Regeneron reports positive data for Dupixent pivotal trial
Regeneron Pharmaceuticals Inc. (REGN) announced supplementary positive data from Part A of a pivotal Phase 3 trial. The study evaluated the investigational use of Dupixent® (dupilumab) in patients 12 years and older with eosinophilic esophagitis. The trial met both of its co-primary endpoints. It also fulfilled all key secondary endpoints. Currently, there is no FDA-approved treatment in the market for eosinophilic esophagitis.
Part A of the trial signed up 81 patients aged 12 years and older with EoE. These patients were administered Dupixent 300 mg weekly for a treatment period of 24 weeks or were given placebo. The new data showed that patients who were given the drug candidate showed fast improvement in ability and comfort of swallowing. Patients reported substantial progress on the Dysphagia Symptom Questionnaire by 4 weeks. The improvement was sustained through 24 weeks.
The data also showed that 64 percent of the patients on Dupixent attained <15 eosinophils/high-power field in comparison to 8% for placebo at 24 weeks. Evan S. Dellon, M.D., M.P.H., Professor of Gastroenterology and Hepatology at the University of North Carolina School of Medicine and principal investigator of the trial said, “The results from this trial show dupilumab significantly improved both patients’ ability to swallow, as well as structural abnormalities in the esophagus, by targeting type 2 inflammation to help reverse tissue damage and scarring that usually worsens over time.”
Dupixent Phase 3 trial is a randomized, double-blind, placebo-controlled study. Part A had 81 patients where 42 were given Dupixent and 39 were given placebo. The co-primary endpoints for the trial evaluated the change from baseline in the DSQ and the proportion of patients achieving peak esophageal intraepithelial eosinophil count of ≤6 eos/hpf at 24 weeks.
Key secondary endpoints of the trial included the evaluation of histopathologic measures of the severity and extent of tissue scarring in the esophagus and the proportion of patients achieving peak esophageal intraepithelial eosinophil count of <15 eos/hpf at 24 weeks. The study also evaluated NES for the relative change from baseline to week 24 in the EoE diagnostic panel and type 2 inflammation transcriptome signatures.
Dupixent was granted Breakthrough Therapy tag in September 2020 to treat patients 12 years and older with EoE. Earlier in 2017, the drug candidate was given Orphan Drug designation for the potential treatment of EoE.
Dupixent is a fully-human monoclonal antibody. It works by controlling the signaling of the interleukin-4 (IL-4) and interleukin-13 (IL-13) proteins. The drug was developed using Regeneron’s proprietary VelocImmune® technology.
Analysis: The eosinophilic esophagitis market is estimated to reach $150 million by 2024, from $100 million in 2019 at a CAGR of 6.2%. It’s but one small pie in a ~$60 billion company like Regeneron. The stock was priced at $567.04 as of last close on 10/28/2020. Over 88% of Regeneron shares are held by institutions, while the public has less than 7% shareholding. The company has a debt of $2.22 billion and cash balance of $3.14 billion, while revenue estimate for the year is $8.28 billion. Cash burn in the TTM was $1.959 billion and revenue cost was $3.9 billion. Net income in the TTM is about $3 billion.
Investment Thesis: Regeneron had been on the go on account of its COVID-19 vaccine development program. However, the company has a well-diversified pipeline which is expected to keep generating significant catalysts to push the stock price up.
CytoDyn reports encouraging data for Leronlimab in stroke patients
CytoDyn Inc. (OTCQB:CYDY) announced positive data for the use of its lead asset Leronlimab in treating stroke patients. The data showed that two patients exhibited significant signs of progress following their treatment with the drug candidate.
Leronlimab has shown the potential to cross the blood-brain barrier for treating various conditions. The data collected from a study of HIV in macaques showed that the drug candidate was able to show nearly 70 to 75 percent CCR5 receptor occupancy in the frontal lobes, parietal lobes, and cerebellum. The impact of CCR5 antagonism in central nervous system pathology is likely to have wide implications in a range of conditions such as multiple sclerosis, Parkinson’s disease, CNS tumors, traumatic brain injury, and stroke recovery.
