On March 18, 2019 Akebia Therapeutics, Inc. (Nasdaq: AKBA), a biopharmaceutical company focused on the development and commercialization of therapeutics for patients with kidney disease, reported preliminary financial results for the full year ended December 31, 2018 and business highlights (Press release, Akebia, MAR 18, 2019, View Source [SID1234534452]).

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"2018 was a transformational year for Akebia," said John P. Butler, President and Chief Executive Officer of Akebia Therapeutics. "We successfully executed a number of strategic initiatives to advance our mission, and with the recent completion of our merger with Keryx, we have emerged as a leading, fully-integrated renal company with the potential to greatly improve care for patients with kidney disease. We are pleased with the robust growth we have seen with Auryxia (ferric citrate) during 2018. Looking ahead, we see the potential for substantial revenue growth in both the hyperphosphatemia and iron deficiency anemia markets."

Mr. Butler continued, "With respect to vadadustat, our lead product candidate, we believe that it has the potential to set a new standard of care for patients with anemia due to chronic kidney disease with a differentiated clinical profile. We recently announced positive top-line results from two pivotal phase 3 studies in Japan conducted by our collaboration partner, Mitsubishi Tanabe Pharma Corporation, which adds to the body of data supporting vadadustat’s potential to serve as a much-needed therapeutic option for patients with anemia due to chronic kidney disease. In addition, we continue to drive our global phase 3 program for vadadustat, with enrollment completed in the larger of the two INNO2VATE studies and enrollment expected to complete in the smaller INNO2VATE study by April 2019. The next 18 months will be a very busy time, with significant catalysts ahead of us."

Business Highlights

Auryxia

Pro forma unaudited Auryxia sales in 2018 were $96 million, representing 72% growth over 2017.

Total Auryxia prescriptions for 2018 were approximately 163,000, representing 85% growth over 2017.

Exit market share for Auryxia tablets in 2018 was 6.6% compared to 4.2% in 2017, exceeding the share gain of all other phosphate binders (branded and generic) in the same period.

Vadadustat Japanese Phase 3 Program

Announced positive top-line results from two phase 3, active-controlled, pivotal studies evaluating vadadustat in Japanese subjects with anemia due to chronic kidney disease (CKD).

Data from these two pivotal studies as well as from two additional single-arm studies in peritoneal dialysis and hemodialysis subjects, also recently announced, is expected to serve as the basis for a Japanese New Drug Application (JNDA) submission by Mitsubishi Tanabe Pharma Corporation (MTPC), expected in 2019.

Vadadustat Global Phase 3 Program

Enrollment in the larger of the two INNO2VATE studies (the "Conversion Study") was completed in February 2019, with a total of 3,554 subjects enrolled. Enrollment in the smaller INNO2VATE study (the "Correction Study"), enrolling approximately 350 subjects, is expected to be completed by April 2019. The company expects to report top-line data from both INNO2VATE studies in the second quarter of 2020, subject to the accrual of major adverse cardiac events (MACE).

The company expects enrollment in the PRO2TECT studies to complete in 2019, with up to approximately 3,700 subjects expected to be enrolled. Top-line results are anticipated in mid-2020, subject to the accrual of MACE.

The two INNO2VATE studies evaluating dialysis-dependent CKD subjects and the two PRO2TECT studies evaluating non-dialysis dependent CKD subjects are global, phase 3, active-controlled, open-label, non-inferiority, cardiovascular outcome studies of vadadustat for the treatment of anemia due to CKD.

Preliminary Financial Results (unaudited)

As a result of the completion of the company’s business combination with Keryx Biopharmaceuticals, Inc. (Keryx) on December 12, 2018 and the time required to complete the allocation of the merger consideration to the fair value of the acquired assets and liabilities as well as the assessment of the associated tax impacts, the company is announcing preliminary results for the full year 2018, which are based on currently available information and are subject to revision, as further discussed below. The company anticipates a delayed filing of its Annual Report on Form 10-K for the fiscal year ended December 31, 2018 (Annual Report) and plans to file a Form 12b-25, Notification of Late Filing, with the Securities and Exchange Commission, which will provide the company with a 15 calendar-day extension.

