Plus Therapeutics Announces Key RNL™ Development and cGMP Drug Manufacturing Collaboration Agreements

On May 4, 2021 Plus Therapeutics, Inc. (Nasdaq: PSTV) (the "Company"), a clinical-stage pharmaceutical company developing novel, targeted therapies for rare and difficult to treat cancers, reported two collaboration agreements to support its process development and analytical chemistry activities for the cGMP manufacturing of Rhenium NanoLiposome (RNL), the Company’s lead investigational asset in clinical development for recurrent glioblastoma (Press release, Cytori Therapeutics, MAY 4, 2021, View Source [SID1234579073]).

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Plus Therapeutics signed a pre-clinical, clinical, and process development agreement with Invicro LLC (Invicro), a Konica Minolta company, a global provider of imaging biomarkers, core lab services, advanced analytics and software solutions for drug discovery and development with best-in-class expertise in radiochemistry. Under this agreement, Invicro will characterize the current manufacturing process and develop in-process manufacturing controls for the RNL active pharmaceutical ingredient (API) and final drug product and provide future clinical trial imaging support and drug development consulting.

In addition, Plus Therapeutics entered into an agreement with Eurofins BioPharma Inc. ("Eurofins"), a market leader in analytical chemistry for discovery pharmacology and advanced materials sciences. Eurofins will develop and validate test methods for purity, composition, and identity of Re-BMEDA, the API in RNL. These test methods will support release testing and compliance with cGMP requirements for new drug substances.

"Process optimization and appropriate quality controls of investigational compounds are very critical aspects in bringing novel drugs to markets," said Marc H. Hedrick M.D., President and Chief Executive Officer of Plus Therapeutics. "We have identified two best-in-class partners in Invicro and Eurofins to help us get one step further in bringing RNL to a registrational clinical trial and ultimately commercial supply."

As previously disclosed, Plus Therapeutics entered into a master services agreement with Piramal Pharma Solutions for the development, manufacture, and supply of RNL intermediate of the drug product.

Invitae Reports $103.6 Million in Revenue Driven by 259,000 in Billable Volume in First Quarter of 2021

On May 4, 2021 Invitae Corporation (NYSE: NVTA), a leading medical genetics company, reported financial and operating results for the first quarter ended March 31, 2021 signaling continued momentum into 2021 (Press release, Invitae, MAY 4, 2021, View Source [SID1234579099]).

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Invitae’s (NVTA) mission is to bring comprehensive genetic information into mainstream medical practice to improve the quality of healthcare for billions of people. www.invitae.com (PRNewsFoto/Invitae Corporation)

"We had a very strong start to the year, experiencing record daily volumes, and we expect that momentum to continue into the coming years," said Sean George, co-founder and chief executive officer of Invitae. "Genetic information is the foundation for personalized medicine, and we believe Invitae is uniquely positioned to deliver that information from a single platform across all stages of life, ushering personalized medicine into the mainstream to improve healthcare for all."

First Quarter 2021 Financial Results

Generated revenue of $103.6 million in the quarter, a 61% increase compared to $64.2 million in the same period in 2020
Reported billable volume of 259,000 in the quarter, a 72% increase compared to 151,000 in the same period in 2020
Reported average cost per billable unit of $290 in the quarter compared to $268 average cost per billable unit in the same period in 2020. Non-GAAP average cost per unit was $242 in the quarter
Achieved gross profit for the first quarter of 2021 of $28.1 million, compared to $23.8 million in the same period in 2020. Non-GAAP gross profit was $40.5 million in the first quarter
Total operating expense, which excludes cost of revenue, for the first quarter of 2021 was $140.5 million compared to $121.6 million in the same period in 2020. Non-GAAP operating expenses, which excludes cost of revenue, for the quarter was $155.4 million.

Net loss for the first quarter of 2021 was $109.5 million, or a $0.56 net loss per share, compared to a net loss of $98.5 million in the first quarter of 2020, or a $0.99 net loss per share. Non-GAAP net loss for the quarter was $122.2 million, or a $0.63 non-GAAP net loss per share.

At March 31, 2021, cash, cash equivalents, restricted cash and marketable securities totaled $681.9 million as compared with $360.7 million as of December 31, 2020. Net increase in cash, cash equivalents and restricted cash for the quarter was $73.0 million. Non-GAAP cash burn was $112.3 million for the quarter. Cash burn for the quarter would have been $94.6 million excluding the cash paid for acquisitions, primarily related to the cash paid to acquire One Codex.

In January, the company announced the sale of approximately 8.9 million shares of its common stock at a price of $51.50 per share, resulting in net proceeds, after underwriting discounts and commissions and offering expenses, of $434.3 million. Subsequent to the close of the first quarter, on April 1, 2021, the company announced that a small group of investors, led by SB Management, a subsidiary of Softbank Group Corp., made an investment of $1.2 billion in convertible senior notes to support the company’s future growth initiatives.

