Pain Therapeutics to try again with REMOXY NDA
Investors love biotech stocks for the lottery ticket-like returns they can offer if a company strikes medical gold or wins the regulator’s blessing. Case in point: Pain Therapeutics, Inc. (NASDAQ:PTIE), whose shares are skyrocketing nearly 160% on Tuesday. The reason? The drug maker announced positive results from a human abuse potential study of its late-stage drug candidate, REMOXY. On back of the results, the company believes it has successfully completed all studies necessary to resubmit the REMOXY new drug application (NDA) to the FDA, and plans to do so shortly.
REMOXY is a proprietary, abuse-deterrent, extended-release oral formulation of oxycodone. The proposed indication for this drug candidate is for "the management of pain severe enough to require daily, around-the-clock, long-term opioid treatment and for which alternative treatment options are inadequate."
The study results indicate that in non-dependent, recreational opioid users, nasal administration of REMOXY resulted in significantly lower abuse potential compared to immediate-release (IR) oxycodone. All study subjects reported reduced ‘Drug Liking’ ‘Take Drug Again’ and ‘Drug High’ for REMOXY compared to oxycodone IR. In addition, nasal administration of REMOXY showed lower exposure to oxycodone, lower peak concentrations (Cmax) and longer time to peak drug concentration (Tmax) against comparator drugs, suggesting comparatively lower abuse potential.
As a reminder, back in September 2016, the FDA sent Pain Therapeutics a Complete Response Letter (CRL) related to the resubmission of the company's NDA for REMOXY. The CRL focused on the abuse-deterrent properties of REMOXY and proposed drug labeling; it made no mention of clinical safety, drug efficacy, manufacturing, stability, bioequivalence, or any other issues from a prior CRL.
Global Blood Therapeutics: A breakthrough designation bestowed by the FDA is good news for investors
Investor optimism remains high, following news that Global Blood Therapeutics Inc's (NASDAQ:GBT) drug candidate voxelotor received FDA breakthrough-therapy status for the treatment of sickle cell disease (SCD). Voxelotor is being developed as a disease-modifying therapy for SCD and previously received European Medicines Agency (EMA) Priority Medicines (PRIME) designation for the treatment of SCD.
Global Blood shares reacted to the news, soaring nearly 20% as of 1:10PM EST.
“The FDA’s decision to grant voxelotor the first Breakthrough Therapy designation for the treatment of sickle cell disease reflects a recognition of the promising efficacy and safety data we have collected to date for this investigational drug, as well as an acknowledgement of the overwhelming need for major advances over available therapies in the treatment of SCD patients,” said Ted W. Love, president and chief executive officer of GBT. “This designation is another significant milestone for GBT as we work to expedite the development of voxelotor.”
The FDA selectively grants BTD to expedite the development and review of drugs that have demonstrated preliminary clinical evidence indicating the potential for substantial improvement over available therapy. The BTD decision for voxelotor was based on clinical data submitted from the following studies:
- Preliminary efficacy and safety data from Part A of the Phase 3 HOPE Study (GBT440-031)
- Phase 1/2 study and open-label extension in adults (GBT440-001/024)
- Ongoing Phase 2 HOPE-KIDS 1 study in children age 6 to 17 (GBT440-007)
- Compassionate Access experience in adults with severe SCD (not eligible for the HOPE Study)
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