Net product revenues for Auryxia from December 12, 2018, the date of our merger with Keryx, through December 31, 2018 were $6.8 million. The company did not have any product revenue prior to our merger with Keryx.

Collaboration revenues for the fourth quarter of 2018 were $53.0 million, compared with $90.6 million during the same period in 2017. The decrease was primarily due to $42.9 million of deferred revenue associated with the MTPC collaboration agreement being recognized as revenue during the fourth quarter of 2017, as the criteria for revenue recognition were satisfied in that quarter. No revenue was recognized under the MTPC collaboration agreement in the fourth quarter of 2018.

Collaboration revenues for the full year 2018 were $200.9 million compared to $181.2 million for the full year 2017. The increase was due to increased revenues recognized under the collaboration agreements with Otsuka Pharmaceutical Co. Ltd. (Otsuka). Through 2018, Otsuka has funded 52.5% of the company’s phase 3 development costs for vadadustat, and in the second quarter of 2019, Otsuka is expected to increase its contribution to 80%.

Cost of goods sold was $6.3 million for the period from December 12, 2018 through December 31, 2018, consisting primarily of costs associated with the manufacture of Auryxia and $4.8 million for the fair-value inventory step-up as a result of the merger accounting.

Research and development expenses were $87.1 million for the fourth quarter of 2018 compared to $68.4 million for the fourth quarter of 2017, and $291.1 million for the full year 2018 compared to $230.9 million for the full year 2017. The increase for both periods is primarily attributable to an increase in external costs related to the continued advancement of the PRO2TECT and INNO2VATE phase 3 program, as well as increased headcount and consulting costs required to support our expanding research and development programs.

Selling, general and administrative expenses were $55.1 million for the fourth quarter of 2018 compared to $7.6 million for the fourth quarter of 2017, and $87.1 million for the full year 2018 compared to $27.0 million for the full year 2017. The increase in selling, general and administrative expenses is primarily attributable to merger-related costs of $49.5 million, of which $41.7 million was incurred in the fourth quarter of 2018, including a non-cash expense of $13.4 million related to the issuance of shares to Baupost Group Securities, L.L.C. in connection with its conversion of Keryx convertible notes. The increase for both periods was also attributable to an increase in costs to support our research and development programs, including headcount, information technology and compensation-related costs.

Akebia reported a pre-tax net loss for the fourth quarter of 2018 of $88.4 million, as compared to a pre-tax net income of $15.5 million for the fourth quarter of 2017. The pre-tax net loss for the fourth quarter of 2018 includes total merger-related costs of $46.5 million. The pre-tax net income reported for the fourth quarter of 2017 was attributable to $42.9 million of collaboration revenue recognized in connection with the MTPC collaboration agreement, as the criteria for revenue recognition was satisfied in the fourth quarter.

For the full year 2018, the Company reported a pre-tax net loss of $171.9 million, as compared to a pre-tax net loss for the full year 2017 of $73.7 million. The pre-tax net loss for the full year 2018 includes total merger-related costs of $54.3 million.

Akebia ended 2018 with cash, cash equivalents and available-for-sale securities of $321.6 million. The company expects its cash resources, including the prepaid quarterly committed cost-share funding from its collaboration partners, to fund its current operating plan into the third quarter of 2020.

Conference Call and Webcast

Akebia management will host its full-year 2018 and business highlights conference call and webcast beginning at 4:30 p.m. Eastern Time today, March 18, 2019.

The conference call can be accessed by dialing (877) 458-0977 within the United States and Canada and (484) 653-6724 for all other locations. The confirmation code is 1376157. Participants should dial in 10 minutes prior to the scheduled start time.

A live webcast of the conference call will be available in the "Investors" section of the company’s website at www.akebia.com.

Beginning the morning of March 19, 2019, the call will be available for replay via telephone and the archived webcast will be available on Akebia’s website. To listen to the telephone replay, dial (855) 859-2056 (U.S. and Canada) or (404) 537-3406 (international) using conference ID number 1376157. The telephone replay will be available for six days following the call.

On March 18, 2019 Transgene (Euronext Paris: TNG), a biotech company that designs and develops virus-based immunotherapies against cancers and infectious diseases, reported that it has secured a €20 million revolving credit facility with Natixis, the French Corporate and Investment bank (Press release, Transgene, MAR 18, 2019, View Source [SID1234621822]).

