On April 2, 2020 Vericel Corporation (NASDAQ:VCEL), a leader in advanced therapies for the sports medicine and severe burn care markets, reported preliminary unaudited product revenue growth for the quarter ended March 31, 2020, and provided business and financial updates related to the COVID-19 pandemic (Press release, Vericel, APR 2, 2020, View Source [SID1234556106]).

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Over the past several weeks, Vericel has implemented several measures to safeguard the health and well-being of its employees, their families, and healthcare providers, while continuing to supply its autologous cell therapy products MACI (autologous cultured chondrocytes on porcine collagen membrane) and Epicel (cultured epidermal autografts) to patients with knee cartilage and severe burn injuries. At this time, all Vericel employees not directly involved in the production and delivery of MACI or Epicel are working from home. For production-related teams, the Company has implemented additional measures to protect the health and safety of its workforce. Vericel representatives will also continue to provide field-based support for surgical cases, as needed, in compliance with applicable government mandated business activity restrictions and facility access rules.

"First and foremost, our thoughts are with those affected by the virus and we are especially thankful to all healthcare workers for their critical efforts to support patients during this challenging time," said Nick Colangelo, President and Chief Executive Officer of Vericel. "While our MACI business has been impacted by the restrictions on elective surgical procedures, the fundamentals of our business remain strong. Prior to cancellations that occurred in the last two weeks of the quarter, MACI was on track to exceed revenue growth guidance and we believe that most patients will reschedule cases to the extent possible following this crisis. In addition, we believe that Epicel may be less directly impacted by the pandemic given the critical nature of severe burn injuries. We are implementing a number of initiatives to maintain our near-term and future growth opportunities while supporting patients and reducing non-essential discretionary spending. Given the strength of our financial position and the underlying fundamentals of our business, we believe that the Company is well-positioned to maintain its leadership position in the sports medicine and severe burn care markets."

Preliminary Unaudited First Quarter Results and 2020 Financial Guidance

Preliminary unaudited total revenues for the quarter ended March 31, 2020 increased approximately 21% compared to the first quarter of 2019, with MACI revenue increasing approximately 21% and Epicel revenue increasing approximately 22%. As a result of various national, state and local restrictions on elective surgical procedures related to the COVID-19 pandemic, beginning in the middle of March there was a significant increase in cancellations of scheduled MACI procedures as well as a slowdown in new MACI orders. The number of MACI procedures scheduled to occur in the first quarter that were cancelled between March 15, 2020 and the end of the quarter reduced the volume of MACI implants for the quarter by approximately 9%.

Due to the significant uncertainty regarding the duration and impact of restrictions on elective procedures related to the COVID-19 pandemic, and the fact that the U.S. Biomedical Advanced Research and Development Authority (BARDA) may adjust the emergency stockpile delivery plan for NexoBrid due to shifting priorities related to the pandemic, the Company is withdrawing its previously announced 2020 financial guidance, which was issued on February 25, 2020. At this time, the Company cannot predict the extent or duration of the impact of the COVID-19 outbreak on its financial and operating results. The Company plans to provide additional information, to the extent practicable, during its first quarter earnings call in May.

Financial Position and Business Continuity

The Company started the year in a strong position across multiple dimensions and is taking prudent measures to ensure a rapid return to normal operations when conditions allow. As of March 31, 2020, the Company had approximately $83 million in cash and investments and carries no debt. Moreover, appropriate expense reduction measures have been implemented.

The Company continues to manufacture MACI and Epicel and maintains a significant safety stock of all key raw materials. At this time there is no indication that supply chain interruptions will impact the Company’s ongoing manufacturing operations. The Company also continues to plan for a mid-2020 submission of the NexoBrid Biologics License Application to the FDA. To drive current and future demand, the Company’s 71 MACI and 10 Epicel sales representatives and clinical support specialists are adapting their practices to support physician education initiatives using virtual tools in regions where executive orders or hospital restrictions preclude their physical presence.

On April 2, 2020 VBL Therapeutics (Nasdaq: VBLT) reported that following the positive interim analysis in the OVAL study, its Japanese licensee NanoCarrier Co., Ltd. (TSE Mothers: 4571), intends to extend the ongoing global Phase 3 OVAL clinical trial in ovarian cancer to patients in Japan (Press release, VBL Therapeutics, APR 2, 2020, View Source [SID1234556105]). NanoCarrier will be responsible for all required operations and expenses related to the study in Japan. VBL and NanoCarrier Co. Ltd. signed an exclusive license agreement in 2017 for the development, commercialization, and supply of VB-111 in Japan. There are more than 10,000 women diagnosed with ovarian cancer in Japan with more than 5,000 associated deaths annually. Therefore, opening clinical sites in Japan will enable to expedite the OVAL trial globally as well as the potential registration of the VB-111 in Japan upon positive outcome.

