On February 27, 2020 Adaptimmune Therapeutics plc (Nasdaq:ADAP), a leader in cell therapy to treat cancer, reported financial results for the fourth quarter and year ended December 31, 2019, and provided a business update (Press release, Adaptimmune, FEB 27, 2020, View Source [SID1234554922]).

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"The last 12 months have been transformative. We reported responses in five different solid tumors, confirming that our SPEAR T-cell platform can treat a wide range of cancers. We also validated the potential of our allogeneic platform by demonstrating that we can generate functional T-cells from stem cells, and by signing our first major strategic deal in five years with Astellas," said Adrian Rawcliffe, Adaptimmune’s Chief Executive Officer. "With our passionate and skilled teams, and our world class capabilities, we are developing our cell therapy pipeline for a range of tumor indications, aiming to launch our first product in 2022 for people with sarcoma."

Responses in five solid tumor indications demonstrate SPEAR T-cell potential to treat cancer

Based on compelling response data in synovial sarcoma from the Phase 1 trial announced in May of last year, and updated at ESMO (Free ESMO Whitepaper) and CTOS, the Phase 2 SPEARHEAD-1 trial was initiated with ADP-A2M4 in synovial sarcoma and myxoid/ round cell liposarcoma (MRCLS). The product was granted Orphan Drug Designation, for the treatment of soft tissue sarcomas, and Regenerative Medicine Advanced Therapy designation, for synovial sarcoma, by the US FDA. The Company aims to launch ADP-A2M4 for sarcoma in 2022.

In January of this year, partial responses in liver, melanoma, gastro-esophageal junction, and head and neck cancers were reported. These early data in multiple indications demonstrate the potential of Adaptimmune’s SPEAR T-cell platform across multiple targets and a range of solid tumors. Further updated data will be presented at upcoming medical / scientific meetings.

Partnerships to develop next-generation and off-the-shelf cell therapies

In January of this year, a co-development and co-commercialization agreement with Astellas, through its wholly owned subsidiary Universal Cells, Inc., was announced for stem-cell derived allogeneic CAR-T and TCR T-cell therapies. The Company has received an upfront payment of $50 million under the agreement and is entitled to receive research funding of up to $7.5 million per year.

This agreement covers the co-development and co-commercialization of up to three T-cell therapies and leverages Astellas’ Universal Donor Cell Platform and Adaptimmune’s stem-cell derived allogeneic T-cell platform. This new collaboration may encompass both CAR-T and TCR T-cell approaches, including Adaptimmune’s novel HLA-independent TCR ("HiT") platform.

In 2019, Adaptimmune announced agreements with Alpine Immune Sciences and Noile-Immune to develop further next-generation products.

Leadership, manufacturing and financial updates strengthen fully integrated cell therapy company position

Adrian Rawcliffe assumed the role of Chief Executive Officer effective September 1, 2019 and John Lunger became Chief Patient Supply Officer effective August 1, 2019. In January 2020, a series of changes to the R&D leadership were announced, including the appointment of Elliot Norry as Chief Medical Officer. These leadership changes strengthen the scientific and clinical organization from early to late stage and accelerate the application of translational science learnings to therapeutic candidates and trials, as Adaptimmune becomes a late-stage cell therapy company aiming to launch a commercial product in 2022.

Adaptimmune’s in-house cell manufacturing facility located at the Navy Yard in Philadelphia, PA, is achieving a 25-day processing time for production of SPEAR T-cells. 95% of patient batches manufactured in 2019 met manufacturing criteria set for those batches. The Navy Yard facility was approved as a manufacturing source for a number of the Company’s clinical trials in Europe. The Company also produced its first GMP batch of lentiviral vector using an in-house, proprietary suspension process at its dedicated manufacturing space within the Cell and Gene Therapy Catapult Manufacturing Centre at Stevenage, UK.

Finally, on January 24, 2020, the Company closed an underwritten public offering of 21,000,000 American Depository Shares (ADSs) which, together with the full exercise by the underwriters on February 7, 2020 of their option to purchase an additional 3,150,000 ADSs, generated net proceeds of approximately $89.8 million. Following the agreement with Astellas and the public offering of ADSs described above, the Company is funded into 2H 2021.

Planned 2020 milestones

First Half of 2020

·Full summary of the ADP-A2M4 Phase 1 trial at a medical conference
·Safety update on Cohorts 1 and 2 of the ADP-A2AFP Phase 1 trial at a medical conference
·Update on Cohort 3 of the ADP-A2AFP Phase 1 trial at a medical conference
·initiation of PD1 / PDL1 inhibitor combination trial with ADP-A2M4 in head & neck cancer
·Data update from allogeneic program at a scientific meeting

Second Half of 2020

·12-month durability data from patients with synovial sarcoma from the ADP-A2M4 Phase 1 trial at a medical conference
·Updates on dose escalation cohorts from the SURPASS trial at medical conferences
·Update on the ADP-A2M4 Phase 1 radiation sub-study at a medical conference
·Update on the ADP-A2AFP expansion cohort at a medical conference

