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Mar 29
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Eloxx Pharmaceuticals Announces Submission of Investigational New Drug (IND) Application for ZKN-013

By Aura Biosciences, Press Release, Private Companies, Publicly Listed
Press Release.

 

  • ZKN-013 in development for treatment of recessive dystrophic epidermolysis bullosa (RDEB) and junctional epidermolysis bullosa (JEB)
  • Additional IND filing for ZKN-013 for treatment of familial adenomatous polyposis (FAP) expected in first half of 2023
  • Recent preclinical results have provided additional support for ZKN-013 in RDEB and FAP

WATERTOWN, Mass., March 28, 2023 (GLOBE NEWSWIRE) — Eloxx Pharmaceuticals, Inc. (NASDAQ: ELOX), a leader in ribosomal RNA-targeted genetic therapies for rare diseases, today announced the submission of an Investigational New Drug (IND) application with the U.S. Food and Drug Administration for ZKN-013 for the treatment of recessive Dystrophic Epidermolysis Bullosa (RDEB) with nonsense mutations. RDEB is a rare skin disease characterized by mutations in Collagen7 gene.

“This IND application is an important milestone towards providing a treatment option for patients with RDEB and JEB, as there are currently no approved disease-modifying treatments,” said Sumit Aggarwal, President and Chief Executive Officer of Eloxx. “We look forward to advancing ZKN-013, our lead TURBO-ZMTM based molecule, into clinical development, as we believe it has the potential to have a positive impact on the treatment of these two painful and debilitating diseases.”

Recent preclinical results demonstrated read-through activity of ZKN-013 in multiple COL7 genotypes across multiple RDEB patient derived fibroblasts and keratinocytes. Read-through activity resulted in up to an 18-fold increase in full-length COL VII protein levels. Prolonged treatment with ZKN-013 further increased COL VII protein levels. Functionality of the restored full-length COL VII protein was confirmed. These results have been accepted for presentation at an upcoming medical conference.

An additional IND filing for ZKN-013 for the treatment of familial adenomatous polyposis (FAP) is planned in the first half of 2023. FAP, a rare inherited disease with no approved drug therapies, is characterized by proliferation of colon polyps. Eloxx is targeting a subset of patients that have nonsense mutations in the Adenomatous Polyposis Coli (APC) gene that is truncated in these patients.

In January 2023, Eloxx published positive results from a study in the APCMin (multiple intestinal neoplasia) model evaluating the potential of ZKN-013 to treat FAP. The APCMin mouse is a translationally validated model for drug development for FAP. In the APCMin model, treatment with ZKN-013 demonstrated a decrease in intestinal polyps and adenomas, resulting in increased survival. The publication also included in vitro and in vivo demonstrating that ZKN-013 inhibited the growth of human colon carcinoma cells with APC nonsense mutations, and promoted read through of premature stop codons in the APC gene, leading to functional restoration of full-length APC protein.

About Eloxx Pharmaceuticals

Eloxx Pharmaceuticals, Inc. is engaged in the science of ribosome modulation, leveraging its innovative TURBO-ZMTM chemistry technology platform in an effort to develop novel Ribosome Modulating Agents (RMAs) and its library of Eukaryotic Ribosome Selective Glycosides (ERSGs). Eloxx’s lead investigational product candidate, ELX-02, is a small molecule drug candidate designed to restore production of full-length functional proteins. The U.S. Food and Drug Administration (FDA) has granted Fast Track designation for ELX-02 for the treatment of CF patients with nonsense mutations. In addition, ELX-02 has also been granted Orphan Drug Designation for the treatment of CF patients with nonsense mutations by the FDA and orphan medicinal product designation by the European Commission. ELX-02 is in clinical development, focusing on cystic fibrosis (US Trial NCT04135495, EU/IL Trial NCT04126473). Eloxx also has preclinical programs focused on select rare diseases, including inherited diseases, cancer caused by nonsense mutations, kidney diseases, including autosomal dominant polycystic kidney disease, as well as rare ocular genetic disorders.

For more information, please visit www.eloxxpharma.com.

Forward-looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements other than statements of present and historical facts contained in this press release, including without limitation, the expected timing of trials of our product candidates and the potential of our product candidate to treat nonsense mutations are forward-looking statements. Forward-looking statements can be identified by the words “aim,” “may,” “will,” “would,” “should,” “expect,” “explore,” “plan,” “anticipate,” “could,” “intend,” “target,” “project,” “contemplate,”

“believe,” “estimate,” “predict,” “potential,” “seeks,” or “continue” or the negative of these terms similar expressions, although not all forward-looking statements contain these words. Forward-looking statements are based on management’s current plans, estimates, assumptions and projections based on information currently available to us. Forward-looking statements are subject to known and unknown risks, uncertainties and assumptions, and actual results or outcomes may differ materially from those expressed or implied in the forward-looking statements due to various important factors, including, but not limited to: our ability to progress any product candidates in preclinical or clinical trials; the uncertainty of clinical trial results and the fact that positive results from preclinical studies are not always indicative of positive clinical results; the scope, rate and progress of our preclinical studies and clinical trials and other research and development activities; the competition for patient enrollment from drug candidates in development; the impact of the global COVID-19 pandemic on our clinical trials, operations, vendors, suppliers, and employees; our ability to obtain the capital necessary to fund our operations; the cost of filing, prosecuting, defending and enforcing any patent claims and other intellectual property rights; our ability to obtain financial in the future through product licensing, public or private equity or debt financing or otherwise; general business conditions, regulatory environment, competition and market for our products; and business ability and judgment of personnel, and the availability of qualified personnel and other important factors discussed under the caption “Risk Factors” in our Quarterly Report on Form 10-Q for the quarter ended September 30, 2022, as any such factors may be updated from time to time in our other filings with the SEC, accessible on the SEC’s website atwww.sec.gov and the “Financials&Filings” page of our website at https://investors.eloxxpharma.com/financials-filings.

All forward-looking statements speak only as of the date of this press release and, except as required by applicable law, we have no obligation to update or revise any forward-looking statements contained herein, whether as a result of any new information, future events, changed circumstances or otherwise.

Contact

Investors
John Woolford john.woolford@westwicke.com 443.213.0506

Media
Laureen Cassidy laureen@outcomescg.com

SOURCE: Eloxx Pharmaceuticals, Inc.

Feb 16
Love0

Aura Biosciences Announces Positive Interim Phase 2 Safety and Efficacy Data of Belzupacap Sarotalocan (Bel-sar) for the First-Line Treatment of Patients with Early-Stage Choroidal Melanoma with Suprachoroidal Administration

By Aura Biosciences, Press Release, Private Companies, Publicly Listed
Press Release.

