Homology Medicine Growth Rely On Successful PKU Gene Therapy Program

 Homology Medicine is a genetic medicine company translating proprietary gene editing and gene therapy technologies into novel treatments for patients with rare diseases. Homology Medicines has a broad, powerful, and proprietary gene editing and gene therapy platform, an experienced and dedicated team, and a commitment to bring new treatments and cures forward for the rare disease community. 


Stock Symbol: FIXX

Platform
The proprietary human hematopoietic stem cell-derived adeno-associated virus vectors (AAVHSCs) to are used to deliver the treatment to patients. 

Q3 2020 Company Highlights
  • Based on Positive Data, Advancing pheNIX Gene Therapy Clinical Trial for PKU to Dose Expansion Phase
  • $60 Million Equity Investment from Pfizer Inc. -
  • Unveiled In Vivo Gene Therapy Program for Hunter Syndrome
Therapeutic Areas
  • Phenylketonuria (PKU)
  • Metachromatic Leukodystrophy
  • Hunter Syndrome (MPS II) 
  • Sickle Cell Disease
  • Homoglobinopathy
  • Opthalmic Targets



Analyst Review and Growth 
Successful PKU Clinical Program is considered as Inflection Point for the company

Analyst Review: Please visit wsj.com

PKU Gene Therapy Clinical Program As Inflection Point 
  • Target Population: Large (Estimated Population- 50,000 worldwide)
  • Competition: Kuvan and Palyniziq are only approved therapy, fewer companies including Biomarin Pharmaceuticals is developing gene therapy for the indication
  • Price: Gene Therapy Products tend to have higher price ranging from 0.5-2.0 millions 
PKU Clinical Program Update (6 November 2020)
  • Marked Reductions in Phe Observed at Two Doses -
  • Achieved Goal with Plans to Advance to Randomized, Concurrently Controlled Expansion Phase of Trial
Cohort 1 (Low-Dose)
Through 52 weeks, patients in Cohort 1 continued to show no meaningful reductions in Phe.

Cohorts 2 and 3 (Mid- and High-Dose)
The mean percent change from baseline in Phe observed in patients in Cohorts 2 and 3 were significant, compared to Cohort 1. These Phe reductions occurred while patients self-liberalized their diets.
Through 48 weeks, one patient in Cohort 2 had Phe levels of <360 μmol/L and/or <600 μmol/L at multiple timepoints and had reached a minimum Phe level of 42 μmol/L, compared with a baseline level of 1,010 μmol/L. Through 13 weeks, one patient in Cohort 3 had a Phe level <360 μmol/L and several Phe levels <600 μmol/L at multiple timepoints and had reached a minimum Phe level of 303 μmol/L, compared with a baseline level of 1,060 μmol/L.

Third Quarter 2020 Financial Update
  • Net loss for the quarter ended September 30, 2020 was $(28.2) million or $(0.62) per share, compared to a net loss of $(29.6) million or $(0.67) per share for the same period in 2019.
  • Collaboration revenues for the quarter ended September 30, 2020 were $0.6 million, compared to $0.4 million for the quarter ended September 30, 2019 and consisted of revenue recognized under the Company’s strategic collaboration with Novartis. Collaboration revenues are being recognized over time consistent with the pattern of performance of research and development activities under the collaboration agreement. Homology and Novartis continue to work together on an ophthalmic program and seek to identify new targets for the collaboration based on the exploratory research component.
  • Total operating expenses for the quarter ended September 30, 2020 were $28.8 million, compared to $31.7 million for the quarter ended September 30, 2019, and consisted of research and development expenses and general and administrative expenses.
  • Research and development expenses for the quarter ended September 30, 2020 were $20.4 million, compared to $25.7 million for the quarter ended September 30, 2019. Research and development expenses decreased due to a decrease in direct research expenses for HMI-102 related to the transition of the manufacturing of clinical trial materials for our Phase 1/2 pheNIX trial from a CMO to our internal GMP facility in 2020, which reduced costs with our CMOs compared to the prior year period. Research and development costs related to laboratory supplies and research materials for our early-stage research programs also decreased. Partially offsetting these decreases were increased costs due to additional employee headcount to support ongoing development programs, research initiatives, technology platform and manufacturing capabilities resulting in increases in salaries, payroll taxes, stock-based compensation expense and recruiting costs.
  • General and administrative expenses for the quarter ended September 30, 2020 were $8.4 million, compared to $6.0 million for the quarter ended September 30, 2019. General and administrative expenses increased primarily due to personnel costs as a result of new hires, increased audit and legal costs and additional costs associated with expanded operations.
  • As of September 30, 2020, Homology had approximately $178.0 million in cash and cash equivalents. Based on current projections, Homology now expects cash resources, together with the anticipated proceeds of $60.0 million from the Pfizer equity investment, to fund operations into the third quarter of 2022.
For details, visit www.homologymedicine.com

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