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Akebia Therapeutics Reports Fourth Quarter and Full-Year 2019 Financial Results and Hosts Conference Call to Discuss Recent Business Highlights

On March 10, 2020 Akebia Therapeutics, Inc. (Nasdaq: AKBA), a biopharmaceutical company focused on the development and commercialization of therapeutics for people living with kidney disease, reported financial results for the fourth quarter and full-year ended December 31, 2019 (Press release, Akebia, MAR 10, 2020, View Source [SID1234555355]). The Company will host a conference call today, Tuesday, March 10, 2020, at 9:00 a.m. Eastern Time to discuss its fourth quarter and full-year 2019 financial results and recent business highlights.

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"2019 was a year of considerable progress for Akebia and with many milestones on the horizon, 2020 is shaping up to be equally, if not more, exciting," stated John P. Butler, Chief Executive Officer of Akebia. "Our highest priority remains the successful execution of our global Phase 3 program for vadadustat, our investigational oral hypoxia-inducible factor prolyl hydroxylase inhibitor (HIF-PHI). We have a tremendous amount of confidence in our vadadustat clinical program and believe we’ve developed an exciting path forward to drive significant value for all our stakeholders. We believe our ability to potentially access a Priority Review Voucher (PRV) for the vadadustat New Drug Application (NDA) to expedite FDA review would meaningfully enhance the potential of bringing vadadustat to patients as quickly as possible, subject to regulatory approval."

Butler continued, "We look forward to sharing data from our global Phase 3 studies of vadadustat, starting with top-line data from INNO2VATE on track for the second quarter of 2020 followed by PRO2TECT in mid-2020. We expect vadadustat to be the first drug of the HIF-PHI class to deliver clear data that directly compare its outcomes to the current standard of care in dialysis-dependent and non-dialysis dependent patients for the treatment of anemia due to chronic kidney disease (CKD)."

Agreement with Vifor Pharma regarding a Priority Review Voucher

In February 2020, Akebia entered into a letter agreement with Vifor (International) Ltd. (Vifor Pharma) relating to Vifor Pharma’s agreement with a third party to purchase a PRV issued by the U.S. Food and Drug Administration (FDA), subject to satisfaction of customary closing conditions. Akebia will pay Vifor Pharma $10.0 million following the closing of the PRV purchase. In exchange, Vifor Pharma is obligated to reserve the PRV for the vadadustat NDA for the treatment of anemia due to CKD in dialysis-dependent and non-dialysis dependent patients until Akebia and Vifor Pharma agree on the financial and other terms under which it will assign the PRV to Akebia or make a mutual decision to sell the PRV. A PRV entitles the holder to priority review of an NDA or a Biologics License Application for a new drug, which reduces the target FDA review time to six months after official acceptance of the submission and could lead to expedited approval.

Jason A. Amello, Chief Financial Officer of Akebia stated, "We are pleased to have extended our cash runway well into 2021 through planned, disciplined spending and the identification of operating efficiencies, coupled with the sales of common stock via our At-the-Market facility over the last few months." The Company’s cash runway, consistent with previous commentary, includes the receipt of a $15.0 million regulatory milestone from Mitsubishi Tanabe Pharma Corporation, Akebia’s development and commercialization collaboration partner in Japan for vadadustat, assuming approval of vadadustat in Japan.

Fourth Quarter and Full-Year 2019 Financial Results

Cash Position: Cash, cash equivalents and available-for-sale securities as of December 31, 2019 were $147.7 million.

Revenues: Total revenue was $69.6 million for the fourth quarter of 2019 compared to $59.9 million for the fourth quarter of 2018(1), and $335.0 million for the full-year 2019 compared to $207.7 million for the full-year 2018(1).

Collaboration revenue was $40.6 million for the fourth quarter of 2019 compared to $53.0 million in the fourth quarter of 2018, and $223.9 million for the full-year 2019 compared with $200.9 million for the full-year 2018. The change in both periods is due to the timing in which vadadustat development expenses are incurred and the associated revenue is recognized on a percentage-of-completion basis.