Financial Results for the fourth quarter and year ended December 31, 2019

·Cash / liquidity position: As of December 31, 2019, Adaptimmune had cash and cash equivalents of $50.4 million and Total Liquidity1 of $89.5 million.
· Revenue: Revenue for the fourth quarter and year ended December 31, 2019 were $0.7 million and $1.1 million compared to $1.5 million and $59.5 million for the same periods of 2018. The revenue recognized for the year ended December 31, 2019 is due to development work on the third target program under the GSK Collaboration and License Agreement. Revenue for the year ended December 31, 2018 included $39.1 million of license revenue and $20.4 million of development revenue due to the performance under the NY-ESO transition program and the PRAME development plan, which were completed in 2018.
·Research and development ("R&D") expenses: R&D expenses for the fourth quarter and year ended December 31, 2019 were $20.4 million and $97.5 million, compared to $22.8 million and $98.3 million for the same periods of 2018. The decrease in R&D expenses for the year ended December 31, 2019 was driven by a reduction in subcontracted expenses and clinical trial costs following the transfer of NY-ESO to GSK in 2018 and a decrease in share-based compensation expenditure, offset by recognition of accrued purchase commitments for clinical materials and payments for in-process research and development (following collaborations with Alpine Immune Sciences, Inc. and Noile-Immune Biotech, Inc.).
·General and administrative ("G&A") expenses: G&A expenses for the fourth quarter and year ended December 31, 2019 were $10.7 million and $43.4 million, compared to $10.8 million and $43.6 million for the same periods of 2018.
·Net loss: Net loss attributable to holders of the Company’s ordinary shares for the fourth quarter and year ended December 31, 2019 was $29.4 million and $137.2 million ($(0.22) per ordinary share) compared to $36.2 million and $95.5 million ($(0.16) per ordinary share) in the same periods of 2018.

Financial Guidance

The Company believes that its existing cash and cash equivalents and marketable securities, Total Liquidity, together with the net proceeds received from the underwritten public offering in January 2020, the additional net proceeds generated from the exercise in full of the underwriters’ option in February 2020 and the upfront payment received under its agreement with Astellas in January 2020, will fund the Company’s current operating plan into the second half of 2021.

Conference Call Information

The Company will host a live teleconference and webcast to provide additional details at 8:00 a.m. EST (1:00 p.m. GMT) today, February 27, 2020. The live webcast of the conference call will be available via the events page of Adaptimmune’s corporate website at www.adaptimmune.com. An archive will be available after the call at the same address. To participate in the live conference call, if preferred, please dial (833) 652-5917 (U.S. or Canada) or +1 (430) 775-1624 (International). After placing the call, please ask to be joined into the Adaptimmune conference call and provide the confirmation code (6083408).

On February 27, 2020 Atara Biotherapeutics, Inc. (Nasdaq: ATRA), a leading off-the-shelf, allogeneic T-cell immunotherapy company developing novel treatments for patients with cancer, autoimmune and viral diseases, reported financial results for the fourth quarter and full year ended December 31, 2019 and recent business highlights (Press release, Atara Biotherapeutics, FEB 27, 2020, View Source [SID1234554921]).

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"2019 was a year of strategic prioritization and significant advancement of our T-cell immunotherapy programs," said Pascal Touchon, President and Chief Executive Officer of Atara. "In 2020 we plan to deliver on key milestones across our pipeline and further establish Atara as a leader in off-the-shelf, allogeneic T-cell immunotherapies through our innovative EBV T-cell platform, next-generation CAR T technologies and state-of-the-art manufacturing capabilities. This work underpins our mission to transform the lives of patients with serious medical conditions through pioneering science, teamwork and a commitment to excellence."

Recent Highlights and Anticipated Upcoming Milestones

Tab-cel (tabelecleucel)

Atara continues to progress tab-cel Phase 3 development for patients with Epstein-Barr virus-associated post-transplant lymphoproliferative disease (EBV+ PTLD)

Atara remains on track to initiate a tab-cel biologics license application (BLA) submission for patients with EBV+ PTLD in the second half of 2020

Atara plans to discuss the totality of tab-cel results with the U.S. Food and Drug Administration (FDA) in a pre-BLA meeting prior to initiating the BLA submission

In the U.S. and Australia, 38 sites are available for enrollment and the Company is preparing to open additional sites in the U.S., Canada and Europe

Atara submitted clinical trial applications (CTAs) to several European countries in November and December 2019 to enable the opening of EU clinical sites in 2020. The Company’s CTAs in the United Kingdom, Spain and Austria were recently approved

Atara recently submitted a Pediatric Investigation Plan (PIP) to the European Medicines Agency (EMA)

Following EMA approval of the PIP, Atara plans to submit a tab-cel EU marketing authorization application for patients with EBV+ PTLD in 2021

Atara continues to see strong tab-cel investigator, physician and patient interest and, for cases in which the Company is not able to enroll patients in its EBV+ PTLD Phase 3 clinical study, Atara is providing tab-cel to patients in need under its expanded access protocol (EAP) and single patient use (SPU) programs

In December 2019, Atara presented long-term clinical results for 61 patients with diverse EBV-associated diseases, including efficacy and safety data for 26 patients with relapsed/refractory EBV+ PTLD and safety findings for 35 patients with other EBV-associated diseases, from a multicenter EAP study of tab-cel at the 61st American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting & Exposition