 

Aura Biosciences Announces Positive Interim Phase 2 Safety and Efficacy Data of Belzupacap Sarotalocan (Bel-sar) for the First-Line Treatment of Patients with Early-Stage Choroidal Melanoma with Suprachoroidal Administration

Boston, USA, February 16 2023 – Aura Biosciences, Inc. (“Aura”) (Nasdaq: AURA), a clinical-stage biotechnology company developing a novel class of virus-like drug conjugate (VDC) therapies for multiple oncology indications, today announced the presentation of positive interim Phase 2 safety and efficacy data of bel-sar with 9-10 months of follow up evaluating two key clinical endpoints: tumor control and visual acuity preservation using the suprachoroidal (SC) route of administration for the first-line treatment of patients with early-stage choroidal melanoma (indeterminate lesions and small choroidal melanoma (IL/CM)). The results were presented at the Macula Society 46th Annual Meeting held February 15-18, 2023, in Miami, FL.

“The data presented today with an average of nine months of follow up for patients treated with three cycles of therapy, show an excellent response to the therapy with 89-100% tumor control. In addition, the safety profile to date has been favorable with only one patient losing visual acuity and no treatment-related SAEs or significant AEs, which is encouraging given that the majority of these patients had tumors close to the fovea or optic disk and would have likely experienced severe and irreversible vision loss with the current standard of care with radiotherapy,” said Dr. Ivana Kim, Director of the Ocular Melanoma Center, Massachusetts Eye and Ear. “These latest results strongly support the potential of bel-sar to be used as a first line treatment option for patients with early-stage choroidal melanoma.”

“We are excited with the interim efficacy data of the Phase 2 study which strongly supports the assumptions for the success of the global Phase 3 trial,” said Dr. Cadmus Rich, Chief Medical Officer of Aura Biosciences. “Collectively, we believe these interim data provide strong confidence to support the launch of a global Phase 3 trial which is on track to begin enrollment this year.”

The presentation can be accessed on the Company’s website: link

Updated Safety and Efficacy Data from the Ongoing Phase 2 Trial with SC Administration

This Phase 2 trial (NCT04417530) is assessing the safety and preliminary efficacy of single- and multiple ascending-doses of bel-sar up to three cycles of treatment via SC administration for the first-line treatment of early-stage choroidal melanoma. A total of 20 adult patients have been enrolled in the trial including the single dose Cohorts 1-3 (n=6) and multiple dose escalation Cohorts 4-6 (n=14). Cohorts 5 and 6 received up to three cycles of therapy, which was considered the therapeutic regimen for evaluation. One patient in Cohort 5 (n=3) received two cycles of therapy and two patients in Cohort 5 received three cycles of therapy (40 μg/dose). All patients from Cohort 6 (n=8) were assigned to receive three cycles of therapy at the highest dose (80 μg/dose). One patient from Cohort 6, who discontinued after one cycle due to unrelated serious adverse events (SAEs), is not included. All patients in Cohorts 5 and 6 had active growth at study entry, as an enrichment strategy to evaluate preliminary efficacy. This group of patients with active growth treated at the therapeutic regimen of three cycles was evaluated for tumor growth rate, tumor control, and visual acuity preservation as the defined clinical endpoints to evaluate preliminary efficacy. The results, with an average of nine months of follow up in patients who received three cycles of therapy in Cohorts 5 and 6, and who match the criteria for the planned global Phase 3 trial, showed a statistically significant reduction in the tumor growth rate (-0.289 mm/yr, p = <0.0001) compared to each patient’s documented growth rate at study entry, and a 100% (8/8) tumor control rate. In addition, the visual acuity preservation rate was 88% (7/8) in these cohorts, with the majority of patients being at high-risk for vision loss with tumors close to fovea or optic disk. The overall tolerability profile of bel-sar was generally favorable, with no dose-limiting toxicities, treatment-related SAEs or significant AEs reported as of January 10, 2023. There was no posterior inflammation and only mild anterior inflammation (Grade 1) in 20% of the patients. Treatment-related AEs were predominantly mild and resolved without sequalae. We believe these interim results indicate that bel-sar may offer a targeted, vision preserving therapy for the first-line treatment of primary CM, where 80% of patients are diagnosed early and have no approved therapies to date.

About Aura Biosciences

Aura Biosciences, Inc. is a clinical-stage biotechnology company developing virus-like drug conjugates (VDCs), a novel class of therapies, for the treatment of multiple oncology indications. Aura’s lead VDC candidate, belzupacap sarotalocan (bel-sar; AU-011), consists of a virus-like particle conjugated with an anti-cancer agent. Bel-sar is designed to selectively target and destroy cancer cells and activate the immune system with the potential to create long-lasting anti-tumor immunity. Bel-sar is currently in development for ocular cancers, and Aura has initiated activities for the global Phase 3 trial evaluating first-line treatment of early-stage choroidal melanoma, a vision- and life-threatening form of eye cancer where standard of care with radiotherapy leaves patients with severe comorbidities, including major vision loss. Aura plans to pursue development of bel-sar across its ocular oncology franchise including for the treatment of patients with choroidal metastasis. In addition, leveraging Aura’s technology platform, Aura is developing bel-sar more broadly across multiple cancers, including in patients with non-muscle invasive bladder cancer (NMIBC). Aura is headquartered in Boston, MA.

Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, and other federal securities laws. Any statements that are not statements of historical fact may be deemed to be forward looking statements. Words such as “may,” “will,” “could”, “should,” “expects,” “intends,” “plans,” “anticipates,” “believes,” “estimates,” “predicts,” “projects,” “seeks,” “endeavor,” “potential,” “continue” or the negative of such words or other similar expressions that can be used to identify forward-looking statements. These forward looking statements include express or implied statements regarding Aura’s future expectations, plans and prospects, including, without limitation, statements regarding the therapeutic potential of bel-sar for the treatment of cancers including choroidal melanoma, non-muscle invasive bladder cancer and choroidal metastases; any express or implied statements regarding the Company’s expectations for the Phase 2 and Phase 3 clinical trials of bel-sar for early-stage choroidal melanoma; and Aura’s expectations regarding the estimated patient populations and related market opportunities for bel-sar.

The forward-looking statements in this press release are neither promises nor guarantees, and investors should not place undue reliance on these forward-looking statements because they involve known and unknown risks, uncertainties, and other factors, many of which are beyond Aura’s control and which could cause actual results to differ materially from those expressed or implied by these forward-looking statements, including, without limitation, an improved quality of life of patients after treatment with bel-sar; a potential paradigm shift in the approach to the treatment of choroidal melanoma; the urgent need for a vision preserving targeted therapy; the potential of bel-sar compared to the existing standard of care for patients with choroidal melanoma; uncertainties inherent in clinical trials and in the availability and timing of data from ongoing clinical trials; the expected timing for submissions for regulatory approval or review by governmental authorities; the risk that the results of Aura’s clinical trials may not be predictive of future results in connection with future clinical trials; the risk that interim data from ongoing clinical trials may not be predictive of final data from completed clinical trials; whether Aura will receive regulatory approvals to conduct trials or to market products; whether Aura’s cash resources will be sufficient to fund its foreseeable and unforeseeable operating expenses and capital expenditure requirements; risks, assumptions and uncertainties regarding the impact of the continuing COVID-19 pandemic on Aura’s business, operations, strategy, goals and anticipated timelines; Aura’s ongoing and planned pre-clinical activities; and Aura’s ability to initiate, enroll, conduct or complete ongoing and planned clinical trials. These risks, uncertainties, and other factors include those risks and uncertainties described under the heading “Risk Factors” in Aura’s most recent Annual Report on Form 10-K and Quarterly Report on Form 10-Q filed with the U.S. Securities and Exchange Commission (SEC) and in subsequent filings made by Aura with the SEC, which are available on the SEC’s website at www.sec.gov. Except as required by law, Aura disclaims any intention or responsibility for updating or revising any forward-looking statements contained in this press release in the event of new information, future developments or otherwise. These forward-looking statements are based on Aura’s current expectations and speak only as of the date hereof and no representations or warranties (express or implied) are made about the accuracy of any such forward-looking statements.

