|Over a week ago|
NeuBase Therapeutics appoints William Mann as COO » 08:1007/2807/28/20
NeuBase Therapeutics announced the appointment of industry veteran William Mann, Ph.D., MBA as COO. Most recently, he served as the president and CEO of Helsinn Therapeutics.
|Over a month ago|
NeuBase Therapeutics added to Russell 3000, Microcap Indexes » 09:0606/2906/29/20
NeuBase Therapeutics announced that it has been added to the Russell 3000 and Russell Microcap Indexes at the conclusion of the 2020 Russell indexes annual reconstitution.
NeuBase Therapeutics initiated with an Outperform at RBC Capital » 06:0006/1606/16/20
RBC Capital analyst Brian…
RBC Capital analyst Brian Abrahams initiated coverage of NeuBase Therapeutics with an Outperform rating and $16 price target. The analyst believes the company's preclinical protein nucleic acids show "considerable promise" and could have broad applicability in a number of diseases, including lead indications Huntington's disease and myotonic dystrophy.
NeuBase Therapeutics reports Q2 EPS (26c), consensus (22c) » 16:2105/1405/14/20
At March 31, the company…
At March 31, the company had cash and cash equivalents of approximately $5.8M, compared with cash and cash equivalents of approximately $10.3M at September 30, 2019. Subsequent to the end of Q2, the Company completed a public equity offering that generated net proceeds of approximately $33.3M. The company believes that its current cash balance will provide sufficient capital to fund operations into Q2 of 2022. "We have generated a solid foundation of data that we expect will enable us to produce mutation-specific genetic medicines for a multitude of diseases. The positive non-human primate pharmacokinetic (biodistribution) and patient-derived cell line pharmacodynamic (activity) data released on March 31st illustrate our ability to create a new class of mutation silencing therapies. In addition, our proprietary targeting technology enables our PATrOL compounds to achieve therapeutically relevant drug concentrations throughout the body, including in the brain," said Dietrich Stephan, CEO of NeuBase. Our therapeutic development programs continue to drive forward, despite the difficult macroenvironment, as evidenced by the recent positive platform validation data and well-received public offering. As we look ahead, our initial focus is to continue advancing our Huntington's disease ("HD") and myotonic dystrophy type 1 ("DM1") programs through lead optimization and IND-enabling studies. We expect to announce the lead candidate for the NT0100 Program for HD by the end of calendar year 2020, followed by the initiation of IND-enabling studies during the first half of calendar year 2021. Finally, our unique biodistribution profile allows us the opportunity to develop therapies against targets and organ systems that we believe other antisense companies cannot currently reach in the body, including into the brain after systemic delivery, which is one of the grand challenges in drug delivery of macromolecules. We believe this unique ability validates our large opportunity in the antisense space."
|Over a quarter ago|
NeuBase Therapeutics 5M share Spot Secondary priced at $6.00 » 09:3304/2804/28/20
The deal size was…
The deal size was increased to $30M in common stock, up from $20M, and priced at the low-end of the $6.00-$6.25 range. Oppenheimer and BTIG acted as joint book running managers for the offering.
NeuBase announces common stock offering, no amount given » 16:0304/2704/27/20
NeuBase Therapeutics announced that it has commenced an underwritten public offering of shares of common stock. NeuBase intends to use the net proceeds from this offering for working capital and general corporate purposes and to advance the development of its product candidates and expand its pipeline. Oppenheimer and BTIG are acting as the joint book-running managers for the offering, and National Securities Corporation is acting as co-manager.
