On June 1, 2020 Protalix BioTherapeutics, Inc. (NYSE American: PLX) (TASE: PLX), a biopharmaceutical company focused on the development, production and commercialization of recombinant therapeutic proteins produced by its proprietary ProCellEx plant cell-based protein expression system, reported financial results for the first quarter ended March 31, 2020, and provided a business update on recent corporate and clinical developments (Press release, Protalix, JUN 1, 2020, View Source [SID1234560744]). The Company’s management will discuss the financial results and provide a clinical, corporate and financial highlights on a conference call and live webcast scheduled for Monday, June 1, 2020 at 8:30 am Eastern Daylight Time (EDT).

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"The first quarter of 2020 has most certainly been transformational for Protalix, despite the COVID-19 pandemic that affected the global markets," said Dror Bashan, Protalix’s President and Chief Executive Officer. "I am proud to say that despite the pandemic, Protalix was able to keep the company running smoothly and adapt quickly to the changing environment."

"During the quarter, we were able to close a $43.7 million private placement," he continued. "Furthermore, the topline results from the completion of our Phase III BRIDGE study and the subsequent BLA submission for PRX–102 announced in May prove that Protalix has actually gained momentum by leaning into this unprecedented challenge. I am convinced now more than ever that our team is positioned for long-term success and look forward to continuing our momentum through the rest of this year and into 2021."

Conference Call and Webcast Information

The Company will host a conference call on Monday, June 1, 2020, at 8:30 am, Eastern Daylight Time, to review the clinical, corporate and financial highlights. To participate in the conference call, please dial the following numbers prior to the start of the call:

The conference call will also be broadcast live and available for replay for two weeks on the Company’s website, www.protalix.com, in the Events Calendar of the Investors section. Please access the Company’s website at least 15 minutes ahead of the conference to register, download, and install any necessary audio software.

First Quarter 2020 and Recent Business Highlights

Clinical and Regulatory Advancements

On May 28, 2020, the Company and its development and collaboration partner, Chiesi Global Rare Diseases, a unit of Chiesi Farmaceutici S.p.A., or Chiesi, announced the submission on May 27, 2020 of a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for pegunigalsidase alfa, or PRX–102, for the treatment of adult patients with Fabry disease via the FDA’s Accelerated Approval pathway. PRX–102 was granted Fast Track designation by the FDA in January 2018. Upon the BLA approval, if approved, the Company will be eligible to receive a milestone payment from Chiesi.
On May 11, 2020, the Company announced positive topline results following the completion of its Phase III BRIDGE clinical trial of PRX–102 for the treatment of Fabry disease. The Phase III BRIDGE clinical trial, a 12-month open-label, single arm switch-over study evaluating the safety and efficacy of PRX–102, 1 mg/kg infused every two weeks, met its main objectives for safety and efficacy, and topline analysis indicated substantial improvement in renal function as measured by mean annualized estimated Glomerular Filtration Rate (eGFR slope) in patients switched from agalsidase alfa to PRX–102.
On February 6, 2020, Protalix and Chiesi announced the receipt of an agreement letter from the FDA for the Initial Pediatric Study Plan (iPSP) for PRX-102 for the treatment of Fabry disease, outlining an agreed-upon approach to address the needs of pediatric Fabry patients.
Corporate & Financial Developments

On March 16, 2020, the Company announced that it has agreed to conduct a feasibility study with Kirin Holdings Company, Limited, or Kirin, to evaluate the production of a novel complex protein utilizing ProCellEx. The Company received a non-refundable payment of $1.0 million and Kirin will provide research funding for the Company’s scientists to conduct cell line engineering and protein expression studies on the target protein.
On March 12, 2020, the Company entered into securities purchase agreements with certain existing and new institutional and other accredited investors in a private placement. Pursuant to such agreements, the Company issued and sold to the purchasers an aggregate of approximately 17.6 million unregistered shares of its common stock at a price per share of $2.485, or aggregate net committed proceeds equal to approximately $41.3 million. Each share of the Company’s common stock issued in the transaction was accompanied by a warrant to purchase an additional share of common stock at an exercise price equal to $2.36.
Financial Results

For the three months ended March 31, 2020, compared to the three months ended March 31, 2019