 

Contacts:

Alex Dasalla
Head of Investor Relations and Corporate Communications
adasalla@aurabiosciences.com

Argot Partners
Matthew DeYoung
aura@argotpartners.com

Source: Aura Biosciences, Inc.

Jan 13
Love0

Pheno Therapeutics Announces Worldwide License From UCB to a Novel Program for Neurodegenerative Disease

By Pheno Therapeutics, Press Release, Private Companies
Press Release.

 

Preclinical program with potential for disease modifying therapeutic

Edinburgh, UK January 11 2023 – Pheno Therapeutics Limited., a biotechnology company focused on the discovery and development of small molecule therapeutics that promote remyelination for the treatment of neurological diseases such as multiple sclerosis, announced today it has entered into an exclusive worldwide license agreement with UCB. The agreement grants Pheno Therapeutics access and rights to develop, manufacture and commercialize a preclinical-stage program of novel small molecules designed to promote remyelination.

“Pheno Therapeutics utilized its proprietary human phenotypic screening platform to discover novel and tractable therapeutic targets to modulate oligodendrocyte biology and promote remyelination. This license allows us to accelerate a promising drug target towards clinical development,” said Fraser Murray, PhD., Chief Executive Officer of Pheno Therapeutics. “Together with our human neurological drug development expertise, there is significant opportunity for Pheno Therapeutics to fast-track this program towards clinical proof-of-concept studies and potentially deliver transformational drugs for the treatment of  demyelination diseases.”

“License agreements like this are a demonstration of the value UCB scientists and our partners are creating through strong research productivity, and we are confident that Pheno Therapeutics, with its expertise in phenotypic screening, stem cell technology and myelin biology, will develop this preclinical program  to its full potential,” said Dhaval Patel, Executive Vice President and UCB’s Chief Scientific Officer.

Pheno Therapeutics will make a one-time upfront payment and will be responsible for development, manufacturing and global commercialization. UCB will receive milestones and tiered royalties on net sales. Further financial details of the agreement were not disclosed.

About the Program:

The preclinical program is a novel small molecule program designed to selectively modulate the activity of an undisclosed target that is preferentially expressed on human oligodendrocytes and is known to play a critical role in the biological pathway and process of myelination.  The therapeutic program is being developed by Pheno Therapeutics under an exclusive worldwide license from UCB.

About Pheno Therapeutics:

Pheno Therapeutics is a discovery and development focused biotechnology company developing small molecule therapeutics that boost the function of human oligodendrocytes and their natural ability to promote remyelination for the treatment of neurological diseases with high unmet medical need such as multiple sclerosis (MS).  MS is a devastating chronic disease with significant individual and societal impact that often manifests in young adults and is associated with a wide range of neurological symptoms which can progress to total physical and cognitive disability.  Pheno Therapeutics is seeking to promote remyelination and reverse the critical demyelination aspect of MS. MS demyelination occurs when the immune system attacks and damages the myelin sheaths that insulate and nourish axons and nerve fibres in the central nervous system, leading to multifocal demyelination, axonal injury and neurodegeneration. For more information on Pheno Therapeutics, please visit https://www.phenotherapeutics.com.

 

Contacts:

Fraser Murray, PhD.
Chief Executive Officer
Pheno Therapeutics
Fraser.Murray@PhenoTherapeutics.com

Katie Odgaard
Account Director
Zyme Communications
Katie.odgaard@zymecommunications.com

+44 (0)7787 502 947

Dec 19
Love0

F2G Announces FDA Filing Acceptance of New Drug Application for Olorofim for the Treatment of Invasive Fungal Infections

By F2G, Press Release, Private Companies
Press Release.

 

Application submitted under priority review with PDUFA target action date set for June 17, 2023

NDA submission is based on positive data from ongoing Phase 2b open-label study of oral olorofim in 100 patients with invasive fungal infections with limited or no treatment options

F2G is continuing preparations for a possible U.S. commercial launch of oral olorofim in 2nd half of 2023

PRINCETON, New Jersey, December 19, 2022 – F2G Inc. today announced that the U.S. Food and Drug Administration (FDA) has accepted for filing its New Drug Application (NDA) for olorofim for the treatment of invasive fungal infections in patients who have limited or no treatment options.

F2G has requested approval of the NDA under the Limited Population Pathway for Antibacterial and Antifungal Drugs (LPAD pathway) for a limited, well-defined population with invasive fungal infections and limited or no treatment options. Under the Prescription Drug User Fee Act (PDUFA), the FDA has set a target action date of June 17, 2023

The NDA is supported by strong efficacy data and a good tolerability profile seen during treatment of the first 100 patients in the Phase 2b open-label study, all of whom had limited or no treatment options for either proven invasive fungal infection (including aspergilllosis, lomentosporiosis, scedosporiosis, Scopulariopsis infections, and refractory extrapulmonary coccidioidomycosis) or probable pulmonary invasive aspergillosis (Study 32, NCT03583164).

“Invasive fungal infections cause substantial morbidity and mortality, particularly among immunosuppressed patients, and can prove to be lethal in also healthy individuals when they get into deeper tissues. Effective therapies do not currently exist for some of these fungi. And even when therapies exist, some patients with invasive infections may be refractory or unable to tolerate existing antifungal treatments, thus underscoring the urgent need for new and effective treatments,” said John H. Rex, MD, chief medical officer of F2G. “Olorofim is a novel mechanism antifungal therapy from the newly discovered orotomide class. It provides a new option for patients who have exhausted treatment alternatives.”

Francesco Maria Lavino, chief executive officer of F2G, added,

“We are committed to addressing rare fungal infections, and the acceptance of filing of olorofim NDA for use in this well-defined and high-need population marks a major milestone toward our goal of bringing new options to these patients. We are building an experienced commercial team in preparation for U.S. launch, pending FDA approval. If approved, olorofim will be the first of a new class of antifungal drugs.”

Olorofim is the only antifungal medication to be awarded Breakthrough Therapy Designation by the FDA. Olorofim works through a novel mechanism of action, different from existing classes of antifungals, exerting fungal cell death through inhibition of the enzyme dihydroorotate dehydrogenase (DHODH) in the pyrimidine synthesis pathway. It is active in vitro against Aspergillus spp. (including azole-resistant and cryptic species), rare molds (e.g., Lomentospora prolificans, Scedosporium spp., Scopulariopsis spp.), and dimorphic fungi (e.g., Coccidioides spp.).

