A big week for CRISPR Therapeutics (CRSP 5.44%) has gotten even bigger as the biotech company is awaiting a crucial decision. The U.S. Food and Drug Administration (FDA) expects to decide on Friday whether to approve what may become CRISPR Therapeutics' very first product -- exa-cel, a gene-editing treatment for blood disorders.
And now, in unrelated news, CRISPR Therapeutics has cut two of its more advanced immuno-oncology candidates to favor second-generation alternatives -- and expand into autoimmune diseases. One of the cancer candidates, CTX-110, was involved in a phase 2 potentially registrational trial, meaning the study could have supported a regulatory request. So, is this latest move a setback for the biotech company or a step forward?
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Positive clinical trial results
It's important to note that CRISPR Therapeutics had reported positive trial results from the now eliminated candidates, CTX-110 AND CTX-130. The FDA had granted both candidates regenerative medicine advanced therapy designation, meant for investigational therapies showing potential to treat serious diseases. The designation helps expedite the development of such potential treatments.
CRISPR Therapeutics' immuno-oncology candidates are chimeric antigen receptor T-cell therapies, in which T cells are genetically altered to better fight cancer cells. The company has been developing CTX-110 and CTX-112 to target the CD19 protein in B cell malignancies and developing CTX-130 and CTX-131 to target protein CD70 in T cell malignancies and solid tumors. Now, the company is dropping the more advanced candidates to focus on the next-generation candidates, CTX-112 and CTX-131.
Though the dropped candidates produced solid clinical trial results so far, CRISPR Therapeutics says the newer ones showed they may be able to do even better. Early trial results demonstrated potential for greater efficacy -- two novel gene edits may boost potency and reduce a slowdown in activity known as CAR T exhaustion.
The next-generation candidates also offer an improved manufacturing profile -- producing a higher number of CAR T cells per batch. So, these candidates may lead to better results for patients and to a more efficient manufacturing process for the company.
Of course, this move may result in a longer road to commercialization, since the company initiated phase 1 trials of these candidates earlier this year -- as mentioned above, CTX-110 already had advanced beyond that point.
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Slow and steady wins the race
Is this a setback or a step forward for CRISPR Therapeutics? Ideally, investors would love for the company to commercialize a candidate from this program as soon as possible. And those initial candidates could have made this happen more quickly than their second-generation versions. But sometimes slow and steady wins the race. The fact that CRISPR Therapeutics recognized the strengths of the newer candidates now actually is great news, even if it means a lengthier time to market.
That's because it allows the company to present its very best candidates for regulatory consideration -- leading to a better chance of approval and a better chance for use among patients. It's better to wait and deliver a game-changing product than to more quickly deliver one that will get lost in the shuffle. Oncology treatment is a highly competitive market, making it crucial for new entrants to stand out from the crowd.
On top of this news, the company also said it's expanding into autoimmune diseases and will begin testing CTX-112 in that area. It will start by launching a clinical trial in systemic lupus erythematosus in the first half of the year.
All of this means that CRISPR Therapeutics' latest news is more of a step forward than anything else. The company has discovered strengths in two of its four immuno-oncology candidates and is going all in on them rather than diluting its efforts across less promising options. And the biotech is even expanding into a new area.
This increases CRISPR Therapeutics' chances for success over time, and that's why this represents great news not only for the biotech company, but for investors, too.