Net product revenue was $28.9 million for the fourth quarter of 2019 compared with $6.8 million in the fourth quarter of 2018(1), and $111.1 million for the full-year 2019 compared to $6.8 million for the full-year 2018(1). Pro forma net product revenue for the full-year 2018, inclusive of pre-merger net product revenue recorded by Keryx Biopharmaceuticals, Inc. (Keryx), was approximately $96 million.

COGS: Cost of goods sold was $38.1 million for the fourth quarter of 2019, which includes non-cash charges, related to the application of purchase accounting as a result of the merger with Keryx, of $18.8 million for inventory step-up and $9.1 million for amortization of intangibles. Cost of goods sold was $145.3 million for the full-year 2019, which includes non-cash charges of $70.4 million for inventory step-up and $36.4 million for amortization of intangibles.

R&D Expenses: Research and development expenses were $80.4 million for the fourth quarter of 2019 compared to $87.1 million for the fourth quarter of 2018(1), and $323.0 million for the full-year 2019 compared to $291.0 million for the full-year 2018(1). The change in both periods was largely attributable to a change in costs associated with our research and development programs, including vadadustat.

SG&A Expenses: Selling, general and administrative expenses were $44.9 million for the fourth quarter of 2019 compared to $55.1 million for the fourth quarter of 2018(1) (which included $41.7 million of merger-related expenses), and $149.5 million for the full-year 2019 compared to $87.1 million for the full-year 2018(1) (which included $49.5 million of merger-related expenses).

Net Loss: Net loss was $94.5 million for the fourth quarter of 2019 compared to $60.1 million for the fourth quarter of 2018(1), and $279.7 million for the full-year 2019 compared to $143.6 million for the full-year 2018(1).

Includes only 18 days of operating results of Keryx following completion of Akebia’s merger with Keryx on December 12, 2018, whereby Keryx became Akebia’s wholly owned subsidiary.

Conference Call

Akebia will host a conference call at 9:00 a.m. Eastern Time today, Tuesday, March 10th, to discuss its fourth quarter and full-year 2019 financial results and recent business highlights. To listen to the conference call, please dial (877) 458-0977 (domestic) or (484) 653-6724 (international) using conference ID number 6572299. The call will also be webcast LIVE and can be accessed via the Investors section of the Company’s website at View Source

A replay of the conference call will be available two hours after the completion of the call through March 16, 2020. To access the replay, dial (855) 859-2056 (domestic) or (404) 537-3406 (international) and reference conference ID number 6572299. An online archive of the conference call can be accessed via the Investors section of the Company’s website at View Source

TRILLIUM THERAPEUTICS REPORTS ANNUAL FINANCIAL AND OPERATING RESULTS

On March 10, 2020 Trillium Therapeutics Inc. (NASDAQ/TSX: TRIL), a clinical stage immuno-oncology company developing innovative therapies for the treatment of cancer, reported financial and operating results, including an update on its transformation program, for the year ended December 31, 2019 (Press release, Trillium Therapeutics, MAR 10, 2020, View Source [SID1234555348]).

2019 Transformation Program

2019 was a critical year in Trillium’s evolution toward a more clinical development-focused CD47 immuno-oncology company. Key developments, as part of a wide-ranging transformation program, were:

Transitioned leadership to a new CEO, Jan Skvarka, who joined in September. Mr. Skvarka is an experienced healthcare executive, who was previously CEO of a PureTech Health portfolio company (Tal Medical), and leading Partner in the Healthcare practice at Bain & Company. He holds an MBA from Harvard Business School.
Established office in Cambridge, MA, which will allow the company to tap the local talent pool. The CEO office and clinical development are now based in Cambridge.
Restructured the company’s footprint by reducing staff by 40% to create a more efficient organization with stronger clinical development focus, as well as extend the cash runway.
Substantially revised the company strategy, namely:
Refocused on intravenous TTI-621 & TTI-622 programs and large hematologic malignancy indications, specifically acute myeloid lymphoma & myelodysplastic syndromes (AML/MDS), peripheral T-cell lymphoma (PTCL), diffuse large B-cell lymphoma (DLBCL) and multiple myeloma;
Deprioritized a lead intratumoral TTI-621 program with intended focus on early-stage cutaneous T-cell lymphoma (CTCL).
Zeroed in on execution of TTI-621 & TTI-622 dose escalation studies, which were declared mission critical path in the near term.
Subsequently, on January 7, 2020, the company announced a data update that confirmed TTI-621 monotherapy activity at initial low dose escalation levels (up to 0.5 mg/kg) across several hematologic malignancies. As such, TTI-621 is the only CD47 blocker that has shown meaningful single agent activity. Further dose optimization is in progress, and TTI-621 is now enrolling patients at a 1.4 mg/kg dose level, or 7 times the initially selected dose at which monotherapy activity was observed.