About Olorofim

Olorofim (formerly, F901318) is F2G’s leading candidate from the orotomide class and is currently being investigated in a Phase 2b open-label study in patients who have limited treatment options for difficult-to-treat invasive, rare fungal mold infections such as azole-resistant aspergillosis, scedosporiosis, lomentosporiosis, and other rare mold infections. F2G has initiated a global Phase 3 trial (“OASIS”) to compare treatment with olorofim versus AmBisome® followed by standard of care (SOC) in patients with proven or probable invasive fungal infection due to Aspergillus species (NCT05101187). Olorofim has received orphan drug designation from the FDA for the treatment of coccidioidomycosis, scedosporiosis (including lomentosporiosis), invasive Scopulariopsis, and invasive aspergillosis. Olorofim has also received orphan drug designation from the European Medicines Agency (EMA) for the treatment of invasive aspergillosis, invasive scedosporiosis (including lomentosporiosis), and invasive Scopulariopsis. Olorofim has been granted Qualified Infectious Disease Product (QIDP) designation for invasive aspergillosis, invasive scedosporiosis, invasive lomentosporiosis, coccidioidomycosis, invasive disease due to Scopulariopsis species, and invasive fusariosis. Olorofim has received Breakthrough Therapy Designation for both “treatment of invasive mold infections in patients with limited or no treatment options, including aspergillosis refractory or intolerant to currently available therapy, and infections due to Lomentospora prolificans, Scedosporium, and Scopulariopsis species” and “treatment of Central Nervous System (CNS) coccidioidomycosis refractory or otherwise unable to be treated with standard of care therapy.” Olorofim is not approved by the FDA or any other regulatory agency.

About F2G

F2G is a biotech company with operations in the UK, US, and Austria focused on the discovery and development of novel therapies to treat potentially life-threatening invasive fungal infections. F2G has discovered and developed a completely new class of antifungal agents called the orotomides which selectively target a key enzyme in the de novo pyrimidine biosynthesis pathway. This is a completely different mechanism from that of the currently marketed antifungal agents and gives the orotomides fungicidal activity against a broad range of rare and resistant fungal mold infections. For more information, please visit: www.f2g.com

Forward-Looking Statements

This announcement contains forward-looking statements. These statements are based on expectations in light of the information currently available, assumptions that are subject to risks and uncertainties which could cause actual results to differ materially from these statements. Risks and uncertainties include general domestic and international economic conditions such as general industry and market conditions, and changes of interest rate and currency exchange rate. These risks and uncertainties particularly apply with respect to product-related forward-looking statements. Product risks and uncertainties include, but are not limited to, completion and discontinuation of clinical trials; obtaining regulatory approvals; claims and concerns about product safety and efficacy; technological advances; adverse outcome of important litigation; domestic and foreign healthcare reforms and changes of laws and regulations. Also, for existing products, there are manufacturing and marketing risks, which include, but are not limited to, inability to build production capacity to meet demand, lack of availability of raw materials and entry of competitive products. The company disclaims any intention or obligation to update or revise any forward-looking statements whether as a result of new information, future events or otherwise.

Media Contact:

Gloria Gasaatura
LifeSci Communications
+1 646 970 4688
ggasaatura@lifescicomms.com

Nov 15
Love0

Artax Biopharma Expands Company’s Scientific Advisory Board

By Artax Biopharma, Press Release, Private Companies
Press Release.

 

Company Welcomes James G. Krueger, M.D., Ph.D., and Ajay Nirula, M.D., Ph.D., Both Experts in T-Cell-Mediated Diseases.

Cambridge, MA, November 15, 2022 – Artax Biopharma, Inc., a clinical stage biotechnology company focused on transforming the treatment of T-cell-mediated diseases, today announces the Company is strengthening its Scientific Advisory Board (SAB) with the appointments of James G. Krueger, M.D., Ph.D., and Ajay Nirula, M.D., Ph.D.

Artax Biopharma has an ongoing Phase 1 clinical trial evaluating AX-158, the Company’s first-in-class, oral small molecule immunomodulating agent that selectively acts at the T-cell receptor to lower self-antigen triggered cytokines, a known cause of autoimmune disease. Importantly, AX-158 has the potential to treat T-cell-mediated diseases without immunosuppression.

“We are honored to welcome global experts Drs. Krueger and Nirula to our Scientific Advisory Board, as they each bring unique research and extensive clinical experience across T-cell-mediated diseases,”

said D. Scott Batty, Jr., M.D., Artax Biopharma’s Chief Medical Officer.

“Drs. Krueger and Nirula bring a profound understanding of T-cell biology and pathophysiology and have success in bringing these effective treatments to patients who need them most. We are very grateful for their expert guidance and developmental insights as we progress AX-158 through clinical trials,”

Dr. Batty, Jr. added.

Dr. Krueger serves as Head of the Laboratory for Investigative Dermatology and as the D. Martin Carter Professor in Clinical Investigation and Dermatology at The Rockefeller University. He is co-director for the Center for Clinical and Translational Science at The Rockefeller University Hospital and has been serving as the Hospital’s Chief Executive Officer since July of 2008. Dr. Krueger uses psoriasis as a model to study inflammatory diseases that involve Th17 cells, a subset of T cells. His work has formed the scientific basis for the highly successful treatment of psoriasis with a range of biologic immune drugs that target the “Type 17” inflammatory immune axis.

Dr. Krueger earned his bachelor’s degree from Princeton University and his Ph.D. degree in Virology and Cell Biology from The Rockefeller University. He received his medical degree from Cornell University Medical College, where he also completed an internship in internal medicine and a residency in dermatology. Dr. Krueger is board certified in dermatology by the American Board of Dermatology and has been the recipient of numerous notable academic and industry honors throughout his career.

Dr. Nirula is Senior Vice President, Immunology, for Eli Lilly and Company based at the Lilly Biotechnology Center in San Diego. Dr. Nirula joined Eli Lilly in 2015 and is responsible for the Company’s discovery, research, and early phase clinical development in immunology. He has also served as the medical leader for Eli Lilly’s work during the COVID pandemic that led to emergency authorization for the Company’s multiple therapeutic neutralizing antibodies. Prior to joining Eli Lilly, Dr. Nirula held leadership positions at Amgen, and at Biogen Idec, and was involved in several research programs and regulatory filings spanning diseases such as rheumatoid arthritis, systemic lupus erythematosus, multiple sclerosis, psoriasis, and vasculitis.

Dr. Nirula earned his undergraduate degree in molecular biology from UC Berkeley, his medical degree from UCLA School of Medicine, and his Ph.D. degree from the University of Texas Southwestern Medical School. He subsequently joined the faculty in the Division of Rheumatology at UCSF Medical Center. Dr. Nirula has published extensively in premier peer-reviewed medical journals such as The New England Journal of Medicine, JAMA, and Nature Immunology.

