On February 24, 2021 Paratek Pharmaceuticals, Inc. (Nasdaq: PRTK), a commercial-stage biopharmaceutical company focused on the development and commercialization of novel life-saving therapies for life-threatening diseases or other public health threats for civilian, government and military use, reported financial results and provided an update on corporate activities for the quarter and year ended December 31, 2020 (Press release, Paratek Pharmaceuticals, FEB 24, 2021, View Source [SID1234575546]).

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"The launch of NUZYRA continued to demonstrate consistent quarter-over-quarter growth through 2020 in a challenging environment created by the ongoing COVID-19 pandemic," said Evan Loh, M.D., Chief Executive Officer at Paratek. "The full year commercial net U.S. sales of NUZYRA, which landed at the high end of our guidance range, reflects the strong health of the underlying core commercial business. With two Biomedical Advanced Research and Development Authority (BARDA) Project BioShield procurements for the Strategic National Stockpile (SNS) now anticipated in 2021 plus the expected continued commercial growth of NUZYRA, we are anticipating a significant ramp up in total revenue for 2021. We believe prescribers recognize NUZYRA as an important life-saving antibiotic that addresses patient needs in the face of the growing global threat from antibiotic resistance."

"In our conversations with clinicians over the past several years, it is clear there is a tremendous unmet need for a well-tolerated, once daily oral broad-spectrum antibiotic that includes coverage against MRSA for skin infections. Current generic antibiotic options are universally challenged by either significant bacterial resistance or serious safety concerns that limit their clinical utility," said Adam Woodrow, Paratek’s President and Chief Commercial Officer. "NUZYRA’s product profile makes it a very attractive treatment option for community use. With access now solidly established with the payers and significant support from infectious disease specialists, we believe our primary care expansion will accelerate and strengthen our sales trajectory, especially as we enter the second half of 2021."

Key 2021 Priorities

Expansion of NUZYRA into Primary Care Setting: Paratek completed the hiring of approximately 40 U.S. sales representatives in February 2021 to enable the expansion of the NUZRYA launch into the primary care setting. The Company expects to execute this expansion within its previously communicated cash runway guidance.
NTM Rare Disease Opportunity: The Company plans to initiate a Phase 2b study, as early as mid-2021, to explore the potential utility of omadacycline as a treatment for non-tuberculous mycobacteria (NTM) infections due to Mycobacterium abscessus (M. abscessus), a rare disease with currently no approved therapies. M. abscessus and based on Company estimates represents a potential $1.0 billion addressable market opportunity in the U.S.
Continued Progress of the BARDA Project BioShield Anthrax Program: The Company expects to continue to execute and deliver on its contract with BARDA to develop NUZYRA for the treatment of pulmonary anthrax, a critical bioterrorism threat deemed important to national security by BARDA. These activities include advancing the U.S. onshoring of NUZYRA manufacturing and the continued execution of certain preclinical research activities supporting the pulmonary anthrax development program. Under Paratek’s five-year agreement with BARDA, the Company also anticipates two procurements of 2,500 treatment courses of NUZYRA each in 2021 for addition to the SNS.
Other Recent Highlights

In December 2020, Paratek entered into a $60 million non-recourse loan agreement with an affiliate of R-Bridge Healthcare Investment Advisory (the R-Bridge Loan) that will be repaid using (i) all proceeds of royalties from the Company’s License and Collaboration Agreement with Zai Lab and (ii) an initial 2.5% revenue interest from the Company’s U.S. net sales of NUZYRA (initial annual cap of $10 million).
The net proceeds of the R-Bridge Loan, together with cash on hand, was used to prepay in full all obligations outstanding under the Company’s Amended and Restated Loan and Security Agreement with Hercules Capital on December 31, 2020.
Fourth Quarter and Full Year 2020 Financial Results