Following the (i) transformation program, (ii) changes in strategic direction, and (iii) encouraging data update, the company raised $117 million in January 2020, in a substantially oversubscribed public offering. Among key investors were experienced healthcare funds, such as Boxer Capital, Logos Capital, New Enterprise Associates, Venrock Healthcare Capital Partners and Vivo Capital. Paul Walker joined the Board of Directors, and Ali Behbahani became Board Observer; both are General Partners at NEA.

Cash Position and Guidance

As of February 28, the company had approximately $130 million in cash and cash equivalents.

Over the next three years (2020-22), the company plans to accomplish the following goals:

Complete TTI-621 and TTI-622 dose escalation studies, expected in 2020 (potentially in 2021, depending on the dose selected);
Conduct 3-4 new hematologic malignancy studies in AML/MDS, PTCL, DLBCL and/or multiple myeloma; and
Initiate and complete a solid tumor exploratory effort with TTI-621 in several key indications.
In 2020, the company will provide the following updates:

TTI-622 study update at the 2020 ASCO (Free ASCO Whitepaper) annual meeting;
TTI-621 study update mid-year and at the 2020 ASH (Free ASH Whitepaper) annual meeting;
TTI-621 and TTI-622 study updates once maximum tolerated doses or recommended phase 2 doses are identified.
Annual 2019 Financial Results:

As of December 31, 2019, Trillium had cash and cash equivalents and marketable securities, and working capital of $22.7 million and $9.8 million, respectively, compared to $33.4 million and $25.1 million, respectively at December 31, 2018. The decrease in cash and cash equivalents and marketable securities, and the decrease in working capital were due mainly to cash used in operations, partially offset by the cash received from the February 2019 public offering. The decrease in working capital was due mainly to cash used in operations and an increase to accounts payable and accrued liabilities due to timing of clinical trial related payments.

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Net loss for the year ended December 31, 2019 of $41.6 million was higher than the loss of $32.9 million for the year ended December 31, 2018. The net loss was higher due mainly to a warrant liability revaluation loss of $5.7 million, the write down of Fluorinov intangible assets of $3.0 million, higher manufacturing costs, and a net foreign currency loss of $0.8 million in the current year compared to a net foreign currency gain of $2.7 million in the prior year. The higher loss was partially offset by lower clinical trial expenses.

In January 2020, the Company completed an underwritten public offering for gross proceeds of $117 million comprising 41,279,090 common shares and 1,250,000 Series II Non-Voting Convertible First Preferred Shares, each issued at $2.75 per share.

Surface Oncology Reports Financial Results and Corporate Highlights for Fourth Quarter and Full Year 2019

On March 10, 2020 Surface Oncology (Nasdaq: SURF), a clinical-stage immuno-oncology company developing next-generation immunotherapies that target the tumor microenvironment, reported financial results and corporate highlights for the fourth quarter and full year 2019, as well as anticipated corporate milestones for 2020 (Press release, Surface Oncology, MAR 10, 2020, View Source [SID1234555347]).

"We have made tremendous progress readying our lead programs, SRF617 and SRF388, for clinical trials. With opened INDs for both of these highly differentiated investigational immunotherapies, we are looking forward to initiating a new phase in our mission to deliver breakthrough treatments to help those affected by cancer," said Jeff Goater, chief executive officer. "Furthermore, with strengthened preclinical data packages across our portfolio, we continue to explore collaborations to enhance our clinical development strategy for each of our lead product programs, an example of which is the clinical collaboration we recently signed with Arcus Biosciences. We look forward to providing initial clinical updates for both SRF617 and SRF388 by the end of 2020."