NeuBase Therapeutics to host conference call » 07:4803/3103/31/20
Management discusses the…
Management discusses the positive pre-clinical data validating its novel, genetic therapy PATrOL Platform on a conference call to be held on March 31 at 8 am. Webcast Link
NeuBase announces preclinical data on genetic therapy PATrOL platform » 07:0803/3103/31/20
NeuBase Therapeutics announced positive preclinical data from its pharmacokinetics studies in non-human primates and in vitro pharmacodynamics data in patient-derived cell lines. NeuBase believes these data validate the key advantages of the proprietary NeuBase peptide-nucleic acid antisense oligonucleotide platform and support the Company's decision to advance the development of its Huntington's disease and myotonic dystrophy type 1 programs, as well as the potential expansion of its therapeutic pipeline into other indications. Non-Human Primate Pharmacokinetic Study: Quantitative whole-body autoradiography was performed on NHPs. A PATrOL-enabled compound was radio-labeled, and the resulting material was injected into NHPs at 5 mg/kg via a bolus tail vein injection. At four hours, twelve hours, and seven days post-dosing, NHPs were sacrificed and sectioned into 40 microm slices. Slices were exposed to autoradiography imaging plates alongside a dilution series of radioactive PNA in whole blood. Upon imaging, the dilution series enabled an analysis of the amount of compound in each of the tissues. In addition, prior to sacrifice, whole blood, urine, and feces were collected from the NHPs at specified timepoints. The major conclusions from this study include: Rapid uptake of compound out of the body's circulation after systemic intravenous administration, with a half-life in circulation of approximately 1.5 hours; Compound penetrates every organ system studied, including the central nervous system and skeletal muscle; Compound crosses the blood-brain barrier and into the key deep brain structures, including the caudate, supporting a key capability for the development of the Company's lead program in HD; Delivery of the compound to skeletal muscle, the primary organ system that is affected in DM1; Because both HD and DM1 have manifestations outside of the primary affected organ, the broad biodistribution of the compounds may enable a potential whole-body therapeutic solution in both indications. Therapeutically relevant doses persist for greater than one week in NHPs after single-dose injection; 96% of administered compound remained in vivo after a one-week period; Redistribution over one week after administration between organ systems enriches concentrations in key brain regions up to two-fold, including in those deep brain structures most relevant for HD; Retention of ~90% of compound concentrations achieved in skeletal muscle over the course of one-week post-single-dose administration; and Sustained concentrations in many other key organ systems throughout the body may indicate the potential for durable therapeutic responses and an infrequent dosing cadence. Patient-Derived Huntington's Cell Line Pharmacodynamic Studies: Multiple Huntington's disease candidate compounds were incubated with HD-derived cells and assayed for their toxicity and their ability to selectively knock down mutant huntingtin protein expression by engaging with the CAG repeat expansion in the huntingtin gene transcript. Multi-well plates were seeded with cells and candidates were added to the culture at various concentrations. Cells were grown for three days and thereafter assayed for cell death. Cell pellets were also collected, lysed, and run on gradient SDS-PAGE gels. Following the transfer of the proteins to a membrane, the membrane was probed with anti-huntingtin and anti-beta-actin antibodies. Secondary antibodies were used to image the immunoblots. The beta-actin bands were used to normalize the amount of protein across the wells. The amounts of mutant and wild type huntingtin protein in treated cells were compared to untreated cells to determine the level of knockdown. The major conclusions from this study include: Activity in engaging target disease-causing transcripts and knocking-down resultant malfunctioning mHTT protein levels preferentially over normal HTT protein knock-down; and Dose limiting toxicities were not observed relative to a control either at or above the doses demonstrating activity in human cells in vitro. In addition, PATrOL enabled compounds were generally well-tolerated in vivo after systemic administration, both after single dose administration in NHPs and multi dose administration in mice for over a month. The intersection of the NHP pharmacokinetic data and the in vitro patient-derived pharmacodynamic data provides a roadmap to create a pipeline of therapeutic candidates which can reach target tissues of interest after systemic administration and achieve the desired activity at that dose. NeuBase believes the data from these studies support the advancement of the Company's HD and DM1 programs into lead optimization and subsequent IND-enabling studies, as well as provide a roadmap for the future expansion of the Company's therapeutic pipeline into other indications, including oncology.
Fly Intel: After-Hours Movers » 19:0003/2603/26/20
BGCP, TXMD, VMW, GPS, LULU, PRGS, HTZ, KBH, GME, SPWH, NBSE, OSS
Check out this…
NeuBase Therapeutics reports Q1 EPS (26c), consensus (32c) » 16:0903/2603/26/20
At December 31, 2019, the…
At December 31, 2019, the company had cash and cash equivalents of approximately $7.7M, compared with cash and cash equivalents of approximately $10.3M at September 30, 2019. The company believes that its current cash balance will provide sufficient capital to fund operations through the end of fiscal FY20.