Moderna Covid Drug Production Deal, And Other News: The Good, Bad And Ugly Of Biopharma

Summary

• Moderna makes deal with Lonza to boost mRNA 1273 production.
• Marker receives orphan drug designation for AML drug.
• Jazz halts defibrotide trial on lack of positive signals.
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Moderna Boosts Production with Lonza Deal

Moderna Therapeutics (NASDAQ:MRNA) announced its new collaboration with Lonza for boosting its production capacity for mRNA 1273. The company had recently struck a deal with the federal government to focus on developing COVID 19 vaccine candidate. The company stands to receive $483 million from BARDA for accelerating the development of the vaccine. Moderna dosed the first patient under its Phase I clinical trial in March.

Under the latest deal with the Swiss firm, Moderna will be able to use Lonza manufacturing facilities in the United States and Switzerland. It is expected that the technology transfer may be started in June while the batch production in the United States may begin in July. Moderna CEO Stéphane Bancel said, “This long-term strategic collaboration agreement will enable Moderna to accelerate, by 10 times, our manufacturing capacity for mRNA-1273 and additional products in Moderna’s large clinical portfolio. Lonza’s global presence and expertise are critical as we scale at unprecedented speed." The agreed longevity of the deal is 10 years and will involve creations of manufacturing suites at various Lonza manufacturing sites.

Moderna also recently announced that it has submitted with the FDA its plan to start Phase 2 and later stage trials of the vaccine candidate. The company has received early feedback for its Phase 2 study design. It is anticipated that the dosing for Phase 2 may start during the second quarter. The company is looking to enroll 600 healthy subjects for assessing the safety and efficacy of the shot with doses administered 28 days apart. Currently, there are a number of pharma companies in the race to produce vaccine for fight against COVID 19.

In response to the latest deal, Lonza chairman and CEO ad interim Albert Baehny said, “The current pandemic illustrates the need to combine the best science with resilient supply chains that can scale. We are fully committed to leveraging our global network and experience in manufacturing technologies to support Moderna’s manufacture of mRNA-1273 as well as collaborating on future Moderna products.”

Moderna’s collaboration with US Biomedical Advanced Research and Development Authority (BARDA) involves development of mRNA 1273, which is an mRNA vaccine that encodes for a prefusion stabilized form of the Spike (NYSE:S) protein. BARDA is a division of the Department of Health and Human Services and its funding will support late stage clinical development programs for the vaccine. In order to scale up, Moderna anticipates to hire up to 150 new employees, including engineers, clinical staff and manufacturing staff.

The company’s new tie up with Lonza is expected to allow the manufacturing of up to a billion doses per year. Lonza is based out of Basel in Switzerland.

Marker Receives Orphan Drug Designation for AML Drug Candidate

Marker Therapeutics (MRKR) announced that the FDA has granted Orphan Drug designation to its acute myeloid leukemia drug candidate MT 401. The orphan drug candidate offers several benefits such as tax credits for qualified clinical trials and 7 years of market exclusivity if the drug candidate is approved. The FDA generally grants this tag to novel biologics and drugs aiming to provide safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the United States.

MT 401 is a multi-tumor associated antigen-specific T cell based product developed for treating AML following allogeneic stem cell transplant. The company CEO and President Peter Hoang said, “In investigator-sponsored trials, our MultiTAA-specific T cell product candidate was well-tolerated and we have observed clinical benefit across various liquid and solid tumors, suggesting the product candidate's ability to induce a patient's own T cells to expand for a more durable anti-tumor effect.” The drug candidate has been given the tag for treating hematological malignancies and solid tumor indications.

Marker Therapeutics reported that it is planning to start its Phase 2 study in subjects with post allegeneic stem cell transplant AML. T cells are obtained from healthy donors and are made to undergo a two-step cell culture process. The process leads to creation of CD4 and CD8 cell mixture which is able to identify multiple tumor specific antigens.

Marker Therapeutics, Inc. is a clinical-stage immuno-oncology company focused on developing next-generation T cell-based immunotherapies for treating hematological malignancies and solid tumor indications. The company’s technology relies upon selective expansion of non-engineered, tumor-specific T cells that recognize tumor associated antigens and kill tumor cells expressing those targets. However, Marker does not genetically engineer its T Cell therapies and therefore its drug candidates are expected to be less expensive to manufacture.

Jazz Pharmaceuticals Draws Blank on Phase 3 Clinical Trial

Jazz Pharmaceuticals Plc (JAZZ) reported that it has halted the enrolment in its Phase 3 clinical study of defibrotide. The company stated that the decision was taken on the recommendation of an Independent Data Monitoring Committee (IDMC) which opined that the study is highly unlikely to produce statistical significance for the primary endpoint of VOD-free survival at Day +30 post-HSCT in the final analysis.

Jazz Pharmaceuticals further stated that the IDMC reached the said recommendation after completing pre-planned interim analysis of the initial 280 patients and where the protocol-specified futility criteria were met. Robert Iannone, M.D., M.S.C.E., executive vice president, research and development of Jazz Pharmaceuticals said, "While enrollment has been discontinued, the trial will run to completion through the follow-up of the more than 370 patients enrolled. VOD is a rapidly progressive and devastating condition that can develop following stem-cell transplantation. As the only approved treatment with demonstrated efficacy based on survival at 100 days after HSCT, defibrotide is an important medicine for this patient population." The company has notified relevant authorities about the decision of discontinuance.

The company stated that the patients already enrolled in the study may be allowed to continue participation if deemed appropriate after further investigation. The relevant Phase 3 was not intended to further assess defibrotide for the treatment of VOD and does not impact the approved indication or other ongoing clinical studies. The Phase 3 study was a randomized, open-label, multi-center trial with an adaptive design. The main aim of the study is to compare the efficacy of defibrotide versus best supportive care in the prevention of hepatic VOD.

Jazz Pharmaceuticals mainly aims to develop medicines for critical diseases with limited treatment options. The main focus of the company is on neuroscience and oncology including movement disorders, sleep medicine and hematologic tumors.

Analyst’s 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.