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Wednesday, September 2, 2020

Muddy Waters goes short on Inovio, citing Covid ‘hype’

Inovio Pharmaceuticals (NASDAQ:INO), up 245% in 2020, is off more than 12% Wednesday as short seller Muddy Waters discloses they are short the name, citing a recent court decision against the company and its recent rise due to the “hype” around a Covid vaccine.

The court decision indicated that the company “lacks manufacturing capacity” to reach 1M doses in 2020 and 100M in 2021.

The competition among the 5 Operation Warp speed companies makes INO’s chances slim absent their ability to force their supplier to transfer manufacturing technology, the research firm wrote.

The Warp speed participants include Sanofi (NASDAQ:SNY), GlaxoSmithkline (NYSE:GSK), Vaxart (NASDAQ:VXRT), Pfizer (NYSE:PFE), Moderna (NASDAQ:MRNA), Merck (NYSE:MRK), Johnson & Johnson (NYSE:JNJ) and AstraZeneca (NYSE:AZN).


New analysis of COVID-19 studies show mortality benefit of cheap steroids

A meta-analysis of pooled data from seven completed studies involving 1,703 critically ill COVID-19 patients showed that the administration of corticosteroids reduced mortality compared to standard-of-care (SOC) treatment. The results were just published online in Journal of the American Medical Association (JAMA).

Patients were randomized to receive systemic dexamethasone, hydrocortisone or methylprednisolone on top of SOC treatment or placebo + SOC. The primary outcome measure was all-cause mortality 28 days after randomization.

The overall 28-day all-cause mortality rate was 38.0% (n=647/1,703). The mortality rate in patients receiving corticosteroids was 32.7% (n=222/678) compared to 41.5% (n=425/1,025) in those receiving SOC only. Results were consistent across the studies.

Results from an Oxford University-led study, RECOVERY, reported almost three months ago, showed a similar mortality benefit from dexamethasone in severely ill patients on ventilators, cutting the risk of death by 28 – 40%. Mortality risk was reduced by 20 – 25% in COVID-19 patients receiving supplemental oxygen. It did not appear to help patients with milder symptoms.

These corticosteroids have been off patent for years and are available at low prices worldwide. In England, for example, a 10-day regimen of dexamethasone only costs ~£5 per patient. In the U.S., a 10-tablet regimen of dexamethasone is available for ~$6, prednisone for a bit less.

By comparison, Gilead Sciences’ (GILD +1.1%) Vektury (remdesivir) costs $390/vial or $2,340/five-day course.

A Phase 3 study, SIMPLE-Severe, showed a mortality rate of 7.6% at day 14 in severely ill COVID-19 patients receiving remdesivir + SOC compared to 12.5% for SOC alone, implying a 39.2% relative reduction in mortality risk (the company reported 62% less risk based on an adjusted odds ratio of 0.38. This approach holds other variables constant and provides the odds ratio for the variable of interest, in this case, mortality).


Bicycle Therapeutics lead drug advances in solid tumor study

The first participant has been dosed in the Phase 2a expansion portion of a Phase 1/2a clinical trial evaluating Bicycle Therapeutics’ (NASDAQ:BCYC) lead candidate BT1718 in patients with MT1-MMP-positive squamous non-small cell lung cancer (NSCLC) and a basket of other MT1-MMP-positive solid tumors. The primary objectives are safety and tolerability.

BT1718 is a Bicycle Toxin Conjugate (BTC) that targets an enzyme called membrane type 1-matrix metalloproteinase (MT1-MMP) that is correlated with malignancy of various tumor types and is believed to mediate cell migration and invasion. It consists of a mono-hindered disulfide cleavable linker and a cytotoxic payload called DM1.


Rocket Pharma advances lead AAV gene therapy in cardiomyopathy study

Rocket Pharmaceuticals (NASDAQ:RCKT) has treated the first patient in the higher-dose cohort in its Phase 1 clinical trial of RP-A501, an adeno-associated viral vector (AAV)-based gene therapy for the treatment of Danon disease, a rare inherited disorder characterized by weakening of heart and skeletal muscles.

The second cohort evaluates RP-A501 at a higher-dose level of 1.1×1014 genome copies/kilogram in male patients 15 years of age and older. The Phase 1 study will assess the safety, tolerability and preliminary efficacy of RP-A501.

Treatment of the higher-dose cohort comes after successful completion of the low-dose cohort and FDA clearance.

Following the review of safety data from the first cohort, all subsequent cohorts will include 2-4 patients per cohort, adjusted down from 3-6 patients in the original protocol.


Twist Bioscience antibodies show encouraging action against SARS-CoV-2

Twist Bioscience (NASDAQ:TWST) announces encouraging in vitro study data on immunoglobulin (IgG) and smaller single domain VHH “nanobodies” (antigen-binding fragments of heavy chain-only antibodies) against SARS-CoV-2. The observed neutralizing effects were comparable to or better than convalescent sera collected from people who recovered from COVID-19. The studies were conducted at Saint Louis University and results verified at Colorado State University.

The studies tested more than 200 well-characterized monoclonal antibodies and VHH nanobodies identified by the company in six weeks using its proprietary synthetic antibody discovery libraries, each containing more than 10B antibody sequences.

CEO Emily Leproust says, “These data are encouraging and provide powerful validation of our ability to generate well characterized and potent antibodies from our proprietary libraries. Importantly, the neutralizing effects seen in these in vitro studies suggest that infections in humans could be blocked. We are now evaluating the best path forward for these neutralizing antibodies to support the fight against COVID-19.”

Saint Louis University, School of Medicine’s James Brien, Ph.D., adds, “All antibodies moving through clinical development for the treatment of COVID-19 are full IgG antibodies and already show promise in early studies. The single domain (VHH) nanobodies included in these neutralization assays may represent a different therapeutic path to treat the disease. Given their very small size in comparison to IgG antibodies, they may be able to access epitopes on the virus that are unavailable to full IgGs.”


NanoViricides to provide COVID-19 drug update on September 3

NanoViricides (NYSEMKT:NNVC) will present an update on the COVID-19 drug development at the LD 500 investor conference on September 3rd at 11:20 AM EDT.

The Company has completed Chemistry, Manufacture, and Controls studies that would be required for an IND application to the FDA.

NNVC is currently conducting studies to finalize its clinical candidate.


ThermoGenesis to outline product strategy in COVID-19 at conference

September 2, 2020

ThermoGenesis Holdings (NASDAQ:THMO) along with its joint venture, ImmuneCyte plans to roll out a comprehensive diagnostic and therapeutic product line to fight COVID-19 pandemic.
The multi-pronged strategy will combine diagnostic, therapeutic and service offerings to better detect, track and fight coronavirus. Further details will be included in the company’s presentation at the LD Micro 500 Conference today at 5 p.m. EST.