Crispr Therapeutics (CRSP) and Vertex Pharmaceuticals (VRTX) stocks initially popped Friday after the biotech companies unveiled promising results for a gene-editing blood-disease treatment.
XThe biotech companies are studying a potential treatment for patients with transfusion-dependent beta thalassemia and sickle cell disease.
Updated test results show 15 months and five months after infusion, two beta thalassemia patients didn't need blood transfusions. One sickle cell disease patient has yet to have a vaso-occlusive crisis — a painful complication of the disease — in the nine months following infusion.
The results are starting to show the potential for gene editing in blood-disease treatment, Crispr Chief Executive Samarth Kulkarni said. Crispr and Vertex's drug, known as CTX001, was the first gene-editing drug to begin testing in humans.
"With these new data, we are beginning to see early evidence of the potential durability of benefit from treatment with CTX001, as well as the consistency of the therapeutic effect across patients," he said in a written statement.
Blood-Disease Treatment Shows Proof
On today's stock market, Crispr stock inched up 0.3% to 59.89. Earlier, shares jumped as high as 6.1%. Vertex stock lifts 1.8% to 268.56.
All three patients in the Crispr and Vertex study experienced side effects. But none were tied to the blood-disease treatment, Vertex said in a news release.
The data show the clinical proof of concept for a gene-editing approach to transfusion-dependent beta thalassemia, Vertex Chief Executive Reshma Kewalramani said in a written statement.
"While these are still early days, these data mark another important milestone for this program and for the field of gene editing," she said.
Now, the biotech companies have opened up enrollment to a broader group of patients. In total, five patients with beta thalassemia and two with sickle cell disease have received treatment. The study runs for two years in total, Vertex spokeswoman Heather Nichols said in an email.
"Each trial can enroll up for 45 patients," she said. "The trial is ongoing and there's been strong demand from both patients and the medical community, so we're pleased with the progress so far."
Biotech Company Faces Alexion
Also Friday, Apellis Pharmaceuticals (APLS) offered an update for its rare blood-disease treatment called pegcetacoplan. Pegcetacoplan is facing off against Alexion Pharmaceuticals' (ALXN) Soliris in improving hemoglobin levels in patients with paroxysmal nocturnal hemoglobinuria, or PNH.
Pegcetacoplan outperformed Soliris at week 16 of Apellis' study. Apellis' drug showed a 53% improvement in hemoglobin at week 16 of treatment, the biotech company said in a news release. The effect was seen across all patients regardless of prior dependence on blood transfusions.
Overall, 85% of pegcetacoplan-treated patients were transfusion-free over 16 weeks vs. just 15% of Soliris-treated patients.
Apellis stock advanced 1.7% to 30.98. Alexion stock fell 0.5% to 109.30.
Pegcetacoplan works by blocking a protein called C3. It differs from Soliris, which inhibits the C5 protein. In an email, Apellis noted C5 inhibitors can control hemolysis — the destruction of red blood cells — in the plasma, but not in the spleen and liver.
As a result, some people living with PNH continue to suffer from debilitating symptoms.
"Pegcetacoplan targets C3, which is upstream from C5 in the complement cascade, a part of the body's immune system," the company said. "By being upstream, C3 can control both intravascular and extravascular hemolysis, thus better controlling the disease and providing relief to patients."
Agios Initially Jumps, Then Falls
Shares of Agios Pharmaceuticals (AGIO) initially jumped as much as 4.8%, but pared those gains and fell 7% at the close, ending the day at 47.14. Representatives of Agios declined to comment on the stock movement.
Agios is working on a blood-disease treatment for alpha thalassemia and beta thalassemia in patients who aren't dependent on blood transfusions. The biotech company also tested its drug, dubbed mitapivat, in patients with sickle cell disease.
In alpha thalassemia, 92.3% of patients achieved a hemoglobin increase between weeks four and 12 of the study. Eight out of nine beta thalassemia patients showed a hemoglobin increase between weeks 12-24, the biotech company said in a news release.
The blood-disease treatment also boosted hemoglobin levels in 63% of sickle cell disease patients.
But one patient with sickle cell disease experienced a vaso-occlusive crisis, according to RBC Capital Markets analyst Kennen MacKay. But he noted there are no published reports of mitapivat leading to these events. The event was reported by a researcher who worked on the study.
Also Friday, Agios announced it sold the royalty it receives on Bristol Myers Squibb's (BMY) Idhifa for $255 million. Now, Royalty Pharma will receive the royalty on sales of the blood cancer treatment.
Follow Allison Gatlin on Twitter at @IBD_AGatlin.
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