Fourth quarter 2020 revenue
Total revenue was $16.0 million for the fourth quarter of 2020, an increase of 17% over the third quarter of 2020 and an increase of 78% over the fourth quarter of 2019.
NUZYRA generated $12.4 million in net U.S. sales during the fourth quarter of 2020, an increase of 14% over the third quarter of 2020 and an increase of 130% over the fourth quarter of 2019.
Government contract service and grant revenue was $2.8 million for the fourth quarter of 2020 versus $2.7 million in the third quarter of 2020. No revenue was generated from the BARDA contract in 2019 as it was executed in December of that year.
Full year 2020 total revenue
Full year 2020 total revenue was $46.9 million, an increase of 184% over full year 2019 total revenue.
NUZYRA generated $38.8 million in net U.S. sales for the full year 2020, an increase of 237% over the full year 2019.
The growth in NUZYRA sales in 2020 reflects the Company’s continued commitment to strong execution and NUZYRA’s attributes that address unmet needs in the marketplace despite the significant disruptions of patient traffic to physician offices and institutions throughout the ongoing COVID-19 pandemic.
In today’s COVID-19 environment, NUZYRA’s ability to minimize hospital stays with the oral formulation is especially relevant for many prescribers and a meaningful benefit for patients.
Government contract service and grant revenue was $6.7 million in 2020. No revenue was generated from the BARDA contract in 2019 as it was executed in December of that year.
Collaboration and royalty revenue of $1.5 million in 2020 is primarily comprised of royalties earned from SEYSARA sales in the U.S. Collaboration and royalty revenue of $5.0 million in 2019 included a $3.0 million milestone earned from Zai Lab in addition to royalties earned from SEYSARA sales in the U.S.
Research and development (R&D) expenses were $6.3 million for the fourth quarter of 2020, compared to $9.1 million for same period in the prior year. R&D expenses were $23.9 million for the full year 2020, compared to $39.6 million in the prior year. The decrease in both periods is primarily the result of lower clinical study costs associated with completion of the Phase 2 UTI program in 2019, partially offset by an increase in expenses incurred under the BARDA contract and third-party manufacturing process scale-up to meet expected future demand for NUZYRA.

Selling, general and administrative (SG&A) expenses were $24.3 million for the fourth quarter of 2020, compared to $21.3 million for the same period in the prior year.  The $3.0 million increase is primarily due to costs incurred to realign our hospital territories and our community expansion.

SG&A expenses were $89.9 million for the full year 2020, compared to $89.1 million in the prior year. The $0.8 million increase is primarily the result of costs for the Company’s contract sales organization and higher product distribution fees, partially offset by lower marketing and promotional program expenses, personnel-related costs and travel due to the COVID-19 pandemic.

Paratek reported a net loss of $25.0 million, or ($0.54) per share, for the fourth quarter of 2020, compared to a net loss of $27.4 million, or ($0.81) per share, for the same period in 2019.

Paratek reported a net loss of $96.5 million, or ($2.19) per share, for the full year 2020 compared to a net loss of $128.8 million, or ($3.93) per share, for the same period in 2019.

Financial Guidance
Paratek also announced its full year 2021 financial guidance.

Paratek estimates 2021 total revenue in the range of $166 to $177 million. This range includes the following components:
2021 NUZYRA net U.S. product sales are expected to be approximately $138 to $144 million, which includes:
U.S. net product sales between $62 to $68 million from the core commercial business
Two SNS procurements by BARDA valued at approximately $76 million
Royalty and collaboration revenue of approximately $8 million
BARDA government contract service and grant revenue between $20 to $25 million
BARDA contract service and grant revenue consists of reimbursement of NUZYRA post-marketing requirements, the anthrax development program and the U.S. onshoring of NUZYRA manufacturing
Paratek estimates 2021 R&D and SG&A expense to be approximately $150 to $155 million. This range includes the following components:
Core business R&D and SG&A expense of $130 million, an increase of 20% over 2020 but consistent with 2019 pre-pandemic levels
The expected increase in R&D and SG&A expense in 2021 when compared to 2020 will be driven by costs associated with the primary care expansion and the initiation of the phase 2b study in NTM
BARDA R&D and U.S. onshoring cost reimbursements between $20 to $25 million
Based upon the Company’s current operating plan as described above, Paratek anticipates its existing cash, cash equivalents and marketable securities of $125.2 million as of December 31, 2020, provide for a cash runway through the end of 2023 with a pathway to cash flow break even.
Company performance and unanticipated events could cause actual results to vary from this forward-looking guidance.

Call and Webcast
Paratek’s earnings conference call for the quarter and year ended December 31, 2020 will be broadcast at 4:30 p.m. EST on February 24, 2021. The live audio webcast can be accessed under "Events and Presentations" in the Investor Relations section of Paratek’s website at www.ParatekPharma.com.

