Kiniksa reports positive results for Mavrilimumab
Kiniksa Pharmaceuticals Ltd. (NASDAQ:KNSA) announced positive data from its global Phase 2 trial. The study achieved both its primary as well as secondary efficacy endpoints with statistical significance. The company believes that its drug candidate mavrilimumab has the potential to emerge as an alternative treatment option for patients suffering from giant cell arteritis.
Phase 2 trial is a randomized, double-blind, placebo-controlled study. It consisted of a six-week screening period and a 26-week double-blind placebo-controlled treatment period. These phases were succeeded by a 12-week washout safety follow-up period. Sanj K. Patel of Kiniksa said, “These data suggest mavrilimumab may offer a treatment option for patients suffering from giant cell arteritis and further demonstrate the potential broad utility of mavrilimumab. We look forward to presenting additional data from this study in a publication or at a future medical conference.”
The trial involved randomizing 70 patients on 3:2 basis and administering mavrilimumab 150 mg or placebo biweekly. Both the options were injected subcutaneously, co-administered with a protocol-defined 26-week oral corticosteroid taper. Patients were then categorized by new onset or relapsing/refractory disease. The primary endpoint of the trial was of time-to-first adjudicated GCA flare. It was found to be statistically significant by Week 26 in all treated patients. However, median time-to-flare by Week 26 could not be assessed for mavrilimumab cohort due to the low number of flares.
The secondary efficacy endpoint was of sustained remission at Week 26 in all treated patients. The endpoint was also found to be statistically significant in the trial. For mavrilimumab recipients, the sustained remission rate at Week 26 was 33.3 percentage points higher than placebo group. For mavrilimumab recipients, the sustained remission rate was reported at 83.2 percent, while for placebo group, it stood at 49.9 percent.
Mavrilimumab was found to be well-tolerated. No drug-related serious adverse events were reported. Also, the rates of drug-related treatment-emergent adverse events between both the groups were similar. The company is currently carrying out the 12-week washout safety follow-up period and supplementary analyses of the Phase 2 trial. Kiniksa will be holding discussions with the FDA for designing its development program in GCA.
The mechanistic rationale of focusing on the granulocyte macrophage colony stimulating factor is amply supported by the preclinical data. It was also shown that the drug candidate was able to impact the production of inflammatory molecules characteristic of GCA pathophysiology in an ex-vivo culture model of arteries from GCA patients. Mavrilimumab also reduced arterial inflammation and gamma interferon production in an in-vivo model of human GCA. The drug candidate was recently given Orphan Drug designation by the FDA.
Mavrilimumab is also being tested as a treatment for COVID-19 pneumonia and hyperinflammation. The company is currently recruiting participants for the Phase 2 portion of Phase 2/3 clinical trial. It is expected that the data from an investigator-initiated study in the U.S. will be available in the fourth quarter of 2020.
Investment Thesis: The company stock has performed strongly in the past one year and is currently trading at attractive valuation. The company has strong pipeline with candidates such as vixarelimab and rilonacept. It has several potential milestones coming up, making the stock an attractive proposition.
Biogen announces CRISPR collaboration with Scribe Therapeutics
Biogen Inc. (NASDAQ:BIIB) has inked a new collaboration with privately held Scribe Therapeutics. The deal is mainly concerned with the development and commercialization of CRISPR-based therapies for addressing underlying genetic reason of amyotrophic lateral sclerosis. The collaboration will focus on leveraging Scribe’s technological platform for this purpose.
Under the terms of this agreement, Scribe and Biogen will cooperate to develop therapeutics for genetically-driven ALS. The collaboration also provides for an option to work on additional neurological disease target as warranted. Benjamin Oakes, CEO and co-founder of Scribe Therapeutics. “We’re proud to collaborate with Biogen and apply our uniquely customized approaches with the goal of developing new, safe and effective genetic medicines for neurodegenerative disease.”
Scribe will be entitled to $15 million upfront payment. It also stands to receive more than $400 million on meeting different development and commercial milestones for two targets of interest. The company may also become eligible to obtain tiered, high-single-digit to sub-teen royalties.
Scribe Therapeutics is a molecular engineering company. It is mainly involved in developing in-vivo therapies for the permanent addressal of underlying causes of a disease. One of its co-founder, Jennifer Doudna, has been conferred Nobel Prize in 2020. The company’s first technology X-Editing offers superior editing, specificity and deliverability capabilities than other currently available CRISPR genome editing tools.
CRISPR molecules come with a guide RNA that is capable of identifying target DNA sequence and an enzyme. The company’s technology is capable to creating treatments which can edit the genome in a safer and more effective manner.
Investment Thesis: This blue-chip stock is expected to retain its stable performance. With its robust product portfolio and development pipeline, Biogen is suitable for a long-term investment plan.
NantKwest reports augmenting its Phase 2 pancreatic cancer trial
NantKwest Inc. (NK) announced adding a new third cohort to its Phase 2 trial for locally advanced or metastatic pancreatic cancer. It is collaborating with ImmunityBio, a privately-held immunotherapy company for this study. The addition of the new cohort will allow cancer patients who have failed all approved standards of care to partake in the trial.
This trial is a randomized, open-label study and seeks to assess safety and efficacy of a combination immunotherapy. This combination therapy comprises NantKwest’s PD-L1 t-haNK, ImmunityBio’s IL-15 superagonist Anktiva (N-803), and aldoxorubicin, plus standard of care. Patrick Soon-Shiong of NantKwest said, “By adding the third cohort to this important study, we’re able to enroll patients at all stages of the disease, even those who experience disease progression after the first- or second-line treatment.”
The trial is currently taking place at three sites. The combination therapy being investigated aims to use the body’s immune system for treating, killing and “remembering” cancer cells. The therapy works on the mechanism to let the body develop its own antibodies for combating the cancer. The company plans to study each treatment setting, each first- and second-line or later maintenance treatment independently. The study plans to enroll 298 subjects for all of its three cohorts.
The primary endpoint for cohorts A and B is progression-free survival and the objective of cohort C is overall survival (OS) per RECIST V1.1. Secondary objectives include overall response rate, complete response rate, durability of response, disease control rate, and overall survival.
Investment Thesis: The stock is quite volatile and is currently trading at discount to its previous highs. It is suitable for investors with strong risk appetite and has the potential to deliver solid returns.
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