On May 6, 2019 Protalix BioTherapeutics, Inc. (NYSE American:PLX) (TASE:PLX), a biopharmaceutical company focused on the development and commercialization of recombinant therapeutic proteins expressed through its proprietary plant cell-based expression system, ProCellEx, reported its financial results for the three months ended March 31, 2019 and provided a corporate update (Press release, Protalix, MAY 6, 2019, View Source;p=RssLanding&cat=news&id=2397172 [SID1234535759]).

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"In the first three months of 2019, we have continued to execute on enrollment in our PRX-102 studies and have worked to prepare ourselves for a potential accelerated approval path," said Mr. Moshe Manor, Protalix’s President and Chief Executive Officer. "In addition, we are encouraged by the initial pharmacokinetic (PK) data from our BRIGHT study, which were presented at the 15th Annual WORLDSymposiumTM 2019 in February 2019, that demonstrate the potential for PRX-102 to be infused once-monthly, compared to the current treatment regimen of every two weeks."

First Quarter 2019 and Recent Clinical and Corporate Highlights

The Company’s BRIGHT phase III clinical trial of pegunigalsidase alfa, or PRX-102, for the treatment of Fabry disease is currently one patient away from completion of enrollment.

The Company’s BALANCE phase III clinical trial of pegunigalsidase alfa for the treatment of Fabry disease is currently eleven patients away from completion of enrollment.

The Company presented results from the BRIGHT Study at the 15th Annual WORLD Symposium showing that infusion of pegunigalsidase alfa every 4 weeks results in the presence of continuous active enzyme throughout the entire infusion interval.

The Company is scheduled to present three posters during the 6th Update on Fabry Disease international conference being held in Prague, Czech Republic, on May 26-28, 2019.

To date, more than 40 patients are being treated in the Company’s various extension studies after opting to continue treatment with pegunigalsidase alfa after they completed an initial study.

The Company plans to meet with the U.S. Food and Drug Administration (FDA) for a follow up meeting during the second quarter of 2019 in connection with the potential accelerated approval filing path for pegunigalsidase alfa.
First Quarter 2019 Financial Results

The Company recorded total revenues of $10.4 million during the three months ended March 31, 2019, compared to $6.7 million for the same period of 2018. The increase is primary attributable to the recognition of $6.9 million of license revenues in the three months ended on March 31, 2019 compared to the recognition of $2.2 million in the same period of 2018.

Research and development expenses for the three months ended March 31, 2019, were $11.7 million, compared to $7.3 million for the same period in 2018. Selling, general and administrative expenses for the three months ended March 31, 2019 were $2.2 million, compared to $2.5 million incurred during the same period in 2018.

Net loss for the three months ended March 31, 2019 was $7.3 million compared to $7.2 million for the three months ended March 31, 2018.
On March 31, 2019, the Company had $30.4 million of cash and cash equivalents, compared to $37.8 million at March 31, 2018, which is currently projected to fund operations into mid-2020. As of March 31, 2019, the Company had outstanding $57.9 million of its 7.50% convertible promissory notes due November 2021.
Conference Call and Webcast Information

The Company will host a conference call on Monday, May 6, 2019, at 8:30 am ET 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: United States: +1-844-358-6760; International: +1-478-219-0004. Conference ID number 6169584.

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.

On May 6, 2019 NuVasive, Inc. (NASDAQ: NUVA), the leader in spine technology innovation, focused on transforming spine surgery with minimally disruptive, procedurally-integrated solutions, reported that management will present at the Bank of America Merrill Lynch Healthcare Conference 2019 at the Encore Hotel in Las Vegas on Wednesday, May 15, 2019, at 1:40 p.m. PT/4:40 p.m. ET (Press release, NuVasive, MAY 6, 2019, View Source [SID1234535777]).

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A live webcast of the presentation will be available online from the Investor Relations page of the Company’s website at www.nuvasive.com. A replay of the presentation will remain available on the website for 30 days after the live webcast.

On May 6, 2019 FibroGen, Inc. (NASDAQ: FGEN) reported that it will report first quarter 2019 financial results on Thursday, May 9, 2019 after market close, and will host a conference call to discuss financial results and provide a business update at 5:00 p.m. ET (2:00 p.m. PT) (Press release, FibroGen, MAY 6, 2019, View Source;p=irol-newsArticle&ID=2397383 [SID1234535839]).

