BiomX Receives Orphan Drug Designation from the U.S. Food and Drug Administration for BX004 for the Treatment of Chronic Pulmonary Infection Caused by Pseudomonas aeruginosa in Patients with Cystic Fibrosis
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Insights
The Orphan Drug Designation (ODD) granted by the FDA to BiomX's phage therapy, BX004, represents a significant regulatory milestone. This designation is typically awarded to drugs that show promise in treating rare diseases, affecting fewer than 200,000 people in the U.S. It provides certain benefits, including tax credits, eligibility for orphan drug grants and a seven-year period of market exclusivity upon approval. The impact on BiomX could be substantial, potentially accelerating the development timeline and reducing costs.
From an industry perspective, the biotech sector often sees increased investor interest in companies that secure ODD, as it reflects a positive regulatory trajectory. For BiomX, the designation for BX004 may enhance its investment profile and could lead to strategic partnerships or funding opportunities to support further clinical development. However, investors should be aware that clinical trials are inherently risky and the eventual FDA approval is not guaranteed.
The market for cystic fibrosis treatments is competitive but also limited due to the specific patient population. BiomX's BX004, targeting chronic pulmonary infections caused by Pseudomonas aeruginosa, addresses a niche yet critical aspect of CF care. The Fast Track designation received earlier, coupled with the new ODD, suggests that BX004 could enter the market with a differentiated profile. This could allow BiomX to carve out a specific segment within the CF treatment market.
Given the positive topline results from their Phase 1b/2a trial, BX004 has the potential to disrupt the current standard of care, which may translate into significant market share and revenue growth for BiomX. However, the long-term impact will depend on the outcomes of subsequent trial phases, the drug's efficacy and safety profile and the company's ability to successfully commercialize the product. Stakeholders should monitor the progress of BX004 closely, as it could influence BiomX's market valuation and competitive stance.
Chronic pulmonary infections caused by P. aeruginosa in cystic fibrosis patients are notoriously difficult to treat due to the bacteria's resistance to multiple antibiotics. Phage therapy, like BX004, offers a novel approach by utilizing viruses that specifically target and kill bacteria. The ODD reflects the FDA's recognition of the potential of BX004 to meet an unmet medical need in CF care.
The positive results from the Phase 1b/2a trials indicate that BX004 could be a significant advancement in the management of chronic P. aeruginosa infections. For medical professionals and patients, the development of such therapies could mean better disease management and improved quality of life. However, the complexity of phage therapy development and the need for extensive clinical validation are important considerations. The medical community will be looking for detailed data on efficacy, dosing and long-term safety to fully assess the potential of BX004 as a treatment option.
CAMBRIDGE, Mass. and NESS ZIONA, Israel, Jan. 04, 2024 (GLOBE NEWSWIRE) -- BiomX Inc. (NYSE American: PHGE) (“BiomX”), a clinical-stage company advancing novel natural and engineered phage therapies that target specific pathogenic bacteria, today announced that its phage cocktail, BX004, has been granted Orphan Drug Designation (“ODD”) by the United States Food and Drug Administration (“FDA”), for the treatment of chronic pulmonary infection caused by Pseudomonas aeruginosa (or P. aeruginosa) in patients with cystic fibrosis (“CF”). As a reminder, in August 2023, the FDA granted BX004 Fast Track designation for the treatment of chronic pulmonary infections caused by P. aeruginosa bacterial strains in patients with CF.
“We are pleased to announce that the FDA has granted BX004 Orphan Drug Designation, which underscores the pressing need to develop new and innovative treatment options for this vulnerable patient population,” said Jonathan Solomon, Chief Executive Officer of BiomX. “Persistent and deadly pulmonary infections from P. aeruginosaremain a major source of morbidity and mortality for CF patients, andBX004 has been designed to address this significant unmet need. Based upon positive topline results observed in our Phase 1b/2a trial, we believe BX004 holds significant potential to improve upon the current standard of care, and we look forward to working with the FDA to further advance the clinical development of BX004.”
