

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Following today’s announcement of the completion of recruitment of Japanese patients, the full patient population, including the entire Japanese cohort, will be included in PMDA’s assessment for a potential market approval in Japan. For a market approval in Japan, the PMDA set a specific requirement for the number of participating Japanese patients of a total of 56 Japanese patients (of which 14 Japanese patients were already included in the primary recruitment of 440 patients) in the AGENT study. The rationale for the specific requirement from PMDA is e.g. that the metabolism of Japanese patients tends to differ from patients in other countries, which is why the effect and potential side effects must be investigated separately.

For the rest of the world, the original 440 patients will be analyzed for efficacy, but the full patient population will be analyzed for safety purposes.

"I am satisfied that we now have completed the recruitment of the Japanese patients, an important step on the way to receive market approval in Japan, the second largest oncology market worldwide. We are now looking forward to continue working with Solasia on the development and registration of arfolitixorin to bring a new treatment option to patients living with mCRC in Japan", said Ulf Jungnelius, M.D, CEO of Isofol.

Solasia Pharma ("Solasia") will fund and supervise clinical development activities in Japan and will be responsible for registrational filing, and following potential regulatory approvals, Solasia will, as the Market Authorization holder, be responsible for the commercialization of arfolitixorin in Japan. Isofol remain the global sponsor of the AGENT study.

"We are very pleased to have completed the recruitment of the target number of patients in Japan in the AGENT study and contributed to the global development of arfolitixorin. I would like to thank all the patients and investigators participating in the study, the CRO in charge of conducting the study, and Isofol, our partner and the sponsor of the AGENT study, for supporting us achieve this important goal. Patient recruitment was completed earlier than expected, and Solasia, together with Isofol, will further proceed development of arfolitixorin for market approval in Japan with the aim of becoming a new treatment option for mCRC patients", said Yoshihiro Arai, President & CEO of Solasia.

Arfolitixorin is evaluated in the AGENT study for the treatment of patients with first-line metastatic colorectal cancer (mCRC). The study is currently being conducted in the U.S., Canada, Europe, Australia and Japan in more than 90 clinics.

The information was submitted for publication, through the agency of the contact person set out above, at 08:30 CET on May 6, 2021.

About the AGENT study

The Phase III AGENT study is a randomized, controlled, multi-centre study assessing the efficacy and safety of arfolitixorin, [6R]-5,10-methylene-THF acid (MTHF), compared to leucovorin, both used in combination with 5- FU, oxaliplatin, and bevacizumab, in first-line metastatic colorectal cancer patients. Patients are randomized in a 1:1 ratio and the primary endpoint is overall response rate (ORR). The key secondary endpoints are progression free survival (PFS) and duration of response (DOR). Other secondary endpoints include overall survival (OS), number of curative metastasis resections, safety, and patient reported outcomes such as quality of life (QoL). Exploratory endpoints include pharmacokinetic (PK) measurements and level of gene expression of folate relevant genes in tumour cells. The study is designed to show superiority for arfolitixorin over leucovorin. The study is ongoing at approximately 90 sites in the U.S., Canada, Europe, Australia and Japan. In December 2020, the last of the AGENT study’s 440 patients was recruited, which is the basis in the statistical analysis plan. Recruitment has since continued in Japan to reach 56 Japanese patients. Isofol is now focusing on completing the ongoing AGENT study where the patients receive first-line standard treatment for metastatic colorectal cancer (mCRC). The company expects that the results of the AGENT study will be available during H1 2022.Further information about the study, including patient eligibility requirements, is available at www.clinicaltrials.gov id: NCT03750786.

About arfolitixorin

Arfolitixorin is Isofol’s proprietary drug candidate being developed to increase the efficacy of standard of care chemotherapy for advanced colorectal cancer. The drug candidate is currently being studied in a global Phase III study, AGENT. As the key active metabolite of the widely used folate-based drugs, arfolitixorin can potentially benefit more patients with advanced colorectal cancer, as it does not require complicated metabolic activation to become effective.