On April 1, 2021 Autolus Therapeutics plc (Nasdaq: AUTL), a clinical-stage biopharmaceutical company developing next-generation programmed T cell therapies, reported that it has received PRIority MEdicines (PRIME) designation from the European Medicines Agency (EMA) for AUTO1, the company’s CAR T cell therapy being investigated in the ongoing FELIX Phase 1b/2 study in relapsed / refractory (r/r) adult B-Acute Lymphocytic Leukemia (ALL) (Press release, Autolus, APR 1, 2021, View Source [SID1234577507]).

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"We are pleased to have received PRIME designation for AUTO1 as it will accelerate the review of a promising therapy targeting unmet medical need," said Dr. Christian Itin, chairman and chief executive officer of Autolus. "The designation comes soon after we presented compelling activity and safety data from the ALLCAR Phase 1 clinical trial at the 62nd American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting. We believe AUTO1 could change standard of care by offering a potentially curative therapy for r/r ALL."

About PRIME
The PRIME program aims to optimize development plans and speed up evaluation of medicines that may offer a major therapeutic advantage over existing treatments or benefit patients without treatment options. The PRIME designation is awarded by the EMA to promising medicines that target an unmet medical need. To be eligible and accepted for PRIME, a medicine has to show its potential to benefit patients with unmet medical needs based on early clinical data coupled with non-clinical data. Through the PRIME program, the EMA offers enhanced support to medicine developers including early interaction and dialogue, and a pathway for accelerated evaluation by the agency. The program is intended to optimize development plans and expedite the review and approval process so that these medicines may reach patients as early as possible.

On April 1, 2021 PTC Therapeutics, Inc. (NASDAQ: PTCT) reported that a $20 million milestone payment was triggered by the first commercial sale of Evrysdi (risdiplam) in the European Union under its License and Collaboration Agreement with Roche (Press release, PTC Therapeutics, APR 1, 2021, View Source [SID1234577524]). Approval for Evrysdi from the European Medicines Agency was received on March 30 for the treatment of spinal muscular atrophy (SMA) in adults and children 2 months and older.

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"We are happy to see the rapid adoption of Evrysdi in the EU which speaks to the need for new treatments for SMA patients," said Stuart W. Peltz, Ph.D., Chief Executive Officer of PTC Therapeutics. "We are delighted that an effective at-home therapy will be available to SMA patients. We recognize that a large proportion of SMA patients in the EU are currently not receiving an approved therapy."

Roche is working closely with reimbursement and assessment bodies in European countries to enable broad and rapid access to SMA patients. Evrysdi is immediately accessible to patients in Germany and will be accessible from early April to patients in France through the cohort Temporary Authorization for Use. Evrysdi has currently been approved in 38 countries and submitted for Health Authority review in a further 33 countries.

Evrysdi is based on PTC science and is commercialized in the United States by Genentech, a member of the Roche Group. Roche led the clinical development of Evrysdi as part of a collaboration with the SMA Foundation and PTC Therapeutics.

About Spinal Muscular Atrophy (SMA)
Spinal muscular atrophy (SMA) is a severe, progressive neuromuscular disease that can be fatal. It affects approximately 1 in 10,000 babies and when untreated is the leading genetic cause of infant mortality. SMA is caused by a mutation of the survival motor neuron 1 (SMN1) gene, which leads to a deficiency of SMN protein. This protein is found throughout the body and is essential to the function of nerves that control muscles and movement. Without it, nerve cells cannot function correctly, leading to progressive muscle weakness over time. Depending on the type of SMA, an individual’s physical strength and their ability to walk, eat or breathe can be significantly diminished or lost.

About Evrysdi (risdiplam)
Evrysdi (risdiplam) is a survival motor neuron 2 (SMN2)-directed RNA splicing modifier designed to treat spinal muscular atrophy (SMA) caused by mutations in chromosome 5q that lead to SMN protein deficiency. Evrysdi is designed to distribute evenly to all parts of the body, including the central nervous system (CNS). Evrysdi is administered daily at home in liquid form by mouth or feeding tube. The U.S. Food and Drug Administration approved Evrysdi for the treatment of SMA for adults and children 2 months and older on August 7, 2020 and the European Medicines Agency approved Evrysdi on March 30 for the treatment of 5q SMA in patients two months of age and older, with a clinical diagnosis of SMA Type 1, Type 2 or Type 3 or with one to four SMN2 copies. Evrysdi is marketed in the United States by Genentech, a member of the Roche Group.

