On March 16, 2020 Arvinas, Inc. (Nasdaq: ARVN), a clinical-stage biotechnology company creating a new class of drugs based on targeted protein degradation, reported financial results for the fourth quarter and full year ended December 31, 2019 and provided a corporate update (Press release, Arvinas, MAR 16, 2020, View Source [SID1234555594]).

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"2019 was an exciting year for Arvinas, marked by tremendous clinical and corporate progress. We were delighted to present the first human clinical data from targeted protein degraders along with preclinical data demonstrating the brain-penetrating potential of our PROTAC protein degraders. These data underscore the potential of our platform to create safe and well-tolerated drugs for the treatment of a variety of life-threatening diseases," said John Houston, Ph.D., Chief Executive Officer of Arvinas.

Business Highlights and Recent Developments

Presented initial clinical safety and pharmacokinetic data for ARV-110 (as a treatment for men with metastatic castration-resistant prostate cancer) and ARV-471 (as a therapy for patients with locally advanced or metastatic ER+/HER2- breast cancer). These data showed that ARV-110 and ARV-471 were well-tolerated and safe for further development at the doses disclosed in October 2019. Further, both PROTAC protein degraders reached exposure levels in human patients that were correlated with tumor inhibition oral regression in preclinical models.

Closed an underwritten public offering of 5,227,273 shares of common stock at a public offering price of $22.00 per share, including the exercise in full by the underwriters of their option to purchase additional shares of common stock. Arvinas received net proceeds of approximately $107.6 million, after deducting underwriting discounts and commissions and offering expenses.

Presented preclinical data demonstrating that oligomer-specific PROTAC molecules degraded aggregates of human alpha-synuclein in primary rat neurons.

Announced the appointment of Laurie Smaldone Alsup, M.D., to Arvinas’ board of directors. Dr. Smaldone Alsup currently serves as Chief Scientific Officer and Chief Medical Officer for NDA Group. She brings experience leading commercially successful product approvals and global regulatory strategies that have successfully advanced programs through all stages of clinical development.

Anticipated Milestones and Expectations

For the ARV-110 program, Arvinas expects to share Phase 1 dose escalation clinical data in the second quarter of 2020.

For the ARV-471 program, Arvinas expects to share Phase 1 dose escalation clinical data in the second half of 2020.

In the second half of 2020, Arvinas expects to provide information about the advancement of additional programs in its robust preclinical pipeline.

Financial Guidance

Based on its current operating plan, Arvinas expects its cash, cash equivalents, and marketable securities will be sufficient to fund its planned operating expenses and capital expenditures into 2022.

Full Year and Fourth Quarter Financial Results

Cash, Cash Equivalents and Marketable Securities Position: As of December 31, 2019, cash, cash equivalents and marketable securities were $280.9 million as compared with $187.8 million as of December 31, 2018. The increase primarily related to net proceeds from a public offering of common stock of $107.6 million, aggregate proceeds of $51.5 million from a collaboration and license agreement with Bayer and the issuance of common stock to Bayer and collaborator milestone and other payments of $7.8 million, partially offset by cash used to fund operations of approximately $67.6 million and cash used to purchase fixed assets and leasehold improvements of $6.2 million. Cash, cash equivalents, and marketable securities increased by $90.4 million in the fourth quarter of 2019. This increase primarily related to net proceeds from a public offering of common stock of $107.6 million and collaborator milestone and other payments of $2.2 million, partially offset by cash used to fund operations of approximately $17.6 million and cash used to purchase fixed assets and leasehold improvements of $1.8 million.

Research and Development Expenses: Research and development expenses were $67.2 million and $20.4 million for the year and quarter ended December 31, 2019, respectively as compared with $45.2 million and $14.6 million for the year and quarter ended December 31, 2018, respectively. The increase in research and development expenses for the year and quarter primarily related to Arvinas’ continued investment in its platform, exploratory and lead optimization programs and its estrogen receptor (ER) and androgen receptor (AR) clinical development programs.

General and Administrative Expenses: General and administrative expenses were $27.3 million and $7.3 million for the year and quarter ended December 31, 2019, respectively, as compared with $12.9 million and $5.8 million for the year and quarter ended December 31, 2018, respectively. The increase in general and administrative expenses for the year and quarter primarily related to increasing infrastructure costs and being a public company for a full year in 2019.