Results from this analysis suggest a high overall response rate (ORR), short time to response and favorable estimated long-term overall survival (OS) rates for tab-cel in patients with EBV+ PTLD following hematopoietic cell transplant (HCT) or solid organ transplant (SOT) who have failed rituximab-based therapy

Tab-cel was shown to be generally well-tolerated in all patients with EBV+ PTLD and other EBV-associated diseases

Studies supporting potential additional tab-cel indications are also advancing

Atara expects to initiate enrollment in a tab-cel Phase 2 multi-cohort study including up to six additional ultra-rare EBV+ diseases in the second half of 2020 based on previous clinical data treating these patients

Atara enrolled the final planned patient in the Phase 1b portion of a Phase 1b/2 clinical study of tab-cel in combination with Merck’s anti-PD-1 (programmed death receptor-1) therapy, KEYTRUDA (pembrolizumab), in patients with platinum-resistant or recurrent EBV-associated nasopharyngeal carcinoma (NPC)

ATA188 for Progressive Multiple Sclerosis (MS)

A Phase 1a clinical study of off-the-shelf, allogeneic ATA188 in patients with progressive MS is ongoing across clinical sites in the U.S. and Australia

Atara expects to present six- and 12-month ATA188 Phase 1a clinical results for cohorts 3 and 4 in the first and second halves of 2020, respectively

Atara retreated the first patients in the open label extension (OLE) portion of the Phase 1a study; OLE design allows patients who complete one year in the Phase 1a dose-escalation portion of the study to be retreated annually using the cohort 3 dose for up to four years

Atara plans to initiate enrollment of a randomized, double-blind, placebo-controlled Phase 1b study in patients with progressive MS in the second or third quarter of 2020

Activation of clinical study sites for the Phase 1b is ongoing with an increased number of leading MS centers expected to participate in the U.S. and Australia

EBV CAR T Platform

In February 2020, an academic off-the-shelf, allogeneic CD19 CAR T clinical study using an EBV T-cell construct for patients with relapsed/refractory B-cell malignancies was presented at the 2020 Transplantation and Cellular Therapy (TCT) Meetings

No cytokine release syndrome or neurotoxicity above Grade 2, and dose-limiting toxicities were observed post-infusion with multiple EBV CD19 CAR T doses administered

No confirmed GvHD was observed in patients who received partially HLA matched third-party donor EBV CD19 CART cells

Investigators observed durable complete responses (CR) with median follow up of 26.9 months for 83 percent (5/6) of patients who received partially HLA matched EBV CD19 CAR T cells manufactured from third-party donors including

100 percent (4/4) response in patients with non-Hodgkin’s lymphoma (NHL)

100 percent (1/1) response in patient with chronic lymphocytic leukemia (CLL)

Findings from this study provide initial clinical proof-of-principle that an EBV T-cell platform has the potential to generate off-the-shelf, allogeneic CAR T immunotherapies with high and durable responses, low risk of toxicity and that can be rapidly delivered to patients

ATA2271/ATA3271 and ATA3219 CAR T Programs

Atara is developing a mesothelin-targeted autologous CAR T (ATA2271) and expects collaborators at Memorial Sloan Kettering Cancer Center (MSK) to submit an Investigational New Drug (IND) application to the FDA for patients with advanced mesothelioma in the second or third quarter of 2020

ATA2271 is designed using a novel 1XX CAR co-stimulatory domain and PD-1 dominant negative receptor (DNR) intrinsic checkpoint inhibition technology

Atara is also developing off-the-shelf, allogeneic CAR T immunotherapies targeting mesothelin (ATA3271) and CD19 (ATA3219) using its next-generation technologies and EBV T cell platform

Started preclinical IND-enabling studies for ATA3219 and ATA3271

Operational

Atara named Kristin Yarema as Chief Commercial Officer; Dr. Yarema brings extensive hematology, oncology, neuroscience and autoimmune disease commercialization experience to Atara as the Company advances commercialization activities for tab-cel

Atara created a Chief Operations Officer role to continue to drive operational excellence across the Company’s programs and platform; Joe Newell, Atara’s current Chief Technical Operations Officer, was appointed to this new role

Atara is executing on its planned commercial supply strategy with continued positive progress on commercial production qualification activities with the Company’s contract manufacturing partner and dedicated, state-of-the-art T cell manufacturing facility in Thousand Oaks, Calif.

Atara continues to scale its EBV T-cell manufacturing platform with current yields of approximately 400 tab-cel doses from a single donor leukapheresis

Fourth Quarter and Full Year 2019 Financial Results

Atara believes that its cash, cash equivalents and short-term investments as of December 31, 2019, together with the net proceeds from the "at-the-market" (ATM) facility in January 2020, are sufficient to fund planned operations into the second quarter of 2021

Cash, cash equivalents and short-term investments as of December 31, 2019 totaled $259.1 million, as compared to $282.9 million as of September 30, 2019

Net cash used in operating activities was $58.7 million and $235.6 million for the fourth quarter and fiscal year 2019, respectively, as compared to $57.4 million and $179.8 million for the same periods in 2018

In the fourth quarter of 2019, the Company sold 2,449,216 shares of common stock pursuant to its ATM facility for net proceeds of $36.3 million and in January 2020 sold 1,371,216 shares of common stock for net proceeds of $21.1 million