Domestic investors wishing to participate in the call should dial: 877-407-0792 and international investors should dial: 201-689-8263. The conference ID is 13716761. Investors can also access the call at View Source

Website Information
Paratek routinely posts important information for investors on the Investor Relations section of its website at www.ParatekPharma.com. Paratek intends to use this website as a means of disclosing material, non-public information and for complying with its disclosure obligations under Regulation FD. Accordingly, investors should monitor the Investor Relations section of Paratek’s website, in addition to following its press releases, U.S. Securities and Exchange Commission (SEC) filings, public conference calls, presentations and webcasts. The information contained on, or that may be accessed through, Paratek’s website is not incorporated by reference into, and is not a part of, this document.

On February 24, 2021 vTv Therapeutics Inc. (Nasdaq:VTVT) reported financial results for the fourth quarter and year ended December 31, 2020, and provided an update on the progress of its clinical programs (Press release, vTv Therapeutics, FEB 24, 2021, View Source [SID1234575545]).

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"Despite the challenges of operating through a global pandemic, 2020 was a successful year for vTv Therapeutics," said Steve Holcombe, president and CEO. "We had positive results in the Phase 2 SimpliciT-1 Study with our lead compound, TTP399, paving the way for our continued development of this asset in patients with type 1 diabetes. In addition, we strengthened our current and future financial position with the initiation of our ATM, agreement with Lincoln Park Capital, and licensing agreement with Anteris Bio."

"In 2021, we look forward to building on these successes as we advance our two lead programs for the treatment of type 1 diabetes and psoriasis. We plan to initiate our first pivotal study of TTP399 along with other supporting studies to demonstrate our unique glucokinase activator’s potential to reduce the incidence of hypoglycemia in people with type 1 diabetes. Furthermore, we have commenced a multiple ascending dose study with HPP737 to be followed by a phase 2 study in patients with psoriasis in order to demonstrate HPP737’s potential to be a well-tolerated, next-generation PDE4 inhibitor."

Recent Achievements and Outlook

Type 1 Diabetes

Mechanistic Study of Ketoacidosis with TTP399. To further support the hypothesis that TTP399 may help reduce the incidence of ketoacidosis, vTv will begin dosing of a mechanistic study of TTP399 in patients with type 1 diabetes during Q1 of 2021 to determine the impact of TTP399 on ketone body formation during a period of acute insulin withdrawal. The FDA agreed with the Company’s recommendation that such a mechanistic study be performed in support of the Company’s planned pivotal trials. vTv expects to report topline results from the mechanistic study in Q2/Q3 2021.
Pivotal Study Planning. The Company is planning to initiate the first of two pivotal, placebo-controlled, six-month clinical trials of TTP399 in approximately 400 subjects with type 1 diabetes in 2H 2021. The studies will be designed to assess TTP399’s ability to reduce the incidence of hypoglycemia when administered as an oral adjunct to insulin therapy.
Publication of SimpliciT-1 Results. Diabetes Care, the American Diabetes Association’s journal of clinical research, published the positive results of the Phase 2 SimpliciT-1 Study of TTP399 as an adjunct therapy to insulin in patients with type 1 diabetes, which showed statistically significant reductions in HbA1c and clinically meaningful reductions in hypoglycemia.
Psoriasis

First-Patient First-Visit of Multiple Ascending Dose Study with HPP737. The Company has begun dosing of a phase 1 multiple ascending dose study to assess the safety, tolerability, and pharmacokinetic profile of HPP737, a PDE4 inhibitor, in healthy volunteers. The phase 1 study will inform dose selection for the planned phase 2 study in psoriasis that the Company expects to begin later this year.
Strategic Partnership with Anteris Bio

License Agreement for Nrf2 Activator HPP971. In December 2020, the Company announced a new license agreement with Anteris Bio for worldwide rights to vTv’s novel, clinical-stage Nrf2 activator compound, HPP971. Anteris will be pursuing indications in renal disease with HPP971.
Azeliragon (TTP488)