The Company recorded revenues from selling goods of $5.0 million during the three months ended March 31, 2020, an increase of $1.5 million, or 43%, compared to revenues of $3.5 million for the same period of 2019. The increase resulted primarily from an increase of $0.8 million in sales of drug product to Brazil as well as an increase of $0.7 million in sales of drug substance to Pfizer Inc.
Revenues from license and R&D services for the three months ended March 31, 2020, were $16.6 million, an increase of $9.7 million, or 140%, compared to revenues of $6.9 million for the same period of 2019. Revenues from the license agreements represent the revenues recognized in connection with previously announced agreements with Chiesi. The increase is primarily due to revenues recognized in connection with the progress of the Company’s clinical trial that have been performed, and with revenues recognized in connection with an updated costs estimation throughout the trials until completion in the amount of $6.7 million.
Cost of goods sold was $3.4 million for the three months ended March 31, 2020, an increase of $1.4 million, or 68%, from cost of goods sold of $2.0 million for the same period of 2019. The increase is primarily due to an increase in sales of goods.
Research and development expenses were $10.3 million for the three months ended March 31, 2020, a decrease of $1.4 million, or 12%, compared to $11.7 million of research and development expenses for the same period of 2019. The decrease was primarily due to a decrease in costs related to manufacturing of our drug in development.
Selling, general and administrative expenses were $3.2 million for the three months ended March 31, 2020, an increase of $1.0 million, or 43%, compared to $2.2 million for the same period of 2019. The increase resulted primarily from a $0.6 million increase in compensation related costs and a $0.2 million increase in professional fees.
Net income for the three months ended March 31, 2020 was $1.7 million, or $0.10 per share, basic and diluted, compared to a net loss of $7.3 million, or $0.50 per share, basic and diluted, for the same period of 2019.

On June 1, 2020 Shanghai Henlius Biotech, Inc. (2696.HK) and Accord Healthcare Limited (Accord) reported that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency ( European Medicines Agency, EMA) issued a favorable opinion that recommended the market for the HLX02 biosimilar (trastuzumab injection) for the treatment of early HER-2-positive breast cancer, HER2-positive metastatic breast cancer and previously untreated HER-2-positive metastatic cancer Contains adenocarcinomas of the stomach or the transition area of ​​the esophagus into the stomach (Press release, Shanghai Henlius Biotech, JUN 1, 2020, View Source;816056005.html [SID1234560743]). According to the EMA-CHMP summary of this positive opinion, HLX02 is the reference product Herceptin (Trastuzumab) very similar. Data show that HLX02 is comparable to Herceptin in terms of quality, safety and effectiveness .

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HLX02 is a monoclonal biosimilar antibody (mAb) developed and manufactured by Henlius in accordance with biosimilar guidelines and will be marketed and marketed by Accord in Europe. The CHMP’s opinion is now being reviewed by the European Commission. A regulatory decision is expected over the next two to three months. As soon as a license is granted , a centralized marketing authorization is granted for all EU member states, Iceland, Norway and Liechtenstein .

Dr. Scott Liu , co-founder and CEO of Henlius , commented: "We are proud of CHMP’s favorable opinion on Henlius, as is the fact that Henlius’ capabilities in developing and manufacturing biological products are recognized by an international drug agency. We look forward to the launch of HLX02 as an alternative, high quality and affordable treatment option for patients with HER-2 positive breast cancer and gastric cancer. We will continue to work with Accord, make HLX02 more available in Europe and other regions, and continue to strive to support patients around the world. "

Dr. James Burt , Executive Vice President of Accord Europe and MENA added: "We are committed to providing oncology patients with access to safe, inexpensive drugs. The CHMP’s positive opinion is due to our in-depth expertise and our extensive portfolio of oncological therapies. Our agreement with Henlius reflects our longstanding commitment to biopharmaceutical development, research and manufacturing. "

On June 1, 2020 EnGeneIC Limited, a clinical-stage biopharmaceutical company advancing its proprietary EDV nanocell platform for targeted cyto-immunotherapy in cancer, reported that an abstract on the Phase I/IIa clinical trial with EGFR-targeted EDV nanocells in patients with recurrent, metastatic pancreatic cancer has been accepted at the 2020 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Virtual Scientific Program, which will be held from May 29-31, 2020 (Press release, EnGeneIC, JUN 1, 2020, View Source [SID1234560742]).

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The abstract, titled, "Interim Data: Phase I/IIa study of EGFR-targeted EDV nanocells carrying cytotoxic drug PNU-159682 (E-EDV-D682) with immunomodulatory adjuvant EDVs carrying α-galactosyl ceramide (EDV-GC) in patients with recurrent, metastatic pancreatic cancer," provides early data from nine patients in the ongoing open label Phase IIa study, the Carolyn Trial, in patients with treatment-refractory metastatic pancreatic cancer. To date, the EDVs carrying the cytotoxic drug and immune adjuvant have been well tolerated and demonstrated an excellent safety profile. Further, early signs point to durable response in patients in this extremely difficult-to-treat patient population, possibly related to the development of an innate and adaptive immune response as a result of direct cytotoxic effects on drug resistant tumor cells.

