TransMedics Reports Positive Data, And Other News: The Good, Bad And Ugly Of Biopharma

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Summary

TransMedics Reports Positive Data from PROTECT Trial

TransMedics Group Inc. (TMDX) announced that it has received positive data from its US pivotal OCS Liver PROTECT Trial. The study is a multicenter, two-armed, prospective trial for assessing the effectiveness and safety of the OCS Liver to preserve and assess donor livers intended for transplantation. The trial studied 300 patients where 153 patients were randomized to transplantation using the OCS Liver while 147 patients were randomized to use cold storage methods.

The study met both its primary and secondary safety and effectiveness endpoints. The primary effectiveness endpoint of the use of OCS Liver showed a significantly lower incidence of early allograft dysfunction with 17.3 percent for OCS and 30.5 percent for the control arm. This data was observed across both the donors after brain death and donors after circulatory death cohorts included in the trial. Waleed Hassanein, MD, President and Chief Executive Officer said, "Data from the trial further support our belief that the OCS Liver System along with our OCS Lung and OCS Heart Systems has the potential to meaningfully improve patient outcomes and increase the number of organ transplants to help end-stage organ failure patients. We are grateful for all the trial investigators, their teams and institutions for completing this seminal trial in liver transplantation."

The trial achieved its three secondary effectiveness endpoints. The OCS Liver maintained an approximate physiologic functioning state and monitored the condition of the liver outside of the human body. The patient survival at 30 days post-transplant stood at 99.3 percent for OCS in comparison to similar 99.3 percent for the control arm. The use of OCS Liver resulted in a significantly lower incidence of ischemic cholangiopathy complications at 6 months post-transplantation with 1.4 percent for OCS and 8.5 percent for Control arm. Ischemic cholangiopathy complications are one of the biggest reasons behind late graft failure after liver transplantation.

The study also met its primary safety endpoint, which was measured as the average liver graft-related serious adverse events per patient. The rate stood for OCS at 0.046 in comparison to 0.075 for Control. The utilization rate was reported at 98.1 percent where 152 patients were transplanted out of 155 donor livers. Dr. Malcolm MacConmara, MD, Director of the Organ Research Lab at UT Southwestern Medical Center and a co-investigator of the OCS Liver PROTECT Trial said, "If approved, this would safely expand the utilization of donor livers and significantly increase the number of livers available for life-saving transplantation."

TransMedics Group Inc. mainly focuses on assessing donor organs for transplantation. It also offers services for portable extracorporeal warm perfusion. The company has developed several technologies for assessing the preserving organ quality prior to carrying out transplant process. Its offerings have the potential of increasing the number of transplant organs found eligible.

Cerecor Announces New Protein Role in COVID-19

Cerecor (CERC) stock jumped as the company made a new announcement pertaining to the role of a novel cytokine in determining the severity and mortality in COVID-19 patients with acute respiratory distress syndrome. The data was collected from a group of 47 COVID-19 patients in hospitals and 30 healthy participants.

Cerecor reported that the quantum of novel cytokine LIGHT is highly correlated with the severity level and mortality of the coronavirus disease. LIGHT levels were found to be significantly higher in the serum of patients hospitalized with COVID-19 in comparison to healthy patients. Dr. Garry Neil, M.D. chief scientific officer, Cerecor commented, "As a company, we recognized the impact of cytokine storm-induced ARDS and the need for treatment options for patients in this area of high unmet need. We remain focused on the CERC-002 clinical program and rapidly moving it forward for the treatment of cytokine storm induced ARDS."

The data showed that the patients requiring ventilator support showed the highest levels of LIGHT. This phenomenon was particularly high in patients above the age of 60 years. The data also showed that higher LIGHT levels were strongly related to the mortality. LIGHT is a cytokine with inflammatory actions encoded by the TNFSF14 gene. It has been demonstrated to have a key role in the immune response to viral pneumonia. It also impacts immune responses in the gut, skin and the lung. LIGHT stimulates B Cell and T Cell responses while also regulating the delivery of other cytokines such as TNF, GM-CSF, IL1, IL6, IL-8 and IL-10.

Cerecor collaborated with Myriad RBM for this study. Myriad RBM is a subsidiary of Myriad Genetics Inc. (NASDAQ:MYGN). The partnership has developed an ultrasensitive assay for the detection of free LIGHT. The assay uses serum and plasma samples and has the ability to measure LIGHT from normal and disease subjects.

Sorrento Announces Approval for Starting Phase 1 Trial CD38 ADC

Sorrento Therapeutics Inc. (SRNE) announced that the US FDA has cleared its Investigational New Drug application for STI-6129. This drug candidate is a CD38-targeting antibody drug conjugate. The company is now planning to start its Phase 1 clinical trial for assessing the drug candidate in patients suffering from advanced relapsed and/or refractory systemic amyloid light chain (AL) amyloidosis.

Sorrento uses its proprietary technology platforms for designing its products such as a CD38 specific antibody. Dr. Hui Li, head of Sorrento's ADC business unit, Levena Biopharma said, "This is Sorrento's first ADC utilizing our site-specific C-LOCK conjugation technology that is advancing into clinical evaluation. STI-6129 demonstrated an improved therapeutic index in animal models, as compared to traditional non-selective conjugates, and we look forward to potentially expanding its utilization into additional ADC programs." The company also said that the primary objective of the upcoming study will be to determine a phase 2 dose for STI-6129 taking into account its safety, preliminary efficacy and pharmacokinetic profile.

Sorrento is a clinical stage biopharmaceutical firm and is mainly focused on developing therapies for malignant cancers. The company uses a multipronged and multimodal approach for this purpose.

Investment Thesis

Cerecor is a mid-size company with its main focus on pediatric healthcare. While its stock has seen considerable drop in its market price on Year to Date basis, the reasons behind this fall are not exclusive to this company. The stock is now looking to make a comeback thanks to positive news such as the latest FDA clearance. The company is also collaborating with other firms such as Myriad Genetics for progressing its product pipeline. Overall, it looks like a decent bet for longer-term investors.

Sorrento Therapeutics Inc. stock has shown over 45 percent growth this year so far. Current valuation seems to be on the higher side. We would avoid.

TransMedics Group stock has lost about a quarter of its value this year so far. However, the main reasons are largely extrinsic to the firm. The company recently announced 61 percent increase in first quarter revenue on a Year over Year basis. With the positive top line data announcement from a pivotal trial, the stock may show positive moves in the near future. However, its latest public offering at discount may send negative signals.

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Disclosure: I/we have no positions in any stocks mentioned, and no plans to initiate any positions within the next 72 hours. I wrote this article myself, and it expresses my own opinions. I am not receiving compensation for it (other than from Seeking Alpha). I have no business relationship with any company whose stock is mentioned in this article.