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Conference Call and Audio Webcast
Interested parties may access a live audio webcast of the conference call via the investor section of the FibroGen website, www.fibrogen.com. It is recommended that listeners access the website 15 minutes prior to the start of the call to download and install any necessary audio software. A replay of the webcast will be available shortly after the call for a period of two weeks. To access the replay, please dial (888) 843-7419 (domestic) or (630) 652-3042 (international), and use passcode 48545313#.

Dial-In Information
Live (U.S./Canada): (888) 771-4371
Live (International): (847) 585-4405
Confirmation number: 48545313

On May 6, 2019 Epizyme, Inc. (Nasdaq: EPZM), a late-stage biopharmaceutical company developing novel epigenetic therapies, reported business and pipeline updates and reported first quarter 2019 financial results (Press release, Epizyme, MAY 6, 2019, View Source [SID1234535736]).

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"This is an incredibly exciting time for our company, and 2019 is slated to be one of the most important years in Epizyme’s evolution," said Robert Bazemore, president and chief executive officer of Epizyme. "Our team is on track to submit two NDAs for tazemetostat this year, first for epithelioid sarcoma in the second quarter, which, if successful, would make tazemetostat the first commercially available EZH2 inhibitor, and then a second submission for follicular lymphoma in the fourth quarter. We are preparing for multiple clinical trials starting mid-year designed to expand the utility of tazemetostat, and to initiate clinical development of our novel G9a inhibitor in the second half of 2019. In addition, we have achieved the first milestone in our partnership with Boehringer Ingelheim, which further validates our research expertise and our collaboration strategy in epigenetic target discovery and drug development. With each milestone accomplished, we are one step closer to fulfilling our mission of bringing new treatments to patients with cancer and other serious diseases."

Tazemetostat Program Updates and Progress

NDA Submission for Epithelioid Sarcoma (ES) on Track for Second Quarter: Epizyme is well underway with its preparations to submit its first New Drug Application (NDA) for accelerated approval to the U.S. Food and Drug Administration (FDA) in the second quarter of 2019 for tazemetostat in patients with ES. This is an ultra-rare and difficult-to-treat sarcoma, and if approved, tazemetostat would be the first treatment specifically indicated for patients with ES. Updated data from the company’s ongoing Phase 2 trial in ES will be presented at the 2019 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting in Chicago; details will be disclosed at a future date.

NDA Submission for All-Comer Follicular Lymphoma (FL) Population Planned for Fourth Quarter: Epizyme plans to submit an NDA for accelerated approval of tazemetostat for patients with FL, regardless of their EZH2 mutational status, who have been previously treated with two or more systemic therapies. The ongoing Phase 2 study has completed enrollment, and the company is working toward the

submission of an NDA for accelerated approval in the fourth quarter of 2019 for this patient population. Updated data from the Phase 2 trial will be presented at a medical meeting in the second quarter of 2019. Details will be disclosed at a future date.

Planned Tazemetostat Clinical Studies in 2019

Epizyme is planning for multiple clinical trials designed to expand the benefit of tazemetostat into earlier treatment lines in follicular lymphoma, and to explore new combinations and potential indications in both FL and solid tumors. Planned clinical trials include:

a combination study of tazemetostat with the chemo-free treatment regimen "R2" (Revlimid plus Rituxan) for patients with relapsed/refractory FL who have received at least one prior therapy;

a combination study of tazemetostat with Rituxan for patients with relapsed/refractory FL;

a combination study of tazemetostat with R-CHOP for front-line patients with FL in collaboration with the Lymphoma Study Association (LYSA);

a combination study of tazemetostat with the standards-of-care for patients with castration-resistant prostate cancer; and

a combination study of tazemetostat with a PARP inhibitor for patients with platinum-resistant solid tumors, such as small-cell lung cancer, triple-negative breast cancer and ovarian cancer.

Initiation of Clinical Development of EZM8266 for Sickle Cell Disease

Upon approval of an Investigational New Application (IND) for EZM8266 for the treatment of patients with sickle cell disease, Epizyme anticipates beginning clinical development in the second half of 2019 with a Phase 1 trial of EZM8266, a novel, first-in-class G9a inhibitor.

