On February 25, 2022 Biohaven Pharmaceutical Holding Company Ltd. (NYSE: BHVN), a biopharmaceutical company with a portfolio of innovative, late-stage product candidates targeting neurological diseases, including rare disorders, reported financial results for the fourth quarter and full year ended December 31, 2021, and provided a review of accomplishments during 2021 and anticipated upcoming milestones (Press release, Biohaven Pharmaceutical, FEB 25, 2022, View Source [SID1234609079]).

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Vlad Coric, M.D., Chief Executive Officer and Chairman of the Board of Biohaven commented, "Last year was transformative for Biohaven as NURTEC ODT achieved an impressive $463 million net revenue in 2021 and we signed a $1.2 billion global commercialization agreement with Pfizer for rimegepant and zavegepant. Achieving a second FDA approval, NURTEC ODT became the first and only migraine therapy approved to both treat and prevent migraine attacks. Our dual indication helped catapult NURTEC ODT to become the market-leading novel migraine therapy in 2021. We delivered over 1,600,000 prescriptions for NURTEC ODT since launch and ensured broad access for patients with approximately 90% commercial insurance coverage. The growth trajectory of the oral CGRP class has dramatically exceeded expectations and we believe our commercial organization is well poised in 2022 to achieve continued growth and market leadership for NURTEC ODT. We are excited about our partnership with Pfizer to bring rimegepant to migraine patients outside the U.S. and look forward to further expanding our migraine franchise with our upcoming NDA filing for intranasal zavegepant, the only intranasal CGRP receptor antagonist in late-stage development."

Dr. Coric continued, "Beyond our migraine franchise, Biohaven has a broad and deep strategic pipeline. With the recent additions to our neuroscience portfolio, we are advancing five novel development platforms – CGRP, Kv7 Ion Channel Activators, MPO, Myostatin and Glutamate Modulation – in addition to fueling a discovery engine in Biohaven Labs. We anticipate a steady flow of clinical results from our nearly 20 programs, plus life cycle management progress and numerous geographic market expansions in 2022 and beyond."