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The Data Safety Monitoring Committee (DSMC) overseeing OVAL recently conducted a preplanned interim analysis and recommended that the study continue. The DSMC made its decision after reviewing un-blinded data and assessing CA-125 response, measured according to the GCIG criteria. The analysis met the interim pre-specified efficacy criterion of an absolute percentage advantage of 10% or higher CA-125 response rate for the VB-111 treatment arm in the first 60 enrolled subjects evaluable for CA-125 analysis.

"We are very pleased that NanoCarrier has chosen to develop VB-111 in ovarian cancer in Japan," said Dror Harats, MD, Chief Executive Officer of VBL Therapeutics. "The extension of the OVAL study into Japan will open up another avenue of patient recruitment and may accelerate commercialization of VB-111 in this important market".

In the previously reported Phase 2 study of VB-111 in platinum resistant ovarian cancer, 58% of the patients treated with VB-111 and paclitaxel demonstrated a CA-125 response. Those patients with a CA-125 response demonstrated a median overall survival of 808 days, versus 351 days for those patients without CA-125 response.

VBLT received an up-front payment of $15 million from NanoCarrier Co. Ltd, at the time the agreement was signed and is entitled to receive more than $100 million in development and commercial milestone payments. If VB-111 is approved, VBL will also receive tiered royalties on net sales in the high-teens.

VBL retains rights to VB-111 in the rest of the world.

About the OVAL study
OVAL is an international Phase 3 randomized pivotal potential registration clinical trial that is comparing a combination of VB-111 and paclitaxel to placebo plus paclitaxel, in patients with platinum-resistant ovarian cancer. The study was designed to enroll approximately 400 patients. Its primary endpoint is overall survival. OVAL is conducted in collaboration with the GOG Foundation, Inc., an independent international non-profit organization with the purpose of promoting excellence in the quality and integrity of clinical and basic scientific research in the field of gynecologic malignancies. For more information, refer to ClinicalTrials.gov identifier NCT03398655.

About VB-111 (ofranergene obadenovec)
VB-111 is a first-in-class, targeted anti-cancer gene-therapy agent that is being developed to treat a wide range of solid tumors. VB-111 is a unique biologic agent that uses a dual mechanism to target solid tumors. Its mechanism combines blockade of tumor vasculature with an anti-tumor immune response. VB-111 is administered as an IV infusion once every 6-8 weeks. It has been observed to be well-tolerated in >300 cancer patients and demonstrated activity signals in an "all comers" Phase 1 trial as well as in three tumor-specific Phase 2 studies. VB-111 has received an Orphan Designation for the treatment of ovarian cancer from the European Commission. VB-111 has also received orphan drug designation in both the US and Europe, and fast track designation in the US for prolongation of survival in patients with rGBM. VB-111 successfully demonstrated proof-of-concept and survival benefit in Phase 2 clinical trials in radioiodine-refractory thyroid cancer and recurrent platinum-resistant ovarian cancer (NCT01711970).

On April 2, 2020 Phio Pharmaceuticals Corp. (Nasdaq: PHIO), a biotechnology company developing the next generation of immuno-oncology therapeutics based on its proprietary self-delivering RNAi (INTASYL) therapeutic platform, reported the closing of its previously announced registered direct offering of 1,713,064 shares of common stock, at a purchase price of $2.335 per share, for gross proceeds of approximately $4.0 million priced at-the-market under Nasdaq rules (Press release, Phio Pharmaceuticals, APR 2, 2020, View Source [SID1234556104]). Phio has also issued to the investors unregistered warrants to purchase up to an aggregate of 1,713,064 shares of common stock.

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H.C. Wainwright & Co. acted as the exclusive placement agent for the offering.

The warrants have an exercise price of $2.21 per share of common stock, are exercisable immediately and will expire five and one-half years following the date of issuance.

The Company intends to use the net proceeds from the offering to fund the development of its immuno-oncology programs, other research and development activities and for general working capital needs.