 

Drs. Krueger and Nirula are welcomed by Artax’s current SAB members:

  • Balbino Alarcón, Ph.D., Artax Founder and Program Director at the National Research Council of Spain (CSIC) and Head of TCR-mediated Signal Transduction Laboratory;
  • Raif Geha, M.D., James L. Gamble Professor of Pediatrics at Harvard Medical School and Chief of the Allergy/Immunology/Rheumatology and Dermatology Division at Children’s Hospital in Boston;
  • Menno de Rie, M.D., Vice-Chair of the Department of Dermatology of the Amsterdam University Medical Centres /University of Amsterdam since 2012;
  • Lawrence Steinman, M.D., Professor of Neurology and Neurological Sciences, Pediatrics, and Genetics at Stanford University; and
  • Cox Terhorst, Ph.D., Professor of Immunology at Harvard Medical School and Chief, Immunology at the Beth Israel Deaconess Medical Center.

 

About Artax Science and Immunomodulation

Artax believes immunomodulation – selective targeting of specific functions and operations of the immune system – holds great potential to address tremendous unmet need in immunology. Our investigational immunomodulating agents act directly at the T-cell receptor (TCR) to selectively target inappropriately activated areas of the immune system, thus eliminating a cause of T-cell-driven diseases while not impacting a patient’s ability to effectively fight foreign pathogens and infections.

T-cells are the central, critical cells orchestrating the immune response, protecting the body against foreign pathogens, infections and malignancy while importantly not reacting to an individual’s tissues and organs. The TCR activates and controls the many functions and responses of the T-cell, serving a critical role for both healthy immune system and T-cell response.

Dysregulated TCR signaling results in increased cytokine signaling, a root cause of T-cell-mediated diseases, including autoimmune diseases. In addition to autoimmune diseases, T-cell-mediated diseases include T-cell malignancies, and induced T-cell pathologies where therapeutic treatments result in unwanted immune reaction side effects. Such induced T-cell immune reactions include stem cell transplants resulting in acute graft-versus-host-disease, and immuno-oncology treatments resulting in immune-related-adverse events. This central role of dysregulated TCR signaling across several disease states therefore makes the TCR a highly attractive target for therapeutic intervention.

 

About AX-158

AX-158 is a first-in-class, oral, small molecule immunomodulating agent in clinical development for the treatment of T-cell-mediated diseases. The immunomodulatory effect of AX-158 is designed to safely return the immune system to a state of rebalance without immunosuppression.

AX-158 is a highly specific (SH3.1 domain) inhibitor of Nck, a protein that naturally amplifies T-cell signaling caused by self-antigens at the TCR. AX-158 inhibits Nck at the TCR, resulting in less TCR activation and lowered self-antigen triggered cytokines, including Th-1, Th-17 and Th-2 type cytokines. High levels of such various cytokines are associated with a broad set of T-cell mediated autoimmune diseases, such as rheumatoid arthritis, psoriasis, atopic dermatitis, inflammatory bowel disease, and many others. Importantly, preclinical data suggests AX-158 is not immunosuppressive, and so does not impact the immune system’s ability to mount a strong response to foreign pathogens and infections.

 

About Artax Biopharma

Artax Biopharma is a clinical-stage biotechnology company transforming the treatment of T-cell-driven diseases through innovative small molecules that modulate the immune system. Artax’s disruptive science holds broad potential to treat T-cell-mediated diseases such as autoimmune diseases, induced T-cell pathologies (such as acute graft-versus-host disease, and immune-oncology treatment-related adverse events), and T-cell malignancies, while simultaneously allowing the body to fight foreign pathogens and infections. For more information, please visit www.artaxbiopharma.com and connect with us on LinkedIn.

 

Contacts:
Media:
Linda Phelan Dyson, MPH
Corporate Communications
lpdyson@verizon.net
M: 973-986-5973

Karen LaRochelle, MBA
Chief Business Officer
Artax Biopharma
klarochelle@artaxbiopharma.com

Nov 07
Love0

Aleta Biotherapeutics Receives Innovation Passport Designation for Biologic CAR T-Cell Therapy Engager ALETA-001

By Aleta Biotherapeutics, Press Release, Private Companies
Press Release.

 

Designation Intends to Accelerate Regulatory Review Process and Facilitate U.K. Patient Access to Medicines for Seriously Debilitating and Life-Threatening Diseases

ALETA-001 Was Developed to Address the Urgent Unmet Need of Patient Relapse After CD19-Targeted CAR T-Cell Cancer Treatment

NATICK, Mass., November 7, 2022 – Aleta Biotherapeutics (Aleta), a privately held immuno-oncology company with novel biologic CAR T engagers that work in synergy with cell therapies to improve outcomes for patients, announces that the U.K. Medicines and Healthcare products Regulatory Agency (MHRA) has granted an Innovation Passport under the Innovative Licensing and Access Pathway (ILAP) for Chimeric Antigen Receptor (CAR) T-cell therapy Engager candidate ALETA-001 for the treatment of patients suffering from the B-cell malignancies, non-Hodgkin lymphoma a (NHL) and Acute Lymphoblastic Leukemia (ALL), and who have failed to respond or have relapsed post-CD19 CAR T-cell therapy. ALETA-001 is expected to enter clinical development in 2023 with Cancer Research UK’s Centre for Drug Development sponsoring and conducting a Phase 1/2a clinical trial.

The Innovation Passport is the first step in the ILAP process, triggering the MHRA and its partner agencies, including The All Wales Therapeutics and Toxicology Centre (AWTTC), National Institute for Health and Care Excellence (NICE), and the Scottish Medicines Consortium (SMC), to chart a roadmap for regulatory and development milestones to enable faster patient access to medicines in the U.K. To receive an Innovation Passport, a medicine must address conditions that are life-threatening or seriously debilitating, and there must be an existing significant patient or public health need.

“This designation for our biologic CAR T-cell therapy engager ALETA-001 marks an important step in addressing the high unmet need for patients who relapse or progress following CD19-targeted CAR T-cell therapy for blood cancers, such as lymphoma and leukemia,”

stated Paul Rennert, Co-Founder, Acting Chief Executive Officer and Chief Scientific Officer, Aleta Biotherapeutics.

“In collaboration with our partner Cancer Research UK, we are excited to move ALETA-001 forward to potentially transform the lives of patients living with blood cancers,”

continued Rennert.

Dr. Nigel Blackburn, Cancer Research UK’s Director of Drug Development, stated,

“We are so pleased to receive this designation for ALETA-001, which reboots CAR T-cell therapy by bridging a patient’s circulating CD19-targeted CAR T-cells to cancer cells expressing CD20.

While CAR T-cell therapy has revolutionized hard-to-treat blood cancer outcomes, a majority of patients will see their cancer return, and this is where the critical potential of ALETA-001 exists. ALETA-001 is a promising approach to address this significant treatment gap for patients who currently lack effective options.”

In June 2021, Aleta Biotherapeutics and Cancer Research UK announced a collaboration in which Cancer Research UK’s Centre for Drug Development will fund, sponsor, and conduct the first-in-human Phase 1/2a clinical trial of ALETA-001, which will be led by Dr. Sridhar Chaganti’s Cellular and CAR T-cell therapies team at the Queen Elizabeth Hospital and University Hospitals Birmingham NHS Foundation Trust, Birmingham UK. In the Cancer Research UK-sponsored trial, patients with B-cell lymphoma/leukemia who have received CD19-targeted CAR-T cell therapy but did not achieve a complete response or who relapsed from a complete response will be enrolled. Aleta retains all rights to further develop and commercialize ALETA-001.