Proforma cash, cash equivalents and short-term investments as of December 31, 2019, including aggregate ATM and option exercise proceeds of $23.6 million in January 2020, was $282.7 million. The number of outstanding shares of common stock and pre-funded common stock warrants as of February 18, 2020 were 58,571,550 shares and 2,888,526 warrants, respectively

Atara reported net losses of $78.5 million, or $1.36 per share, and $291.0 million, or $5.67 per share, for the fourth quarter and fiscal year 2019, respectively, as compared to $80.0 million, or $1.75 per share, and $230.7 million, or $5.27 per share, for the same periods in 2018

Total operating expenses include non-cash expenses of $14.0 million and $58.8 million for the fourth quarter and fiscal year 2019, respectively, as compared to $11.0 million and $37.5 million for the same periods in 2018

Research and development expenses were $61.6 million and $216.1 million for the fourth quarter and fiscal year 2019, respectively, as compared to $62.3 million and $167.5 million for the same periods in 2018

The decrease in the fourth quarter 2019 was primarily due to one-time license fees of $12.5 million incurred in the fourth quarter of 2018 for exclusive rights to the next-generation CAR T program targeting mesothelin, with no such corresponding costs incurred in fourth quarter of 2019, partially offset by higher employee-related and overhead costs from increased headcount and operating activities

The increase in fiscal year 2019 was primarily due to costs associated with the Company’s continuing expansion of research and development activities, including:

Clinical study, manufacturing and outside service costs related to MS programs

Research and process development costs related to CAR T programs

Higher employee-related and overhead costs from increased headcount and operating activities

Research and development expenses include $7.0 million and $26.8 million of non-cash stock-based compensation expenses for the fourth quarter and fiscal year 2019, respectively, as compared to $5.2 million and $16.2 million for the same periods in 2018

General and administrative expenses were $18.1 million and $79.6 million for the fourth quarter and fiscal year 2019, respectively, as compared to $19.6 million and $69.7 million for the same periods in 2018. The decrease in the fourth quarter 2019 was primarily due to a decrease in outside services costs, partially offset by an increase in employee-related costs driven by increased headcount. The increase in the fiscal year 2019 was primarily due to an increase in employee-related costs driven by increased headcount to support the Company’s expanding operations

General and administrative expenses include $5.0 million and $24.9 million of non-cash stock-based compensation expenses for the fourth quarter and fiscal year 2019, respectively, as compared to $4.3 million and $17.6 million for the same periods in 2018

Conference Call and Webcast Information

Atara will host a live conference call and webcast today at 8:00 a.m. EST to discuss the Company’s financial results and recent operational highlights. Analysts and investors can participate in the conference call by dialing (888) 540-6216 for domestic callers and (734) 385-2715 for international callers, using the conference ID 1554668. A live audio webcast can be accessed by visiting the Investors & Media – News & Events section of atarabio.com. An archived replay will be available on the Company’s website for approximately 14 days following the live webcast.

On February 27, 2019 Athenex, Inc. (NASDAQ: ATNX), a global biopharmaceutical company dedicated to the discovery, development and commercialization of novel therapies for the treatment of cancer and related conditions, reported its financial results and business highlights for the fourth quarter ended December 31, 2019 (Press release, Athenex, FEB 27, 2020, View Source [SID1234554920]).

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"Following a very successful and productive 2019, our team’s immediate focus is to execute on regulatory approval of our two most advanced candidates, Oral Paclitaxel and tirbanibulin ointment. Both of these products have the potential to change the standard of care in their respective markets," stated Dr. Johnson Lau, Chief Executive Officer and Chairman of Athenex. "We have been making the necessary investments in our manufacturing and commercial infrastructure to ensure a successful launch for Oral Paclitaxel upon approval. Our goal is to establish Oral Paclitaxel as the treatment of choice for patients receiving chemotherapy for metastatic breast cancer."

Fourth Quarter 2019 and Recent Business Highlights:

Clinical Programs:

Tirbanibulin ointment for actinic keratosis (AK)

A New Drug Application (NDA) was submitted to the FDA.

Oral Paclitaxel for metastatic breast cancer

Results presented at 2019 San Antonio Breast Cancer Symposium (SABCS) showed that Oral Paclitaxel had superior response and overall survival benefit compared to IV paclitaxel in the treatment of metastatic breast cancer.

Incidence and severity of neuropathy were less frequent with Oral Paclitaxel compared to IV paclitaxel.

Oral Paclitaxel abstract accepted for Press Program at SABCS.

Final FDA meeting has been scheduled for early April and the Company plans to submit an NDA in the US shortly thereafter.

Corporate Announcements:

Expanded strategic partnership with Guangzhou Xiangxue Pharmaceutical through a licensing agreement for Oral Paclitaxel, Oral Irinotecan, and tirbanibulin ointment, in China, Hong Kong and Macao. Athenex is entitled to receive aggregate payments of up to $200 million, including an initial license payment of $30 million, and double-digit tiered royalties on commercial sales.

In December 2019, closed a private placement equity offering of 3.95 million shares of common stock at a price of $15.30 per share. Net proceeds from the offering were approximately $59.4 million.

Commercial Business:

Athenex Pharmaceutical Division (APD) currently markets a total of 30 products with 59 SKUs.

Athenex Pharma Solutions (APS) currently markets 5 products with 16 SKUs.