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The credit facility will have a 30-month term and Transgene will be able to draw on and repay the facility at its discretion.

Transgene has used its shares in the Chinese biotech company Tasly Biopharmaceuticals as collateral for this loan. As a reminder, Transgene became a Tasly Biopharmaceuticals shareholder in July 2018 and holds 2.5% of its capital as the result of a series of agreements under which Transgene transferred to Tasly Biopharmaceuticals its Chinese rights to T601 and T101, two immunotherapies discovered by Transgene and which are currently being developed by Tasly Biopharmaceuticals in Greater China. Tasly Biopharmaceutials has announced its intention to list its shares on the Hong Kong Stock Exchange.

Jean-Philippe Del, Vice President, Finance, said: "I am glad that we have been able to monetize our shareholding in Tasly Biopharmaceutical to extend our cash runway. With this new loan facility, we now have the funds needed to support our clinical and pre-clinical activities until mid-2020."

On March 18, 2019 Alligator Bioscience (Nasdaq Stockholm: ATORX), reported that they will present preclinical data for the drug candidate ATOR-1017 at the 4th Annual Immuno-oncology Summit Europe, to be held in London on March 18-22, 2019 (Press release, Alligator Bioscience, MAR 18, 2019, View Source [SID1234538668]).

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ATOR-1017 is a monoclonal antibody in development for the treatment of metastasizing cancer. It activates the costimulatory receptor 4-1BB and its immunostimulatory function is dependent on cross-linking to Fc-gamma receptors on immune cells.

The new data show that ATOR-1017, in an experimental model for colon cancer (MC38), displays potent anti-tumor effects. Furthermore, ATOR-1017 induces a dose-dependent effect on tumor growth inhibition and survival. Existing preclinical data have already shown that ATOR-1017 stimulates both NK (Natural Killer) and T cells, both of which contribute to an effective immune-mediated killing of tumor cells.

"ATOR-1017 is designed to have a superior safety and efficacy profile through its tumor-directed properties and these latest data support the positioning of ATOR-1017 as a best-in-class 4-1BB antibody with the potential to reduce side effects, but also to generate a potent and long-lasting immune response. This provide us with a strong preclinical package to move into the next phase of development. We expect to start clinical trials in cancer patients later this year," said Christina Furebring, SVP Preclinical Development, at Alligator Bioscience.

Dr Karin Enell Smith, Senior Scientist Immuno-oncology at Alligator, will hold an oral presentation with the title: "ATOR-1017 – A Tumor Directed Fcγ-Receptor Cross Linking Dependent 4-1BB Agonistic Antibody" on Monday, March 18, 5:45 p.m GMT (6:45 p.m. CET).

For further information, please contact:
Cecilia Hofvander, Director Investor Relations & Communications
Phone +46 46 540 82 06
E-mail: [email protected]

This information is such information as Alligator Bioscience AB (publ) is obliged to make public pursuant to the EU Market Abuse Regulation. The information was submitted for publication, through the agency of the contact person set out above, at 1:30 p.m. CET on March 18, 2019.

About ATOR-1017
ATOR-1017 is an immunostimulatory antibody (IgG4) that binds to the costimulatory receptor 4-1BB (also known as CD137) expressed on tumor-specific T cells and NK cells.
4-1BB has the capacity to support the immune cells involved in tumor control, making 4-1BB a particularly attractive target for cancer immunotherapy.

ATOR-1017 is differentiated from other 4-1BB antibodies, partly because of its unique binding profile, but also because its immunostimulatory function is dependent on cross-linking to Fc-gamma receptors on immune cells. The aim is to achieve effective tumor-targeted immune stimulation with minimum side effects. ATOR-1017 is planned to enter clinical studies 2019.

On March 18, 2019 Stemline Therapeutics, Inc. (Nasdaq: STML), a commercial-stage biopharmaceutical company focused on the development and commercialization of novel oncology therapeutics, reported that Ivan Bergstein, M.D., Stemline’s CEO, was selected to participate as a panelist on "The Brave New World of Myeloid Tumors (Press release, Stemline Therapeutics, MAR 18, 2019, View Source [SID1234534420])." The panel will take place on Monday, March 18 at 3 PM PT during the 31st Annual ROTH conference at the Ritz-Carlton Laguna Niguel, CA.

