Three years ago, biotechs focused on gene editing faced skepticism from many investors. Despite some making clinical progress, only a few had successfully launched products. Since then, the landscape has evolved with more clinical successes and several companies bringing gene-editing products to market.
However, Intellia Therapeutics (NASDAQ: NTLA) has not emerged as one of the top performers in this niche. The biotech’s shares have experienced a 30% decline over the past year. The question arises: Is it a prudent decision to invest in Intellia Therapeutics’ shares at this point? Let’s explore.
Intellia Therapeutics’ promising programs
In the challenging landscape of clinical-stage biotechs, Intellia Therapeutics finds itself at a crucial juncture. Despite limited revenue and consistent losses, the company holds promise with its advanced programs, particularly as they progress towards potential approval.
At the forefront of Intellia’s pipeline is NTLA-2001, designed to address transthyretin amyloidosis, a rare ailment causing diverse symptoms, including muscle weakness. The collaboration with biotech giant Regeneron adds significant strength to this program, offering financial support without resorting to dilutive financing measures.
Enrolling patients for a phase 3 study targeting the non-hereditary form of transthyretin amyloidosis, with plans to dose the first patient in the first quarter, Intellia is also gearing up for a late-stage study for hereditary transthyretin amyloidosis. With a patient population exceeding 350,000 for this condition and the potential for NTLA-2001 to become the first one-time curative treatment, the stakes are high.
While it may take an estimated two years (optimistically) to launch NTLA-2001 if late-stage trials yield positive results, the therapeutic landscape for transthyretin amyloidosis, lacking a cure, holds immense potential. Intellia Therapeutics is not solely reliant on NTLA-2001; it has another candidate, NTLA-2002, in clinical trials for hereditary angioedema, with data expected this year and a phase 3 study on the horizon.
Lots of patience required
As of the end of 2023, Intellia Therapeutics reported having $1 billion in cash, equivalents, and marketable securities, a decrease from $1.3 billion the previous year. The management anticipates that this financial cushion will suffice until mid-2026. While positive outcomes from ongoing clinical trials or regulatory advancements could significantly impact the stock price positively, these prospects come with inherent risks.
Despite the collaboration with Regeneron and the recent approval of Casgevy, the first CRISPR-based gene-editing therapy, there are no guarantees of success for Intellia Therapeutics’ programs. The CRISPR-focused platform shows promise, but potential pitfalls remain. Investors considering a position in this biotech stock should be prepared to withstand substantial risks before deciding to initiate a small investment.
