On August 6, 2019 Aurinia Pharmaceuticals Inc. (NASDAQ: AUPH / TSX:AUP) ("Aurinia" or the "Company"), a late- stage clinical biopharmaceutical company focused on advancing voclosporin in multiple indications, reported financial results for the three and six months ended June 30, 2019 and recent operational highlights (Press release, Aurinia Pharmaceuticals, AUG 6, 2019, View Source [SID1234538319]). Amounts, unless specified otherwise, are expressed in U.S. dollars.

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"During the past quarter, the Aurinia team has taken multiple steps forward preparing the organization for the next phase of its evolution into a commercial-stage entity. In anticipation of the AURORA Phase 3 results in lupus nephritis, we are appropriately scaling the organization ahead of data, which if confirmatory, sets the stage for a NDA filing during the first half of next year. If approved, we project a commercial launch of voclosporin in early 2021 as a potentially first-line treatment, in combination with mycophenolate mofetil and low-dose steroids for lupus nephritis," commented Peter Greenleaf, President and Chief Executive Officer of Aurinia.

Michael Martin, Chief Operating Officer stated, "In parallel, we are taking all of the steps necessary to initiate a Phase 2/3 clinical trial with VOS for the potential treatment of DES. This Phase 2/3 trial follows a well-established development path for ophthalmic drugs in this therapeutic area."

Mr. Greenleaf further said, "With respect to our ongoing proof-of-concept study for FSGS, we have added additional clinical trial sites and have modified the study protocol to broaden the inclusion of patients who have received limited corticosteroids exposure prior to enrollment. We expect that these actions will lead to enhanced enrollment."

VOS Phase 2/3 AUDREY ("Aurinia Dry Eye Study") Clinical Study in DES

Based upon the VOS exploratory Phase 2a results, Aurinia has initiated plans for a Phase 2/3 study which is expected to enroll its first patient in the fourth quarter of 2019. This study will include certain critical regulatory requirements that the FDA has traditionally required for DES product approval, which include both dose-optimization along with a comparison to vehicle.

The Phase 2/3 clinical study otherwise known as, "THE AUDREY STUDY" is designed as a randomized, double-masked, vehicle-controlled, dose ranging study to be conducted in the U.S., AUDREY will evaluate the efficacy and safety of VOS in subjects with DES. Approximately 480 subjects are to be enrolled. The study will consist of four arms and in a 1:1:1:1 randomization schedule to either 0.2% VOS, 0.1% VOS, 0.05% VOS or vehicle, dosed twice daily for 12 weeks. The primary outcome measure for the study is the proportion of subjects with 10mm improvement in Schirmer Tear Test ("STT") at 4 weeks.

Secondary outcome measures, which will be assessed at multiple time points include, STT, Fluorescein Corneal Staining ("FCS"), change in eye dryness, burning/stinging, itching, photophobia, eye pain and foreign body sensation, change in Symptom Assessment in Dry Eye ("SANDE") score and additional safety endpoints.

Voclosporin for Focal Segmental Glomerulosclerosis ("FSGS")

Aurinia initiated a Phase 2 proof-of-concept, open-label study for FSGS in June 2018. The study was designed to evaluate the role of voclosporin for treatment-naïve patients diagnosed with primary FSGS. In response to slower than anticipated enrollment, Aurinia has recently opened additional clinical trial sites outside of the United States and has amended the study to allow enrollment of FSGS patients who have previously received limited corticosteroid exposure. Up to approximately 20 patients are expected to be enrolled with interim results anticipated in 2020.

Financial Liquidity at June 30, 2019

As at June 30, 2019, Aurinia had cash and cash equivalents of $131.5 million compared to $144.3 million of cash, cash equivalents and short-term investments at March 31, 2019 and $125.9 million at December 31, 2018. Net cash used in operating activities was $13.3 million for the second quarter ended June 30, 2019 compared to $12.3 million for the second quarter ended June 30, 2018.

The Company believes, that based on its current plans that it has sufficient financial resources to fund the existing LN program, including the AURORA trial and the AURORA 2 extension trial, complete the NDA submission to the FDA, conduct the ongoing Phase 2 study for FSGS, initiate the AUDREY Phase 2/3 study, and fund operations into the second half of 2020.