Revenues: Revenue was $43.0 million and $4.9 million for the year and quarter ended December 31, 2019, respectively, as compared with $14.3 million and $3.4 million for the year and quarter ended December 31, 2018, respectively. Revenue for the year ended December 31, 2019 included $24.7 million of revenue recognized from the Arvinas contribution of the license to the joint venture between Bayer and Arvinas to pursue the PROTAC technology in agricultural applications (the Joint Venture). The remaining revenue of $18.3 million and revenue of $4.9 million for the year and quarter ended December 31, 2019, respectively, was generated from the license and rights to technology fees and research and development activities related to the collaboration and license agreement with Bayer that was initiated in July 2019, the collaboration and license agreement with Pfizer that was initiated in January 2018, and the amended and restated option, license and collaboration agreement with Genentech that was initiated in November 2017.

Loss from Equity Method Investment: Loss from equity method investment for the year ended December 31, 2019 was $24.7 million, which related to the loss from the equity method investment in the Joint Venture. The loss was generated from the Joint Venture’s expensing the values associated with the contributed intellectual property from the Joint Venture partners.

Net Loss: Net loss was $70.3 million and $21.0 million for the year and quarter ended December 31, 2019, respectively, as compared with $41.5 million and $16.1 million for the year and quarter ended December 31, 2018, respectively. The increase in net loss for the year and quarter primarily related to Arvinas’ continued investment in its platform, exploratory and lead optimization programs, its ER program, its AR program and increase in general and administrative infrastructure costs related to the first full year as a public company offset by an increase in revenue primarily related to the Bayer collaboration that was initiated in July 2019.

About ARV-110

ARV-110 is an orally bioavailable PROTAC protein degrader designed to selectively target and degrade the androgen receptor (AR). ARV-110 is being developed as a potential treatment for men with metastatic castration-resistant prostate cancer (mCRPC). Arvinas’ Phase 1 trial of ARV-110 will assess its safety, tolerability, and pharmacokinetics, and will also include measures of anti-tumor activity and pharmacodynamic readouts as secondary endpoints.

ARV-110 has demonstrated activity in preclinical models of AR mutation or overexpression, both common mechanisms of resistance to currently available AR-targeted therapies.

About ARV-471

ARV-471 is an orally bioavailable PROTAC protein degrader designed to specifically target and degrade the estrogen receptor (ER) for the treatment of women with metastatic ER+/HER2- breast cancer. Arvinas’ Phase 1 trial of ARV-471 will assess its safety, tolerability, and pharmacokinetics, and will also include measures of anti-tumor activity and pharmacodynamic readouts as secondary endpoints. In preclinical studies, ARV-471 demonstrated near-complete ER degradation in tumor cells, induced robust tumor shrinkage when dosed as a single agent in multiple ER-driven xenograft models, and showed superior anti-tumor activity when compared to a standard of care agent, fulvestrant, both as a single agent and in combination with a CDK4/6 inhibitor.

On March 16, 2020 Seelos Therapeutics, Inc. (Nasdaq: SEEL), a clinical-stage biopharmaceutical company focused on the development of therapies for central nervous system disorders and rare diseases, reported the closing of its previously announced underwritten public offering of 7,500,000 shares of its common stock, at a price to the public of $0.60 per share (Press release, Apricus Biosciences, MAR 16, 2020, View Source [SID1234555593]). The net proceeds to Seelos from this offering are expected to be approximately $3.9 million, after deducting underwriting discounts and commissions and other estimated offering expenses payable by Seelos. Seelos intends to use the net proceeds from the offering for general corporate purposes and to advance the development of its product candidates.

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Benchmark Company acted as sole book-running manager for the offering.

The securities described above were offered by Seelos pursuant to a "shelf" registration statement on Form S-3 (File No. 333-221285) previously filed with the Securities and Exchange Commission (the "SEC") on November 2, 2017, amended on December 1, 2017 and declared effective by the SEC on December 7, 2017. A final prospectus supplement and the accompanying prospectus relating to and describing the offering was filed with the SEC. Electronic copies of the final prospectus supplement and the accompanying prospectus may be obtained by visiting the SEC’s website at www.sec.gov or by contacting The Benchmark Company, LLC, Attn: Prospectus Department, 150 E. 58th Street, 17th floor, New York, NY 10155, by calling (212) 312-6700 or by e-mail at [email protected].