Goal is to launch 7 products in 2020, including a major 503B product.

Financial Results for the Quarter Ended December 31, 2019

Product sales for the three months ended December 31, 2019 were $14.1 million, compared with $19.0 million for the three months ended December 31, 2018, a decrease of $4.9 million or 26%. This decrease was primarily attributable to lower sales of vasopressin and Chongqing API. License and other revenue recorded in the three months ended December 31, 2019 was $20.3 million, compared to $2.3 million in the same period in 2018. The increase was primarily due to a $20.0 million milestone achieved during the three-months ended December 31, 2019 in connection with the Almirall license agreement.

Cost of sales for the three months ended December 31, 2019 totaled $15.7 million, an increase of $1.4 million, or 10%, as compared to $14.3 million for the three months ended December 31, 2018. The increase in cost of sales was primarily due to an increase in the cost of specialty product sales of $4.6 million, offset by a decrease in the cost of 503B and API sales of $3.2 million. The increase in cost of specialty product sales was in line with the increase in specialty product revenue, while the cost of 503B and API sales decreased at a slower rate than the respective sales due to continued fixed costs despite decreased production. Changes in availability of products and market demand could increase or decrease our revenue and gross profit in the future.

Research and development expenses for the three months ended December 31, 2019 were $21.8 million as compared to $20.8 million for the three months ended December 31, 2018. This was primarily due to an increase in clinical and preclinical costs related to encequidar, tirbanibulin ointment, and TCR-T development, offset by a decrease in product development costs related to the scale up of 503B operations, API research and development, and the launch of certain specialty products in 2018.

Selling, general and administrative expenses for the three months ended December 31, 2019 totaled $18.1 million, as compared to $11.6 million for the three months ended December 31, 2018, an increase of $6.5 million, or 56%. This was primarily due to an increase of $6.3 million related to the costs of preparing to commercialize our proprietary drugs, together with a small increase in general administrative expenses including professional fees, rent, utilities, insurance, and office expenses.

Net loss attributable to Athenex for the three months ended December 31, 2019 was $21.7 million, or ($0.28) per diluted share, compared to a net loss of $27.1 million, or ($0.41) per diluted share, in the same period last year.

At December 31, 2019, the Company had cash, cash equivalents and short-term investments of $160.8 million, which included $7.8 million funded by New York State for the construction of the Dunkirk facility for which the company has recorded a corresponding liability, compared to $107.4 million of cash, cash equivalents and short-term investments at December 31, 2018. The amount at December 31, 2019 does not include the $30 million license fee payment the Company expects to receive as part of the expanded license agreement with Guangzhou Xiangxue Pharmaceutical. Based on the current operating plan, the Company expects that its cash and cash equivalents and short-term investments as of December 31, 2019, will enable it to fund operations into the first quarter of 2021.

Financial Results for the Year Ended December 31, 2019

Product sales for the year ended December 31, 2019 were $80.5 million, an increase of $24.1 million, or 43%, as compared to $56.4 million for the year ended December 31, 2018. The increase was due to a $20.0 million increase in specialty product sales from growing sales volume of existing products and the launch and sales of new products along with an $11.8 million increase in 503B sales mainly attributable to vasopressin sales in the first half of 2019, offset by a $5.2 million decrease in sales of API products and a $2.5 million decrease in medical device sales.

Total revenue for the year ended December 31, 2019 was $101.2 million, an increase of $12.1 million as compared to $89.1 million for the year ended December 31, 2018.

We recognized $20.0 million and $30.0 million in collaboration and license revenue for the years ended December 31, 2019 and 2018, respectively, pursuant to the license agreement entered into with Almirall in December 2017. We recognized zero and $2.0 million in collaboration and license revenue for the years ended December 31, 2019 and 2018, respectively, pursuant to the license agreement we entered into with PharmaEssentia to develop and commercialize oral paclitaxel, oral irinotecan, and oral docetaxel in Taiwan, Singapore, and Vietnam.

Cost of sales totaled $69.6 million for the year ended December 31, 2019, an increase of $22.6 million, or 48%, as compared to $47.0 million for the year ended December 31, 2018. This was primarily due to an increase in the cost of specialty product sales and 503B product sales of $21.5 million and $7.0 million, respectively, and was offset by a decrease in cost of API product sales of $5.9 million. The increase in cost of sales was in line with the increase in product sales. Changes in availability of products and market demand could increase or decrease our revenue and gross profit in the future.

Research and development expenses totaled $84.4 million for the year ended December 31, 2019, a decrease of $35.5 million, or 30%, as compared to $119.9 million for the year ended December 31, 2018. This was primarily due to a decrease in licensing fees, product development, and clinical operations and included the following: $30.0 million decrease in drug in-licensing fees primarily related to a $29.5 million license fee related to the license of TCR-T technology in connection with the establishment of Axis; $8.4 million decrease of product development costs related to the scale up of 503B operations, API research and development, and the launch of certain specialty products in 2018; and $3.7 million decrease of clinical development costs related to encequidar and tirbanibulin ointment due to the winding down of the two AK Phase 3 studies. The decrease in these R&D expenses was offset by an increase of $4.7 million of preclinical development costs related to the Arginine Deprivation Therapy and TCR-T Immunotherapy platforms and a $1.9 million increase of R&D related compensation expense.

