ICPT = Intercept Pharmaceuticals, Inc. (Nasdaq:ICPT) announced that the U.S. Food and Drug Administration (FDA) has accepted Intercept’s New Drug Application (NDA) for obeticholic acid (OCA) seeking accelerated approval for the treatment of fibrosis due to nonalcoholic steatohepatitis (NASH) and granted priority review. The FDA grants priority review to drugs that have the potential to treat a serious condition and, if approved, would provide a significant improvement in safety or effectiveness.
“If approved, OCA would be the first available therapy for patients with fibrosis due to NASH, a condition that is expected to become the leading cause of liver transplant in the U.S. as soon as 2020,” said Mark Pruzanski, M.D., President and Chief Executive Officer of Intercept. “It is exciting to achieve this critical regulatory milestone that brings us one step closer to our goal of delivering the first approved therapeutic to those living with this devastating disease. From OCA’s prior designation as a Breakthrough Therapy to the grant of priority review today, our work with FDA continues to set an important precedent for the field, and we look forward to working with the agency over the coming months as they review the first NDA in NASH.”
The FDA has assigned a Prescription Drug User Fee Act (PDUFA) target action date of March 26, 2020 for the NDA. In the NDA filing acceptance notification letter, the FDA also indicated that it currently plans to hold an advisory committee meeting to discuss the application. A date for the advisory committee meeting has not been finalized and the timeline for the review of the NDA by the FDA remains subject to change.
OCA is the only investigational therapy to have received Breakthrough Therapy designation from the FDA for NASH with fibrosis. The NDA filing for OCA is supported by positive interim analysis results from the pivotal Phase 3 REGENERATE study in patients with liver fibrosis due to NASH. In the study, OCA 25 mg demonstrated robust improvement in liver fibrosis (by ≥1 stage) with no worsening of NASH at 18 months.
ARWR = As previously reported, Baird analyst Madhu Kumar upgraded Arrowhead Pharmaceuticals (ARWR) to Outperform from Neutral with a price target of $70, up from $39, after Medicines Company (MDCO) announced a definitive agreement to be acquired by Novartis (NVS). That deal forces Kumar to re-evaluate Arrowhead’ RNAi platform, which is heavily levered to lipid-modifying RNAi drugs for cardiovascular disease, the analyst tells investors. Given the early positive efficacy for Arrowhead’s ANGPTL3 and APOC3 RNAi drugs presented at the American Heart Association meeting, Kumar believes these programs have “the chance to dominate the ANGPTL3 and APOC3 landscapes,” though cautions both are “quite early.”
SSI = Still moving higher after reporting quarterly earnings on November 21, 2019. Third quarter highlights:
- Comparable sales increased 17.4%.
- Net loss was $15.9 million compared to net loss of $31.4 million in the third quarter 2018.
- Adjusted net loss improved by $26.3 million to $4.2 million.
- Adjusted EBITDA improved $28.5 million to $15.3 million.
- Converted 17 department stores to Gordmans off-price, bringing total year-to-date conversions to 89.
- Excess availability under the credit facility at quarter end was $101 million, an increase of approximately $35 million compared to the end of the second quarter 2019.
- Amazon Counter was expanded to approximately 700 of our stores, generating convenience for customers and increased store traffic.
“Our outstanding third quarter results give us further confidence in our decision to convert the entire company to an off-price business model,” commented Michael Glazer, Chief Executive Officer. “The 17.4% increase in third quarter comparable sales was driven by a variety of factors that encompass our guests’ positive reaction to the new Gordmans stores and our pre-conversion activities. The 89 department stores converted to off-price during 2019 delivered a combined sales increase of nearly 40% in the third quarter.
“Our results also reflect that guests in our department stores are responding quite favorably as we celebrate sales and events prior to their store converting. Our expanded home business in department stores delivered a comparable sales increase of 180% in the third quarter. In the fourth quarter, we expect excitement to grow with our holiday marketing focusing on celebrating the last holiday season at our department stores with messages including ‘Last Black Friday Sale Ever’ and ‘Last Christmas Sale Ever.’ Notably, our pre-conversion promotional efforts do not involve incremental couponing or markdowns and as a result, retail margins have not been negatively impacted. In fact, in the third quarter, retail margins increased 130 basis points.
“Additionally, we are thrilled with the early results of our Amazon partnership which is popular with Amazon customers and our loyal guests. Over 700 of our stores are now active pick-up points for Amazon Counter just in time for the holiday season. This will increase our traffic and provide Amazon shoppers with fast, flexible and convenient package pick-up.”