Discontinuation of azeliragon for Alzheimer’s disease. On December 15, 2020, vTv announced that the phase 2 Elevage Study of azeliragon did not meet its primary objective. vTv has discontinued development of azeliragon for Alzheimer’s disease, but the Company is evaluating potential alternative indications in partnership with other interested parties.
Capital Raising

At-the-Market (ATM) Offering. In January, the Company filed a prospectus supplement for $5.5 million of additional capacity under the Controlled Equity Offering Sales Agreement with Cantor Fitzgerald & Co. As of the date of this release, the full $5.5 million remains available.
Lincoln Park. The Company continues to leverage its partnership with Lincoln Park to raise capital to fund its on-going and planned clinical trials and corporate operations on an opportunistic basis.
Fourth Quarter 2020 Financial Results

Cash Position: The Company’s cash position as of December 31, 2020, was $5.7 million compared to $1.8 million as of September 30, 2020.
Revenue: Revenue for the fourth quarter was $6.4 million and was insignificant for the third quarter of 2020. The revenue for the fourth quarter was primarily attributable to the upfront consideration, consisting of cash and an equity interest, received in connection with the Company’s license agreement with Anteris Bio.
R&D Expenses: Research and development expenses were $2.5 million and $1.8 million for the three months ended December 31, 2020 and September 30, 2020, respectively. This increase of $0.8 million was driven primarily by the reversal of certain performance-based compensation accruals which occurred in the third quarter.
G&A Expenses: General and administrative expenses were $2.0 million for the fourth quarter of 2020 and $1.1 million for the third quarter, respectively. The increase of $1.0 million was driven by the reversal of certain performance-based compensation accruals in the third quarter.
Net Income/(Loss) Before Non-Controlling Interest: Net income before non-controlling interest was $1.6 million for the fourth quarter of 2020 compared to a net loss of $2.3 million for the third quarter of 2020.
Net Income/(Loss) Per Share: Diluted net income per share was $0.02 for the three months ended December 31, 2020 compared to a diluted net loss per share of ($0.03) for the three months ended September 30, 2020, based on weighted-average diluted shares of 74.4 million and 48.2 million for the three-month periods ended December 31, 2020 and September 30, 2020, respectively. Non-GAAP net income per fully exchanged share was $0.02 for the three months ended December 31, 2020 compared to a net loss per fully exchanged share of ($0.02) at September 30, 2020, based on non-GAAP fully exchanged weighted-average shares of 74.4 million and 71.3 million for the three months ended December 31, 2020 and September 30, 2020, respectively.
Full Year 2020 Financial Results

Revenue: Revenues were $6.4 million and $2.8 million for the years ended December 31, 2020 and 2019, respectively. The 2020 revenue is attributable to the upfront payment and fair value of the equity interest received by the Company in connection with the license agreement with Anteris Bio. The revenue earned during 2019 primarily relates to the recognition of amounts deferred for the license agreement with Reneo and a milestone received under the license agreement with Newsoara.
R&D Expenses: Research and development expenses were $11.0 million and $15.1 million for the years ended December 31, 2020 and 2019, respectively. This decrease was attributable primarily to lower spending on clinical trials for azeliragon and TTP399 in 2020 coupled with the impact of reversals of performance-based compensation accruals in 2020.
G&A Expenses: General and administrative expenses were $7.3 million and $8.5 million for the years ended December 31, 2020 and 2019, respectively. Such decreases were primarily driven by lower compensation costs related to a reversal of accruals for performance-based compensation and lower share-based compensation expense in 2020.
Net Loss Before Non-Controlling Interest: Net loss before non-controlling interest was $12.8 million and $21.9 million for the years ended December 31, 2020 and 2019, respectively.
Net Loss Per Share: GAAP net loss per share was $0.18 and $0.59 for the years ended December 31, 2020 and 2019, respectively, based on weighted-average shares of 47.1 million and 30.3 million for the years ended December 31, 2020 and 2019, respectively. Non-GAAP net loss per fully exchanged share was $0.17 and $0.37 for the years ended December 31, 2020 and 2019, respectively, based on non-GAAP fully exchanged weighted-average shares of 70.2 million and 53.4 million for the years ended December 31, 2020 and 2019, respectively.