Second Quarter 2019 Financial Results

For the three months ended June 30, 2019 Aurinia reported a consolidated net loss of $15.9 million or $0.17 per common share compared to a consolidated net loss of $15.7 million or $0.19 per common share for the same period in 2018.

Research and development expenses (R&D) increased slightly to $11.2 million for the three months ended June 30, 2019, compared to $10.5 million for the three months ended June 30, 2018. The increase in these expenses reflected higher costs incurred for the AURORA 2 extension trial, the drug-drug interaction ("DDI") study preparation costs associated with the planned NDA submission for LN and preparation costs for the Phase 2/3 DES clinical study.

Corporate, administration and business development expenses increased to $4.9 million for the three months ended June 30, 2019, compared to $3.5 million for the same period in 2018. The increase was primarily due to an increase in consulting fees related to recruitment fees and pre-commercial activities, such as market and payor research, and higher personnel and sponsorship costs.

Non-cash stock compensation expense was $2.0 million for the second quarter ended June 30, 2019 as compared with $2.0 million for the same period in 2018 and is included in both research and development and corporate, general and business development expenses.

Aurinia incurred other expenses of $720,000 during the three months ended June 30, 2019 associated with the successful defense of a proxy contest in connection with its June 26, 2019 annual general meeting. There was no similar expense in the comparable period.

Aurinia also recorded a non-cash reduction of $625,000 in the estimated fair value of derivative warrant liabilities which reduced the loss for the second quarter ended June 30, 2019 compared to an increase of $1.9 million in the estimated fair value of derivative warrant liabilities which increased the loss for the second quarter ended June 30, 2018. The derivative warrant liabilities will ultimately be eliminated on the exercise or forfeiture of the warrants and will not result in any cash outlay by the Company.

Financial Results for Six Months Ended June 30, 2019

For the six months ended June 30, 2019, Aurinia reported a consolidated net loss of $28.3 million or $0.31 per common share compared to a consolidated net loss of $31.2 million or $0.37 per common share for the comparable period in 2018.

R&D expenses were $21.8 million for the six months ended June 30, 2019 compared to $19.4 million for the same period in 2018. The increase in these expenses reflected higher costs incurred for the AURORA 2 extension trial, the DDI study and preparation costs associated with the planned LN NDA submission partially offset by lower AURORA trial costs as this trial nears completion.

Corporate, administration and business development expenses were $8.8 million for the six months ended June 30, 2019 compared to $7.3 million for the same period in 2018. The increase reflects the same items as noted in the second quarter corporate, administration and business development expenses.

Non-cash stock compensation expense totaled $3.6 million for the six months ended June 30, 2019 as compared with $4.1 million for the same period in 2018 and is included in both research and development and corporate, general and business development expenses.

For the six months ended June 30, 2019 Aurinia recorded a decrease of $2.4 million in the estimated fair value of derivative warrant liabilities compared to an increase of $4.6 million for the comparable period in 2018.

This press release should be read in conjunction with our unaudited interim condensed consolidated financial statements and the Management’s Discussion and Analysis for the second quarter ended June 30, 2019 which are accessible on Aurinia’s website at www.auriniapharma.com, on SEDAR at www.sedar.com or on EDGAR at www.sec.gov/edgar.

Aurinia will host a conference call and webcast to discuss the second quarter ended June 30, 2019 financial results today, Tuesday, August 6, 2019 at 4:30 p.m. ET. This event can be accessed on the investor section of the Aurinia website at www.auriniapharma.com.

On August 6, 2019 Agenus Inc. (NASDAQ: AGEN), an immuno-oncology company with a pipeline of immune checkpoint antibodies and cancer vaccines, reported that it will release its second quarter 2019 financial results before the market opens on Thursday, August 8, 2019 (Press release, Agenus, AUG 6, 2019, View Source [SID1234538185]). Agenus executives will host a conference call and webcast at 8:30 a.m. ET the same day.

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To access the live call, dial 1-844-492-3727 (U.S.) or 1-412-317-5118 (International) and ask to be joined into the Agenus call. The call will also be webcast and will be accessible from the Company’s website at View Source or with this link View Source

A replay will be available on the Company’s website approximately two hours after the call and will remain available until November 7, 2019.