Recent Corporate Highlights:

Entered into a clinical collaboration with Arcus Biosciences (NYSE: RCUS) in January 2020, to evaluate SRF617 (targeting CD39) in combination with AB928 (a dual A2a/A2b adenosine receptor antagonist) in clinical trials
FDA clearance of the Investigational New Drug applications (INDs) for both SRF617 and SRF388 (targeting IL-27) in January 2020
Continued progression of the ongoing phase 1/1b trial of NZV930 (targeting CD73) by Surface Oncology’s partner Novartis
Promotion of Liisa Nogelo to chief legal officer and Alison O’Neill to senior vice president, clinical development
Selected 2019 Corporate Highlights:

Filed INDs for both SRF617 and SRF388
Strengthened its team and Board of Directors with key additions, including the board appointment of Ramy Ibrahim, M.D., the chief medical officer of the Parker Institute for Cancer Immunotherapy
Announced a development candidate, SRF813 (CD112R), targeting the activation of natural killer and T cells
Published a peer-reviewed manuscript in the scientific journal ImmunoHorizons1 describing the biological activity of IL-27, an immunosuppressive cytokine and the target of SRF388
Gave multiple preclinical data presentations related to Surface Oncology’s pipeline programs at key scientific conferences, including the Society for the Immunotherapy of Cancer (SITC) (Free SITC Whitepaper)’s (SITC) (Free SITC Whitepaper) 34th Annual Meeting and the Brisbane Immunotherapy 2019 Conference
Secured a debt financing facility for up to $25 million from K2 HealthVentures
Held Surface Oncology’s inaugural Investor and Analyst Day
Selected Anticipated 2020 Corporate Milestones:

Initiation of phase 1 trial for SRF617 in the first half of 2020
Initiation of phase 1 trial for SRF388 in the first half of 2020
Multiple preclinical data presentations anticipated at key medical and scientific conferences throughout 2020, including at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) annual meeting in April
Initial clinical updates for both SRF617 and SRF388 anticipated by the end of 2020
Financial Results:

As of December 31, 2019, cash, cash equivalents and marketable securities were $105.2 million, compared to $158.8 million on December 31, 2018.

Research and development (R&D) expenses were $11.7 million for the fourth quarter ended December 31, 2019, compared to $10.5 million for the same period in 2018. This increase was primarily driven by additional spend incurred for SRF617 and SRF388 associated with the IND filings in the fourth quarter of 2019. R&D expenses were $52.1 million for the full year 2019, compared to $52.5 million for the same period in 2018. This decrease was primarily driven by a reduction of manufacturing spend on the SRF231 (CD47) program, which was partially offset by increased spend on SRF617 and SRF388 associated with the IND filings in the fourth quarter of 2019. R&D expenses included $2.4 million in stock-based compensation expense for the full year 2019.

General and administrative (G&A) expenses were $5.1 million for the fourth quarter ended December 31, 2019, compared to $4.8 million for the same period in 2018. G&A expenses were $20.6 million for the full year 2019, compared to $16.1 million for the same period in 2018. The increase in G&A expenses for both the fourth quarter of 2019 and the full year 2019 was primarily due to increased personnel costs and professional fees. G&A expenses included $3.6 million in stock-based compensation expense for the full year 2019.

For the fourth quarter ended December 31, 2019, net loss was $16.0 million, or basic and diluted net loss per share attributable to common stockholders of $0.57. Net loss was $4.7 million for the same period in 2018, or basic and diluted net loss per share attributable to common stockholders of $0.17. For the full year ended December 31, 2019, net loss was $54.8 million, or basic and diluted net loss per share attributable to common stockholders of $1.97. Net loss was $6.6 million for the same period in 2018, or basic and diluted net loss per share attributable to common stockholders of $0.33.

Financial Outlook:

Following the strategic restructuring implemented in January, Surface Oncology’s current cash and cash equivalents are projected to fund the Company into 2022. Anticipated milestones under the NZV930 collaboration with Novartis and additional capital potentially available under the K2 HealthVentures debt financing, in aggregate, would extend Surface Oncology’s cash runway into the second half of 2022.

