Tuesday, April 28, 2020 @ 7:00 am
Polyphor AG (SIX: POLN) a research-driven clinical-stage, Swiss biopharmaceutical company committed to discovering and developing first-in-class molecules in oncology and antimicrobial resistance today announced its financial results for the full-year of 2019.
Balixafortide – Start of the FORTRESS Phase III clinical study followed by strong progress
In June 2019, Polyphor announced that the first patient had been enrolled in the FORTRESS Phase III clinical study evaluating balixafortide (POL6326) in combination with eribulin for the treatment of patients with HER2-negative, locally recurrent or metastatic breast cancer. The study enrollment is progressing ahead of plan (264 patients randomized, 69% total) as a result of strong execution and the company expects the first data-cut for the first co-primary end point, Objective Response Rate (ORR), from the trial around end of Q1 2021, which could lead to an accelerated approval in the US, while Progression Free Survival (PFS) is expected in Q4 2021 and will be the basis for a regular Marketing Authorization submission in the US and EU.
Polyphor firmly believes that the potential positive ORR and PFS results will not only create a significant business opportunity in the initial indication but also give the opportunity to build a pipeline around balixafortide. The company plans to investigate balixafortide as a novel treatment option in earlier lines of metastatic breast cancer and tumors with other combinations beyond breast cancer. The company started a series of collaborations to generate preclinical evidence together with world-renowned institutions, such as the MD Anderson Cancer Center. Moving forward, Polyphor expects to continue to broaden academic and industry collaborations in the mid-term.
Beyond balixafortide, the company has identified a number of novel oncology targets which can be engaged by peptides from Polyphor’s leading macrocycle technology platform and is currently assessing the opportunity to nominate development candidates post balixafortide’s ORR results in the beginning of 2021.
Inhaled murepavadin – Completion of the preclinical program and decision to submit a Clinical Trial Application in Q4 2020
In 2019, Polyphor completed the preclinical experiments on inhaled murepavadin for the treatment of patients with cystic fibrosis (CF). The program is partially funded by the Innovative Medicines Initiative (IMI), a partnership between the European Union and the European pharmaceutical industry. Having diligently assessed the available non-clinical and translational studies suggesting significantly higher safety margins (at least 5-10 times) versus the I.V. formulation, the likelihood of inhaled murepavadin to cause kidney damage at planned doses in human is expected to be low.
As a result, a Clinical Trial Application is expected in the next 6 months (Q4 2020) and the start of a Phase I study shortly after, expanding Polyphor’s clinical pipeline. If approved for commercial use, inhaled murepavadin would represent the first new class of antibiotics for Gram-negative pathogens in the last 50 years targeting specifically Pseudomonas aeruginosa bacteria.
Renewed strategy for research and preclinical antibiotic programs
In December 2019, Polyphor completed the preclinical experiments on the company’s novel Outer Membrane Protein Targeting Antibiotics (OMPTA) candidate POL7306, a medium spectrum antibiotic targeting all WHO Priority 1 pathogens. Despite the promising efficacy package, the company has decided not to submit a CTA for the POL7306 program due to estimated therapeutic margins. After a thorough analysis of the learnings from the research and development efforts Polyphor has decided to reprioritize the remaining antibiotics programs with strong focus on formulation and peptide design optimization to achieve broader therapeutic margins before moving into clinical trials. In doing so, the company will apply lessons learned from the POL7306 program as well as the murepavadin I.V. program which the company has decided to stop in the beginning of 2020.
In addition, Polyphor initiated a new target program within the OMPTA class, thanatin derivatives, targeting specifically Enterobacteriaceae including multidrug resistant strains, one of the most common and resistant pathogens. Moving forward, the company seeks to continue research and development for the antibiotics pipeline with the support of existing and future non-dilutive and/or external financing to ensure external scientific validation while minimizing the cost of these programs to the cash position in the near future.
Polyphor started 2019 with a strong financial position, that together with a swift change in the strategic direction and proactive resource allocation, enabled the company to shift strategic priorities and reallocate resources to the promising assets in oncology, cystic fibrosis and anti-infectives.
In 2019, the total loss of the period amounted to CHF 64.7 million. R&D costs increased by 36% compared to last year to a total of CHF 60.7 million. R&D costs were primarily driven by the murepavadin Phase III trials (PRISM) which were closed in July 2019 and the initiation of the pivotal trial of balixafortide (FORTRESS). Revenues decreased by CHF 6.5 million from the last reporting period which included a licensing agreement with Santhera Pharmaceuticals Ltd. This brings the company’s total cash position to CHF 77.4 million (cash and cash equivalents) as of December 31, 2019, which will finance operations throughout 2020 and well into Q1 2021.
Statement on the impact of the COVID-19 outbreak
Polyphor’s first priority remains the health and safety of its employees and clinical study participants. Regarding the Phase III Pivotal Study FORTRESS, the company has taken all appropriate measures to safeguard patients, study conductors, investigators and the conduct of the study in general. As continuity of the trial remains crucial for patients, investigators and the company, Polyphor will continue to take proactive measures related to COVID-19 to ensure uninterrupted supply and mitigate any impact on the study. As of end of April, the company is on schedule to complete the recruitment of 384 patients in the Fortress study by end of September 2020 and start the murepavadin inhaled study in healthy volunteers in Q4 2020.
Commenting on the company’s outlook, Polyphor CEO Gökhan Batur says: “We have demonstrated the capacity to rapidly transform our strategic priorities and build capabilities to successfully execute a Phase III oncology study that is currently ahead of plan in its enrollment and achieved positive first Data Safety Monitoring Board (DSMB) recommendation. The data readout on balixafortide is an important milestone and key priority for our company.” Batur continues: “With our renewed strategy, we aspire not only to continue on our balixafortide Phase III trial, but also to establish a pipeline of potentially transformative new oncology and antibiotic candidates and new indications for balixafortide that are in clinical development in the near future. These additional efforts in progressing our mid-term pipeline in oncology and antibiotics will bring substantial benefit to patients and high value to our company and our shareholders.”