Full Year and Recent Business Highlights

CGRP Antagonist Platform – Milestones and Next Steps

Full-year 2021 net product revenue from sales of NURTEC ODT totaled $462.5 million – The Company recognized $190.0 million in net product revenue from sales of NURTEC ODT in the fourth quarter of 2021, a 40% increase compared to the third quarter of 2021, and a 441% increase compared to the fourth quarter of 2020.
Entered strategic collaboration with Pfizer for the commercialization of rimegepant and zavegepant outside of the United States– In November 2021, Biohaven and Pfizer announced a strategic commercialization arrangement for NURTEC ODT in markets outside of the United States upon approval. The strategic commercialization arrangement closed in January 2022. At closing, Pfizer made an upfront payment to Biohaven of $500.0 million, consisting of $150.0 million cash and $350.0 million in the purchase of Biohaven equity. Biohaven is also eligible to receive up to $740.0 million in future milestones and tiered, double-digit royalties on ex-U.S. net sales. In addition to the tiered double-digit royalties owed to Biohaven on net sales outside of the U.S., Pfizer will compensate Biohaven for a pro-rata share of certain of its sales-based milestone obligations owed to Bristol-Myers Squibb Company ("BMS") and for the related royalties on net sales outside of the U.S. owed under Biohaven’s license and funding agreements with BMS and Royalty Pharma.
NURTEC ODT approved in the U.S. for the preventive treatment of migraine – In May 2021, the Company announced that the FDA approved NURTEC ODT for the preventive treatment of episodic migraine. This milestone approval makes NURTEC ODT the first and only medication approved to both treat and prevent migraine attacks, expanding the product label to include the use of NURTEC ODT 75 mg up to 18 doses per month. In the pivotal Phase 3 clinical trial, NURTEC ODT rapidly and effectively prevented migraine, reducing migraine days by 30% after just 1 week of every other day treatment, and approximately half of patients experienced at least a 50% reduction in moderate-to-severe migraine days by 3 months of treatment.
Received positive opinion recommending the granting of a marketing authorization for rimegepant 75 mg for the acute and preventive treatment of migraine, from the Committee for Medicinal Products for Human Use, a committee of the European Medicines Agency – In February 2022, the Company was notified that CHMP adopted a positive opinion, recommending the granting of a marketing authorization in the European Union ("EU") for rimegepant 75 mg (available as an orally dissolving tablet), intended for the prophylaxis and acute treatment of migraine. If approved, VYDURA(rimegepant) will be the commercial name for rimegepant in the EU. The full indication for VYDURA is the acute treatment of migraine with or without aura in adults and preventive treatment of episodic migraine in adults who have at least four migraine attacks per month. Biohaven expects to receive determination regarding its Marketing Authorization Application in the EU for rimegepant in the first half of 2022.
Reported positive topline results from pivotal trial of NURTEC ODT for the acute treatment of migraine in China and South Korea– In February 2022, the Company announced positive topline results from China and South Korea, Phase 3 clinical trial of NURTEC ODT in adults for the acute treatment of migraine. Led by BioShin Limited, a subsidiary of Biohaven, the randomized, double-blind, placebo-controlled, regional, multi-center study met the co-primary endpoints evaluating the efficacy and safety of the orally dissolving tablet ("ODT") formulation of rimegepant, an oral CGRP receptor antagonist. This is the fifth positive pivotal study of rimegepant and the first to be conducted in Asia Pacific. The study met its co-primary endpoints of freedom from pain (p<0.0001) and freedom from most bothersome migraine–associated symptom ("MBS") including either nausea, phonophobia or photophobia (p<0.0001) at 2-hours following a single oral dose of rimegepant. The early onset and durable 48-hour efficacy observed in China and South Korea were consistent with previous clinical trial results. In addition to the co-primary endpoints, NURTEC ODT preliminary data demonstrated significant relief of multiple migraine symptoms with rapid onset efficacy that was statistically superior to placebo as early as 45 minutes for both pain relief and freedom from MBS (p<0.05), at 60 minutes for return to normal function (p = 0.0023), and at 90 minutes for pain freedom (p = 0.0012). NURTEC ODT demonstrated sustained efficacy on all four of these clinically important efficacy outcomes through 48 hours. The use of rescue medication within 24 hours was significantly lower for NURTEC ODT-treated patients than for placebo (p < 0.0001). Rimegepant also showed a favorable safety and tolerability profile among study participants that was consistent with prior clinical trial results in the United States. Detailed data from the study will be presented at future medical meetings to help inform ongoing and future research.
NURTEC ODT approved in Israel for both acute and preventative treatment of migraine – In December 2021, the Company announced that the Ministry of Health of Israel approved NURTEC ODT for preventative treatment, the first global approval outside of the U.S. for prevention. In March 2021, the Company announced approval in the region for the acute treatment of migraine.
NURTEC ODT approved in Kuwait for the acute treatment of migraine – In November 2021, the Company announced that NURTEC ODT obtained registrational approval from the Kuwait Ministry of Health. This marks the third approval in the Middle East region in 2021, joining prior market approvals in both Israel and the United Arab Emirates in March.
Investigational New Drug ("IND") Applications filed for NURTEC ODT for the indications of chronic rhinosinusitis and temporomandibular disorder ("TMD") – In the first quarter of 2022, Biohaven received "Study May Proceed" communications from the FDA regarding the Company’s proposed clinical trials for the use of NURTEC ODT in chronic rhinosinusitis and TMD. Both clinical trials are expected to commence in the first quarter of 2022.
Reported positive topline results from pivotal Phase 3 trial of intranasal zavegepant for the acute treatment of migraine – In December 2021, the Company announced positive topline results from its pivotal Phase 3 trial of intranasal zavegepant for the acute treatment of migraine. The results of the study showed that zavegepant was statistically superior to placebo on the co-primary endpoints of pain freedom (24% vs 15%, p <.0001) and freedom from most bothersome symptom (40% vs 31%, p = 0.0012) at 2 hours. Zavegepant was superior to placebo demonstrating pain relief as early as 15 minutes, with patients achieving return to normal function as early as 30 minutes after dosing (p < 0.006). The efficacy benefits of zavegepant were durable, including superiority versus placebo (p < 0.05) on: sustained pain freedom 2 to 24 hours; sustained pain freedom 2 to 48 hours; sustained pain relief 2 to 24 hours; and sustained pain relief 2 to 48 hours. Previously, the efficacy and safety of intranasal zavegepant were shown in a randomized controlled Phase 2/3 dose-ranging trial with a total of over 1,000 patients treated. Biohaven plans to file a New Drug Application ("NDA") for intranasal zavegepant with the U.S. FDA in the first half of 2022 and other countries thereafter.
Enrolled first patient in Phase 2/3 clinical trial of oral zavegepant for the preventive treatment of migraine – In March 2021, the Company enrolled the first patient in a Phase 2/3 clinical trial for the use of oral zavegepant for the preventive treatment of migraine. As a part of the previously announced funding agreement with Royalty Pharma to advance the development of zavegepant, Biohaven concurrently received a $100.0 million milestone payment. The randomized, double-blind, placebo-controlled trial will enroll approximately 2,900 people with migraine and will evaluate the efficacy and safety of 100 mg and 200 mg doses of oral zavegepant. Topline results for the Phase 2/3 clinical trial is expected in the second half of 2022. Biohaven’s zavegepant program also encompasses intranasal zavegepant as well as oral formulations of zavegepant for non-migraine indications.
Kv7 Platform Acquisition – Milestones and Next Steps