Selling, general, and administrative expenses totaled $66.7 million for the year ended December 31, 2019, an increase of $17.7 million, or 36%, as compared to $49.0 million for the year ended December 31, 2018. This was primarily due to an increase of $15.6 million related to the costs of preparing to commercialize our proprietary drugs, an increase of $1.3 million of general administrative expenses including rent, utilities, insurance, and office expenses, and an increase of $0.9 million of professional fees including legal and accounting fees.

Net loss attributable to Athenex for the year ended December 31, 2019 was $123.7 million, or ($1.67) per diluted share, compared to a net loss of $117.4 million, or ($1.82) per diluted share, for the year ended December 31, 2018.

Outlook and Upcoming Milestones:

Final FDA meeting for Oral Paclitaxel in metastatic breast cancer has been scheduled for early April and the Company plans to submit an NDA in the US shortly thereafter.

Plan to submit additional data from ongoing Phase 1 study of Oral Paclitaxel in cutaneous angiosarcoma for presentation at a medical meeting (H1-2020).

Enroll patients in dose expansion part of Phase 1 trial of Oral Paclitaxel in combination with pembrolizumab for the treatment of advanced solid tumors (H2-2020).

Financial Guidance:

Athenex’s product sales in 2019 amounted to $80.5 million, representing a 43% year-over-year increase which was above the 35%-40% guidance range provided on the last earnings call in November 2019. The Company expects 2020 year-over-year product sales growth to be in the mid-single digits, from $80.5 million in 2019. While the Company does not give quarterly guidance, product sales are expected to be back-end loaded in 2020. The product sales guidance for 2020 has taken into account the discontinuation of vasopressin sales and the suspension of operations at the Taihao API plant in 2019, which had meaningful contributions in the first nine months of 2019.

Conference Call and Webcast Information:

The Company will host a conference call and live audio webcast today, Thursday, February 27, 2020, at 8:00am Eastern Time to discuss the financial results and provide a business update.

To participate in the call, dial 877-407-0784 (domestic) or 201-689-8560 (international) fifteen minutes before the conference call begins and reference the conference passcode 13698005. The live conference call and replay can also be accessed via audio webcast at this link View Source and also via the Investor Relations section of the Company’s website, located at View Source

On February 27, 2020 Allogene Therapeutics, Inc. (Nasdaq: ALLO), a clinical-stage biotechnology company pioneering the development of allogeneic CAR T (AlloCAR T) therapies for cancer, provided a corporate update and reported fourth quarter and full-year 2019 financial results for the periods ended December 31, 2019 (Press release, Allogene, FEB 27, 2020, View Source [SID1234554919]).

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"2019 was a very successful year for Allogene. We accomplished all of our corporate goals, which includes the establishment of world-class capabilities across multiple new functions and the initiation of our first clinical trials in patients with relapsed or refractory aggressive non-Hodgkin lymphoma and multiple myeloma," said David Chang, M.D., Ph.D., President, Chief Executive Officer and Co-Founder of Allogene. "Our momentum and focus towards making AlloCAR T therapies and their life-saving potential a reality for patients will continue at full speed in 2020. This year will be an important one as we look to demonstrate proof-of-concept data from two key programs and plan for potential pivotal studies."

Recent Highlights

ALLO-501 (anti-CD19 AlloCAR T)
•In 2019, Allogene initiated the ALLO-501 Phase 1 ALPHA trial in patients with relapsed/refractory non-Hodgkin lymphoma (NHL). This study utilizes ALLO-647, Allogene’s anti-CD52 monoclonal antibody (mAb) as a part of the lymphodepletion regimen. Initial data is planned to be presented at a medical meeting in Q2 2020.
•The U.S. Food and Drug Administration cleared the Investigational New Drug Application (IND) for ALLO-501A, the Company’s next generation anti-CD19 AlloCAR T construct devoid of the rituximab off-switch. ALLO-501A, previously referred to as ALLO-501.1, was created to eliminate the rituximab recognition domains in ALLO-501, allowing for use in a broader patient population, including those NHL patients with recent rituximab exposure.
•The Company expects to initiate an abbreviated Phase 1 portion of the ALLO-501A trial (ALPHA2) in the second quarter of the year. The ALPHA2 trial will leverage the findings of the ALPHA trial to finalize the ALLO-501A cell dose and the ALLO-647 based lymphodepletion regimen for a potential pivotal Phase 2 trial.

ALLO-715 (anti-BCMA AlloCAR T)
•The Company has created a robust anti-BCMA strategy centered around ALLO-715 for the treatment of multiple myeloma (MM).
•In 2019, Allogene initiated the ALLO-715 Phase 1 UNIVERSAL trial in patients with relapsed/refractory MM, designed to assess the safety and tolerability of ALLO-715. This trial is also intended to identify the optimal dose of ALLO-647 and includes the potential to evaluate lymphodepletion regimens that do not include fludarabine and cyclophosphamide. UNIVERSAL continues to accrue as planned with initial data anticipated in Q4 2020.
•In January 2020, Allogene entered into a clinical collaboration with SpringWorks Therapeutics to evaluate ALLO-715 in combination with SpringWorks’ investigational gamma secretase inhibitor, nirogacestat, in patients with relapsed/refractory MM. The Company expects to initiate this combination trial in the second half of 2020.
•The Company nominated ALLO-605 as its first TurboCARTM candidate and part of its larger MM strategy. The TurboCAR technology provides the effects of cytokine stimulation to AlloCAR T cells with the goal of enhancing

their potency, expansion and persistence. ALLO-605 is advancing in preclinical development with a potential IND in 2021.