Professor Vinod Ganju, the Principal Investigator on the study at Frankston Private Hospital, Melbourne, said, "There have been no significant advances in the treatment of pancreatic cancer and the mortality rate remains very high. The EDV therapeutic is showing a remarkable safety profile and some patients have had prolonged periods of disease control without toxicity. Currently, modifications to dose and schedule are ongoing with the goal of achieving further substaintial improvement in anti-tumour efficacy."

Dr. Himanshu Brahmbhatt, Joint-CEO of EnGeneIC, stated, "this is the first time a super-cytotoxic drug designed to overcome serious multi-drug resistance in late stage cancers, has been safely delivered in patients. One aspect of the EDV’s unique mechanism of action was recently published in Cancer Cell in March 2020 and the associated cover art depicts the cyto-immunotherapeutic pathway in pictorial form (Cancer Cell, 37: 354-370 (2020)."

Details of abstract can be found at the ASCO (Free ASCO Whitepaper) Meeting Library.

Abstract Number: 4632
Title: "Interim Data: Phase I/IIa study of EGFR-targeted EDV nanocells carrying cytotoxic drug PNU-159682 (E-EDV-D682) with immunomodulatory adjuvant EDVs carrying α-galactosyl ceramide (EDV-GC) in patients with recurrent, metastatic pancreatic cancer"

Session: Gastrointestional Cancer, Gastroesophageal, Pancreatic, and Hepatobillary

Trial: View Source

On June 1, 2020 BERG, a clinical-stage biotech company that uses Artificial Intelligence (AI) to research diseases and develop innovative treatments, reported results from a Phase 1 clinical study of its investigational drug, BPM 31510 (novel ubidecarenone formulation) in glioblastoma (GBM) (Press release, Berg, JUN 1, 2020, View Source [SID1234560741]). The study, presented at the 2020 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting (ASCO) (Free ASCO Whitepaper), highlights safety, tolerability and the impact of BPM 31510-IV influence on metabolism in patients, supporting planned Phase 2 clinical development.

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"At BERG, we understand the toll cancer takes not only on a patient, but also their loved ones, and we want to lead in the battle to fight the disease," said Dr. Niven R. Narain, BERG Co-founder, President and Chief Executive Officer. "BPM 31510 is a unique therapeutic modality targeting cell metabolism that is consistent with our proprietary Interrogative Biology platform. We are excited at the opportunities the findings will have in refining clinical development among multiple cancer indications, especially in brain cancers."

GBM is a destructive type of brain cancer often acknowledged for its steep morbidity and mortality rates. There are few effective treatment options available in the marketplace, thus substantiating the urgent need for novel therapeutic approaches to improve outcomes for this disease. BPM 31510 is a unique therapeutic modality which specifically targets cell metabolism and shifts the cancer’s glycolytic dependency toward mitochondrial oxidative phosphorylation. When applied, it induces oxidative stress and causes cell death, specifically of cancer cells. BPM 31510 serves as particularly relevant indication for GBM’s glycolytic dependency.

The study was led by Seema Nagpal, M.D. Clinical Associated Professor of Neurology & Neurological Sciences, and Lawrence Recht, M.D., Professor of Neurology & Neurological Sciences, both of Stanford University School of Medicine, with other Stanford Medicine researchers and in collaboration with BERG.

Details of the BERG presentation:
This Poster is available to registered 2020 ASCO (Free ASCO Whitepaper) Virtual Scientific Program attendees and can be viewed at:
View Source

In its commitment to serve patients afflicted with cancer, BERG has collaborated on other projects with leading institutions, like MD Anderson Cancer Institute (clinical trials) and Harvard/BIDMC (Project Survival), among others.

On June 1, 2020 Kazia Therapeutics Limited (ASX: KZA;NASDAQ: KZIA), an Australian oncology-focused biotechnology company, reported to share a poster presentation of interim data from the ongoing phase II study of paxalisib (formerly GDC-0084) in glioblastoma, the most common and most aggressive form of primary brain cancer (Press release, Kazia Therapeutics, JUN 1, 2020, View Source [SID1234560740]). Top-line data from this interim analysis was previously announced to ASX on 7 April 2020.