On August 6, 2019 CytomX Therapeutics, Inc. (Nasdaq:CTMX), a clinical-stage oncology-focused biopharmaceutical company pioneering a novel class of investigational antibody therapeutics based on our Probody therapeutic technology platform, reported that it will present at the 2019 Wedbush PacGrow Healthcare Conference (Press release, CytomX Therapeutics, AUG 6, 2019, View Source [SID1234538203]). Sean McCarthy, D.Phil., president, chief executive officer and chairman, will present a corporate overview on August 13, 2019, at 10:20 a.m. ET.

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A live audio webcast of the presentation will be available through the Investors and News section of CytomX’s website. An archived replay will be available for 30 days following the event.

On August 6, 2019 Gamida Cell Ltd. (Nasdaq: GMDA), a leading cellular and immune therapeutics company, reported financial results for the quarter ended June 30, 2019 (Press release, Gamida Cell, AUG 6, 2019, View Source [SID1234538219]). The company also highlighted continued progress in advancing its clinical development candidates: omidubicel, an investigational advanced cell therapy in Phase 3 clinical development designed to enhance the life-saving benefits of hematopoietic stem cell (bone marrow) transplant for patients with hematologic malignancies, and GDA-201, an investigational, natural killer (NK) cell-based cancer immunotherapy in Phase 1 development in patients with non-Hodgkin lymphoma and multiple myeloma.

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"During the past quarter, Gamida Cell made important progress toward its goal of transforming the treatment landscape for patients with blood cancers and rare, serious hematologic diseases," stated Julian Adams, Ph.D., chief executive officer of Gamida Cell. "We are continuing to progress our multi-center, randomized Phase 3 study of omidubicel to enable a topline data readout, which is expected in the first half of 2020. As we look ahead toward the potential submission of a biologics license application next year, we are advancing key activities required to bring omidubicel to patients in a commercial setting. To help ensure that we will have sufficient and reliable commercial supply, we established a commercial manufacturing supply agreement with Lonza and engaged Biopharmax, a biopharmaceutical design and construction firm, to initiate the construction of our own commercial manufacturing facility in Israel."

"Our second cell therapy program, GDA-201, is also moving forward. We anticipate additional data from the ongoing Phase 1/2 clinical study in the second half of the year. We are also on track with our plans to develop a cryopreserved formulation of GDA-201 to enable a multi-center clinical study in patients with non-Hodgkin lymphoma," Dr. Adams continued. "Both omidubicel and GDA-201 are based on our proprietary cell expansion platform, which has the potential to further expand our pipeline. In June, we appointed Dr. Tracey Lodie to our team as chief scientific officer to set our scientific strategy and lead new translational research to further elucidate the potential of our technology and clinical development programs."

Program Highlights

Continued to advance the Phase 3 clinical study of omidubicel: Patient enrollment continued to progress in the Gamida Cell’s Phase 3 study of omidubicel in patients with high-risk hematologic malignancies. The international, randomized, multi-center study is designed to evaluate the safety and efficacy of omidubicel compared to standard umbilical cord blood for allogeneic bone marrow transplant in approximately 120 patients with no available matched donor. The company anticipates completing patient enrollment by the end of this year with topline data anticipated in first half of 2020.
Established agreements to support commercial manufacturing for omidubicel: In June, Gamida Cell and Lonza announced that the companies entered into a strategic manufacturing agreement for the future commercial production after potential FDA approval of omidubicel. This agreement follows a successful multi-year clinical manufacturing relationship and provides Gamida Cell with a path to commercial supply of omidubicel. Under this multi-year agreement, Lonza will construct and dedicate production suites at its Geleen, NL site for the anticipated commercial launch. Additionally, the agreement enables Gamida Cell to increase the number of dedicated production suites over time to ensure commercial supply. Gamida Cell also has the option of expanding further into Lonza’s global cell and gene therapy manufacturing network. In August, Gamida Cell signed an agreement with Biopharmax for the construction of suites for the commercial manufacture of omidubicel after potential FDA approval of omidubicel at a Gamida Cell-operated facility in Israel.
Initiated enrollment for Cohort 2 in the Phase 1/2 study of omidubicel in patients with severe aplastic anemia: In June, patient enrollment began in Cohort 2 of the investigator-sponsored, Phase 1/2 clinical study of omidubicel in patients with severe aplastic anemia, a rare and life-threatening blood disorder. Earlier this year, encouraging data from Cohort 1 were reported at the 2019 Transplantation & Cellular Therapy (TCT) Meeting. All three patients enrolled in Cohort 1 successfully underwent a bone marrow transplant consisting of omidubicel plus a haploidentical stem cell graft. The rapid engraftment, sustained hematopoiesis and accelerated immune recovery observed in these patients enabled the initiation of Cohort 2, where patients will be treated with omidubicel as a stand-alone graft.
Demonstrated continued progress with GDA-201 clinical development program: Gamida Cell continued to make progress with the GDA-201 clinical development program. The investigator-sponsored, Phase 1/2 clinical study of GDA-201 in patient with non-Hodgkin lymphoma and multiple myeloma is ongoing, with additional data expected in the second half of 2019. The company is developing a cryopreserved formulation of GDA-201 to enable a multi-center, multi-dose Phase 1/2 clinical study in patients with non-Hodgkin lymphoma, which is expected to begin next year.
Corporate Highlights