 

About Biologic CAR T-Cell Therapy Engager (CTE) ALETA-001

ALETA-001 is an off-the-shelf preclinical biologic program developed to treat and prevent cell therapy relapse of existing CD19-targeted CAR T-cell therapies, termed CAR19 T cells. ALETA-001 bridges CAR19 T cells to a second antigen, CD20. ALETA-001 binds to B-cell lymphomas and leukemias expressing CD20 antigens and restores tumor expression to CD19. ‘Recoating’ CD20-expressing cancer cells to express CD19 addresses the critical issues of tumor CD19 antigen loss and density and holds the potential to restore potent killing in patients who are no longer responding to previously administered, circulating CD19-targeted CAR T-cell therapy due to reduction or loss of tumor CD19 expression. In June 2021, Aleta and Cancer Research UK announced a collaboration in which Cancer Research UK will fund, sponsor, and conduct the Phase 1/2a clinical trial of ALETA-001.

 

About Aleta Biotherapeutics

Aleta Biotherapeutics is an immune-oncology company with a portfolio and platform of novel off-the-shelf biologic CAR T engagers (CTEs) that work in synergy with cell therapies to improve outcomes for patients. Aleta’s CTEs bridge CAR T-cell therapies to target multiple tumor antigens, binding to existing tumor antigens and changing the tumor antigen expression to match the CAR T receptor. Aleta’s CTEs address the critical issues of tumor antigen loss, density and heterogeneity, which optimizes the potential for potent killing by separately administered cell therapies, including existing CAR19 T-cell therapies.

ALETA-001 and ALETA-005 are designed to treat and prevent cell therapy relapse of circulating CAR19 and B-cell maturation antigen (BCMA) T-cell therapies by restoring tumor antigen expression to match CAR T receptors. ALETA-004 is Aleta’s first CTE program to bind CTEs to multiple tumor antigens and fundamentally change tumor cell antigen expression, thereby bridging the tumors to match a specific CAR T-cell therapy. In the case of ALETA-004, Aleta’s CTE changes the expression of Acute Myeloid Leukemia (AML), a non-B cell tumor, to express CD19, which allows the potential for CAR CD19 cell therapies to treat AML. http://www.aletabio.com

 

About Cancer Research UK’s Centre for Drug Development

Cancer Research UK has an impressive record of developing novel treatments for cancer. The Cancer Research UK Centre for Drug Development has been pioneering the development of new cancer treatments for 25 years, taking over 140 potential new anti-cancer agents into clinical trials in patients. It currently has a portfolio of 21 new anti-cancer agents in preclinical development, Phase I or early Phase II clinical trials. Six of these new agents have made it to market including temozolomide for brain cancer, abiraterone for prostate cancer and rucaparib for ovarian cancer. Two other drugs are in late development Phase III trials. www.cruk.org.uk/cdd

Contacts:

Media:
Linda Phelan Dyson, MPH
973-986-5973
lpdyson@verizon.net

Karen LaRochelle, MBA
Chief Business Officer
Aleta Biotherapeutics
klarochelle@aletabio.com

Oct 19
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PIC Therapeutics Completes $35 Million Series A Financing to Develop Treatments for Drug-Resistant Breast Cancer

By PIC Therapeutics, Press Release, Private Companies
Press Release.

 

Financing Round Led by OrbiMed with participation from Lumira Ventures, Harrington Discovery Institute and existing investors Advent Life Sciences and Belinda Termeer

Therapy development focused on addressing a fundamental mechanism in cancer-driving oncogenes

Natick, Mass. – October 19, 2022 – PIC Therapeutics, Inc., a biopharmaceutical company dedicated to developing life-changing medicines for patients with cancer, today announced the closing of a $35 million Series A financing led by OrbiMed. Other new investors participating in this financing include Lumira Ventures and Harrington Discovery Institute. The company’s existing investors including Advent Life Sciences and Belinda Termeer also participated and provided initial seed financing instrumental to meeting key milestones.

Proceeds from the financing will be used to advance the company’s development-stage small molecule drug, an allosteric protein translation modulator targeting eIF4E, into first-in-man, first-in-mechanism clinical studies in advanced metastatic breast cancer. The proceeds will also support expansion of the company’s pipeline of emerging oncology indications.

PIC Therapeutics is targeting a fundamental mechanism at the convergence of many oncogenic signaling pathways that results in apoptotic cancer cell death while sparing normal cells. Allosteric modulation of eIF4E offers many advantages to previous approaches, and simultaneously addresses multiple drivers of pharmacology, allowing the company’s small molecules to truly drive differential CAP dependent translation in target cells.

Preclinical studies show that PIC compounds modulate, but do not block, protein translation. PIC compounds mechanistically modulate cellular proteomes, leading to rapid and significant reduction in cancer cell viability via apoptosis. Inducing apoptosis rather than senescence is an important distinguishing feature of PIC’s approach to this elusive target.

“This financing from a committed and distinguished investor syndicate, which includes new and existing investors, underscores the progress we’ve made to advance our lead program toward our goal of cancer therapies that broadly address cancer-driving oncogenes via a fundamental mechanism in protein translational modulation”

said Katherine Bowdish, Chief Executive Officer of PIC Therapeutics.

“We are well positioned to build a leading mechanistic-based oncology company that brings promising science to cancer patients with drug resistant tumors.”

“We are excited to partner with PIC Therapeutics as they build a differentiated targeted oncology company, and we look forward to supporting the team as they work towards achieving key development goals over the coming years,”

said Tal Zaks, Partner of OrbiMed Advisors.

“Founded on the scientific work of Dr. Gerhard Wagner at Harvard University and Dr. Nahum Sonenberg at McGill University, PIC Therapeutics has developed a truly novel approach for eIF4E, an important target in resistant cancers. We are pleased to work with the PIC team and its investors to enable new frontiers in targeted mechanistic oncology with the potential to transform the treatment paradigm for cancer patients”

commented Gerry Brunk, Managing Director of Lumira Ventures.

 

About PIC Therapeutics

PIC Therapeutics is a biotechnology company pioneering the discovery and development of first-in mechanism, first-in-class small molecule medicines focused on fundamentally changing how we treat cancer by developing therapeutics that modulate protein translation. PIC Therapeutics targets a “master switch” of cancer signaling pathways, selectively impacting oncogene protein production by altering the Pre-Initiation Complex (PIC) that drives their mRNA translation. PIC Therapeutics’ selective approach modulates cancer cell proteomes, impacting multiple dysregulated oncogenic drivers leading to a powerful new generation of cancer-treating therapeutics. Our agents offer the opportunity to address both drug resistance and tumor heterogeneity, issues that plague many existing treatments.

For more information visit www.pictherapeutics.com. PIC is guided by a dedication to improving cancer patient outcomes and to realizing the potential of our programs to their benefit.