About the Evrysdi (risdiplam) Clinical Studies

FIREFISH (NCT02913482) is an open-label, two-part pivotal clinical trial in infants with Type 1 SMA. Part 1 was a dose-escalation study in 21 infants with the primary objective of assessing the safety profile of risdiplam in infants and determining the dose for Part 2. Part 2 is a pivotal, single-arm study of risdiplam in 41 infants with Type 1 SMA treated for two years followed by an open-label extension. The primary objective of Part 2 was to assess efficacy as measured by the proportion of infants sitting without support after 12 months of treatment, as assessed in the Gross Motor Scale of the Bayley Scales of Infant and Toddler Development – Third Edition (BSID-III) (defined as sitting without support for five seconds). The study met its primary endpoint.

SUNFISH (NCT02908685) is a two part, double-blind, placebo controlled pivotal study in people aged 2 to 25 years with Types 2 or 3 SMA. Part 1 (n=51) determined the dose for the confirmatory Part 2. Part 2 (n=180) evaluated motor function using the Motor Function Measure 32 (MFM-32) scale at 12 months. MFM-32 is a validated scale used to evaluate fine and gross motor function in people with neurological disorders, including SMA. The study met its primary endpoint.

Clinical Trial Safety Data
The safety profile of Evrysdi was established across FIREFISH and SUNFISH pivotal trials. The most common adverse reactions in later-onset SMA (incidence of at least 10 percent of patients treated with Evrysdi and more frequently than control) were fever, diarrhea, and rash. The most common adverse reactions in infantile-onset SMA were similar to those observed in later-onset SMA patients. Additionally, the most common adverse reactions (incidence of at least 10 percent) were upper respiratory tract infection, pneumonia, constipation, and vomiting.

In addition to FIREFISH and SUNFISH, Evrysdi is being evaluated in a broad range of people with SMA, including in:

JEWELFISH (NCT03032172) is an open-label exploratory trial designed to assess the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) in people with SMA aged 6 months to 60 years who received other investigational or approved SMA therapies for at least 90 days prior to receiving Evrysdi. The study has completed recruitment (n=174).

RAINBOWFISH (NCT03779334) is an open-label, single-arm, multi-center study, investigating the efficacy, safety, pharmacokinetics, and pharmacodynamics of risdiplam in babies (~n=25), from birth to 6 weeks old (at first dose), with genetically diagnosed SMA, who are not yet presenting symptoms. The study is currently recruiting.

On April 1, 2021 Arbutus Biopharma Corporation (NASDAQ: ABUS), X-Chem, Inc. (X-Chem) and Proteros biostructures GmbH (Proteros) reported that they have entered into a discovery research and license agreement focused on the discovery of novel inhibitors targeting the SARS-CoV-2 nsp5 main protease (Mpro) (Press release, X-Chem, APR 1, 2021, View Source [SID1234577492]). The agreement is designed to accelerate the development of pan-coronavirus agents to treat COVID-19 and potential future coronavirus outbreaks.

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This collaboration brings together Arbutus’ expertise in the discovery and development of antiviral agents with X-Chem’s industry leading DNA-encoded library (DEL) technology and Proteros’ protein sciences, biophysics and structural biology capabilities and provides important synergies to potentially identify safe and effective therapies against coronaviruses including SARS-CoV-2. The collaboration will allow for the rapid screening of one of the largest small molecule libraries against Mpro (an essential protein required for the virus to replicate itself) and the use of state-of-the-art structure guided methods to rapidly optimize Mpro inhibitors, which Arbutus could potentially progress to clinical candidates. Financial terms of the transaction were not disclosed.