The shares of common stock (but not the warrants or the shares of common stock underlying the warrants) were offered by the Company pursuant to a "shelf" registration statement on Form S-3 (File No. 333-224031) previously filed with the Securities and Exchange Commission (the "SEC") on March 29, 2018 and declared effective by the SEC on April 6, 2018. A final prospectus supplement and accompanying prospectus relating to the shares of common stock being offered were filed with the SEC. Electronic copies of the final prospectus supplement and accompanying prospectus may be obtained on the SEC’s website at View Source or by contacting H.C. Wainwright & Co., LLC at 430 Park Avenue, 3rd Floor, New York, NY 10022, by phone at (646) 975-6996 or e-mail at [email protected].

The warrants described above were offered in a private placement under Section 4(a)(2) of the Securities Act of 1933, as amended (the "Act"), and Regulation D promulgated thereunder and, along with the shares of common stock underlying the warrants, have not been registered under the Act, or applicable state securities laws. Accordingly, the warrants and underlying shares of common stock may not be offered or sold in the United States except pursuant to an effective registration statement or an applicable exemption from the registration requirements of the Act and such applicable state securities laws.

This press release shall not constitute an offer to sell, or the solicitation of an offer to buy, nor shall there be any sale of these securities in any state or jurisdiction in which such an offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction.

On April 2, 2020 Pfizer Inc. (NYSE: PFE) reported that the European Commission (EC) has approved RUXIENCE (rituximab), a monoclonal antibody (mAb) and biosimilar to MabThera (rituximab), for the treatment of non-Hodgkin’s lymphoma (NHL), chronic lymphocytic leukemia (CLL), rheumatoid arthritis (RA), granulomatosis with polyangiitis (GPA) and microscopic polyangiitis (MPA), and pemphigus vulgaris (PV).1,2,3 (Press release, Pfizer, APR 2, 2020, View Source [SID1234556103])

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"The approval of biosimilars such as RUXIENCE is an important development for the treatment of certain cancers and autoimmune conditions," said Igor Aurer, M.D., Ph.D., Professor of Medicine and Head of Hematology Division, University Hospital Centre Zagreb, Croatia. "It’s a step toward allowing clinicians an additional treatment option which can help improve access for patients in need of this established medicine."

The EC approval is based on a comprehensive data package which demonstrated biosimilarity of RUXIENCE to the reference product. This includes results from the REFLECTIONS B3281006 clinical comparative study, which evaluated the efficacy, safety and immunogenicity, pharmacokinetics and pharmacodynamics of RUXIENCE and found no clinically meaningful differences in safety or efficacy compared to the reference product in patients with CD20-positive, low tumor burden follicular lymphoma.4

"Biosimilars like RUXIENCE exhibit a similar safety and efficacy profile to the originator product and have the potential to improve treatment access while reducing healthcare costs," said Masum Hossain, regional president, Oncology International Developed Markets at Pfizer. "Building on our ongoing commitment to bring biosimilars to market, we look forward to making RUXIENCE available to patients in the EU in the coming months."

Biosimilars have been a significant catalyst for change for the healthcare industry over the last decade, with the potential to help create more sustainable healthcare systems. With more than 10 years of global in-market experience and a portfolio which now includes seven approved biosimilar products in Europe, Pfizer is proud to be a leader and at the forefront of this vital healthcare segment. This approval follows the positive recommendation from the Committee for Medicinal Products for Human Use (CHMP) in January 2020.5 RUXIENCE was recently made available to adult patients in the United States for the treatment of NHL, CLL, GPA and MPA and also launched in Japan in January 2020.

About RUXIENCE (rituximab biosimilar)

RUXIENCE is a monoclonal antibody (mAb) biosimilar to MabThera which works by targeting a protein called CD20, which is present on the surface of B lymphocytes, also known as B cells. When it attaches to CD20, rituximab helps destroy these B cells.

RUXIENCE safety information

Do not use RUXIENCE if you are allergic to rituximab or other proteins which are like rituximab or any of the other ingredients of this medicine, if you have a severe active infection at the moment or if you have a weak immune system.

RUXIENCE should also not be used if you have severe heart failure or severe uncontrolled heart disease and have rheumatoid arthritis, granulomatosis with polyangiitis, microscopic polyangiitis or pemphigus vulgaris.

