On April 1, 2021 Celsion Corporation (NASDAQ: CLSN), a clinical-stage company focused on DNA-based immunotherapy and next-generation vaccines, reported that a poster highlighting the Company’s ongoing Phase I/II OVATION 2 Study with GEN-1 in advanced ovarian cancer was presented last week at the Virtual Annual Meeting on Women’s Cancer, sponsored by the Society of Gynecologic Oncology (Press release, Celsion, APR 1, 2021, View Source [SID1234577506]). The poster, titled "A Phase I/II Study Evaluating Intraperitoneal GEN-1 in Combination with Neoadjuvant Chemotherapy [NACT] in Patients with Newly Diagnosed Advanced Epithelial Ovarian Cancer (EOC)," can be viewed here.

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GEN-1 is Celsion’s DNA-mediated interleukin-12 (IL-12) immunotherapy designed using TheraPlas, its proprietary, synthetic, non-viral nanoparticle delivery system platform. The poster was presented by Premal Thaker, MD, Study Chair of the OVATION 2 Study and Professor of Obstetrics and Gynecology, Director of Gynecological Oncology Clinical Research, Division of Gynecologic Oncology, Washington University School of Medicine. Additional authors were R.W. Holloway, L. Kuroki, S. E. DePasquale, W.H. Bradley, A. ElNaggar, M.C. Bell, R.P. Rocconi, A. Bregar, M.D. Indermaur, C. Gunderson, B. Pothuri, R. Agajanian, D. Warshal, D. Provencher, M. McHale, V. John, M. Bergman, S. Lau, L. Musso, K. Anwer, N. Borys and C.A. Leath III.

"Currently advanced ovarian cancer has a low survival rate and a lack of effective therapies. PARP inhibitors have made an important contribution in a subset of patients. For the majority of patients there is hope that an immunotherapy such as GEN-1 will provide a valuable new treatment option to improve both the quality of life and the life expectancy for these women," commented Dr. Thaker. "OVATION 2 Study data now show R0 resections in 14 of 17 patients, or 82%, in the GEN-1 + NACT arm, compared with seven of 12 patients, or 58%, in the NACT alone arm. We view R0 resections as a good predictor of survival, and R0 resections in the GEN-1 + NACT arm are encouraging thus far," Dr. Thaker added.

The poster describes the OVATION 2 Study, which combines GEN-1 with standard-of-care neoadjuvant chemotherapy (NACT) in patients newly diagnosed with Stage III/IV ovarian cancer. NACT is designed to shrink the cancer as much as possible for optimal surgical removal after three cycles of chemotherapy. Following NACT, patients undergo interval debulking surgery, followed by three adjuvant cycles of chemotherapy and up to nine additional weekly GEN-1 treatments, the goal of which is to delay progression and improve overall survival. The OVATION 2 Study is an open-label, 1-to-1 randomized trial, 80% powered to show the equivalent of a 33% improvement in progression-free survival (PFS) (HR=0.75), the primary endpoint, when comparing the treatment arm (standard of care + GEN-1) with the control arm (standard of care alone).

The Company recently announced that it has enrolled approximately 40% of the anticipated 110 patients to be enrolled into the OVATION 2 Study. To date, 29 patients have had their interval debulking surgery with the following results:

14 of 17, or 82%, of patients treated with GEN-1 had a R0 resection, which indicates a microscopically margin-negative complete resection in which no gross or microscopic tumor remains in the tumor bed.
7 of 12 patients, or 58%, of patients in the control arm had an R0 resection.
This interim data represents a 41% improvement in R0 resection rates for GEN-1- patients compared with control arm patients and is consistent with the reported improvement in resection scores noted in the encouraging Phase I OVATION 1 Study, the manuscript of which has been submitted for publication in a peer-reviewed journal.
Celsion also recently announced that GEN-1 had received Fast Track designation from the U.S. Food and Drug Administration (FDA). This designation is intended to facilitate the development and expedite the regulatory review of drugs to treat serious conditions and fill an unmet medical need.

About GEN-1 Immunotherapy

GEN-1, designed using Celsion’s proprietary TheraPlas platform technology, is an IL-12 DNA plasmid vector encased in a nanoparticle delivery system, which enables cell transfection followed by persistent, local secretion of the IL-12 protein. IL-12 is one of the most active cytokines for the induction of potent anti-cancer immunity acting through the induction of T-lymphocyte and natural killer (NK) cell proliferation. The Company has previously reported positive safety and encouraging Phase I results with GEN-1 given as monotherapy or a combination therapy in patients with advanced peritoneally metastasized primary or recurrent ovarian cancer, and recently completed a Phase Ib dose-escalation trial (OVATION 1 Study) of GEN-1 in combination with carboplatin and paclitaxel in patients with newly diagnosed ovarian cancer.