 

About OrbiMed

OrbiMed is a leading healthcare investment firm, with approximately $18 billion in assets under management. OrbiMed invests globally across the healthcare industry, from start-ups to large multinational corporations, through a range of private equity funds, public equity funds, and royalty/credit funds. OrbiMed seeks to be a capital provider of choice, providing tailored financing solutions and extensive global team resources to help build world-class healthcare companies. OrbiMed’s team of over 130 professionals is based in New York City, San Francisco, Shanghai, Hong Kong, Mumbai, Herzliya, and other key global markets.
www.orbimed.com

 

About Lumira Ventures

Lumira Ventures is a North American healthcare venture capital firm whose companies have brought dozens of biomedical innovations to patients worldwide over the past two decades. Lumira invests across multiple stages, sectors and therapeutics areas, partnering with mission-driven entrepreneurs and co-investors to build transformative healthcare companies. Lumira has offices in Toronto, Montréal, Vancouver and Boston. For more information please visit www.lumiraventures.com

 

Contacts

Matt Burke
mattdavidburke@gmail.com

Sep 12
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Fierce Biotech Names Amphista Therapeutics as One of its “Fierce 15” Biotech Companies of 2022

By Amphista Therapeutics, Press Release, Private Companies
Press Release.

 

Cambridge, UK – September, 12, 2022 – Amphista Therapeutics, a leader in next generation targeted protein degradation (TPD) approaches, today announced that Fierce Biotech has named it as one of 2022’s Fierce 15 biotechnology companies, designating it as one of the most promising early-stage biotechnology companies in the industry.

Amphista is a world leading next-generation Targeted Protein Degradation (TPD) company with the mission to unlock the full therapeutic potential of TPD by addressing the limitations of first-generation approaches. Amphista advantages include: targeting of a non-cereblon component with a more consistent expression profile that enables increased tissue reach, broader disease applicability and potentially greater clinical efficacy. This mechanistic approach, leveraging an essential protein, offers the potential for reduced clinical resistance.  Amphista’s TPD therapeutics are designed to be drug-like molecules with oral bioavailability and the potential for CNS penetration.  Amphista has built a strong IP foundation for the platform including its proprietary warheads, forming the basis of a true platform approach to TPD.

Nicki Thompson, CEO of Amphista, said

“We are delighted to be selected as a ‘Fierce 15’ company in a year where we have made outstanding progress in advancing our proprietary Eclipsys™ platform and therapeutic portfolio and in expansion of our world-leading team.  This also comes in a year where our technology has been further recognized and validated by our strategic collaborations with global pharmaceutical companies Merck Healthcare, a division of Merck, and Bristol Myers Squibb worth more than $2 billion. We remain dedicated to advancing our ground-breaking science with the potential to open up this exciting modality by delivering medicines to patients across a wider range of diseases.”

Every year Fierce Biotech evaluates hundreds of early-stage companies from around the world for its annual Fierce 15 list, which is based on a variety of factors such as the strength of its technology, partnerships, venture backers and a competitive market position.  The Fierce 15 celebrates the spirit of being “fierce” – championing innovation and creativity, even in the face of intense competition.

About Fierce Biotech
Fierce Biotech is the biotech industry’s daily monitor, an email newsletter and web resource providing the latest biotech news, articles, and resources related to clinical trials, drug discovery, FDA approval, FDA regulation, patent news, pharma news, biotech company news and more. More than 450,000 top biotech professionals rely on Fierce Biotech for an insider briefing on the day’s top stories. Signup is free at www.fiercebiotech.com/signup.

About Amphista Therapeutics

Amphista Therapeutics is focused on transforming the lives of patients with severe diseases including cancer. The company is applying its proprietary Amphista degrader platform to advance new approaches in targeted protein degradation (TPD) that  address the challenges faced by earlier stage TPD research and to realise the full therapeutic potential of this transformational approach. Founded by Advent Life Sciences, Amphista is a spin-out of TPD expert Professor Alessio Ciulli’s labs at the University of Dundee. The company has raised over $60M to date and is funded by leading life science investors including Forbion, Gilde Healthcare, Novartis Venture Fund, Advent Life Sciences, BioMotiv and Eli Lilly & Company.

For more information, please visit: https://amphista.com/

CONTACTS:
Lynn Granito
Berry & Company Public Relations
lgranito@berrypr.com
212 253 8881

Sep 12
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AviadoBio selected by Fierce Biotech as a ‘Fierce 15’ 2022 winner

By AviadoBio, Press Release, Private Companies
Press Release.

 

AviadoBio selected by Fierce Biotech as a ‘Fierce 15’ 2022 winner

Award reinforces AviadoBio’s position as one of the most promising early-stage biotech companies in the gene therapy industry

London, UK; Monday, September 12, 2022 — AviadoBio, a pioneering, pre-clinical stage gene therapy company focused on developing and delivering transformative medicines for people with neurodegenerative disorders, today announced that it has been selected by Fierce Biotech as a ‘Fierce 15’ 2022 winner, designating it one of the most promising early-stage biotechnology companies in the industry.

The award validates AviadoBio’s progress over the past year and recognizes the mission of the company, and its aspiration to develop gene therapy treatments for patients living with neurodegenerative diseases such as frontotemporal dementia (FTD) and amyotrophic lateral sclerosis (ALS) who currently have few-to-no therapeutic options.

“This award acknowledges the immense progress AviadoBio has made this year, and is a testament to the hard work of everyone at the company,”

said Lisa Deschamps, Chief Executive Officer.

“This year has marked some important milestones, including securing orphan drug designation from the FDA and EMA, as we move AVB-101, our investigational AAV gene supplementation therapy AVB-101 for frontotemporal dementia (FTD), into the clinic in late 2022.”

The Fierce 15 celebrates biotech companies who champion innovation and creativity. Every year Fierce Biotech evaluates hundreds of early-stage companies from around the world for its annual Fierce 15 list, which is based on a variety of factors such as the strength of technology, partnerships, venture backers and a competitive market position.

 

About Aviadobio

At AviadoBio, our mission is to transform the lives of people living with neurodegenerative disorders by developing and delivering transformative gene therapies for diseases including frontotemporal dementia (FTD) and amyotrophic lateral sclerosis (ALS). The Company’s technology is based on pioneering research from King’s College London and the UK Dementia Research Institute. AviadoBio’s unique platform combines next-generation gene therapy design with deep neuroscience expertise and a novel neuroanatomy-led approach to drug delivery. AviadoBio’s investors include New Enterprise Associates (NEA), Monograph Capital, Advent Life Sciences, EQT Lifesciences, Dementia Discovery Fund (DDF), F-Prime Capital, Johnson & Johnson Innovation – JJDC, Inc. (JJDC), and LifeArc.

The company is developing AVB-101 for patients with FTD-GRN. AVB-101 is an investigational AAV gene therapy designed to slow or stop disease progression by delivering a functional copy of the GRN gene throughout the central nervous system to restore progranulin levels.

For more information, please visit www.aviadobio.com and follow us at Twitter @AviadoBio and LinkedIn AviadoBio.

Aug 04
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F2G Announces $70 Million Financing to Advance Development and Commercialization of New Antifungal Agent Olorofim

By F2G, Press Release, Private Companies
Press Release.