On February 24, 2021 West Pharmaceutical Services, Inc. (NYSE: WST), a global leader in innovative solutions for injectable drug administration, reported that the Company’s Board of Directors has approved a second-quarter 2021 dividend of $0.17 per share (Press release, West Pharmaceutical Services, FEB 24, 2021, View Source [SID1234575544]). The dividend will be paid on May 5, 2021, to shareholders of record as of April 21, 2021.

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On February 24, 2021 CytomX Therapeutics, Inc. (Nasdaq: CTMX), a clinical-stage oncology-focused biopharmaceutical company pioneering a novel class of investigational conditionally activated antibody therapeutics based on its Probody technology platform, reported fourth quarter and full year 2020 financial results and provided a business update (Press release, CytomX Therapeutics, FEB 24, 2021, View Source [SID1234575543]).

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"2020 was a highly productive year for CytomX in which we saw our clinical-stage pipeline advance to now encompass Phase 2 evaluations of four Probody therapeutics across nine cancer types, all while contending with the challenges posed by the COVID-19 pandemic. We have demonstrated that our Probody masking technology has the potential to widen or create a therapeutic window for first-in-class and validated oncology targets and we continue to execute on our strategic plan of delivering on the promise of our technology platform for transforming the lives of people with cancer," said Sean McCarthy, D.Phil., president, chief executive officer and chairman of CytomX Therapeutics. "Our leadership in the research, discovery and development of conditionally activated antibody therapeutic candidates positions us well for future growth as we now drive to important Phase 2 datasets for praluzatamab ravtansine (CX-2009) and CX-2029, directed against the targets CD166 and CD71, respectively, which have historically been considered to be undruggable. We are also pleased with the ongoing progress within our strategic partnerships including recent commitments from our foundational partner, Bristol Myers Squibb, to expand the evaluation of anti-CTLA-4 antibody, BMS-986249, into additional tumor types," continued Dr. McCarthy.

Business Highlights and Recent Developments

Presented at the 2020 San Antonio Breast Cancer Symposium updated data from the Phase 1 study of the anti-CD166 conditionally activated antibody-drug conjugate (ADC), praluzatamab ravtansine (CX-2009), in patients with human epidermal growth factor receptor 2 (HER2)-non-amplified breast cancer and translational data demonstrating measurable levels of activated praluzatamab ravtansine in tumor tissue, which supported the launch in December 2020 of a three-arm Phase 2 study. Arms A and B will study praluzatamab ravtansine as a single agent in patients with hormone receptor-positive (HR+), HER2-non-amplified breast cancer and triple-negative breast cancer (TNBC), respectively. Arm C will examine the combination of praluzatamab ravtansine and pacmilimab (CX-072), the Company’s proprietary conditionally activated anti-PD-L1 therapeutic candidate, in TNBC.
Continued patient enrollment in the Phase 2 expansion study of CX-2029, in partnership with AbbVie, evaluating the anti-CD71 conditionally activated ADC as a single agent in four cohorts: squamous non-small cell lung cancer, head and neck squamous cell carcinoma, esophageal and gastro-esophageal junction cancers, and diffuse large B-cell lymphoma.
Our partner, Bristol Myers Squibb, continued enrollment in its ongoing, randomized Phase 1/2a study of BMS-986249 in patients with previously-untreated unresectable stage III-IV melanoma and expanded the scope of the Part 2b evaluation to include three new cohorts, enrolling patients with advanced hepatocellular carcinoma, metastatic castration-resistant prostate cancer, and unresectable locally advanced or metastatic TNBC. BMS also continued enrollment into a Phase 1 study of a second anti-CTLA-4 Probody, BMS-986288.
Advancement of our third conditionally activated ADC, CX-2043, into investigational new drug (IND)-enabling studies. CX-2043 is directed against the epithelial cell adhesion molecule (EpCAM/Trop-1), a high potential target with elevated expression on a wide variety of tumor types.
Continued IND-enabling studies for CX-904, our most advanced program in the new and promising modality of T-cell engaging bispecific antibodies. CX-904, partnered with Amgen, targets the epidermal growth factor receptor on tumor cells and the CD3 receptor on T cells.
Continued drug discovery activities for conditionally activated T-cell engaging bispecific antibodies as part of our strategic collaboration with Astellas.
Appointed new Board member Dr. Mani Mohindru.
Strengthened balance sheet with approximately $108 million raised from a follow-on public equity offering.
Anticipated Events