Bicycle Therapeutics Reports Fourth Quarter and Full Year 2019 Financial Results and Corporate Updates

On March 10, 2020 Bicycle Therapeutics plc (NASDAQ:BCYC), a biotechnology company pioneering a new and differentiated class of therapeutics based on its proprietary bicyclic peptide (Bicycles) technology, reported financial results for the fourth quarter and full year ended December 31, 2019 and discussed recent corporate updates (Press release, Bicycle Therapeutics, MAR 10, 2020, View Source [SID1234555346]).

"Last year was transformational for Bicycle as the completion of our initial public offering and the strengthening of corporate leadership enabled successful execution against our pipeline development strategy," said Kevin Lee, Ph.D., Chief Executive Officer of Bicycle Therapeutics. "This year is already off to a strong start, with two new immuno-oncology collaborations designed to expand upon our wholly owned portfolio of systemic immune cell agonists and tumor-targeted immune cell agonists (TICAs). We look forward to the momentum continuing as we progress multiple Bicycle Toxin Conjugates through the clinic this year and prepare to begin clinical development of our first TICA. We believe our anticipated near-term milestones will further demonstrate the role Bicycles could play in creating a much-needed new treatment paradigm for people living with cancer and other serious diseases."

Fourth Quarter 2019 and Recent Highlights

Entered into Strategic Collaboration with Genentech to Discover, Develop and Commercialize Novel Bicycle-based Immuno-oncology Therapies. In February 2020, Bicycle entered into a strategic collaboration agreement with Genentech. Under the terms of the agreement, Bicycle will be responsible for discovery research and early preclinical development up to candidate selection. Bicycle will receive a $30 million upfront payment. The upfront payment and potential milestone payments could total up to $1.7 billion. Bicycle will also be eligible to receive tiered royalties. None of Bicycle’s wholly owned oncology assets, including its immuno-oncology candidates, are included in the collaboration.
Announced Collaboration with Cancer Research UK to Develop New Bicycle Immuno-oncology Candidate, BT7401. In January 2020, Bicycle announced a second collaboration with Cancer Research UK, the world’s largest independent funder of cancer research. Cancer Research UK will fund and sponsor development of BT7401, a multivalent Bicycle CD137 agonist, through a Phase I/IIa clinical study.
Dosed First Patient in Phase I/II Trial of BT5528, a Second-generation Bicycle Toxin Conjugate (BTC) Targeting EphA2, in Patients with Advanced Solid Tumors. In November 2019, Bicycle announced that the first patient had been dosed in the Phase I dose escalation portion of its Phase I/II trial of BT5528 in patients with advanced solid tumors associated with EphA2 expression. BT5528 is the first second-generation BTC to enter the clinic and has demonstrated promising anti-tumor activity and tolerability across a broad range of preclinical studies.
Presented New Preclinical Data for Novel, Fully Synthetic TICAs at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper)’s (SITC) (Free SITC Whitepaper) 2019 Annual Meeting. In an oral presentation at SITC (Free SITC Whitepaper), Bicycle presented new data showing that its lead immuno-oncology candidate, BT7480, a TICA targeting Nectin-4 and agonizing CD137, rapidly penetrates tumors and effects powerful anti-tumor activity in preclinical models.
Anticipated Key Events in 2020

Initiation of Phase IIa trial of BT1718, a BTC targeting MT1-MMP, in patients with solid tumors expressing MT1 (trial sponsored by Cancer Research UK)
Interim BT5528 Phase I data readout from the ongoing Phase I/II trial
Initiation of nivolumab arm in BT5528 Phase I portion of the ongoing Phase I/II trial
Initiation of a Phase I/II trial of BT8009, a second-generation BTC targeting Nectin-4, a tumor antigen shown to be overexpressed in tumor cells, in patients with solid tumors
Financial Results