 

MANCHESTER, UK, 4 August 2022 – F2G Ltd, a clinical-stage biopharmaceutical company focused on the discovery and development of novel therapies to treat life-threatening rare fungal infections with a high unmet medical need, today announces a $70 million financing. The financing was co-led by new investors Forbion and Sofinnova Partners with strong participation from existing investors, Novo Holdings, Morningside Ventures, Cowen Healthcare Investments and Advent Life Sciences. The proceeds from the financing in addition to the $480 million from the recent strategic collaboration with Shionogi & Co., Ltd., will enable F2G to advance late-stage development and commercialization in the US of olorofim, a novel oral antifungal therapy to treat invasive aspergillosis (IA) and other rare mold infections. Nanna Lüneborg of Forbion and Joe Anderson of Sofinnova Partners will join the F2G Board of Directors.

Olorofim represents the first novel class of antifungals developed in the past 20 years and is the only antifungal medication to be awarded a Breakthrough Therapy Designation for multiple indications by the US Food and Drug Administration (FDA). Olorofim works through a unique mechanism of action, different from existing classes of antifungals, exerting fungicidal activity through inhibition of the pyrimidine synthesis pathway. It is anticipated to be used to treat patients with a serious invasive, rare fungal disease where existing treatments are inappropriate or no longer effective. Data from F2G’s open Phase 2b study in patients with rare and resistant molds, who have limited treatment options, will be presented at ID Week annual conference in October.

Francesco Maria Lavino, Chief Executive Officer of F2G, said:

“We are delighted to attract this additional capital from such high caliber late-stage investors to F2G, and welcome Nanna and Joe to the Board. Their experience will be invaluable as we move to our next stage of growth. This is a pivotal year for the Company. We are building a world class team with commercial, operational and dealmaking experience as we prepare for final development and commercialization of olorofim in the US. If approved, olorofim is expected to be the first new class of anti-fungal with a novel, differentiated mechanism of action in more than 20 years and will address genuine unmet needs in conditions with high morbidity and mortality.”

Nanna Lüneborg, General Partner at Forbion, commented:

“The Forbion Growth Opportunities Fund II focuses on promising late-stage European life sciences companies like F2G. We have been very impressed with the significant progress achieved to date and we are pleased to support this highly impactful Company in its next stage of growth as it pursues commercialization of olorofim in the US.”

Joe Anderson, Partner at Sofinnova Partners, said:

“We aim to support outstanding companies developing innovative treatments for life-threatening disease. With its highly experienced team, F2G has made significant progress over recent years and is now close to bringing its breakthrough product to market for patients with limited treatment options. We look forward to working with management, the board and our co-investors as the company moves into this important phase in its development and growth.”

In May 2022, F2G entered a $480million strategic collaboration with Shionogi & Co., Ltd. to develop and commercialize olorofim in Europe and Asia which included $100 million in upfront payment and $380 million in regulatory and commercialization milestones plus double-digit royalties on sales.

END

For further information please contact:

F2G Ltd
Francesco Maria Lavino | Chief Executive Officer
Ralf Schmid | Chief Financial Officer
Tel: +44 (0)161 785 1271 (UK) / +43 (0)1 997 4267 (A)

Optimum Strategic Communications
Mary Clark / Charlotte Hepburne-Scott / Manel Mateus / Zoe Bolt
Email: F2G@optimumcomms.com
Tel: +44 (0) 203 882 9621

Notes to Editors

About F2G

F2G is a biotech company with operations in the UK, US, and Austria focused on the discovery and development of novel therapies to treat potentially life-threatening invasive fungal infections. F2G has discovered and developed a completely new class of antifungal agents called the orotomides which selectively target a key enzyme in the de novo pyrimidine biosynthesis pathway. This is a completely different mechanism from that of the currently marketed antifungal agents and gives the orotomides fungicidal activity against a broad range of rare and resistant fungal mold infections. For more information, please visit: www.f2g.com

About olorofim

Olorofim (formerly, F901318) is F2G’s leading candidate from the orotomide class and is currently in a Phase 2b open-label study (ClinicalTrials.gov Identifier: NCT03583164) in patients who have limited treatment options for difficult-to-treat invasive, rare fungal mold infections such as azole-resistant aspergillosis, scedosporiosis, lomentosporiosis, and other rare mold infections. F2G has initiated a global Phase 3 trial (“OASIS”)2 to compare treatment with olorofim versus AmBisome® followed by standard of care (SOC) in patients with lower respiratory tract invasive fungal disease caused by proven or probable infection with Aspergillus species. Olorofim has received orphan drug status from the European Medicines Agency for the treatment of invasive aspergillosis and invasive scedosporiosis. Olorofim has also received orphan drug status from the FDA for the treatment of coccidioidomycosis scedosporiosis, and invasive aspergillosis. Olorofim has been granted Qualified Infectious Disease Product (QIDP) designation for invasive aspergillosis, invasive scedosporiosis, invasive lomentosporiosis, coccidioidomycosis, invasive disease due to Scopulariopsis species, and invasive fusariosis.

About invasive aspergillosis

Aspergillosis is a fungal infection caused by Aspergillus species of mold which are commonly found all over the world. Most of these molds, in most people, are harmless. However, aspergillus is transmitted to humans through inhalation and may cause a broad spectrum of disease ranging from hypersensitivity reactions to direct invasion and destruction of tissue. Invasive aspergillosis is a rare disease that can occur in over 10% of some high-risk immunosuppressed populations with mortality exceeding 80%.

About Forbion
Forbion is a dedicated life sciences venture capital firm with offices in The Netherlands, Germany and Singapore. Forbion invests in life sciences companies that are active in the (bio-) pharmaceutical space. Forbion manages well over €2 billion across multiple fund strategies that cover all stages of (bio-) pharmaceutical drug development. Forbion’s current team consists of over 30 life sciences investment professionals that have built an impressive performance track record since the late nineties with successful investments in 92 companies. The firm is a signatory to the United Nations Principles for Responsible Investment. Besides financial objectives, Forbion selects investments that will positively affect the health and well-being of patients. Its investors include the EIF, through its European Recovery Programme (ERP), LfA, Dutch Venture Initiative (DVI), AMUF and EFSI facilities and KfW Capital through the Programme, “ERP – Venture Capital Fonds investments.” Forbion operates a joint venture with BGV, the manager of seed and early-stage funds, especially focused on Benelux and Germany.
For more information, please visit: www.forbion.com

About Sofinnova Partners
Sofinnova Partners is a leading European venture capital firm in life sciences, specializing in healthcare and sustainability. Based in Paris, London and Milan, the firm brings together a team of professionals from all over the world with strong scientific, medical and business expertise. Sofinnova Partners is a hands-on company builder across the entire value chain of life sciences investments, from seed to later-stage. The firm actively partners with ambitious entrepreneurs as a lead or cornerstone investor to develop transformative innovations that have the potential to positively impact our collective future.

Founded in 1972, Sofinnova Partners is a deeply established venture capital firm in Europe, with 50 years of experience backing over 500 companies and creating market leaders around the globe. Today, Sofinnova Partners has over €2.5 billion under management. For more information, please visit: www.sofinnovapartners.com.