About the Virtual Annual Meeting on Women’s Cancer

The Society of Gynecologic Oncology (SGO) 2021 Annual Meeting on Women’s Cancer will be a fully virtual meeting, allowing participants to access high-quality content and engage remotely from around the world in a platform that will be designed explicitly for this meeting. SGO is working hard to provide a great virtual experience with the latest gynecologic cancer research and education you have come to expect at the Annual Meeting on Women’s Cancer. All education sessions will be recorded and available through the virtual platform for attendees who register for the meeting by March 25, 2021.

On April 1, 2021 Enveric Biosciences, Inc. (NASDAQ: ENVB) ("Enveric" or the "Company"), a patient-first biotechnology company developing novel cannabinoid (CBD)_medicines to improve quality of life for cancer patients, reported its financial results for the year ended December 31, 2020 and provided shareholders with an update on its accomplishments in 2021 thus far (Press release, Enveric Biosciences, APR 1, 2021, View Source [SID1234577523]).

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David Johnson, Chairman and Chief Executive Officer, said, "Since the closing of our going public transaction in late December 2020, our team has achieved several critical milestones that have positioned our Company to accelerate the execution of our vision to extend and enhance the quality of life for cancer patients in need through researching and developing novel supportive care therapies."

Mr. Johnson continued, "Our ability to strengthen our balance sheet through the closing of approximately $22.8 million in gross proceeds during the first quarter of 2021 has allowed us the opportunity to not only accelerate the research and development of our cannabinoid-based therapies, but also the optionality to evaluate a robust pipeline of strategic asset acquisitions and partnerships. During the first quarter of 2021, we launched a development collaboration and supply agreement with PureForm and shortly thereafter acquired the exclusive license to five molecules focused on pain and dermatology indications. Our team remains focused on advancing several pivotal studies throughout the remainder of 2021 in the large, unmet, supportive care market for cancer."

Corporate Updates:

Strengthened balance sheet with the closing of two registered direct offering totaling $22.8 million in gross proceeds from the closing of $10 million on January 14, 2021 and $12.8 million on February 11, 2021.

Acquired an exclusive, perpetual license from Diverse Biotech for five molecules, four of which are dermatology-focused and one that is pain-focused. As part of the agreement, Enveric will gain access to scientists and formulators to help with the research and development of these assets through pre-clinical and clinical studies to alleviate certain side effects resulting from cancer treatment.

Launched development collaboration and exclusive supply agreement with PureForm Global to support cannabinoid clinical programs aimed to treat pain and inflammation resulting from cancer treatments initially targeting supportive care indications that include radiodermatitis, chemotherapy-induced neuropathy, and glioblastoma.

Assembled a talented, world-class Executive Leadership Team, Board of Directors and Scientific Advisory Board with experience having held positions at Bristol Myers Squibb, Pfizer, Merck, Abbott, Baxter and other global healthcare and biotechnology companies. Collectively, the team has successfully led multiple therapies throughout the entire regulatory process, with substantial expertise in product development, dermatology, wound healing, oncology, intellectual property, and capital markets.
Milestones for the Remainder of 2021:

Glioblastoma Multiforme (GBM)

Q3 ’21 –We intend to seek approval from Israeli Ministry of Health (MOH), Center for Cannabis, to move forward with a Phase I/II trial
Q4 ’21 – We intend to begin enrollment of Phase I/II trial, an open label evaluation of temozolomide with clomiphene and CBD in GBM
Radiation Dermatitis

Q3 ’21 – We intend to an investigational new drug application
Q4 ’21 – We intend to initiate a Phase I/II Trial
Financial Results for the Year Ended December 31, 2020:

Net cash used in operating activities was $3,888,785 during the year ended December 31, 2020, which consisted primarily of a net loss of $6,864,676, offset by amortization of note discount of $288,631, stock-based compensation of $1,977,155, induced conversion of warrants of $802,109, amortization of intangible assets of $120,872, increases in prepaid expenses and other current assets for $636,497, and increases in accounts payable and accrued liabilities of $267,002.

Enveric’s operating expenses increased to $5,617,317, for the year ended December 31, 2020 from $2,296,534 for the year ended December 31, 2019, for an increase of $3,320,783, or 145%. This change was primarily driven by an increase in general and administrative fees of $3,146,700 and an increase in research and development costs of $174,083.

Net cash provided by financing activities was $5,531,270 during the year ended December 31, 2020. Cash as of December 31, 2020 totaled $1,578,460 and the Company currently has no debt.

Subsequent to December 31, 2020, the Company completed two registered direct offerings for gross proceeds of $22.8 million. On March 10, 2021, the Company also received $3,267,245 from the exercise of warrants to purchase 851,099 shares of common stock.

As of March 29, 2021, the Company had 19,450,507 shares of common stock outstanding.

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."