Wave Life Sciences have announced financial results for the first quarter ended March 31, 2018, and provided a business update.

“The strong momentum we achieved in 2017 with our clinical development programs and as an organization continued through the first quarter,” said Paul Bolno, MD, MBA, Chief Executive Officer and President of Wave Life Sciences. “We are seeing significant interest from patients and the medical community in our first three clinical studies in Huntington’s disease and Duchenne muscular dystrophy and look forward to initiating three additional development programs by the end of the year.”

The global Phase 1 clinical trial, testing WVE-210201 for the treatment of Duchenne muscular dystrophy (DMD) patients amenable to exon 51 skipping, continues to enroll patients. Safety data from the trial are anticipated in the third quarter of 2018.

  • Wave’s manufacturing facility completes inaugural good manufacturing practice (GMP) campaigns

    Wave is now producing clinical material in the GMP manufacturing suite at its Lexington, Massachusetts facility. The company recently completed manufacturing runs and material from these campaigns will support the development programs, including those for ALS and FTD.

    In July 2017, Wave took occupancy of the new facility which was designed to provide greater independence and flexibility in conducting clinical trials, secure material for current and future development activities and provide the capability for commercial-scale manufacturing. The company is continuing to build out the 90,000-square foot facility with process development, quality control and analytical development laboratories.     
  • Takeda collaboration takes effect: at least $230 million in committed cash

    In April 2018, Wave’s global strategic collaboration with Takeda Pharmaceutical Company Limited to discover, develop and commercialize nucleic acid therapies for disorders of the central nervous system took effect when Wave closed on the issuance and sale of 1,096,892 ordinary shares to Takeda and received aggregate cash proceeds of $60 million. Wave also received an upfront payment of $110 millionin cash under the collaboration. In addition, Takeda is required to fund at least $60 million of Wave research over a four-year period to advance multiple preclinical targets.
  • Neuromuscular research collaboration with Deep Genomics established to identify novel splicing targets

    In April, Wave formed a collaboration with Deep Genomics, Inc. to identify novel therapies to be developed by Wave for the treatment of genetic neuromuscular disorders. Under the collaboration, the companies will analyze and test oligonucleotides against potential therapeutic targets within multiple genes implicated in neuromuscular disorders. The collaboration aims to increase the size of patient populations that may be eligible for treatment by expanding the universe of druggable splicing targets beyond DMD and spinal muscular atrophy.

First Quarter 2018 Financial Results and Financial Guidance

Wave reported a net loss of $35.2 million in the first quarter of 2018 as compared to $21.1 million in the same period in 2017. The increase in net loss in the first quarter of 2018 was primarily driven by increases in research and development efforts, infrastructure investments and employee headcount to support Wave’s corporate goals.

Research and development expenses were $29.2 million in the first quarter of 2018 as compared to $14.7 million in the same period in 2017. The increase in research and development expenses in the first quarter of 2018 was primarily driven by increases in research, preclinical and clinical investments, further expansion of our manufacturing capabilities and facility-related expenses to continue to advance Wave’s expanding pipeline.

General and administrative expenses were $8.0 million in the first quarter of 2018 as compared to $5.9 million in the same period in 2017. The increase in general and administrative expenses in the first quarter of 2018 was primarily driven by the continued growth in Wave’s employee headcount, as well as increases in facility-related expenses and other general operating expenses.

Wave ended the first quarter of 2018 with $110.5 million in cash and cash equivalents as compared to $142.5 million as of December 31, 2017. The decrease in cash and cash equivalents in the first quarter of 2018 was primarily the result of Wave’s quarterly net loss of $35.2 million.

The company expects that its cash and cash equivalents, together with the $170.0 million of cash received from Takeda in April 2018, will enable it to fund its operating and capital expenditure requirements to the end of 2020.