Acquired Kv7 channel platform for treatment of epilepsy and other neurologic disorders from Channel Biosciences, LLC – In February 2022, the company announced that it had entered into a definitive agreement with Channel Biosciences, LLC, a subsidiary of Knopp Biosciences, LLC, to acquire a Kv7 channel targeting platform, adding the latest advances in ion-channel modulation to Biohaven’s growing neuroscience portfolio. BHV-7000 (formerly known as KB-3061) is the lead asset from the Kv7 platform and is a potentially best-in-class potassium channel activator with a profile suggestive of a wide therapeutic index, high selectivity, and significantly reduced GABA-ergic activity. Biohaven intends to bring BHV-7000 to the clinic in 2022 in preparation for a development program in focal epilepsy. In consideration for the transaction, Biohaven will make an upfront payment comprised of $65 million in Biohaven common shares and $35 million in cash to Knopp Biosciences. Biohaven has also agreed to make additional success-based earnout payments (i) up to $325 million based on BHV-7000 developmental and regulatory epilepsy milestones through approvals in the US, EU and Japan, (ii) up to an additional $250 million based on developmental and regulatory milestones for the Kv7 pipeline development in other indications and additional country approvals, and (iii) up to $562.5 million for commercial sales-based milestones of BHV-7000, the total of which will be achieved if annual worldwide net sales exceed $3 billion. Biohaven has also agreed to make scaled royalty payments for BHV-7000 and the pipeline programs, starting at high single digits and peaking at low teens for BHV-7000 and starting at mid-single digits and peaking at low double-digits for the pipeline programs.
Taldefgrobep Alfa Platform License – Milestones and Next Steps

Entered into a worldwide license agreement with BMS for taldefgrobep alfa, a Phase 3-ready anti-myostatin adnectin for SMA – In February 2022, the Company announced that it entered into a worldwide license agreement with BMS for the development and commercialization rights to taldefgrobep alfa (also known as BMS-986089), a novel, Phase 3-ready anti-myostatin adnectin. Taldefgrobep is a muscle-targeted experimental treatment developed for neuromuscular disease and offers the opportunity for combination therapy. The in-licensing of taldefgrobep expands Biohaven’s portfolio of innovative, late-stage product candidates for the treatment of neurologic, neuroinflammatory, and psychiatric indications. Under the terms of the agreement, Biohaven will receive worldwide rights to taldefgrobep alfa and BMS will be eligible for regulatory approval milestone payments, as well as tiered, sales-based royalties beginning in the high-teens. Biohaven plans to initiate a Phase 3 clinical trial of taldefgrobep alfa in SMA in 2022.
Glutamate Modulation Platform – Milestones and Next Steps

Initiated pivotal Phase 3 trial of troriluzole in OCD– In January 2021, the Company announced that it had commenced enrollment in a Phase 3 clinical trial assessing the efficacy and safety of troriluzole in patients with Obsessive Compulsive Disorder ("OCD"). Biohaven is advancing the 280 mg once daily dose of troriluzole into two double-blind, placebo-controlled Phase 3 clinical trials with identical study designs and plans to enroll approximately 600 patients in each of these adjunctive treatment trials across study sites in both the United States and Europe. Phase 3 trial enhancements include increased sample size to adequately power for previously observed treatment effect, a higher dose of troriluzole and optimized clinical trial design to minimize placebo effect.
Myeloperoxidase ("MPO") Platform – Milestones and Next Steps

Completed enrollment of verdiperstat arm of pivotal HEALEY ALS trial – In November 2021, the Company announced that approximately 160 individuals with Amyotrophic Lateral Sclerosis ("ALS") were enrolled in the verdiperstat treatment arm of the pivotal HEALEY ALS Platform Trial. Verdiperstat was selected as one of the inaugural investigational treatments to be evaluated in this first-ever platform trial in ALS, which began enrolling patients in July 2020 across 52 sites from the Northeast ALS Consortium ("NEALS"). Topline results for verdiperstat in the treatment of ALS are anticipated in mid-2022.
Biohaven Labs and Other Collaborations