"It is well accepted that in addition to the availability of vaccines, effective and safe therapies are needed to successfully combat the COVID-19 pandemic and any future coronavirus outbreaks," stated Dr. Michael Sofia, Arbutus’s Chief Scientific Officer. "Arbutus, X-Chem and Proteros have complementary and valuable expertise that makes this collaboration particularly well-suited for small molecule drug discovery targeting coronaviruses. Our goal is to identify unique and differentiated pan-coronavirus assets targeting the main coronavirus protease which, when combined with assets arising from our internal nucleoside program targeting the SARS-CoV-2 nsp12 viral polymerase, could deliver a much-needed all-oral antiviral treatment for SARS-CoV-2 and any potential future coronavirus outbreaks."

"We are delighted of this joint discovery research collaboration with Arbutus and X-Chem, which has the potential to identify unique small molecule treatment options for COVID-19 and other possible coronavirus related respiratory diseases" said Dr. Torsten Neuefeind, Proteros’ CEO. "The complementary strengths of all parties gives us a strong position to potentially inhibit a key enzyme with a central role in the viral life cycle in a specific and effective manner."

"The discovery and development of novel drugs to combat infections caused by coronavirus is an incredibly important and challenging task", added Matt Clark, PhD, Chief Executive Officer of X-Chem. "We are exhilarated to join forces with industry leaders Arbutus and Proteros in this effort and bring our drug discovery expertise to this important area of antiviral research."

On April 1, 2021 PharmaCyte Biotech, Inc. (OTCQB: PMCB), a biotechnology company focused on developing cellular therapies for cancer and diabetes using its signature live-cell encapsulation technology, Cell-in-a-Box, announced today that it has successfully completed the twelve-months product stability testing required by the U.S. Food and Drug Administration (FDA) for its CypCaps final clinical trial product (Press release, PharmaCyte Biotech, APR 1, 2021, View Source [SID1234577508]). This product will be used in the company’s planned clinical trial in locally advanced, inoperable pancreatic cancer upon the FDA lifting the clinical hold on PharmaCyte’s Investigational New Drug Application (IND).

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PharmaCyte’s Chief Executive Officer, Kenneth L. Waggoner, said of the completed twelve-month stability study, "We are pleased that our CypCaps product successfully completed all of the required stability tests and that the product has now shown itself to be stable and active after being stored for 1 year at -80oC. The study will continue in order to determine the maximum shelf life of the CypCaps product. It should be noted that this data is exceptional for a product of this kind."

As reported previously, the ongoing stability study is designed to determine the shelf life of the Cell-in-a-Box encapsulated cell product, CypCaps, stored frozen at -80oC. Upon analysis after 12 months in storage at -80oC, the unfrozen CypCaps product passed all of the specified tests, including cell viability, enzyme activity and cell potency as well as pH, label check, capsule appearance, and integrity. This twelve-month data, as well as all future longer-term time points of the shelf life analyses, such as the next time point to be evaluated after 18-months of storage at -80oC, will be reported to the FDA. This ongoing stability study was initiated prior to the submission of the company’s IND to the FDA, and the information and data will form part of the package of information together with data from additional studies requested by the FDA.

In a survey of European biotech/biomedical companies developing advanced therapeutic medicinal products (i.e., products containing living cells like CypCaps), shelf life issues were reported to be one of the major challenges that such products face (ten Ham et al., 2018, Molecular Therapy: Methods & Clinical Development vol. 11, pp121-130). Laboratory data on non-GMP produced encapsulated cells have suggested that the shelf life of Cell-in-a-Box products stored at -80oC is even longer than 1 year.

To learn more about PharmaCyte’s pancreatic cancer treatment and how it works inside the body to treat locally advanced inoperable pancreatic cancer, we encourage you to watch the company’s documentary video complete with medical animations at: View Source

On April 1, 2021 Inhibikase Therapeutics, Inc. (Nasdaq: IKT), a clinical-stage pharmaceutical company developing therapeutics to modify the course of Parkinson’s disease (PD) and related disorders inside and outside of the brain, reported financial results for the fourth quarter and full year ended December 31, 2020 and highlighted recent developments (Press release, Inhibikase Therapeutics, APR 1, 2021, View Source [SID1234577525]).