Completed public follow-on offering of approximately $40 million in gross proceeds: In July, Gamida Cell announced that the company closed an underwritten public offering of 7,000,000 ordinary shares and that the underwriters exercised in full their option to purchase an additional 1,050,000 ordinary shares at the public offering price of $5.00 per share. The aggregate gross proceeds to Gamida Cell from the offering, including the shares sold pursuant to the underwriters’ option, before deducting underwriting discounts and commission and offering expenses, were $40.3 million.
Bolstered management team with appointment of Tracey Lodie, Ph.D., as chief scientific officer: In June, the company announced the appointment of Tracey Lodie, Ph.D., as chief scientific officer. Prior to joining Gamida Cell, Dr. Lodie served as senior vice president, translational immunology at BlueRock Therapeutics, where she helped to advance their universal pluripotent stem cell platform into central nervous system, cardiovascular, and autoimmune therapeutic areas. She also served as vice president of immunology at Syros Pharmaceuticals, where she developed new autoimmunity and immuno-oncology research programs. Prior to Syros Pharmaceuticals, Dr. Lodie spent over 14 years at Sanofi-Genzyme, where she held roles of increasing responsibility. She obtained a PhD. in immunology and pathology at Boston University School of Medicine before completing a post-doctoral fellowship at Beth Israel Deaconess Medical Center in the Department of Hematology/Oncology.
Shawn Cline Tomasello and Stephen Wills elected to Board of Directors, reflecting company’s progress toward commercialization: In June, Shawn Cline Tomasello and Stephen T. Wills were elected to Gamida Cell’s board of directors. Ms. Tomasello has extensive experience in commercializing first-in-class medicines for the treatment of cancer, including Yescarta (at Kite Pharma, now part of Gilead Sciences) and Imbruvica (at Pharmacyclics, now part of AbbVie). Mr. Wills has extensive operational, financial and transactional experience over nearly three decades in the life sciences and accounting industries. He has served as chief financial officer of Palatin Technologies, a publicly-traded biotechnology company developing peptide therapeutics, since 1997 and also serves as Palatin’s chief operating officer and executive vice president.
Anticipated 2019-2020 Milestones

Gamida Cell’s anticipated program milestones in 2019-2020 are as follows:

Omidubicel

Complete enrollment in Phase 3 study of omidubicel in patients with hematologic malignancies by the end of 2019
Report topline data from the Phase 3 study of omidubicel in patients with hematologic malignancies in the first half of 2020
Complete BLA submission for omidubicel in hematologic malignancies in the second half of 2020, should Phase 3 data be positive
GDA-201

Complete patient enrollment in the ongoing Phase 1 study in the second half of 2019
Present additional data at a medical meeting in the second half of 2019
Initiate multi-center, Phase 1/2 clinical study in patients with NHL in 2020
Second Quarter 2019 Financial Results