Cash was $92.1 million as of December 31, 2019, compared with $63.4 million as of December 31, 2018. Cash at December 31, 2019 does not include the $30 million upfront payment from Genentech.
Research and development expenses were $6.6 million for the three months ended December 31, 2019 and $25.5 million for the year ended December 31, 2019, compared to $6.5 million for the three months ended December 31, 2018 and $20.8 million for the year ended December 31, 2018. The increase of $0.1 million for the three months ended December 31, 2019 as compared to the same period in the prior year was primarily due to an increase in personnel-related expenses, including $0.4 million of incremental non-cash share-based compensation expense, offset by a decrease of $0.9 million in direct program spending. The increase of $4.8 million for the year ended December 31, 2019 as compared to the same period in the prior year was primarily due to an increase of $2.1 million in direct program spending as well as an increase in personnel-related expenses, including $0.8 million of incremental non-cash share-based compensation expense.
General and administrative expenses were $3.4 million for of the three months ended December 31, 2019 and $14.6 million for the year ended December 31, 2019, compared to $2.1 million for of the three months ended December 31, 2018 and $8.1 million for the year ended December 31, 2018. The increase of $1.3 million for the three months ended December 31, 2019 as compared to the same period in the prior year is primarily due $0.9 million in personnel-related costs, including $0.4 million of incremental non-cash share-based compensation expense, as well as $1.6 million in professional fees and costs related to operations as a public company, offset by a $1.2 million favorable effect of foreign exchange rates. The increase of $6.4 million for the year ended December 31, 2019 as compared to the same period in the prior year was primarily due to an increase in personnel-related expenses, including $1.2 million of incremental non-cash share-based compensation expense, as well as an increase in professional fees and costs related to operations as a public company, offset by a $0.6 million favorable effect of foreign exchange rates.
Net loss was $4.4 million, or $(0.25) basic and diluted net loss per share, for the three months ended December 31, 2019 and net loss was $30.6 million, or $(2.77) basic and diluted net loss per share for the year ended December 31, 2019, compared to net loss of $6.6 million, or $(13.19) basic and diluted net loss per share for the three months ended December 31, 2018, and net loss of $21.8 million, or $(49.78) basic and diluted net loss per share for the year ended December 31, 2018.

Precision BioSciences Reports Fourth Quarter and Fiscal Year 2019 Financial Results and Provides Business Update

On March 10, 2020 Precision BioSciences, Inc. (Nasdaq: DTIL) ("Precision"), a life sciences company dedicated to improving life through the application of its pioneering, proprietary ARCUS genome editing platform, reported financial results for the fourth quarter and fiscal year ended December 31, 2019, and provided a business update (Press release, Precision Biosciences, MAR 10, 2020, View Source [SID1234555344]).

"The considerable progress we made in 2019, our first year as a public company, positions Precision BioSciences to further accelerate development across all areas of our portfolio," commented Matt Kane, CEO and co-founder of Precision BioSciences. "We now have three clinical-stage allogeneic CAR T candidates in development for the treatment of NHL, B-ALL, CLL/SLL and multiple myeloma, and have the clinical team and resources to focus on rapidly advancing these programs. We look forward to providing meaningful updates on their progress during 2020. Over the past year, we also built and opened our first-in-class cGMP-compliant manufacturing facility, advanced our partnered and wholly-owned in vivo gene-correction programs, and achieved validating milestones at our food-focused subsidiary, Elo Life Systems. I am very proud of the achievements of the entire Precision team in 2019 and am excited to continue this impressive pace of progress in 2020."

Recent Developments and Upcoming Milestones

Allogeneic CAR T portfolio

Presented initial clinical data for lead CD19-targeted allogeneic CAR T candidate PBCAR0191; dose escalation continuing on-track with further updates expected during 2020. IND applications cleared for wholly-owned CD20 and BCMA targeted CAR T programs. PBCAR20A clinical trial set to begin at higher dose level, accelerating development