About Orphan Drug Designation (ODD)
An orphan drug is defined in the 1984 amendments of the U.S. Orphan Drug Act (ODA) as a drug intended to treat a condition affecting fewer than 200,000 persons in the United States. Orphan designation qualifies the sponsor of the product for seven-year marketing exclusivity to the first sponsor obtaining FDA approval of a designated drug, a tax credit equal to
About BX004
BiomX is developing BX004, utilizing its proprietary BOLT platform, for the treatment of CF patients with chronic pulmonary infections caused by P. aeruginosa, a main contributor to morbidity and mortality in patients with CF. In September 2021, BX004 was cleared by the FDA to initiate a Phase 1b/2a study in CF patients with chronic pulmonary infections caused by P. aeruginosa. The Phase 1b/2a trial was composed of two parts. Part 1 of the study evaluated the safety, pharmacokinetics, and microbiologic/clinical activity of BX004 in nine CF patients in a single ascending dose and multiple dose design. Part 2 of the study evaluated the safety and efficacy of BX004 in 34 CF patients randomized to treatment or placebo in a 2:1 ratio. Positive topline results from Part 2 of the study were announced on November 29, 2023. In August 2023, the FDA granted BX004 Fast Track designation for the treatment of chronic pulmonary infections caused by P. aeruginosa bacterial strains in patients with CF.
About BiomX
BiomX is a clinical-stage company developing both natural and engineered phage cocktails designed to target and destroy bacteria in the treatment of chronic diseases. BiomX discovers and validates proprietary bacterial targets and customizes phage compositions against these targets. For more information, please visit www.biomx.com, the content of which does not form a part of this press release.
Safe Harbor
This press release contains express or implied “forward-looking statements” within the meaning of the “safe harbor” provisions of the U.S. Private Securities Litigation Reform Act of 1995. Forward-looking statements can be identified by words such as: “target,” “believe,” “expect,” “will,” “may,” “anticipate,” “estimate,” “would,” “positioned,” “future,” and other similar expressions that predict or indicate future events or trends or that are not statements of historical matters. For example, when BiomX discusses the safety, tolerability and efficacy of BX004 and its potential ability to treat CF patients, as well as the potential to advance the BX004 program to a larger, pivotal Phase 2b/3 trial, if such trial is initiated, including, among other things, timing, design, enrollment, regulatory approvals and funding of such trial, BiomX is making forward-looking statements. Forward-looking statements are neither historical facts nor assurances of future performance. Instead, they are based only on BiomX management’s current beliefs, expectations and assumptions. In addition, past and current pre-clinical and clinical results, as well as compassionate use, are not indicative and do not guarantee future success of BiomX clinical trials. Because forward-looking statements relate to the future, they are subject to inherent uncertainties, risks and changes in circumstances that are difficult to predict and many of which are outside of BiomX’s control. Actual results and outcomes may differ materially from those indicated in the forward-looking statements. Therefore, investors should not rely on any of these forward-looking statements and should review the risks and uncertainties described under the caption “Risk Factors” in BiomX’s Annual Report on Form 10-K filed with the Securities and Exchange Commission (the “SEC”) on March 29, 2023, and additional disclosures BiomX makes in its other filings with the SEC, which are available on the SEC’s website at www.sec.gov. Forward-looking statements are made as of the date of this press release, and except as provided by law BiomX expressly disclaims any obligation or undertaking to update forward-looking statements.
1 Source: https://www.fda.gov/media/83372/download
BiomX Contacts:
Investor Relations:
LifeSci Advisors, LLC
John Fraunces
(917) 355-2395
jfraunces@lifesciadvisors.com, or
LifeSci Advisors, LLC
Brian Mullen
(203) 461-1175
bmullen@lifesciadvisors.com
BiomX, Inc.
Anat Primovich
Corporate Project Manager
+972 (50) 697-7228
anatp@biomx.com
Source: BiomX Inc.
FAQ
What is the Orphan Drug Designation granted to BiomX's BX004 by the FDA?
What previous designation was granted to BX004 by the FDA?
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