Leronlimab has been given a Fast Track designation by the FDA for two potential indications including as a combination therapy with HAART for HIV-infected patients and for metastatic triple-negative breast cancer. Nader Pourhassan of CytoDyn said, “We continue to focus our energies on our most important task of enrolling more patients in our severe-to-critical COVID-19 Phase 3 trial as quickly as possible.”
CytoDyn has long-term planning for its lead asset. The company reported that it has received the clearance from the Medicines & Healthcare product Regulatory Agency (MHRA) of the U.K. government to file Biologics License Application as a One injection per week treatment for multi-drug resistance HIV patients in the U.K.
Analysis: CytoDyn’s lead asset leronlimab is set to be a major player in the CCR5 inhibitors/antagonist market. The company has a market capitalization of $1.38 billion on a stock price of $2.42, which is in the lower quadrant of the 52-week range from $0.26 to $10.01. Over 86% of the 571 million outstanding shares are held by the public, while institutions, corporations and insiders hold 4%, 3.7% and 5.68% shares respectively. CytoDyn has a debt of $32.4 million, and cash burn in the TTM has been $87.6 million, whereas cash balance is very low at $18.20 million.
Investment Thesis: While CytoDyn is deriving synergies by developing its lead candidate Leronlimab for different indications, its COVID-19 program is also expected to yield positive results.
Turning Point Therapeutics announces positive results from TPX-0022 trial
Turning Point Therapeutics Inc. (TPTX) reported positive clinical data from its SHIELD-1 study of TPX-0022. The trial involved twenty-two patients and it tested four dose levels of TPX-0022. The group consisted of 13 MET-altered non-small cell lung cancer, 4 colorectal cancer, 4 gastric or gastroesophageal junction cancer and one glioblastoma patients.
The data from the preliminary efficacy analysis was based on 15 evaluable patients while total 22 patients with MET-dysregulated advanced solid tumors were treated with TPX-0022 at dose levels from 20 mg once daily (QD) to 120 mg QD. Mohammad Hirmand, M.D., chief medical officer said “MET driven cancers affect a large and growing population of patients who have limited therapeutic options. Based on these early study findings, we look forward to advancing the development of TPX-0022.”
Turning Point expects to start Phase 1 dose expansion after the recommended Phase 2 dose is determined. The company plans to begin discussions with the FDA to convert the ongoing Phase 1 into a registrational Phase 1/2 design. It is anticipated that the company may begin the Phase 2 portion in the second half of 2021.
Turning Point Therapeutics is a clinical-stage precision oncology company. Its development portfolio mainly consists of cancer therapies aiming to fill the gaps in the current cancer treatment regimen. Its main drug candidate repotrectinib is a next-generation kinase inhibitor which targets the ROS1 and TRK oncogenic drivers of non-small cell lung cancer and advanced solid tumors. The drug candidate is currently in a registrational Phase 2 study called TRIDENT-1.
Analysis: Turning Point’s candidate TPX-0022 is a MET/CSF1R/SRC inhibitor that is set to gain significant share in the ~$100 billion cancer market set to double by 2026. 8 Wall Street analysts are very bullish on the company with an average score of 4.62/5 and a price target of $117.13. The current price of the stock is $104.42, taking the market capitalization to $4.88 billion. Over 74% shares are held by institutions, hedge funds, PE/VC firms and corporations hold about 6% shares each, while insiders hold over 8% shares. Turning Point has a cash balance of $710.43 million, a negligible debt burden of $4.46 million, and cash burn of $108 million in the TTM. The company has a comfortable cash run for a few years.
Investment Thesis: The company currently has three drug candidates in different developmental stages. Currently, Turning Point does not have any revenue-generating product in the market, however, it has a robust liquidity profile to keep its development programs going.
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