On December 9, 2019, Precision presented initial results from the ongoing Phase 1/2a trial of its lead allogeneic chimeric antigen receptor (CAR) T product candidate, PBCAR0191. Data included adult patients with relapsed or refractory (R/R) non-Hodgkin lymphoma (NHL) or R/R B-cell precursor acute lymphoblastic leukemia (B-ALL) treated at the lowest two dose levels (Dose Level 1, 3×105 cells/kg; Dose Level 2, 1×106 cells/kg). No dose-limiting toxicities or cases of graft-versus-host disease were observed. Early evidence of dose dependent, cell-mediated anti-cancer activity was observed. In the NHL cohort, four out of six patients treated across Dose Levels 1 and 2 achieved an objective response at day 28+, for an objective response rate of 67%. Two of three NHL patients treated at Dose Level 1 achieved partial responses at day 28+. Responses at day 28+ were seen to deepen in the three NHL patients treated at Dose Level 2, with one patient achieving a complete response and one patient achieving a partial response. In the B-ALL cohort, one of three patients treated at Dose Level 2 achieved a complete response at day 28+; the remaining two B-ALL patients with progressive disease had high disease burden and poor prognostic indicators on entry into the trial.

The PBCAR0191 trial is ongoing and dosing is progressing as planned. Based on the data observed at Dose Levels 1 and 2, and after discussion with the U.S Food and Drug Administration (FDA), Precision recently implemented an amendment to the PBCAR0191 trial protocol designed to further optimize clinical activity. The amended trial design is intended to specifically address key clinical questions. These include assessing the impact of higher total doses of cells on clinical activity and/or the impact of modified lymphodepletion on the ability to achieve durable clinical benefit with associated CAR T cell expansion and persistence.

The most important modification is the inclusion of two additional dose levels: Dose Level 4 (6×106 cells/kg) and Dose Level 5 (9×106 cells/kg). These higher doses will employ a split dosing strategy following a single lymphodepletion, with the intention of optimizing clinical activity, while preserving a favorable safety profile. Dose Level 4 will comprise two infusions of 3×106 cells/kg, and Dose Level 5 will comprise three such infusions.

In addition, Precision now has the option to increase or decrease the doses of fludarabine and cyclophosphamide used in the lymphodepletion protocol if data suggest that CAR T cell rejection limits efficacy.

The final modification allows for the option to re-dose patients following evidence of clinical response but with subsequent disease progression.

Precision expects to announce additional clinical data from the PBCAR0191 trial for both the NHL and B-ALL cohorts during 2020. PBCAR0191 is being developed in collaboration with Servier, an international pharmaceutical company.

In September 2019, the FDA cleared the Investigational New Drug (IND) application for PBCAR20A, Precision’s second and wholly-owned CAR T therapy candidate, which is expected to enter the clinic in Q1 2020. Based on the safety profile so far observed for PBCAR0191, the FDA has permitted Precision to begin dosing with PBCAR20A at Dose Level 2 (1×106 cells/kg), which is expected to accelerate the trial. PBCAR20A is an allogeneic anti-CD20 CAR T therapy candidate in development for the treatment of (1) NHL and (2) chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL). PBCAR20A has received Orphan Drug Designation from the FDA for the treatment of mantle cell lymphoma.

In January 2020, the FDA also cleared Precision’s IND application for its third CAR T therapy candidate, PBCAR269A, which is also wholly-owned by Precision and which targets BCMA for the treatment of patients with multiple myeloma. PBCAR269A has received Orphan Drug Designation from the FDA, and dosing of patients in a Phase 1/2a clinical trial is expected to begin in 2020.

In vivo gene correction portfolio

Lead in vivo gene correction program identified, clinical candidate selection expected in 2020

In January 2020, Precision announced that it expects to advance its first wholly-owned in vivo gene correction program for the treatment of primary hyperoxaluria type 1 (PH1), a rare genetic disease. In collaboration with Dr. Jim Wilson’s group at the University of Pennsylvania, Precision has generated encouraging preclinical data demonstrating that one-time administration of an ARCUS nuclease can efficiently knock out the validated target gene HAO1 in hepatocytes of non-human primates, and can reduce urine oxalate, a known PH1 biomarker, by up to 70% in a mouse model. In 2020, Precision expects to select a clinical candidate for this program to advance into human trials.

In partnership with Gilead, Precision is developing an in vivo gene correction program to potentially cure chronic infection with the hepatitis B virus. Submission of an IND for this product candidate is currently targeted for 2021.