Research and development (R&D) expenses in the second quarter of 2019 were $7.0 million and were also $7.0 million in the same period in 2018. R&D expenses were higher in the second quarter of 2019 compared to the same period in 2018 due to the advancement of omidubicel and GDA-201 but were offset by a $2.0 million increase in grants related to the Israeli Innovation Authority (IIA).
General and administrative expenses were $3.8 million for the second quarter of 2019, compared to $2.9 million in the same period in 2018. The difference was attributable mainly to a $0.4 million increase in cash and non-cash expenses related to hiring and establishing the U.S. headquarters as well as a $0.5 million increase in professional services, including an increase in expenses associated with being a publicly-traded company.
Finance income, net, was $16.8 million for the second quarter of 2019, compared to finance expenses, net, of $3.2 million in the same period in 2018. The net increase was primarily due to non-cash income resulting from the re-valuation of warrants, offset by non-cash expenses from the re-valuation of the IIA royalty-bearing grant liability.
Net income for the second quarter of 2019 was $6.0 million, compared to a net loss of $13.1 million in the same period in 2018.
As of June 30, 2019, Gamida Cell had total cash, cash equivalents and available-for-sale securities of $41.7 million, compared to $60.7 million as of December 31, 2018. The June 30, 2019, cash position excludes the aggregate gross proceeds from the company’s recent public follow-on offering, which were $40.3 million.

2019 Financial Guidance
Gamida Cell continues to expect cash used for ongoing operating activities in 2019 to range from $35 million to $40 million, reflecting anticipated expenditures to advance the company’s clinical programs.

Gamida Cell expects that its cash, cash equivalents and available-for-sale securities will support the company’s ongoing operating activities into the fourth quarter of 2020. This cash runway guidance is based on the company’s current operational plans and excludes any additional funding that may be received or business development activities that may be undertaken.

Conference Call Information
Gamida Cell will host a conference call today, August 6, 2019, at 8:30 a.m. ET to discuss these financial results and company updates. A live webcast of the conference call can be accessed in the "Investors" section of Gamida Cell’s website at www.gamida-cell.com. To participate in the live call, please dial 866-930-5560 (domestic) or 409-216-0605 (international) and refer to conference ID number 2127937. A replay of the webcast will be available for approximately 30 days.

About Omidubicel
Omidubicel (formerly known as NiCord), the company’s lead clinical program, is an advanced cell therapy under development as a potential life-saving allogeneic hematopoietic stem cell (bone marrow) transplant solution for patients with hematologic malignancies (blood cancers). Omidubicel is the first bone marrow transplant product to receive Breakthrough Therapy Designation from the U.S. Food and Drug Administration and has also received Orphan Drug Designation in the U.S. and EU. In a Phase 1/2 clinical study, omidubicel demonstrated rapid and durable time to engraftment and was generally well-tolerated.1 A Phase 3 study evaluating omidubicel in patients with leukemia and lymphoma is ongoing in the U.S., Europe and Asia.2 Omidubicel is also being evaluated in a Phase 1/2 clinical study in patients with severe aplastic anemia.3 The aplastic anemia investigational new drug application is currently filed with the FDA under the brand name CordIn, which is the same investigational development candidate as omidubicel. For more information on clinical trials of omidubicel, please visit www.clinicaltrials.gov.

About GDA-201
Gamida Cell applied the capabilities of its NAM-based cell expansion technology to develop GDA-201 (formerly known as NAM-NK), an innate natural killer (NK) cell immunotherapy for the treatment of hematologic and solid tumors in combination with standard of care antibody therapies. GDA-201 addresses key limitations of NK cells by increasing the cytotoxicity and in vivo retention and proliferation in the bone marrow and lymphoid organs of NK cells expanded in culture. GDA-201 is in Phase 1 development through an investigator-sponsored study in patients with refractory non-Hodgkin lymphoma and multiple myeloma.4

Omidubicel and GDA-201 are investigational therapies, and their safety and efficacy have not been evaluated by the U.S. Food and Drug Administration or any other health authority.

On August 6, 2019 United Therapeutics Corporation (Nasdaq: UTHR) reported that Martine Rothblatt, Ph.D., Chairman and Chief Executive Officer of United Therapeutics, will provide an overview and update on the company’s business at the 2019 Wedbush PacGrow Healthcare Conference in New York City (Press release, United Therapeutics, AUG 6, 2019, View Source [SID1234538235]).

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The presentation will take place on Tuesday, August 13, 2019, at 10:20 AM Eastern Time, and can be accessed via a live webcast on the United Therapeutics website at View Source under the "Investors" tab in the "Events and Presentations" section. An archived, recorded version of the presentation will be available approximately twenty-four hours after the presentation ends and can be accessed at the same location for 90 days. [uthr-g]