| | • | | DMD exon 53 targeting program |
Wave is leveraging learnings from its ongoing DMD development and discovery efforts to advance its program to address DMD in boys amenable to exon 53 skipping, including recent data presented at the Project Parent Muscular Dystrophy (PPMD) Annual Conference. The company will present new data from its exon 53 skipping program at the 23rd International Annual Congress of the World Muscle Society in October 2018 and expects to deliver a clinical data readout for this program in 2020.
| | • | | PRECSION-HD Phase 1b/2a clinical trials |
ThePRECISION-HD program, which consists of two global Phase 1b/2a clinical trials evaluating investigational therapiesWVE-120101 andWVE-120102 for patients with Huntington’s disease, continues to enroll patients at sites in the United States, Europe and Canada, and the company is on track to report topline data in the first half of 2019.
| | • | | Promisingin vivo data presented for ongoing and planned programs in DMD, ALS and FTD |
At the PPMD Annual Conference on June 29, 2018, the company presentedin vivo data demonstrating that its murine-specific stereopure oligonucleotide restored 70% to 90% of natural dystrophin in a dystrophin deficient mouse model for DMD (mdx23) with substantial protein expression in the heart and diaphragm. Wave also provided an update on its clinical and discovery programs in DMD at the 2018 New Directions in Biology and Disease of Skeletal Muscle Conference.
In the last three months, Wave has presentedin vivo preclinical study results forWVE-3972-01, the company’s investigational stereopure antisense oligonucleotide designed to target the pathogenic allele of theC9ORF72gene for the treatment of amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD). Animal data forWVE-3972-01 demonstrating potent, sustained and preferential knockdown of toxic biomarkers associated with ALS and FTD were presented at the Alzheimer’s Association International Conference, RNA Therapeutics Conference 2018: From Base Pairs to Bedside and the 5th International Conference on Molecular Neurodegeneration. The company intends to initiate clinical trials ofWVE-3972-01 in ALS and FTD in the fourth quarter of 2018.
| | • | | Continued strengthening of intellectual property position |
Wave’s advances with its core chemistry and stereochemistry platform continue to strengthen its intellectual property position relating to the design, synthesis and manufacture of stereopure nucleic acid therapeutic candidates, including the allowance of sequence-independentcomposition-of-matter claims in the United States.
Second Quarter 2018 Financial Results and Financial Guidance
Wave reported a net loss of $35.9 million in the second quarter of 2018 as compared to $24.6 million in the same period in 2017. The increase in net loss in the second quarter of 2018 was largely driven by increased research and development efforts and the continued growth of employee headcount to support Wave’s programs.
Research and development expenses were $32.5 million in the second quarter of 2018 as compared to $19.1 million in the same period in 2017. The increase in research and development expenses in the second quarter of 2018 was largely driven by increases in research, preclinical and clinical investments, further expansion of our manufacturing capabilities and facility-related expenses, along with the continued growth of employee headcount to support Wave’s programs.
General and administrative expenses were $8.9 million in the second quarter of 2018 as compared to $6.7 million in the same period in 2017. The increase in general and administrative expenses in the second quarter of 2018 was mainly driven by the increase in Wave’s employee headcount, as well as increases in professional service expenses and other general operating expenses.