This press release does 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 offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of any such state or jurisdiction.

On March 16, 2020 Abeona Therapeutics Inc. (Nasdaq: ABEO), a fully-integrated leader in gene and cell therapy, reported fourth quarter and full year 2019 financial results, as well as business updates, which will be discussed on a conference call scheduled for Tuesday, March 17 at 10:00 a.m. ET (Press release, Abeona Therapeutics, MAR 16, 2020, View Source [SID1234555592]). Interested parties are invited to participate in the call by dialing 844-369-8770 (domestic) or 862-298-0840 (international) or via webcast at View Source

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"We have started 2020 strongly by opening enrollment in the pivotal Phase 3 VIITALTM study evaluating EB-101 for RDEB and we remain on track to dose the first patient in the first quarter," said João Siffert, M.D., Chief Executive Officer of Abeona. "A majority of study participants have been pre-screened and interest from the RDEB community has been high. The recently published long-term follow-up data leave us confident that VIITALTM will demonstrate EB-101 has the unique potential to durably heal the most challenging large and chronic wounds faced by RDEB patients."

Dr. Siffert continued, "We have also made great strides in our MPS III gene therapy programs. Positive interim data from the ongoing Phase 1/2 Transpher A study demonstrated that ABO-102 preserved neurocognitive skills up to two years after treatment in MPS IIIA patients treated early in life. In MPS IIIB, dose-dependent and sustained reductions in disease-specific biomarkers demonstrated a clear biologic effect of ABO-101 treatment in the Phase 1/2 Transpher B study, which is now enrolling a third dose cohort. Finally, our $103.5 million gross underwritten public offering fully funds operations well into 2021 and enables the continued progress and momentum across our robust clinical pipeline."

Fourth Quarter and Recent Highlights

Opening of enrollment in the Phase 3 VIITALTM study evaluating EB-101 for RDEB, which is on track to treat the first patient in Q1. The majority of study participants have been pre-screened and preparations for an additional clinical site initiation are ongoing.

Presentation of positive interim data from ABO-102 gene therapy program in MPS IIIA at WORLDSymposium:

●Three young patients treated in high-dose cohort 3 (at ages 27 months, 19 months, and 12 months) continued to show preserved neurocognitive skills 18 months to two years post treatment, compared with natural history.

●Across all cohorts (n=14), biomarker improvements included rapid and sustained, dose-related reductions in CSF-HS that reached lower limit of quantitation in Cohort 3 (n=2); a reduction in plasma HS levels; and a durable, dose-dependent reduction in liver volume with up to 2 years of follow up.

●ABO-102 has been well-tolerated, with long-term safety remaining favorable 15-45 months post treatment. There have been no treatment-related severe adverse events and no clinically-significant adverse events reported.

Presentation of positive interim data from ABO-101 gene therapy program in MPS IIIB at WORLDSymposium:

●Initial improvements in multiple disease-specific biomarkers including decreased cerebrospinal fluid heparan sulfate (HS) levels, reduced plasma and urine HS and glycosaminoglycans, reduced liver volume.

●ABO-101 has been well-tolerated to date, with no treatment-related severe adverse events and no clinically-significant adverse events reported (n=8).

Completion of cohort 2 and enrollment in cohort 3 of the ABO-101 Transpher B study in MPS IIIB.

Closing of $103.5 million underwritten public offering strengthening balance sheet to support the VIITALTM study through data readouts and the advancement of additional clinical programs.

Receipt of EMA PRIME designation for ABO-102 program in MPS IIIA.

Issuance of two U.S. Patents for AIM AAV capsids.

Completion of strategic review.

Fourth Quarter and Full Year Summary Financial Results

Cash, cash equivalents and marketable securities as of December 31, 2019 were $129.3 million, compared to $47.9 million as of September 30, 2019. The increase in cash of $81.4 million was driven primarily by $103.5 million gross underwritten public offering.

Net loss was $0.30 per share for the fourth quarter of 2019, compared to $0.36 per share in the comparable period in 2018. For the twelve months ended December 31, 2019, net loss was $1.51 per share compared to $1.19 per share in the same period in 2018.