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Key Points

Analysis of Stage 1 of the study (n=9) shows median overall survival (OS) of 17.7 months. This compares very favourably with temozolomide, the existing standard of care, which has a reported median OS of 12.7 months in this patient population
Progression-free survival (PFS) in Stage 1 was 8.4 months, which represents a clinically material advantage over the 5.3 months associated with temozolomide
The longest-treated patient remains on therapy and progression-free some 19 months after diagnosis
Safety profile is consistent with prior clinical experience. Rash, mucositis, and hyperglycemia are the most common toxicities, and 60mg, once daily, orally, was confirmed as the maximum tolerated dose (MTD)
Professor Patrick Wen from Dana-Farber Cancer Institute, who was the lead author on the poster presentation, commented, "These are encouraging early signals. We anticipate paxalisib will move into a pivotal study later this year and look forward to reviewing further data as it emerges."

Kazia CEO, Dr James Garner, added, "We are pleased to be able to share these extremely promising data with clinicians and partners, albeit in the novel forum of a virtual academic meeting. As we have previously said, the gold standard for any new cancer drug is the ability to extend life, and we are seeing evidence from this study that paxalisib may achieve this very challenging goal. We expect to begin recruitment to the international GBM AGILE pivotal study in the second half of this year. In the meantime, we expect several further data read-outs over the next two quarters."

Summary of Paxalisib Data in Comparison to Temozolomide (existing standard of care)

Temozolomide
(FDA-approved treatment)

Paxalisib

(Stage 1 of Phase II Study)

Progression-Free Survival (PFS)

Measures ability of a drug to slow
growth of a tumour

5.3 months

8.4 months

Overall Survival (OS)

Measures ability of a drug to
prolong life

12.7 months

17.7 months

ASCO Conference

The American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting is one of the premier scientific conferences in the world for research and treatment of cancer. It is typically attended by more than 30,000 clinicians, researchers, industry executives, and patient advocates. In 2020, the meeting is being conducted in a virtual format due to the ongoing COVID-19 pandemic.

The Kazia data is presented in Abstract 2550 (NCT03522298). In addition to Kazia’s paxalisib poster, abstracts are being presented by the Global Coalition for Adaptive Research on the GBM AGILE clinical trial (Abstract TPS2579; NCT03970447), and by the Alliance for Clinical Trials in Oncology on their genomically-guided study in brain metastases (Abstract TPS2573; NCT03994796), in which paxalisib is one of three participating drug candidates.

The Kazia poster is available for download via the Kazia website at View Source

Background

The reported overall survival (OS) figure of 17.7 months represents a strong signal of clinical efficacy. The existing, FDA-approved standard of care, temozolomide, is associated with an OS of 12.7 months in this patient population[1]. Comparison between different studies is always imprecise, but the magnitude of the numerical difference provides powerful evidence that treatment with paxalisib may extend life in this patient group.

The reported progression-free survival (PFS) figure of 8.4 months compares favourably with the PFS of 5.3 months that is associated with temozolomide in this patient population. In April 2020, Kazia reported an interim analysis showing a PFS of 8.5 months in the overall study population (n=30). This poster only reports the first stage of the study (n=9), and the figure in this part of the study was 8.4 months.

Before losing patent protection, temozolomide achieved peak sales in excess of US$ 1 billion per annum, which provides an indication of the commercial opportunity associated with a new treatment for glioblastoma.

The Kazia study is being conducted in newly-diagnosed glioblastoma patients, following surgery and radiotherapy. Only those patients with an unmethylated MGMT promotor have been recruited. This genetic marker renders patients effectively resistant to temozolomide and is present in approximately two-thirds of patients.

Thirty patients were enrolled to this study, comprising 9 in Stage 1 , and 21 in Stage 2. Data reported here are provisional figures from Stage 1 (for OS) and from the entire study population (for PFS), but may change as ongoing patients proceed through the study. The study has been conducted at leading centers of excellence in the United States.

The safety of paxalisib remained broadly consistent with prior experience, with hyperglycaemia (raised blood sugar), oral mucositis (mouth ulcers), and low-grade rash among the most common drug-related toxicities.

In addition to this phase II study in glioblastoma, four other studies are underway with paxalisib in different forms of brain cancer, and it is anticipated that several of these will provide initial efficacy data during CY 2020.

Investors are referred to Kazia’s announcement of 7 April 2020 for further discussion of these results.

Next Steps

The phase II study remains ongoing, with approximately half of the total enrolled patient population still receiving drug at the time of analysis and a number of additional patients still in follow-up. Kazia expects to complete the study in 1H CY2021.

Kazia had previously had an abstract accepted the AACR (Free AACR Whitepaper) Annual Meeting, which had originally been scheduled for April 2020. This meeting has now been rescheduled to several virtual meetings, and Kazia will present a poster at the ‘AACR Virtual Annual Meeting II’ on 22-24 June 2020.