Editas Medicine Prepares to Make History With EDIT-101

After another solid quarter of progress, the genome-editing company looks forward to a phase 1/2 study for its revolutionary CRISPR medicine.

Steve Symington
Steve Symington
Mar 1, 2019 at 8:00AM
Health Care

Editas Medicine (NASDAQ:EDIT) released fourth-quarter 2018 results on Thursday after the market closed, detailing its latest progress toward both the commercialization of its primary EDIT-101 drug candidate, as well as efforts to expand its repertoire with other novel CRISPR medicines.

Let's throw on our lab coats, then, to zoom in on its latest results and better understand the state of the genome-editing leader's business.

A DNA strand on a teal background.

IMAGE SOURCE: GETTY IMAGES.

Editas Medicine results: The raw numbers

Metric

Q4 2018

Q4 2017

Year-Over-Year Growth

Collaboration and other R&D revenue

$6.119 million

$3.667 million

66.9%

GAAP net income (loss)

($25.054 million)

($36.189 million)

N/A

GAAP net income (loss) per share

($0.52)

($0.84)

N/A

Data source: Editas Medicine. R&D = research and development; GAAP = generally accepted accounting principles.

Check out the latest earnings call transcript for Editas Medicine.

What happened with Editas Medicine this quarter?

  • The increase in revenue was driven by $1.6 million in revenue recognized under Editas' strategic alliance with Allergan, as well as $0.8 million in revenue related to its collaboration with Juno Therapeutics.
  • R&D expenses declined 27.3% year over year to $19.2 million, driven by a combination of lower success payment expenses, stock-based compensation, and process and platform development costs.
  • Editas ended the year with cash, cash equivalents, and marketable securities of $369 million, good for at least 24 months of funding for operating expenses and capital expenditures -- or, as the company puts it, to "fund advancement of multiple transformative experimental medicines."
  • The U.S. Food and Drug Administration (FDA) approved Editas' investigational new drug (IND) application for its EDIT-101 drug candidate. As it noted would be the case during last quarter's call, Editas received a $25 million milestone payment from Allergan per the terms of their alliance. 
  • Editas and Allergan now expect to enroll 10 to 20 patients in a phase 1/2 open-label, dose-escalation study in the second half of 2019 to evaluate EDIT-101 for treating Leber Congenital Amaurosis type 10 (LCA10), the leading cause of childhood blindness. Edit-101 would then be the first-ever in vivo -- or editing inside the body -- CRISPR medicine administered to patients.
  • In late April and early May, Editas plans to present in vivo proof-of-concept data for a CRISPR gene-editing approach to treat Usher syndrome 2A (USH2A) at the 22nd Annual Meeting of the American Society of Gene & Cell Therapy. 
  • This year, Editas also expects to begin IND-enabling activities for an experimental medicine candidate to treat sickle cell disease and beta thalassemia.
  • The company further anticipates moving forward research programs centered around allogeneic engineered natural killer cell medicines for cancer in 2019.
  • Following the resignation of CEO Katrine Bosley in January, Editas named recently appointed board member Cynthia Collins as its interim CEO. An executive search firm has been retained to find a permanent successor.

What management had to say

Collins stated:

2018 was a year of significant achievements for Editas Medicine as we advanced our efforts to bring transformative medicines to patients. The IND application for our lead experimental medicine, EDIT-101 for LCA10, was accepted by the FDA upon initial review. We advanced a potentially best-in-class CRISPR medicine candidate to treat sickle cell disease and beta-thalassemia; and, we strengthened our organization with key additions to our senior leadership and Board of Directors. In 2019, we plan to dose patients with EDIT-101, making it the first in vivo CRISPR medicine administered to patients in history. We are also well positioned to advance the Company's EM22 long-range goals.

Looking forward

Editas doesn't provide specific quarterly financial guidance. But the company demonstrated considerable progress toward its primary goals this quarter, particularly as it not only moves its EDIT-101 drug candidate toward its first phase 1/2 study later this year, but also advances its pipeline of supplementary novel medicines with the help of its rock-solid balance sheet. Thus, I think patient, long-term Editas Medicine shareholders still have every reason to be excited for what's to come.