Before starting treatment with RUXIENCE, talk to your doctor, pharmacist or nurse if:

you have ever had or might now have a hepatitis infection. This is because in a few cases, RUXIENCE could cause hepatitis B to become active again, which can be fatal in very rare cases. Patients who have ever had hepatitis B infection will be carefully checked by their doctor for signs of this infection
you have ever had heart problems (such as angina, palpitations or heart failure) or breathing problems
you are pregnant, think that you might be pregnant or are planning to become pregnant
Tell your doctor, pharmacist or nurse if you are taking, have recently taken or might take any other medicines. This includes medicines obtained without a prescription and herbal medicines. In particular, tell your doctor if:

you are taking medicines for high blood pressure
you have ever taken medicines which affect your immune system – such as chemotherapy or immune-suppressive medicines
If you have been diagnosed with rheumatoid arthritis, granulomatosis with polyangiitis, microscopic polyangiitis or pemphigus vulgaris, also tell your doctor if:

you think you may have an infection, even a mild one like a cold. The cells that are affected by RUXIENCE help to fight infection and you should wait until the infection has passed before you are given RUXIENCE
you had a lot of infections in the past or suffer from severe infections
you think you may need any vaccinations in the near future, including vaccinations needed to travel to other countries. Some vaccines should not be given at the same time as RUXIENCE or in the months after you receive RUXIENCE
If you are not sure, talk to your healthcare professional before you are given RUXIENCE.

Like all medicines, RUXIENCE can cause side effects, although not everybody gets them. Most side effects are mild to moderate, but some may be serious and require treatment. Rarely, some of these reactions have been fatal.

Infusion reactions

RUXIENCE is given by intravenous infusion into your vein. During or within the first 24-hours of the infusion you may develop fever, chills and shivering. Less frequently, some patients may experience pain at the infusion site, blisters, itching, sickness (nausea), tiredness, headache, breathing difficulties, blood pressure raised, wheezing, throat discomfort, tongue or throat swelling, itchy or runny nose, vomiting, flushing or palpitations, heart attack or low number of platelets. If you have heart disease or angina, these reactions might get worse.

Tell the person giving you the infusion immediately if you develop any of these symptoms, as the infusion may need to be slowed down or stopped. You may require additional treatment such as an antihistamine or paracetamol.

Infections

You might get infections more easily during your treatment with RUXIENCE. Tell your doctor immediately if you get signs of an infection including:

fever, cough, sore throat, burning pain when passing urine or feeling weak or generally unwell
memory loss, trouble thinking, difficulty walking or sight loss – these may be due to a very rare, serious brain infection, which has been fatal (Progressive Multifocal Leukoencephalopathy or PML)
If you are being treated for rheumatoid arthritis, granulomatosis with polyangiitis, microscopic polyangiitis or pemphigus vulgaris, you will also find this information in the Patient Alert Card you have been given by your doctor. It is important that you keep this Alert Card and show it to your partner or caregiver. Your doctor will also give you a patient booklet with more detailed information on the risks and symptoms of infections and PML.

Skin reactions

Very rarely, severe blistering skin conditions that can be life-threatening may occur. Redness, often associated with blisters, may appear on the skin or on mucous membranes, such as inside the mouth, the genital areas or the eyelids, and fever may be present. Tell your doctor immediately if you experience any of these symptoms.

Other very common side effects of RUXIENCE treatment include:

If you are being treated for non-Hodgkin’s lymphoma or chronic lymphocytic leukemia:

bacterial or viral infections, bronchitis
low number of white blood cells, with or without fever or blood cells called "platelets"
feeling sick (nausea)
bald spots on the scalp, chills, headache
lower immunity – because of lower levels of antibodies called "immunoglobulins" (IgG) in the blood which help protect against infection
If you are being treated for rheumatoid arthritis:

infections such as pneumonia (bacterial)
pain on passing water (urinary tract infection)
allergic reactions that are most likely to occur during an infusion, but can occur up-to 24-hours after infusion
changes in blood pressure, nausea, rash, fever, feeling itchy, runny or blocked nose and sneezing, shaking, rapid heartbeat, and tiredness
headache
changes in laboratory tests carried out by your doctor. These include a decrease in the amount of some specific proteins in the blood (immunoglobulins) which help protect against infection
If you are being treated for granulomatosis with polyangiitis or microscopic polyangiitis:

infections, such as chest infections, pain on passing water (urinary tract infections), colds and herpes infections
allergic reactions that are most likely to occur during an infusion, but can occur up-to 24-hours after infusion
diarrhea
coughing or shortness of breath
nose bleeds
raised blood pressure
painful joints or back
muscle twitches or shakiness
feeling dizzy
tremors (shakiness, often in the hands)
difficulty sleeping (insomnia)
swelling of the hands or ankles
If you are being treated for pemphigus vulgaris:

allergic reactions that are most likely to occur during an infusion, but can occur up to 24 hours after infusion
long lasting depression
loss of hair
RUXIENCE may also cause changes in laboratory tests carried out by your doctor. If you are having RUXIENCE with other medicines, some of the side effects you may get may be due to the other medicines.