Additional Pipeline Updates
•The Company nominated ALLO-316, an anti-CD70 AlloCAR T, as its next clinical candidate for potential development in acute myeloid leukemia, T-cell malignancies and/or renal cell carcinoma. Submission of the IND for ALLO-316 is expected by year end 2020.

Fourth Quarter Financial Results
•As of December 31, 2019, Allogene had $588.9 million in cash, cash equivalents, and investments.
•Research and development expenses were $49.4 million for the fourth quarter of 2019, which includes $6.4 million of non-cash stock-based compensation expense and $10.0 million in expenses associated with the signing of our collaboration with Notch Therapeutics. For the full year of 2019, research and development expenses were $144.5 million. Research and development expense for the year includes $19.4 million of non-cash stock-based compensation expense.
•General and administrative expenses were $15.2 million for the fourth quarter of 2019, which includes $7.5 million of non-cash stock-based compensation expense. For the full year of 2019, general and administrative expenses were $57.5 million, which includes $26.6 million of non-cash stock-based compensation expense.
•Net loss for the fourth quarter of 2019 was $61.0 million, or $0.58 per share, including non-cash stock-based compensation expense of $13.9 million. For the full year of 2019, our net loss was $184.6 million, or $1.83 per share, including non-cash stock-based compensation expense of $46.1 million.

2020 Financial Guidance
•Allogene expects full year GAAP net losses to be between $260 million and $280 million including estimated non-cash stock-based compensation expense of $70 million to $75 million and excluding any impact from potential business development activities.

Conference Call and Webcast Details
Allogene will host a live conference call and webcast today at 5:30 a.m. Pacific Time / 8:30 a.m. Eastern Time to discuss financial results and provide a business update. To access the live conference all by telephone, please dial 1 (866) 940-5062 (U.S.) or 1 (409) 216-0618 (International). The conference ID number for the live call is 2981059. The webcast will be made available on the Company’s website at www.allogene.com under the Investors tab in the News and Events section. Following the live audio webcast, a replay will be available on the Company’s website for approximately 30 days.

On February 27, 2020 IVERIC bio, Inc. (Nasdaq: ISEE) reported financial and operating results for the fourth quarter and full year ended December 31, 2019 and provided a general business update (Press release, Ophthotech, FEB 27, 2020, View Source [SID1234554918]).

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"We made tremendous progress last year as we build a diversified portfolio in retinal diseases that includes both therapeutics and gene therapy, setting the stage for IVERIC bio to be a leader in developing transformative therapies to treat retinal diseases," stated Glenn P. Sblendorio, Chief Executive Officer and President of IVERIC bio. "We achieved a major milestone with our positive Zimura pivotal clinical trial results in geographic atrophy secondary to dry AMD. Our goal is to continue to build on this momentum. Following the positive data, our team quickly started working on our second Zimura pivotal clinical trial in GA with plans to enroll the first patient next month. Our lead gene therapy programs in rhodopsin mediated adRP and BEST1 related retinal diseases continue to advance towards Phase 1/2 clinical trials and we expect to identify our lead minigene construct for LCA10 later in the year."

Therapeutics Programs

Zimura (avacincaptad pegol): Complement C5 Inhibitor

On October 28, 2019, the Company announced that Zimura (avacincaptad pegol) met its pre-specified primary efficacy endpoint and reached statistical significance in an international, multicenter, randomized, double masked, sham controlled clinical trial in geographic atrophy (GA) secondary to dry age-related macular degeneration (AMD), referred to as the OPH2003 trial. Zimura was generally well tolerated after 12 months of administration. The Company believes that the safety and efficacy results from this trial could meet regulatory agencies’ requirements to serve as one of the two pivotal clinical trials typically required for marketing approval.

January 2020, the Company announced the design of its second pivotal clinical trial of Zimura in GA secondary to dry AMD, ISEE2008. The Company plans to enroll approximately 400 patients in this international, multicenter, double masked, sham controlled clinical trial. Patients will be randomized to receive either monthly administration of Zimura 2mg or sham during the first 12 months of the trial, at which time the primary efficacy analysis of the mean rate of change of GA growth at 12 months will be performed. If the 12 month results are positive, the Company plans to file an application with the U.S. Food and Drug Administration and the European Medicines Agency for marketing approval of Zimura for GA following receipt of that data. At month 12, the Company plans to re-randomize patients in the Zimura 2 mg arm to receive either monthly or every other month administration of Zimura 2 mg. All the patients who

were initially randomized to the sham control arm will continue with monthly administration of sham. The final evaluation will take place at month 24.

The Company plans and is on track to enroll the first patient in the ISEE2008 trial next month.

The Company’s ongoing Phase 2b clinical trial of Zimura for the treatment of autosomal recessive Stargardt disease, an orphan inherited retinal disease, is on track for top-line data to be available during the second half of 2020.