On March 16, 2020 Galectin Therapeutics Inc. (NASDAQ: GALT), the leading developer of therapeutics that target galectin proteins, reported financial results and provided a business update for the year ended December 31, 2019 (Press release, Galectin Therapeutics, MAR 16, 2020, View Source [SID1234555580]). These results are included in the Company’s Annual Report on Form 10-K, which has been filed with the U.S. Securities and Exchange Commission and is available at www.sec.gov.

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Harold H. Shlevin, Ph.D., President and Chief Executive Officer of Galectin Therapeutics, said, "The focus of the past few months has been on finalizing the refinements to our planned NASH-RX trial based on feedback from the U.S. Food and Drug Administration (FDA). Our Adaptive-Designed Phase 2b/3 trial protocol is nearly complete, and we anticipate initiating the trial in the second quarter of 2020. We have been very successful working with our partners to modify our initial trial design, incorporating a new biostatistics element to verify the rate of varices development and accordingly adjust trial sizing to help assure a key assumption related to the rate of varices development is met. Most recently, we strengthened our executive team with the addition of Dr. Pol F. Boudes, a Chief Medical Officer who has experience running NASH drug trials. In addition, as a result of the modifications to our trial, our clinical research organization Covance has been able to identify additional international clinical trial sites. Drug manufacturing capacity has been established to meet the needs of the entire trial. Belapectin (formerly known as GR-MD-02) is the first drug that has been shown to prevent the development of esophageal varices in patients with compensated NASH cirrhosis. If confirmed, these results would constitute a significant benefit for patients."

Richard E. Uihlein, Chairman of the Board, added, "I am extremely pleased with the progress achieved over the past few months. We are now in the final stages of finalizing our NASH-RX trial, which improves the likelihood of showing belapectin’s effects. And, with the recent addition of Dr. Boudes as Chief Medical Officer, we have a strong executive with extensive experience conducting trials of this nature. As always, our goal is to provide a therapy for the growing NASH epidemic around the world."

NASH-RX Trial Update

The NASH-RX trial is planned to use an adaptive design, confirm dose selection and reaffirm the efficacy data observed in the NASH-CX trial and, with pre-planned adaptations, inform the larger Phase 3 trial component. The adaptive design being considered allows pre-planned adjustments of the trial that may include, amongst other factors, optimization of dose selection, confirmation of efficacy and proof of concept observed in the NASH-CX trial, optimized sizing and statistical powering of the Phase 3 component, and possible inclusion of more advanced cirrhotic patients. We believe that these adaptations taken together should optimize conduct of the NASH-RX trial giving belapectin (GR-MD-02) the best opportunity to show a positive therapeutic effect. If the results of the NASH-RX trial are compelling, there could be the potential for accelerated FDA approval and/or partnership opportunity with a large pharmaceutical company.

The trial protocol is based on feedback from several interactions with the FDA during the last few months of 2019, including the November 14, 2019, telephone conference which included the FDA and Company representatives along with its co-primary investigators, biostatistical experts and other experts at Covance. In this meeting, the FDA indicated the new design was reasonable (subject to review of the protocol), and FDA indicated that they were still supportive of the surrogate end-point concepts originally proposed.

We believe the study design potentially could improve the likelihood of showing drug efficacy because:

It clarifies and reaffirms NASH-CX efficacy and safety at two distinct drug doses supported by robust pharmacokinetic analysis

It provides for appropriate selection of optimal dose – e.g., single dose (2 or 4 mg/kg) for Phase 3 component

A separate Hepatic Impairment study may allow inclusion of more severe patients who are believed to have a much higher rate of esophageal varices progression and bleeding and other decompensating events

Reduced frequency of esophagogastroduodenoscopys (EGD), elimination of biopsy endpoints and elimination of hepatic venous pressure gradient (HVPG) testing may make it easier to enroll trial patients and retain these patients during the duration of the trial.

Adaptation to size and power calculations based on sample size re-estimation and the interim analysis will allow better estimates of Phase 3 cohort sizing and of statistical power

A planned interim analysis after 18 months of completed treatment will assess affirmation of Phase 2 efficacy and safety results, help select a single optimal dosage, and inform the Phase 3 stage of the study, including its size.