JAN = JanOne announced a licensing agreement for TV1001SR, a treatment for Peripheral Artery Disease, commonly called PAD. The agreement with LSU Health Shreveport, UAB Research Foundation, and TheraVasc, gives JanOne a worldwide, exclusive license for TV1001SR along with a portfolio of 30 patents and other intellectual property relating to the sustained release of sodium nitrite. The company anticipates TV1001SR will be a groundbreaking treatment for those with PAD, an often painful disease affecting more than 200M people worldwide and 8.5M in the United States. If the company is ultimately granted FDA market approval, it believes that TV1001SR would immediately disrupt the $4.37B a year PAD market in the United States.
PTCT = PTC Therapeutics (PTCT) announced that the FDA has granted priority review for the New Drug Application, or NDA, for risdiplam for the treatment of spinal muscular atrophy, or SMA. The Prescription Drug User Fee Act, or PDUFA, goal date for a decision by the FDA is May 24, 2020. The filing acceptance by the FDA triggers a $15M milestone payable to PTC by Roche (RHHBY). The FDA has granted risdiplam priority review status, which is designated to drugs that, if approved, would represent significant improvements in the safety or effectiveness of the treatment, diagnosis, or prevention of serious conditions when compared to standard applications.
“The FDA’s acceptance of the NDA is an important step towards making risdiplam available to SMA patients in the U.S.,” said Stuart W. Peltz, Ph.D., Chief Executive Officer of PTC Therapeutics. “We are proud that risdiplam, the first oral small molecule targeting splicing, was produced from our proprietary splicing platform. Risdiplam’s NDA submission includes results from a broad SMA patient population, including type 1, type 2 and type 3 SMA patients demonstrating improvements in motor functions and developmental milestones, and a compelling safety profile. We believe that an oral therapeutic that reaches all affected tissues in the body would mark a significant advancement in the treatment for SMA patients and their families.”
MRTX = Still attracting buyers after November 10, 2019, when Piper Jaffray analyst Tyler Van Buren maintained an Overweight rating and $125 price target on Mirati Therapeutics (MRTX) following the company’s presentation of new data for the combination of sitra + nivo in chemo and PD-1 refractory bladder cancer patients and in oral cavity cancer patients at SITC. In a note to investors titled “The Sitra + Nivo Combo Regimen Appears Active In Bladder And Oral Cancers,” Van Buren said that the combo is “potentially promising in both of these patients populations,” but he wants to see more data to start including the opportunities in his model. The analyst also noted that sitra was being evaluated in combination with Beigene’s (BGNE) tislelizumab/PD1 in multiple solid tumor types and he expects “to see additional Phase I/II data over the next year.”
MTNB = Moving higher after company announced on November 18, 2019, that Jerome D. Jabbour, Chief Executive Officer, has been invited to present a company overview, as well as host investor meetings, at the 31st Annual Piper Jaffray Healthcare Conference.
Date: Tuesday, December 3, 2019
Presentation Time: 10:30 a.m. ET
Place: Lotte New York Palace Hotel, New York
Investors interested in arranging a meeting with the Company’s management during this conference should contact the conference coordinator.
A live audio webcast of the presentation will be available on the Events page of the Investors section of the Company’s website (www.matinasbiopharma.com). A webcast replay will be accessible for 90 days following the live presentation.
TNK = Still attracting buyers interest after November 18, 2019, when BofA/Merrill analyst Ken Hoexter issued a double-upgrade on Teekay Tankers to Buy from Underperform with a price target of $3, up from $2. Despite the 83% run-up in the stock price since October, the analyst contends that shares can “re-rate higher” with crude tanker markets current being in the “early stages of a robust recovery” and given the company’s “attractive operating leverage” to rate inflections. Hoexter adds that Teekay’s mid-sized vessels will also benefit from an improving crude tanker market, with the current tanker demand expected at 6% FY20 that should “materially outpace muted fleet supply growth of 1-2%.”
Note: Teekay Tankers Ltd. just completed a one-for-eight reverse stock split.
DMPI = DelMar Pharmaceuticals, Inc. (Nasdaq: DMPI), a biopharmaceutical company focused on the development of new solid tumor cancer therapies, today announced interim data on its two Phase 2 trials of VAL-083, the Company’s lead compound for the treatment of glioblastoma multiforme (GBM). The data were presented in two posters at the 2019 Society for NeuroOncology Annual Meeting in Phoenix, Ariz.