Elo Life Systems

Advanced Elo Life Systems’ key food and agriculture programs

Elo’s partnered program with Cargill focuses on developing canola oil with ultra-low saturated fatty acids. In 2019, the partnership was able to demonstrate progress towards the goal of achieving a product with <2.5% saturate levels. The program remains ongoing to further reduce saturate levels.

In 2020, Elo expects to advance its ZeroMelonTM program to develop sustainable and scalable alternatives for zero-calorie sweeteners.

Corporate activities

In-house cGMP-compliant manufacturing facility opened and operational. Enhanced leadership team, Board of Directors and Scientific Advisory Board

In July 2019, Precision announced the opening of its in-house Current Good Manufacturing Practice (cGMP) compliant manufacturing facility, located in Research Triangle Park, North Carolina. The Manufacturing Center for Advanced Therapeutics (MCAT) is designed to manufacture clinical trial material for Precision’s planned Phase 1/2 clinical trials starting in 2020, beginning with its BCMA CAR T program. In the longer term, Precision believes MCAT has the potential to be a commercial launch facility. MCAT is expected to provide various operational and strategic advantages to the company as its CAR T and gene correction portfolios continue to progress.

Precision made key appointments to its leadership team in 2019, including Chris Heery, MD, Chief Medical Officer; Dario Scimeca, General Counsel; and Nicholas Riddle, MD, PhD, Vice President, Financial Strategy and Investor Relations. In addition, David Thomson, PhD, was appointed to the position of Chief Operating Officer after serving as Precision’s Chief Development Officer since 2017.

Precision added new members to its Board of Directors in 2019, including Kevin J. Buehler, former CEO of Alcon Laboratories, and Raymond Schinazi, PhD, Hon DSc, the Frances Winship Walters Professor of Pediatrics and Director of the Laboratory of Biochemical Pharmacology at Emory University.

Precision formed a Scientific Advisory Board of leading experts in immuno-oncology and infectious disease, including Raymond Schinazi, PhD; Kenneth C. Anderson, MD, the program director of the Jerome Lipper Multiple Myeloma Center and LeBow Institute for Myeloma Therapeutics at the Dana-Farber Cancer Institute; Hagop Kantarjian, MD, Chair of the Department of Leukemia at The University of Texas MD Anderson Cancer Center; and Cameron Turtle, MBBS, PhD, Associate

Member at Fred Hutchinson Cancer Research Center and Associate Professor at the University of Washington.

Upcoming Corporate Presentations

Precision’s senior leadership team will be participating in the following upcoming conferences:

Barclays Global Healthcare Conference, March 12, 2020

H.C. Wainwright Global Life Sciences Investor Conference, April 21, 2020 in London, UK

Jefferies 7th Annual Cell Therapy Summit, April 30, 2020 in New York, NY

Fiscal Year 2019 Financial Results

Cash and Cash Equivalents: As of December 31, 2019, Precision had approximately $180.9 million in cash and cash equivalents. The Company expects that existing cash and cash equivalents will be sufficient to fund operating expenses and capital expenditure requirements into the second half of 2021.

Revenues: Total revenues for the year ended December 31, 2019 were $22.2 million, compared to $10.9 million for the year ended December 31, 2018. This increase was primarily due to research funding from Precision’s joint development collaboration partners.

Research and Development Expenses: Research and development expenses were $82.4 million for the year ended December 31, 2019, as compared to $45.1 million for the same period in 2018. This increase of $37.3 million was primarily due to increases in platform development and early stage research expenses, including increases in personnel costs, laboratory supplies and services and expenses to support Precision’s technology platform development and manufacturing capabilities.

General and Administrative Expenses: General and administrative expenses were $27.0 million for the year ended December 31, 2019, as compared to $13.7 million for the same period in 2018. The increase of $13.3 million was primarily due to an increase in employee-related costs for additional personnel and facility costs associated with the Company’s growing infrastructure needs.

Net Loss: Net loss was $92.9 million, or $(2.21) per share, for the year ended December 31, 2019, compared to a net loss of $46.0 million, or $(2.92) per share, for the same period in 2018.