Report initial data from the praluzatamab ravtansine (CX-2009) Phase 2 study in the fourth quarter of 2021.
Report initial data from the CX-2029 Phase 2 expansion study in the fourth quarter of 2021.
Submit IND applications for CX-2043 and CX-904 in late 2021.
Virtual analyst and investor briefing with Key Opinion Leaders in April 2021 to discuss our Probody technology platform with focus on praluzatamab ravtansine and CX-2029.
Fourth Quarter and Full Year 2020 Financial Results
Cash, cash equivalents and short-term investments totaled $316.1 million as of December 31, 2020, compared to $296.1 million as of December 31, 2019. In January 2021, the Company closed on its previously announced underwritten public offering of common stock with net proceeds of approximately $93.6 million. In February 2021, the underwriters exercised in full the option to purchase additional shares of common stock resulting in additional net proceeds of $14.1 million to the Company.

Total revenues were $16.4 million and $100.4 million for the three months and year ended December 31, 2020, respectively, compared to $8.3 million and $57.5 million for the corresponding periods in 2019. The net increase in total revenues were primarily driven by an increase in the percentage of completion of the CD71 Co-Development and Licensing Agreement with AbbVie and the recognition of revenue from the Collaboration and License Agreement with Astellas entered into in March 2020.

Research and development expenses decreased by $14.4 million and $18.7 million during the three months and year ended December 31, 2020, respectively, to $22.0 million and $112.9 million, compared to $36.4 million and $131.6 million for the corresponding periods in 2019. The decreases were largely attributed to a decrease in clinical trial activities primarily due to the COVID-19 pandemic.

General and administrative expenses were essentially flat during the three months and year ended December 31, 2020, amounting to $9.1 million and $36.0 million, respectively, compared to $9.2 million and $36.8 million for the corresponding periods in 2019.

Conference Call & Webcast Information
CytomX management will host a conference call today at 5:00 p.m. ET (2:00 p.m. PT). Interested parties may access the live webcast of the conference call from the Events and Presentations page of CytomX’s website at www.cytomx.com or by dialing 1-877-809-6037 (U.S. and Canada) or 1-615-247-0221 (International) using the passcode 5558715. An archived replay of the webcast will be available on the Company’s website until March 3, 2021.

On February 24, 2021 Zymeworks Inc. (NYSE: ZYME), a clinical-stage biopharmaceutical company developing multifunctional biotherapeutics, reported financial results for the year ended December 31, 2020 and provided a summary of recent business and clinical highlights (Press release, Zymeworks, FEB 24, 2021, View Source [SID1234575542]).

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"Throughout 2020, we initiated and advanced several clinical trials and achieved important regulatory milestones for zanidatamab and ZW49," said Ali Tehrani, Ph.D., Zymeworks’ President & CEO. "This has set up 2021 to be a data-rich year for both of our lead clinical assets as well as new preclinical candidates and therapeutic platforms, and we are well resourced to deliver on our priorities."