Corporate and Scientific Highlights

In April, the company announced plans to open a new laboratory and production facility near Research Triangle Park in North Carolina to further expand its capacity to strengthen our operations and help us offer industry-leading turnaround times – including for customers across the Atlantic and in South America.
Made two additions to our platform:
Acquired genomics company Genosity to support the speed, efficiency and flexibility needed for mainstream global adoption of personalized cancer care and monitoring to help transform how cancer is diagnosed, treated and monitored; and
Added One Codex’s microbiome and infectious disease testing capabilities to offer a high-quality, low-cost, end-to-end metagenomics product (sequencing and results) and enable the development of future offerings in infectious disease, preterm birth and wellness.
Joined The eMERGE Network, a nationwide effort organized and funded by the National Human Genome Research Institute, connecting genetic data and electronic medical record (EMR) systems to advance the use of genetics in healthcare. Invitae is providing genetic testing for a study of up to 25,000 people to evaluate the impact of population screening, especially on underserved populations.
Issued Invitae’s inaugural Environmental, Social and Governance (ESG) report, highlighting the company’s commitment and increased focus on sustainability issues and in recognition of their impact on the business and patient health.
Presented additional results at the 2021 American College of Medical Genetics and Genetics (ACMG) Annual Clinical Genetics Meeting from a landmark study conducted in collaboration with Mayo Clinic. The study evaluating colorectal cancer patients found one in six people harbor genetic changes that are known to substantially increase the risk for cancer, especially in patients who were younger when diagnosed.
Added digital health AI company Medneon’s risk assessment tools to our education and clinical support offerings to further support cancer patients by making it easier for clinicians to determine who should get testing and how to use genetic information to individualize treatment.
Launched a project with Bristol Myers Squibb, Janssen Research & Development, LLC (Janssen), Novartis and Genentech, a member of the Roche Group, to develop a standardized panel for Molecular Residual Disease (MRD) detection in patients with Acute Myeloid Leukemia (AML) to support clinical trial testing across multiple drug development programs in order to better establish the clinical utility of MRD as a biomarker in AML.
Expanded the capabilities of Invitae’s advanced clinical chatbot Gia to enable clinician use of Gia to guide their patients in an intuitive, enjoyable and telemedicine-friendly conversation through receiving and understanding genetic test results and possible next steps, including scheduling a call with a genetic counselor.
Signed 25 biopharma partnership deals in the quarter, including the introduction of a new sponsored testing program to provide no-charge genetic testing to individuals at risk for or suspected of having the most common adult Neurodegenerative conditions, including Parkinson’s disease, amyotrophic lateral sclerosis and early-onset Alzheimer’s disease, in the United States, Canada, Australia and Brazil.
Webcast and Conference Call Details
Management will host a conference call and webcast today at 4:30 p.m. Eastern / 1:30 p.m. Pacific to discuss financial results and recent developments. To access the conference call and webcast, please register at the link below:

View Source

Upon registering, each participant will be provided with call details and a registrant ID. Reminders will also be sent to registered participants via email.

The live webcast of the call and slide deck, may be accessed here or by visiting the investors section of the company’s website at ir.invitae.com. A replay of the webcast and conference call will be available shortly after the conclusion of the call and will be archived on the company’s website.

VBL Therapeutics to Report First Quarter Financial Results on May 11

On May 4, 2021 VBL Therapeutics (Nasdaq: VBLT) reported that it will release its first quarter financial results for the period ended March 31, 2021 on Tuesday, May 11, 2021 before market open (Press release, VBL Therapeutics, MAY 4, 2021, View Source [SID1234579115]). Professor Dror Harats, M.D, Chief Executive Officer and Amos Ron, Chief Financial Officer, will host a conference call at 8:30am EDT the same day to discuss the results and provide a corporate update.

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Protagonist Reports First Quarter 2021 Financial Results and Provides Corporate Update

On May 4, 2021 Protagonist Therapeutics (Nasdaq: PTGX) ("Protagonist" or the "Company") reported financial results for the first quarter of 2021 ended March 31, 2021 and provided a corporate update (Press release, Protagonist, MAY 4, 2021, View Source [SID1234579132]).

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"Our progress this quarter affirms the strength of our pipeline and execution capabilities," said Dinesh Patel, PhD, Protagonist’s President and Chief Executive Officer. "For our lead drug candidate, rusfertide in polycythemia vera, we have completed enrollment for our Phase 2 study and confirmed the registrational path forward based on recent engagement with both U.S. and European regulators. We look forward to providing an interim update from our ongoing Phase 2 study at a major medical conference in the second quarter of 2021."

Dr. Patel continued, "We are very pleased with the rate of enrollment in our ongoing Phase 2 study of the oral alpha-4-beta-7-integrin antagonist PN-943. For our oral IL-23 receptor antagonists, we expect to dose the first subject in the Phase 1 study of PN-232 in the second quarter of 2021 and intend to complete both this trial and the Phase 1 trial of PN-235 during the second half of 2021. There is excellent movement forward across our full portfolio, driven by our commitment to patients in need of new and better therapeutic options."