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Key Business and Clinical Highlights

Completed Initial Public Offering: In December 2020, Inhibikase successfully completed its initial public offering (IPO) of 1,800,000 shares of common stock at a public offering price of $10.00 per share. The Company received aggregate net proceeds of approximately $14.60 million after deducting offering costs, underwriting discounts and commissions.

Commenced Dosing of Patients in Phase 1 Study of IkT-148009 for the treatment of PD and associated GI Disorders: In February 2021, Inhibikase commenced patient dosing in older and elderly healthy volunteers in a Phase 1 randomized single ascending dose and multiple ascending dose study to determine the safety, tolerability and pharmacokinetics of IkT-148009. IkT-148009 is a novel brain penetrant Abelson tyrosine kinase, or c-Abl, inhibitor intended to be used to modify Parkinson’s disease and its gastrointestinal complications.

Accelerated timelines for completion of the Phase 1 trial and initiation of dosing in PD patients. Early data from this study have led to a reduction in the timelines for completion of the study by 6 or more months, providing the opportunity to obtain regulatory approval to commence dosing of PD patients much earlier than previously anticipated.

Commenced chronic toxicology studies of IkT-148009 to permit long-term dosing in patients. In January, 2021 Inhibikase commenced 3 month and 6 month long-term toxicology studies of IkT-148009 in rats and 3 month and 9 month long-term toxicology studies of IkT-148009 in monkeys as required to obtain regulatory approval for chronic administration of IkT-148009 in patients. These studies are expected to be completed in the fourth quarter of 2021.

Commenced clinical batch manufacturing and pill formulation of IkT-001Pro in preparation for an Investigational New Drug application filing in the second quarter of 2021. In February, 2021 Inhibikase commenced clinical batch manufacturing and final product formulation of IkT-001Pro as a film-coated tablet in preparation for regulatory filing with the Food and Drug Administration to initiate clinical development. This regulatory filing is anticipated to be completed near the end of the second quarter of 2021, with initiation of clinical development 30 days after the filing, subject to FDA agreement and issuance of a Study May Proceed notification.
"2020 was a transformative year for Inhibikase, as we successfully completed our IPO and worked diligently to advance our novel programs to treat neurodegenerative diseases towards the clinic," commented Milton Werner, Ph.D., President and Chief Executive Officer of Inhibikase. "Just recently, we were pleased to announce the advancement of our lead candidate, IkT-148009, into a Phase 1 dose escalation study to evaluate the safety, tolerability and pharmacokinetics in elderly volunteers. Early data from this study has been encouraging and we are shortening the timeline to completion by six or more months, with an expectation to move into early patient studies this year. We believe that IKT-148009 could be a transformative therapy for millions of Parkinson’s patients worldwide. Simultaneously, we have advanced our oncology clinical candidate, IkT-001Pro, into manufacturing and expect to initiate clinical development early in the third quarter. Our highly dedicated team has exceeded all of our internal expectations to move these clinical programs forward for the benefit of patients."

Fourth Quarter and Full Year 2020 Financial Review

Net Loss: Net loss for the fourth quarter ended December 31, 2020, was $1.21 million, or $0.15 per share, compared to a net loss of $0.43 million, or $0.05 per share for the fourth quarter 2019. For the full year ended December 31, 2020, net loss was $2.85 million, or $0.35 per share, compared to a net loss of $5.72 million, or $0.70 per share, for the same period in 2019.

R&D Expenses: Research and development expenses were $0.23 million for the fourth quarter 2020, compared to $0.27 million for the same period in 2019. For the full year 2020, research and development expenses were $0.89 million, compared to $2.55 million for the same period in 2019. The decrease was primarily driven by a decline in grant related research expenditures and non-grant related research.

SG&A Expenses: Selling, general and administrative expenses for the fourth quarter 2020 were $1.14 million compared to $0.39 million for the fourth quarter 2019. For the full year 2020, selling, general and administrative expenses were $2.62 million, compared to $4.27 million for the same period in 2019. The decrease was primarily related to a non-recurring 2019 charge of $1.59 million of deferred IPO costs in connection with the 2018 abandoned IPO effort plus net decrease in all other selling, general and administrative expenses of $0.06 million.

Cash Position: Cash and cash equivalents were $13.95 million as of December 31, 2020.