2020 Business Highlights and Recent Developments

Clinical Data Continues to Support Goal of Establishing Zanidatamab as the Foundational HER2 Targeted Therapy Across Multiple HER2-Positive Cancers
Updated clinical data was recently presented at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Gastrointestinal Cancers Symposium for zanidatamab, in both HER2-expressing biliary tract cancer (BTC) and gastroesophageal adenocarcinoma (GEA). Overall zanidatamab was well tolerated with the majority of treatment-related adverse events being mild or moderate in severity. With respect to antitumor activity, both refractory BTC and GEA compare favorably to current standard of care and emerging treatments. These data support zanidatamab’s broad therapeutic potential as a foundational therapy across multiple HER2-expressing cancers.
Zanidatamab Advances in Accelerated Pivotal Trial Paving the Way for Commercialization
The U.S. Food and Drug Administration granted Breakthrough Therapy designation to zanidatamab for BTC, enabling an Accelerated Approval pathway for the global pivotal trial (HERIZON-BTC-01) for zanidatamab monotherapy in patients with previously treated HER2 gene-amplified BTC and submission of a Biologics License Application (BLA) as early as 2022. A second pivotal trial evaluating zanidatamab as first-line treatment for advanced HER2-positive GEA is on track to launch mid-2021. Zanidatamab received additional drug review special designations in the U.S. and the European Union that are expected to help to expedite clinical development of both trials.
ZW49 Demonstrates Antitumor Activity and Differentiated Safety Profile; Expansion Cohorts Enrolling while Dose Escalation Continues
ZW49, a bispecific antibody-drug conjugate targeting HER2, has begun enrolling patients into the expansion cohort portion of the ongoing Phase 1 clinical trial. Interim data were recently presented and showed no dose limiting toxicities or treatment-related hematologic, pulmonary, or liver toxicity. The majority of treatment-related adverse events were mild or moderate in severity with the most common being keratitis, fatigue, and diarrhea. ZW49 demonstrated antitumor activity at all dose levels evaluated in the once every three week regimen, including confirmed partial responses and stable disease per RECIST 1.1., and dose escalation is continuing at 3 mg/kg once every three weeks. Three indication-specific expansion cohorts (HER2-positive breast cancer, HER2-positive GEA, and a basket cohort of other HER2-positive cancers) utilizing the 2.5 mg/kg once every three week regimen have also been initiated to better ascertain antitumor activity in more homogeneous patient populations.
Milestone Payments Received and Deal Values Increase for Partnership Deals
Throughout 2020, partnerships continued to expand and additional milestones were achieved upon BeiGene initiating multiple clinical studies with zanidatamab, Merck and BMS expanding Azymetric and EFECT collaborations, and Iconic/Exelixis entering into a licensing deal for a ZymeLink ADC. Zymeworks has nine active collaborations that could result in up to $8.6 billion in potential milestone payments in addition to royalties on potential product sales.
Strengthened Balance Sheet
In January 2020, Zymeworks completed an upsized $320.8 million public financing to accelerate and expand global development of its lead clinical candidates, zanidatamab and ZW49, and support further advancement of its novel preclinical programs.
Financial Results for the Year Ended December 31, 2020

Revenue was $39.0 million in 2020 compared to $29.5 million in 2019. For both years, revenue related to non-recurring upfront fees, milestone payments, option fees, research support and other payments under our licensing and collaboration agreements. Revenue for 2020 included $15.0 million from BeiGene for development milestones, $12.0 million from BMS for an expansion fee and $12.0 million from other partners for research support, partner revenue, drug supply and other payments. Revenue for 2019 included $8.0 million from Lilly for achievement of a development milestone, $7.5 million and $3.5 million from BMS and Daiichi Sankyo, respectively, for exercise of commercial license options, $3.5 million for recognition of deferred revenue relating to our licensing and collaboration agreement with BeiGene, and $7.0 million from our partners for other development milestones, research support and other payments.

Research and development expense was $168.5 million in 2020 compared to $115.9 million in 2019. The $52.6 million increase related primarily to additional clinical trial activities and associated drug manufacturing costs for zanidatamab, as well as an increase in salaries and benefits expense resulting from a higher headcount. Expenses also increased in 2020 for higher development activity for ZW49 and an increase in-licensing and milestone payments for discovery and research activities. Research and development expense included non-cash stock-based compensation expense of $12.3 million in 2020 and $14.3 million in 2019.

General and administrative expense was $57.9 million in 2020 compared to $64.2 million in 2019. General and administrative expense included non-cash stock-based compensation expense of $16.1 million in 2020 and $34.2 million in 2019. Excluding stock-based compensation expense, general and administrative expense increased by $11.9 million year over year primarily due to an increase in salaries and benefits expense resulting from a higher headcount, as well as higher insurance and professional services expenses.

Net loss was $180.6 million in 2020 compared to $145.4 million in 2019. The increase in net loss was primarily due to the increases in research and development expenses referred to above partially offset by lower general and administrative expense and higher revenue and other income.

Zymeworks expects research and development expenditures to increase over time in line with the advancement and expansion of the Company’s clinical development of its product candidates, as well as its ongoing preclinical research activities. Additionally, Zymeworks anticipates continuing to receive revenue from its existing and future strategic partnerships, including technology access fees and milestone-based payments. However, Zymeworks’ ability to receive these payments is dependent upon either Zymeworks or its collaborators successfully completing specified research and development activities.

As of December 31, 2020, Zymeworks had $451.6 million in cash resources consisting of cash, cash equivalents, short-term investments and certain long-term investments.