PRODUCT DEVELOPMENT AND CORPORATE UPDATE

Disorders of Red Blood Cells and Iron Regulation

Rusfertide (PTG-300)
Investigational, injectable, hepcidin mimetic discovered through our peptide technology platform. Hepcidin is a natural hormone that regulates iron homeostasis and controls the absorption, storage, and distribution of iron in the body. Rusfertide is currently being evaluated for disorders associated with iron overload and excessive erythrocytosis (red blood cell production).

In March 2021, following an end-of-Phase 2 meeting with the U.S. Food and Drug Administration ("FDA") and written comments from the European Medicines Agency ("EMA"), Protagonist has advanced its preparations for a global pivotal study of rusfertide in polycythemia vera ("PV"). The Company plans to initiate this Phase 3 study in early 2022.
In April 2021, Protagonist announced completion of enrollment in the ongoing Phase 2 study of rusfertide in PV. The Company plans to present updated interim data from this study at an upcoming medical meeting in the second quarter of 2021.
In the second half of 2021, Protagonist expects to announce preliminary results from the ongoing Phase 2 open-label proof-of-concept study of rusfertide in patients with hereditary hemochromatosis ("HH").
In addition to ongoing studies in PV and HH, the Company expects to select a third indication for rusfertide in 2021.
Inflammatory Bowel Diseases

PN-943
Investigational, orally delivered, gut-restricted alpha-4-beta-7 specific integrin antagonist for inflammatory bowel diseases.

The 150-patient Phase 2 "IDEAL" study evaluating the safety, tolerability and efficacy of PN-943 in patients with moderate to severe ulcerative colitis is underway, and the study is on track for completion in 2022.
Oral IL-23 Receptor Antagonists

PTG-200; PN-235; PN-232 (Janssen collaboration)
Investigational, orally delivered, IL-23 receptor antagonists. Protagonist has entered into a worldwide agreement with Janssen to co-develop and commercialize these drug candidates.

In the second quarter of 2021, Protagonist expects to dose the first subject in the Phase 1 study of PN-232. The Company expects to complete both this trial and the Phase 1 trial of PN-235 in the second half of 2021. PN-232 and PN-235 are part of the oral IL-23 pathway blocker portfolio strategy, pursued in collaboration with Janssen.
Enrollment continues for the Phase 2a proof-of-concept PRISM study of PTG-200, a first-generation drug candidate for patients with moderate to severe Crohn’s disease.
First Quarter 2021 Financial Results

Cash, Cash Equivalents and Marketable Securities: Cash, cash equivalents and marketable securities as of March 31, 2021 were $279.7 million. The Company expects current cash, cash equivalents and marketable securities to be sufficient to fund its planned operating and capital expenditures through the first half of 2024.
License and Collaboration Revenue: License and collaboration revenue was $6.2 million for the first quarter of 2021 compared to $3.6 million for the same period of 2020. The increase was primarily due to the additional services provided to Janssen under the collaboration agreement during 2021 related to PN-232 and PN-235.
Research and Development ("R&D") Expenses: R&D expenses for the first quarter 2021 were $24.2 million as compared to $18.8 million for the same period of 2020. The increase was primarily due to costs associated with advancing our clinical trials with our pipeline assets of rusfertide and PN-943, as well as our three IL-23 receptor antagonist assets under the Janssen collaboration (PTG-200, PN-235 and PN-232).
General and Administrative ("G&A") Expenses: G&A expenses for the first quarter 2021 were $6.0 million as compared to $4.6 million for the same periods of 2020. The increase was primarily related to professional fees and employee compensation related expenses supporting the growth in our operations.
Net Loss: The first quarter net loss was $24.0 million, or a net loss of $0.54 per share, compared to the first quarter of 2020 net loss of $20.1 million, or a net loss of $0.72 per share.

VAXIMM Announces Participation at Scientific and Industry Events in May

On May 4, 2021 VAXIMM AG, a Swiss/German biotech company focused on developing an oral plug and play DNA vaccination technology to stimulate patients’ cytotoxic T-cells targeting a wide range of cancer-related antigens, reported that the Company will participate in several scientific and industry events this month (Press release, Vaximm, MAY 4, 2021, View Source [SID1234579171]).

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Dr. Heinz Lubenau, Chief Executive Officer of VAXIMM, and the management team will attend the following conferences:

18th CIMT (Free CIMT Whitepaper) Annual Meeting
May 10-12, 2021, Virtual Event
– To schedule a meeting with the Company, please contact [email protected].

21st Bio€quity Europe
May 17-19, 2021, Virtual Event
– Company presentation.
– To request a meeting, please sign up through the event’s partneringONE meeting system.

SACHS FORUM: 7th Annual Immuno-Oncology Innovation Forum
May 18-20, 2021, Virtual Event
– Company presentation.
– Panel discussion "Latest Advances in Cell & Gene Therapies.", 6:50 pm CEST, 19 May.
– To schedule a meeting, please sign up in the event’s online meeting system.

17th Annual Anglonordic Life Science Conference
May 25-26, 2021, Virtual Event
– Company presentation during session, "Biotech Investment and Technology Investment," 12 pm CEST, 26 May.
– To schedule a meeting, please sign up in the online meeting system.

For updates, please see the Events section of the Company’s website.