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About CD123
CD123 is a cell surface target expressed on a wide range of myeloid tumors including blastic plasmacytoid dendritic cell neoplasm (BPDCN), certain myeloproliferative neoplasms (MPNs) including chronic myelomonocytic leukemia (CMML) and myelofibrosis (MF), acute myeloid leukemia (AML) (and potentially enriched in certain AML subsets), myelodysplastic syndrome (MDS), and chronic myeloid leukemia (CML). CD123 has also been reported on certain lymphoid malignancies including multiple myeloma (MM), acute lymphoid leukemia (ALL), hairy cell leukemia (HCL), Hodgkin’s lymphoma (HL), and certain Non-Hodgkin’s lymphomas (NHL). In addition, CD123 has been detected on some solid tumors as well as autoimmune disorders including cutaneous lupus and scleroderma.

About ELZONRIS
ELZONRIS (tagraxofusp), a CD123-directed cytotoxin, was approved by the Food and Drug Administration (FDA) on December 21, 2018 for the treatment of adult and pediatric patients, two years and older, with blastic plasmacytoid dendritic cell neoplasm (BPDCN). For full prescribing information in the U.S., visit www.ELZONRIS.com. In November 2018, the European Medicines Agency (EMA) granted ELZONRIS accelerated assessment to the marketing authorization application (MAA), which was submitted to, and validated by, the EMA in January 2019. ELZONRIS is also being evaluated in additional clinical trials in other indications including chronic myelomonocytic leukemia (CMML), myelofibrosis (MF) and acute myeloid leukemia (AML).

About BPDCN
BPDCN is an aggressive hematologic malignancy with historically poor outcomes and an area of unmet medical need. BPDCN typically presents in the bone marrow and/or skin and may also involve lymph nodes and viscera. The BPDCN cell of origin is the plasmacytoid dendritic cell (pDC) precursor. The diagnosis of BPDCN is based on the immunophenotypic diagnostic triad of CD123, CD4, and CD56, and other markers. For more information, please visit the BPDCN disease awareness website at www.bpdcninfo.com.

On March 18, 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 full-year ended December 31, 2018 and provided a corporate update (Press release, Protalix, MAR 18, 2019, View Source [SID1234534453]).

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"Throughout 2018 and into early 2019, we significantly advanced our clinical development program for PRX-102 and, as of today, have enrolled 127 Fabry disease patients across all of our PRX-102 clinical trials. Most recently, we had a very productive meeting with the U.S. Food and Drug Administration (FDA) to discuss the potential filing of an application for accelerated approval and, at the FDA’s request, we intend to hold a follow up meeting by the end of the second quarter of 2019 to discuss the data and content of the potential filing for accelerated approval. We are very pleased with the collaborative manner our engagement with the FDA has been to date and cautiously optimistic about the prospects of our discussions with the agency," said Mr. Moshe Manor, Protalix’s President and Chief Executive Officer. "We are very excited as we move forward into 2019 which could be a transformational year for us, including the read outs from our Fabry trials and the potential for establishing the path for accelerated approval for PRX-102 with the FDA."

2018 Clinical and Corporate Highlights

Pegunigalsidase alfa (PRX-102) for the treatment of Fabry Disease

·Recently the Company held a very productive meeting with the FDA regarding the potential path for accelerated BLA approval for PRX-102.
·The FDA confirmed that the Company’s PRX-102 program could rely on surrogate endpoints as part of the basis for a potential accelerated BLA approval.
·The FDA urged the Company to apply for a follow up Type C meeting as soon as possible to review with the agency the data and content of such potential accelerated filing application.
·The discussion with the FDA revolved around the data the Company has generated from all of its PRX-102 clinical trials to date, primarily the kidney biopsy results and the eGFR data, that could be included in the potential application for accelerated approval.
·In January 2018, the Company received fast track designation from the FDA for PRX-102.
·In May 2018, the Company reported on the baseline characteristics for its BALANCE phase III clinical trial for the treatment of Fabry disease highlighting that PRX-102 is less inhibited by preexisting neutralizing antibodies compared to Fabrazyme, and, therefore, has the potential to attenuate renal decline and/or stabilize renal function in patients who have not had an optimal clinical response to Fabrazyme.
·In July 2018, the Company expanded its partnership with Chiesi Farmaceutici S.p.A., or Chiesi, to include exclusive U.S. rights for the development and commercialization of PRX-102. The terms of the agreement include an up-front payment of $25 million, up to $20 million in development costs, up to $760 million, in the aggregate, in regulatory and commercial milestone payments and tiered royalties ranging from 15 to 40%.

