(RTTNews) - Today's Daily Dose brings you news about FDA denying approval to Zosano Pharma's migraine treatment; CRISPR Therapeutics' CARBON trial results; and the progress in NantKwest's COVID-19 vaccine candidate.
1. CRISPR Therapeutics' CARBON trial Proves a Point but Stock gets Whacked
CRISPR Therapeutics (CRSP) announced positive top-line results from its ongoing phase I trial of CTX110, an investigational allogeneic CAR-T cell therapy, in adult patients with relapsed or refractory non-Hodgkin lymphoma, dubbed CARBON.
Data reported were from 11 patients spread across 4 dose levels of CTX110. Dose Levels 1 & 2 had 3 patients each, Dose Level 3 had 4 patients and Dose level 4 had 1 patient.
The primary endpoints of the CARBON trial include safety as measured by the incidence of dose-limiting toxicities (DLTs) and overall response rate. Key secondary endpoints include the duration of response, progression-free survival, and overall survival.
According to the trial results, CTX110 has shown dose-dependent efficacy and response rates.
Complete response (CR) was achieved at Dose Levels 2, 3, and 4 - with the rates being 33%, 50%, and 100%, respectively. In other words, 1 out of the 3 patients in Dose Level 2; 2 out of the 4 patients in Dose Level 3, and the lone patient in Dose Level 4 achieved a complete response.
Complete response refers to the disappearance of all signs of cancer in response to treatment. This does not always mean the cancer has been cured. (Source: National Cancer Institute).
In Dose Levels 1 to 3, no dose-limiting toxicities were observed. In these levels, Cytokine Release Syndrome (CRS) occurred in three patients all of which were resolved. Two additional serious adverse events that occurred after CTX110 infusion were determined to be unrelated to disease progression or CTX110 and they too were resolved.
The lone patient in Dose Level 4 also experienced Cytokine Release Syndrome which resolved in 5 days and as mentioned above he had also achieved a complete response. However, on Day 26, the patient was hospitalized with febrile neutropenia and developed symptoms of short-term memory loss and confusion. In due course, he also developed serious complications like HHV-6 encephalitis and succumbed to death a few days later.
Commenting on the trial results, Joseph McGuirk, investigator in the CARBON trial, said, "While longer follow-up is required, these early data support the potential for CTX110 to become an effective off-the-shelf CAR-T therapy for patients with relapsed or refractory B-cell malignancies."
CRSP closed Wednesday's trading at $92.22, down 13.57%.
2. NantKwest's COVID-19 Vaccine on Track
NantKwest Inc. (NK) and ImmunityBio a privately-held immunotherapy company, announced that the first patient has been dosed in their phase I clinical trial of hAd5-COVID-19, a novel COVID-19 vaccine candidate.
hAd5-COVID-19 targets the inner nucleocapsid (N) and the outer spike (S) protein and uses a second-generation adenovirus that delivers multiple proteins of the SARS-CoV-2 with the potential for long-term immunity through memory T cells.
The phase I trial of hAd5-COVID-19, which is being conducted at the Hoag Hospital in Newport Beach, California, is designed to enroll 35 healthy adults aged 18 to 55 years old.
NK closed Wednesday's trading at $7.61, down 9.94%.
3. Zosano plunges as FDA gives Thumbs Down To Migraine Drug Candidate
Shares of Zosano Pharma Corporation (ZSAN) plunged more than 27% on Wednesday, following the FDA's refusal to approve Qtrypta, proposed for the acute treatment for migraine.
Qtrypta is a proprietary formulation of Zolmitriptan, administered via the company's intracutaneous microneedle-array drug-delivery system. Zolmitriptan as a tablet and an orally disintegrating tablet (tablet that dissolves quickly in the mouth) is already used to treat acute migraine headaches in adults.
Last month, in a preliminary communication related to Qtrypta, the FDA had raised questions regarding unexpected high plasma concentrations of Zolmitriptan observed in five study subjects from two pharmacokinetic studies and the differences in Zolmitriptan exposures observed between subjects receiving different lots of Qtrypta in the company's clinical trials.
The reasons cited in the complete response letter for denying approval to Qtrypta are consistent with FDA's preliminary communication in September.
ZSAN closed Wednesday's trading at $0.44, down 27.74%.
4. Stocks That Touched New Highs/Lows
ProPhase Labs Inc. (PRPH) closed at a new 52-week high of $7.96, up 12.75%.
Phreesia Inc. (PHR) closed at an all-time high of $36.28, up 11.56%.
Tarsus Pharmaceuticals Inc. (TARS) closed at a new high of $24.50, up 15.51%.
Codiak BioSciences Inc. (CDAK) closed at a new low of $9.75, down 16.02%.
Baudax Bio Inc. (BXRX) closed at an all-time low of $1.42, down 10.13%.
The views and opinions expressed herein are the views and opinions of the author and do not necessarily reflect those of Nasdaq, Inc.