Business Updates

Epizyme recently earned a $5.5 million milestone payment from Boehringer Ingelheim, following the selection of a lead optimization candidate for the shared program targeting an enzyme within the helicase family. The companies are jointly researching and developing this program, with both parties sharing U.S. commercialization responsibilities and Boehringer Ingelheim assuming responsibility for commercialization outside the U.S. The companies will also share research responsibilities for a histone acetyltransferase (HAT) program that is under development.

In March of this year, Epizyme raised $172.50 million in aggregate gross proceeds, before deducting underwriting discounts and offering expenses, from two concurrent underwritten public offerings.

First Quarter 2019 Financial Results

Cash Position: Cash, cash equivalents and marketable securities were $371.1 million as of March 31, 2019, as compared to $247.9 million as of March 31, 2018.

Revenue: Collaboration revenue for the first quarter of 2019 was $7.9 million. There was no collaboration revenue recognized for the first quarter of 2018. The increase in collaboration revenue is due to the company’s collaboration with Boehringer Ingelheim, which was initiated in November 2018.

R&D Expenses: Research and development (R&D) expenses were $26.9 million for the first quarter of 2019, compared to $25.6 million for the first quarter of 2018. The increase is

primarily due to greater tazemetostat manufacturing costs and costs incurred in preparation for two NDA submissions offset by decreases in clinical trial expenses.

G&A Expenses: General and administrative (G&A) expenses were $12.0 million for the first quarter of 2019, compared to $9.4 million for the first quarter of 2018. The increase is primarily due to a rise in medical affairs and commercial costs as a result of organizational development in preparation for tazemetostat commercialization.

Net Loss Attributed to Common Stockholders: Net loss attributable to common stockholders was $32.3 million, or $0.39 per share, for the first quarter of 2019, compared to $34.1 million, or $0.49 per share, for the first quarter of 2018.

Financial Guidance

Following its March financing, and based on its current operating plan, Epizyme expects its cash runway to extend into at least the first quarter of 2021.

The company will not hold a conference call in conjunction with these results.

About the Epizyme-Boehringer Ingelheim Collaboration

Epizyme and Boehringer Ingelheim established a worldwide collaboration agreement in November 2018 to develop novel epigenetic oncology therapies. Under the terms of the agreement, Boehringer Ingelheim and Epizyme will jointly research and develop a helicase program, with both parties sharing U.S. commercialization responsibilities and Boehringer Ingelheim assuming responsibility for commercialization outside the U.S. Epizyme and Boehringer Ingelheim will also share research responsibilities for a histone acetyltransferase (HAT) program, with Boehringer Ingelheim assuming responsibility for worldwide development and commercialization. Epizyme received an upfront payment of $15 million and will receive an additional $5 million in research funding in 2019, and is eligible to receive up to $280.5 million in research, development and commercialization milestones. For the helicase program, Epizyme will fund a portion of the global development costs, retain a share of U.S. profits and receive tiered royalties on ex-U.S. sales. For the HAT program, Epizyme is eligible to receive tiered royalties on worldwide sales.

On May 6, 2019 Syndax Pharmaceuticals, Inc. (Nasdaq: SNDX), a clinical stage biopharmaceutical company developing an innovative pipeline of cancer therapies, reported its financial results for the first quarter ended March 31, 2019. In addition, the Company provided a clinical and business update (Press release, Syndax, MAY 6, 2019, View Source [SID1234535760]). As of March 31, 2019, Syndax had $92.7 million in cash, cash equivalents and short-term investments.

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"We are pleased to report that E2112, our Phase 3 registration trial of entinostat plus exemestane in HR+, HER2- breast cancer, has passed its fourth interim overall survival analysis," said Briggs W. Morrison, M.D., Chief Executive Officer of Syndax. "E2112 was designed to replicate the compelling overall survival results obtained in the Phase 2b ENCORE 301 trial which led to Breakthrough Therapy designation. The next overall survival assessment is expected in the fourth quarter of this year. We remain confident in the potential that the addition of entinostat to exemestane will result in a positive survival benefit, which would allow us to file for full regulatory approval in this indication."

Dr. Morrison added, "We also look forward to filing an IND for SNDX-5613, our targeted menin inhibitor, later this quarter. Supported by a robust preclinical dataset, we believe this therapeutic class has the potential to make a meaningful impact for patients with genetically-defined acute leukemias for whom limited effective therapies exist."