Please refer to the European Summary of Product Characteristics for RUXIENCE for complete safety information.

On April 2, 2020 Oncolytics Biotech Inc. (NASDAQ: ONCY) (TSX: ONC), currently developing pelareorep, an intravenously delivered immuno-oncolytic virus, reported positive clinical data published in a peer-reviewed journal highlighting that the combination of FOLFIRI, bevacizumab and pelareorep was well tolerated, with promising efficacy signals in colorectal cancer patients with KRAS mutated tumors (Press release, Oncolytics Biotech, APR 2, 2020, View Source [SID1234556102]). The article, entitled "Elucidation of Pelareorep Pharmacodynamics in a Phase I Trial in Patients with KRAS Mutated Colorectal Cancer," authored by Dr. Sanjay Goel, Department of Medical Oncology, Montefiore Medical Center, Albert Einstein College of Medicine, Bronx, NY, et al., was published on March 10, 2020, in Molecular Cancer Therapeutics.

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The study enrolled 36 patients with KRAS mutation in a dose-escalation trial, of which 30 patients were assessable for response. The combination of FOLFIRI, bevacizumab and pelareorep was well tolerated, with promising signals of efficacy. Six patients received the recommended phase 2 dose (RP2D), at which a 50% overall response rate and a median overall survival (OS) of 25.1 months were observed, which compares favorably to the historical OS of 13.5 months (an 86% improvement). Among 30 evaluable patients, 6 (20%) had a partial response (PR) and 22 patients (73.3%) had stable disease (SD) as their best response, for a clinical benefit rate (PR +SD) of 93.3%.

Enhanced efficacy elicited by the administration of pelareorep was supported by evidence of an adaptive immune response occurring after each cycle of pelareorep treatment. Rapid maturation of dendritic cells was observed at 48 hours, from a baseline mean of 4.5% to a mean of 18.6% (4.1 fold change, p=0.000016), followed by an increase in absolute CD8 (2.4 fold change, p=0.00015) and CD4 (3.5 fold change, p=0.00015), on day 4. The most important observation was the activation of CD8 cells (CD8+ CD70+) on day 8, from a baseline mean of 1.5% to a mean of 18.8% (12.9 fold change, p=0.0009). These dramatic immune responses were only seen after pelareorep administration and not with the other medications alone, strongly suggesting that pelareorep is influencing these responses. In addition, on-treatment tumor biopsies revealed replicating virus (pelareorep), thereby demonstrating successful and efficient intravenous (systemic) delivery.

"Pelareorep combined with FOLFIRI, bevacizumab triggers a robust adaptive immune response, highlighting a unique pattern of dendritic cell maturation followed by CD8 T cell activation that was observed after every dose of pelareorep," said Dr. Rita Laeufle, Chief Medical Officer at Oncolytics Biotech. "Importantly, we have received great interest from key opinion leaders who are eager to work with pelareorep in gastrointestinal cancers, and this paper supports that interest. Our biomarkers of T cell clonality and CEACAM6, along with these promising efficacy findings, have allowed us to craft a clear clinical strategy in gastrointestinal cancers, and we look forward to providing updates on those plans in the near future."

Molecular Cancer Therapeutics, a monthly medical journal published by the American Association for Cancer Research (AACR) (Free AACR Whitepaper), Inc. (AACR) (Free AACR Whitepaper), strives to be the top choice for publishing the best science in the discovery and preclinical development of novel therapeutic agents for oncology. A copy of the paper can be found on our website: View Source

About Pelareorep

Pelareorep is a non-pathogenic, proprietary isolate of the unmodified reovirus: a first-in-class intravenously delivered immuno-oncolytic virus for the treatment of solid tumors and hematological malignancies. The compound induces selective tumor lysis and promotes an inflamed tumor phenotype through innate and adaptive immune responses to treat a variety of cancers and has been demonstrated to be able to escape neutralizing antibodies found in patients.