In the Phase 3 component of this trial, the primary endpoint is development of new esophageal varices. Patients already enrolled for the Phase 2b component of the trial will continue on the selected single dose into the Phase 3 component of the trial. Patient selection for both Phase 2b and 3 components will be based on routine clinical signs of portal hypertension, including, amongst others, the presence or absence of varices, depressed platelet count (thrombocytopenia), enlargement of the spleen size and evidence of collateral blood vessels by imaging. The current study design and protocol are subject to modification after review by FDA.

The focus and goal of the therapeutic program is to prevent the development of large esophageal varices, which are strongly correlated with patient mortality due to sudden and severe bleeding. Based on the results of the NASH-CX trial, the clinical program will focus on patients who are at increased risk of developing varices, i.e. patients who have clinical signs of portal hypertension, such as low platelet counts or increased spleen size (splenomegaly).

The key milestones and associated target dates for the NASH-RX trial will be announced as elements of design of the trial are finalized based on the recent FDA feedback. However, we currently expect the first patient to be initiated in the second quarter of 2020. The study overall will likely involve approximately 130 sites in 11 countries in North America, Europe, Asia, and Australia.

Other Updates

Announced that Pol F. Boudes, M.D. has been appointed Chief Medical Officer – a key development for the company as it nears launch of its NASH-RX trial, an adaptively-designed Phase 3 trial in NASH cirrhosis. Dr. Boudes’ diverse background in drug development, especially his experience in NASH and other liver diseases, bolsters Galectin’s global advanced clinical development of belapectin for NASH cirrhosis.

Peer-reviewed publication, Scientific Presentations and Conferences

Gastroenterology, a prominent journal in the field of gastrointestinal disease, published a peer reviewed paper titled, "Effects of Belapectin, an Inhibitor of Galectin-3, in Patients with Nonalcoholic Steatohepatitis with Cirrhosis and Portal Hypertension," highlighting the potential prevention of esophageal varices of its NASH-CX Phase 2 clinical trial in NASH cirrhosis. We were greatly honored that such a prestigious, peer-reviewed publication felt the quality of our science merited the industry-wide attention they provide.

Initial results of the efforts at Galectin Sciences LLC (our majority-owned subsidiary) were presented by Dr. E. Zomer, Ph.D., Vice President, Discovery Research and Product Development, at the 3rd Annual Anti-Fibrotic Drug Development (AFDD) Summit regarding Galectin’s discovery program of its next generation of oral galectin-3 inhibitors. The presentation entitled "Therapeutic Integrin Inhibition," discussed the next generation of galectin-3 inhibitors, as well as the discovery of functional allosteric inhibitors.

Financial Results

For the year ended December 31, 2019, the Company reported a net loss applicable to common stockholders of $13.6 million, or ($0.26) per share, compared to a net loss applicable to common stockholders of $15.0 million, or ($0.38) per share for the full year 2018. The decrease is largely due a decrease in general and administrative, primarily stock-based compensation, and preferred stock dividends, somewhat offset by an increase in research and development expense.

Research and development expense for 2019 was $7.5 million compared with $6.5 million for 2018. The increase was primarily due to costs related to our NASH-RX clinical trial planning and site start-up and qualification processes globally, along with preparations and some preclinical activities incurred in support of the planned clinical program, such as development and reproductive toxicity studies, clinical supplies and other supportive activities, somewhat offset by lower non-cash stock compensation expenses. General and administrative expenses for 2019 were $6.0 million, down from $7.1 million for the full year 2018, primarily due to a decrease in non-cash stock-based compensation expenses.

As of December 31, 2019, the Company had $47.5 million of cash and cash equivalents. During 2019, the company effected a Rights Offering which, together with other common stock and warrants issued, raised $50.5 million in net proceeds. The Company also has a $10 million unsecured line of credit, under which no borrowings have been made to date. The Company believes it has sufficient cash, including availability under the line of credit, to fund currently planned operations and research and development activities through at least September 30, 2021.

The Company expects that it will require more cash to fund operations after September 30, 2021 and believes it will be able to obtain additional financing as needed. The total cost to obtain the interim efficacy data of the planned trial, including general overhead, is currently estimated to be approximately $125 million; however, the costs and timing of such trial are not yet completely finalized. These costs will require additional funding. There can be no assurance that we will be successful in obtaining financing to support our operations beyond September 30, 2021, or, if available, that any such financing will be on terms acceptable to us.