·In October 2018, the Company presented positive preliminary data from its BRIDGE phase III clinical trial for the treatment of Fabry disease indicating a significant improvement in kidney function in patients switched from agalsidase alfa (Replagal) to PRX-102 at the 1st Canadian Symposium on Lysosomal Diseases.
·In December 2018, the enrollment for the BRIDGE phase III clinical trial for the treatment of Fabry disease was completed.
·As of today, the BALANCE trial is over 80% enrolled and the BRIGHT trial is over 90% enrolled.
·Based on the FDA discussion during our recent meeting we believe that the potential filing for accelerated approval might be based on data the Company has already generated in its clinical trials of PRX-102.
·Currently, substantially all patients treated in the BRIGHT trial have remained on the once-monthly 2mg/kg dosing regimen. All of the 13 patients that have completed the 12-month study have opted, together with their treating physician’s advice, to continue with once-monthly dosing in an extension study rather than switching back to the 1mg/kg every two weeks regimen.

Oral antiTNF (OPRX-106) for Ulcerative Colitis

·Throughout 2018, the Company reported positive results from its phase II clinical trial of OPRX-106 for the treatment of ulcerative colitis. Final results demonstrated as follows:
oclinical response in 67% of patients and clinical remission in 28% of patients;
omucosal improvement in 61% of patients, with 33% achieving mucosal healing; and
o89% of the patients experienced a reduction in Mayo Score, and 61% of the patients experienced a reduction in endoscopic sub score.
·The Company is evaluating the best path forward which could include initiating next-stage development internally or collaborate with potential parties.

Alidornase alfa (PRX-110) for the treatment of Cystic Fibrosis

·In 2018, the Company received valuable feedback on PRX-110 from potential partners. While the data generated to date is very encouraging, further analysis will likely be required to maximize the potential value of this asset. Given the Company’s focused cash utilization, it does not plan to conduct further development of PRX-110 at this time.

Full-Year 2018 Financial Results

·During the preparation of the 2018 annual report, the Company reevaluated its revenue recognition policies and determined that certain revenues generated under the Company’s license agreements should be recognized for accounting purposes. Previously, the Company did not recognize those revenues.

·The Company recognized revenues from license and R&D services equal to $2.2 million, $2.8 million and $11.7 million over the first, second and third quarters of 2018, respectively. The restatement is expected to decrease the Company’s loss for each of those periods.

·The Company recorded total revenues of $34.2 million during the year ended December 31, 2018, which was comprised of $9.0 million from selling goods and $25.2 million from license revenues, compared to $19.2 million from selling goods, and $1.8 million from license and R&D services for the same period of 2017.

·Research and development expenses for the year ended December 31, 2018, were $33.3 million, compared to $28.8 million for the same period of 2017. Selling, general and administrative expenses for the year ended December 31, 2018 were $10.9 million, compared to $11.5 million incurred during the same period of 2017.

·Operating loss for the year ended December 31, 2018 was $19.3 million compared to $34.5 million for the year ended December 31, 2017.

·For the year ended December 31, 2018, the Company reported a net loss of $26.5 million, or $0.18 per share, basic and diluted, compared to $45.4 million, excluding a one-time, non-cash net charge of $38.1 million in connection with the remeasurement of a derivative, or $0.35 per share, basic and diluted, for the same period of 2017.

·On December 31, 2018, the Company had $37.8 million of cash and cash equivalents, compared to $51.2 million on December 31, 2017, which is currently projected to fund operations into mid-2020. As of December 31, 2018, a total of $57.9 million aggregate principal amount of the Company’s 7.5% convertible notes due November 2021 was outstanding.

Conference Call and Webcast Information

The Company will host a conference call on Monday, March 18, 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 9583103.

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.