The first poster outlined the open-label, Phase 2 study of VAL-083 as a first-line treatment in newly-diagnosed, unmethylated GBM patients. This study has enrolled 23 out of a planned 30 patients as of the data cut-off date of November 2, 2019. For the 22 patients who had completed at least one cycle of treatment as of that date, median progression-free survival (PFS) with VAL-083 is currently 9.9 months (confidence interval, or CI 7.3-12.0 months). For the 18 patients initially receiving the intended treatment dose (30 mg/m2/day on days 1, 2 and 3 of a 21-day cycle) median PFS is currently 10.4 months (CI 6.0-12.0 months). While this is not a head-to-head trial, historically, temozolomide (TMZ) has been demonstrated to have 6.9 months PFS in unmethylated GBM patients. Other doses were also examined as part of the dose escalation aspect of the study, and all but the 20 mg/m2/day dose also demonstrated superior PFS to the historical comparator. A median of eight cycles of treatment has been received by all patients who had either completed treatment or remain in active treatment. Nine patients have received more than 10 cycles. This study is being conducted at Sun Yat-sen University Cancer Center in China.
The second poster outlined interim data from two groups of patients receiving VAL-083 in the open-label, Phase 2 study in recurrent and adjuvant unmethylated GBM settings. The recurrent group is receiving second-line therapy with VAL-083 following TMZ failure. Sixty-two patients (out of a planned 83) have been enrolled as of the data cut-off of November 15, 2019, with 35 patients having received an initial dose of 40 mg/m2/day and 27 (out of a planned 48) having received an initial dose of 30 mg/m2/day (on days 1, 2 and 3 of a 21-day cycle). Median overall survival (mOS) for the 60 patients who have completed at least one cycle of treatment is currently 7.5 months (CI 6.0-11.5 months). For the 25 of those patients who initially received the intended treatment dose of 30 mg/m2/day, mOS is currently 10.6 months (CI 5.8-10.6 months). While this is not a head-to-head trial, historically lomustine, which is the most commonly used chemotherapy for these patients, has demonstrated a mOS of 7.2 months.
The second arm of this study, in which patients receive VAL-083 as adjuvant therapy following treatment with radiation and TMZ, was initiated in July 2019. As of the data cut-off of November 15, 2019, five patients (out of a planned 20) have been enrolled and all patients remain alive on continued therapy. The study in recurrent and adjuvant GBM is being conducted at M.D. Anderson Cancer Center in Houston, Tex.
Similar to prior experience with VAL-083, myelosuppression has been the most common adverse event observed. Four subjects have experienced a serious adverse event (SAE) possibly related to VAL-083 in the newly-diagnosed group, eleven subjects have experienced a possibly drug-related SAE in the recurrent group, and no patients have experienced a possibly drug-related SAE in the adjuvant group as of the relevant data cut-off dates.
During a review of the data, which was conducted at the SNO conference, Dr. David Reardon, clinical director of the Center for Neuro-Oncology at the Dana-Farber Cancer Institute, Professor of Medicine at the Harvard Medical School and member of DelMar’s Scientific Advisory Board stated, “Putting it into the perspective of what has benefitted our patients historically, which has been a cytotoxic treatment approach like Delmar’s VAL-083, we have had other classes of anticancer therapies that have unfortunately been quite disappointing. So, with VAL-083 we have a mechanism of action that has a proven track record and a rationale for why it may be superior to what we have currently. All of those factors explain why it has been challenging to move the bar in this disease. That said, I think there is some reason to be hopeful that this has a likelihood of further confirming the signals we have seen so far.”
Dr. Nick Butowski, director of translational research in neuro-oncology and a researcher at the Brain Tumor Center and also a member of DelMar’s Scientific Advisory Board added, “Upon reviewing the analysis from both studies, mechanistically, survival data-wise, imaging-wise, quality of life and safety data are all very intriguing and exciting. Obviously, we need to see the completed data and then move on to the next steps.”
Saiid Zarrabian, CEO of DelMar Pharmaceuticals stated: “We continue to be encouraged with the interim outcomes for both of our ongoing Phase 2 trials of VAL-083 in GBM. Our first-line treatment study continues to show outstanding results and we are particularly pleased to see VAL-083 at the 30 mg dose currently showing a full three months longer progression-free survival. This represents an improvement of around 30% over temozolomide, the current standard of care. We are also encouraged by the 30 mg dose in the recurrent patient cohort, currently showing median overall survival of more than three months or approximately a 30% improvement over historical published results from the current standard of care. We look forward to providing subsequent updates in 2020 at the American Academy of Cancer Research and American Society of Clinical Oncology conferences.”
The Company’s current cash resources are expected to be sufficient to fund the Company’s planned operations into the fourth quarter of calendar year 2020, and to allow for funding to top-line results for our newly-diagnosed and recurrent setting studies, and for full enrollment for the adjuvant study arm patients.
To get these stocks much faster (real-time) with alerts, check out the tool we use here. There’s nothing like it on the market.