HtrA1 Inhibitor

The Company has identified a lead compound in this preclinical program to address GA secondary to dry AMD and is developing the formulation and manufacturing process with the goal of filing an IND during 2021.

Gene Therapy Programs in Orphan Inherited Retinal Diseases

IC-100: Rhodopsin-Mediated Autosomal Dominant Retinitis Pigmentosa (RHO-adRP)
Natural history studies and IND-enabling activities for IC-100 are ongoing. The Company plans to initiate a Phase 1/2 clinical trial for IC-100 in patients with rhodopsin mediated adRP during the fourth quarter of 2020.

IC-200: BEST1-Related IRDs
Natural history studies and IND-enabling activities for IC-200 are ongoing. The Company plans to initiate a Phase 1/2 clinical trial for IC-200 in patients with BEST1 related retinal diseases during the first half of 2021.

miniCEP290: Leber Congenital Amaurosis Type 10 (LCA10)
IVERIC bio, in collaboration with the University of Massachusetts Medical School (UMass Medical School), is continuing to optimize the minigene constructs with the goal of identifying a lead construct by mid-year 2020.

miniABCA4 Program for Stargardt Disease (STGD1)
IVERIC bio, through its collaborative sponsored research agreement with UMass Medical School, is evaluating several ABCA4 minigene constructs in both in vitro and in vivo experiments. The Company has received preliminary results and expects to receive additional results for the miniABCA4 program during the second half of 2020.

miniUSH2A: USH2A-Related IRDs Including Usher Syndrome Type 2A (Usher 2A) and USH2A-Associated Nonsyndromatic Autosomal Recessive Retinitis Pigmentosa
This research program targets IRDs associated with mutations in the USH2A gene, including Usher 2A and USH2A-associated nonsyndromatic autosomal recessive retinitis pigmentosa. The Company expects to receive preliminary results during the second half of 2020.
Corporate Update
In December 2019, the Company completed an underwritten public offering in which it sold 7,750,000 shares of its common stock at a price of $4.00 per share, and it also sold to certain investors pre-funded warrants to purchase 3,750,000 shares of its common stock at a price of $3.999 per share underlying each warrant. The Company raised approximately $42.6 million in net proceeds from this offering. During the fourth quarter of 2019, IVERIC bio appointed Guangping Gao, PhD as Chief Strategist, Gene Therapy, and Abraham Scaria, PhD as Chief Scientific Officer.

Fourth Quarter and Year End 2019 Financial Results and 2020 Cash Guidance

As of December 31, 2019, the Company had $125.7 million in cash and cash equivalents. The Company estimates that its year-end 2020 cash and cash equivalents will range between $60 million and $70 million. The Company also estimates that its cash and cash equivalents will be sufficient to fund its operations and capital expenditure requirements as currently planned into the beginning of 2022. These estimates are based on the Company’s current business plan, including initiation of the Zimura ISEE2008 trial and the continuation of the Company’s other on-going research and development programs. These estimates do not reflect any additional expenditures, including associated development costs, in the event the Company in-licenses or acquires any new product candidates or commences any new sponsored research programs.

2019 Financial Highlights

R&D Expenses: Research and development expenses were $11.6 million for the quarter ended December 31, 2019, compared to $16.1 million for the same period in 2018. For the year ended December 31, 2019, research and development expenses were $39.6 million compared to $41.7 million for the same period in 2018. Research and development expenses decreased primarily due to decreased costs associated with the Company’s Zimura OPH2003 clinical trial in GA and OPH2005 clinical trial in Stargardt disease, as earlier-enrolled patients completed those trials, and decreased costs related to the Company’s acquisition of Inception 4 and its HtrA1 inhibitor program during 2018. These decreases were offset by increases in costs associated with the Company’s gene therapy programs.

G&A Expenses: General and administrative expenses were $6.3 million for the quarter ended December 31, 2019, compared to $5.7 million for the same period in 2018. For the year ended December 31, 2019, general and administrative expenses were $21.6 million compared to $23.6 million for the same period in 2018. General and administrative expenses decreased primarily due to decreases in costs to support the Company’s operations and infrastructure.

Net Income (loss): The Company reported a net loss for the quarter ended December 31, 2019 of $17.5 million, or ($0.39) per diluted share, compared to a net income of $104.1 million, or $2.62 per diluted share, for the same period in 2018. In the quarter and year ended December 31, 2018, the Company recognized a gain on extinguishment of a royalty purchase liability of $125 million due to its December 2018 termination of a royalty and sales agreement with Novo Holdings A/S. For the year ended December 31, 2019, the Company reported a net loss of $58.9 million or ($1.39) per diluted share, compared to a net income of $63.1 million or $1.70 for the same period in 2018.

Conference Call/Web Cast Information
IVERIC bio will host a conference call/webcast to discuss the Company’s financial and operating results and provide a business update. The call is scheduled for February 27, 2020 at 8:00 a.m. Eastern Time. To participate in this conference call, dial 800-458-4121 (USA) or 323-794-2598 (International), passcode 6010573. A live, listen-only audio webcast of the conference call can be accessed on the Investors section of the IVERIC bio website at www.ivericbio.com. A replay will be available approximately two hours following the live call for two weeks. The replay number is 888-203-1112 (USA Toll Free), passcode 6010573.