Pipeline Updates

Entinostat

ECOG-ACRIN has informed the Company that following its fourth preplanned interim overall survival (OS) analysis, the E2112 trial will continue as planned until either an OS benefit is observed, or the final target number of events occur. E2112 is Syndax’s NCI-sponsored, ECOG-ACRIN led Phase 3 registration trial of entinostat, a Class I selective HDAC inhibitor, plus exemestane in advanced hormone receptor positive, human epidermal growth factor receptor 2 negative (HR+, HER2-) breast cancer. The next interim analysis for the OS endpoint is scheduled for 4Q19, with a final OS assessment, if necessary, to be conducted in 2Q20. Any positive OS assessment would enable the Company to file for full regulatory approval. The E2112 trial design was informed by the Phase 2b ENCORE 301 trial, the results of which led to entinostat’s Breakthrough Therapy designation in HR+, HER2- breast cancer, in which patients receiving the entinostat/exemestane combination demonstrated a statistically significant OS benefit.

At the American Association of Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting held March 29 – April 3, 2019, Syndax presented data from the non-small cell lung cancer (NSCLC) and melanoma cohorts of the ENCORE 601 trial of entinostat in combination with KEYTRUDA (pembrolizumab), Merck’s anti-PD-1 (programmed death receptor-1) therapy. These data provided further evidence that the addition of entinostat to pembrolizumab may overcome resistance to immunotherapy in melanoma and NSCLC patients whose disease progressed on or after anti-PD-1 therapy. As the Company has previously indicated, following availability of positive E2112 OS results, it will determine whether to advance its entinostat-PD-1 combination programs into one or more registration trials.

SNDX-5613

Syndax continues to expect to file an Investigational New Drug (IND) application with the U.S. Food and Drug Administration (FDA) for its targeted menin inhibitor, SNDX-5613, later this quarter, with the initiation of a Phase 1 clinical trial in a defined subset of acute leukemia patients expected to follow shortly thereafter.

SNDX-6352

The Company continues to anticipate initial results from the Phase 1 dose escalation trial of SNDX-6352, Syndax’s anti-CSF-1R monoclonal antibody, in patients with chronic graft versus host disease (cGVHD) in the second half of the year. The objectives of this trial are to evaluate the safety and preliminary efficacy of SNDX-6352 in cGVHD and to identify a recommended Phase 2 dose and schedule.

First Quarter 2019 Financial Results

As of March 31, 2019, Syndax had cash, cash equivalents and short-term investments of $92.7 million and 31.6 million shares and share equivalents issued and outstanding.

In March 2019, Syndax issued 4.5 million shares of its common stock and prefunded warrants at an offering price of $6.00, as well as warrants to purchase up to 4.5 million shares of its common stock, with half at an exercise price of $12.00 per share and the remaining half at an exercise price of $18.00 per share. As a result of the offering, Syndax received aggregate net proceeds of approximately $27.4 million.

First quarter 2019 research and development expenses decreased to $11.3 million from $15.3 million. The first quarter decrease was primarily due to reduced CMC activities and decreased clinical activities.

General and administrative expenses for the first quarter 2019 decreased to $3.9 million from $4.8 million. The decrease was primarily due to decreased pre-commercialization expenses and decreased professional fees.

For the three months ended March 31, 2019, Syndax reported a net loss attributable to common stockholders of $14.3 million or $0.53 per share compared to $19.4 million or $0.79 per share for the prior year period.

Financial Guidance

Today the Company provided operating expense guidance for the second quarter and full year 2019. For the second quarter and full year 2019, research and development expenses are expected to be $9 to $10 million and $46 to $50 million, respectively, and total operating expenses are expected to be $13 to $14 million and $60 to $64 million, respectively.

Conference Call and Webcast

In connection with the earnings release, Syndax’s management team will host a conference call and live audio webcast at 4:30 p.m. ET today, Monday, May 6, 2019.

The live audio webcast and accompanying slides may be accessed through the Events & Presentations page in the Investors section of the Company’s website at www.syndax.com. Alternatively, the conference call may be accessed through the following:

Conference ID: 4292817
Domestic Dial-in Number: 855-251-6663
International Dial-in Number: 281-542-4259
Live Webcast: View Source

For those unable to participate in the conference call or webcast, a replay will be available for 30 days on the Investors section of the Company’s website, www.syndax.com.