On March 16, 2020 Zai Lab Limited (NASDAQ: ZLAB), a China and U.S.-based innovative commercial stage biopharmaceutical company, reported the China National Medical Products Administration (NMPA) has accepted its supplemental New Drug Application (sNDA) for ZEJULA (niraparib) as a maintenance treatment of adult patients with advanced epithelial ovarian, fallopian tube, or primary peritoneal cancer who are in a complete or partial response to first-line platinum-based chemotherapy (Press release, Zai Laboratory, MAR 16, 2020, View Source [SID1234555578]).

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"We believe ZEJULA is a potential best-in-class PARP inhibitor due to its compelling efficacy, once-daily dosing and superior pharmacokinetic properties including its ability to cross the blood brain barrier," said Dr. Samantha Du, Founder and Chief Executive Officer of Zai Lab. "The NMPA’s acceptance of our sNDA submission for ZEJULA as a first-line monotherapy treatment after surgery and platinum-based chemotherapy has the potential to both fundamentally change how women with ovarian cancer are treated in China and significantly expand ZEJULA’s market opportunity. Zai Lab remains committed to make a meaningful impact on the way cancer is treated in China and globally, and we plan to continue to develop and bring many new and innovative treatment options to patients in need."

The PRIMA study conducted by our partner GlaxoSmithKline plc (GSK) demonstrated that ZEJULA treatment resulted in a 38% reduction in the risk of disease progression or death in the overall study population when compared to placebo. ZEJULA also demonstrated benefits in all patient subgroups. For patients whose cancer is associated with homologous recombination deficiency (HRD) positive status, ZEJULA treatment resulted in a 57% reduction in the risk of disease progression or death.

GSK submitted a sNDA to the U.S. FDA for the use of ZEJULA in ovarian cancer as first-line maintenance treatment based on the PRIMA study, and the application was accepted in February 2020 and is being reviewed under the Real-Time Oncology Review (RTOR) pilot program.

About Ovarian Cancer

Ovarian cancer is one of the most common gynecologic cancers in China with more than 52,000 newly diagnosed cases and 23,000 deaths each year. While platinum-based chemotherapy is effective at inducing an initial response in ovarian cancer, the disease will recur in the majority of women. New agents that prolong the duration of response following platinum-based treatment and delay the inevitable relapse of ovarian cancer will benefit patients with ovarian cancer in China.

About ZEJULA (niraparib)

ZEJULA (niraparib) is indicated as monotherapy for the maintenance treatment of adult patients with platinum-sensitive relapsed high grade serous epithelial ovarian, fallopian tube, or primary peritoneal cancer who are in response (complete or partial) to platinum-based chemotherapy. The NMPA recently accepted Zai Lab’s sNDA for niraparib is a monotherapy maintenance treatment in adult patients with advanced epithelial ovarian, fallopian tube, or primary peritoneal cancer who are in a complete or partial response to first-line platinum-based chemotherapy.

Zai Lab has ongoing pivotal studies in Chinese patients for first-line and second-line maintenance therapies in ovarian cancer. Zai Lab also conducted a Phase 1 pharmacokinetic (PK) study of niraparib in Chinese patients with ovarian cancer. This study was published in August 2019 in The Oncologist and demonstrated that the PK profile of niraparib in Chinese patients were comparable to that of patients evaluated in ZEJULA’s global PK study.

Zai Lab in-licensed rights to ZEJULA from GSK for Mainland China, Hong Kong and Macau. The NDA for recurrent ovarian cancer was accepted by the NMPA on December 12, 2018, granted priority review status on January 29, 2019 and approved on December 27, 2019. Zai Lab has obtained approvals to market ZEJULA in Mainland China, Hong Kong and Macau for maintenance therapy in patients with platinum-sensitive, recurrent ovarian cancer.

Since the Hong Kong launch of ZEJULA in October 2018, it has rapidly gained market share in the region despite being launched more than two years behind Lynparza. Based on IQVIA (formerly IMS) data, ZEJULA is now the market leading PARP inhibitor with market share in Hong Kong of 71% for the full year ended December 31, 2019.