Entered exclusive license and research collaboration agreement with KU Leuven to develop and commercialize first-in-class TRPM3 antagonists for the treatment of chronic pain – In January 2022, the Company announced that it entered into an exclusive global license and research agreement with the Center for Drug Design and Discovery ("CD3") and the Laboratory of Ion Channel Research ("LICR") at Katholieke Universiteit Leuven ("KU Leuven") to develop and commercialize first-in-class transient receptor potential melastatin-3 ("TRPM3") channel antagonists. BHV-2100 is the lead TRPM3 antagonist from the platform and an orally-bioavailable small molecule TRPM3 antagonist.
Initiated Phase 1a/1b clinical trial of BHV-1100 for the treatment of Multiple Myeloma – In October 2021, Biohaven announced the enrollment of the first patient in a Phase 1a/1b clinical trial of BHV-1100 in combination with autologous cytokine induced memory-like ("CIML") natural killer ("NK") cells and immunoglobulin ("Ig") to target and kill multiple myeloma cells expressing the cell surface protein CD38. BHVN-1100 is the lead asset from Biohaven’s ARMTM (Antibody Recruiting Molecule) Platform. The trial plans to enroll 25 patients for this single-center, open-label study, and will enroll newly diagnosed multiple myeloma patients who have minimal residual disease ("MRD+") in first remission prior to an autologous stem cell transplant ("ASCT").
Announced BHV-1200 demonstrates effective neutralization of multiple strains of COVID-19 – In February 2021, the Company announced that a hyperimmune globulin mimic developed with Biohaven’s proprietary Multimodal Antibody Therapy Enhancer ("MATE") platform has demonstrated functional binding and neutralization of the SARS-CoV-2 virus, including the strains known as the "English" and "South African" variants (also known as B.1.1.7 and B.1.351, respectively). The Company intends to advance BHV-1200 into a full clinical development program. Accelerated development of the COVID-19 MATE program has been supported by the Bill and Melinda Gates Foundation. In addition, the in vitro data indicate that BHV-1200 may activate important immune system components including antibody-dependent cellular phagocytosis and antibody dependent cellular cytotoxicity. Biohaven’s proprietary MATE-conjugation technology could also be used against other infectious diseases by changing the targeting moiety of its antibody binders.
Corporate Updates:

Appointed CEO Vlad Coric as Chairman of the Board of Directors – In December 2021, the Board of Directors unanimously elected Vlad Coric, MD to the role of Chairman of the Board of Directors following the retirement of Declan Doogan, MD.
Appointed Matthew Buten as Chief Financial Officer– In December 2021, the company announced the appointment of Matthew Buten to the role of Chief Financial Officer ("CFO") effective on December 20, 2021, following the retirement of CFO James Engelhart.
Appointed Kishen Mehta to Board of Directors – In June 2021, the Company announced the appointment of Kishen Mehta, Portfolio Manager at Suvretta Capital Management, LLC, to its Board of Directors. Mr. Mehta previously served as a strategic advisor to Biohaven.
Upcoming Milestones:

Biohaven is continuing to support the launch of NURTEC ODT and develop its product candidates through clinical and preclinical programs in several common and rare disorders. The Company expects to reach significant pipeline milestones with its CGRP receptor antagonist, glutamate modulator, and myeloperoxidase inhibitor platforms in 2022.

Biohaven expects to:

Submit an NDA to the FDA for intranasal zavegepant for acute treatment of migraine in the first quarter of 2022.
Receive a determination regarding its Marketing Authorization Application in the E.U. for rimegepant in the first half of 2022.
Submit an NDA for the acute treatment of migraine in China and South Korea in the second half of 2022.
Report topline of troriluzole in Spinocerebellar Ataxia ("SCA") in the first half of 2022.
Report topline of verdiperstat in ALS in the second half of 2022.
Capital Position:

At the start of 2022, Biohaven has approximately $1.0 billion of available liquidity. On January 4, 2022, we received $500.0 million in upfront proceeds from Pfizer relating to the strategic collaboration arrangement, consisting of $150.0 million cash and $350.0 million in proceeds from the purchase of Biohaven common shares at a 25% market premium. Cash, cash equivalents, and marketable securities as of December 31, 2021, were $364.6 million, excluding $2.4 million of restricted cash, compared to $355.3 million as of December 31, 2020. We also have $125.0 million in non-dilutive committed capital from our credit facility with Sixth Street.

Fourth Quarter 2021 Financial Highlights

Product Revenue, Net: Net product revenue was $190.0 million for the three months ended December 31, 2021, compared to $35.1 million for the three months ended December 31, 2020. The increase of $154.9 million in net product revenue is due to both increased NURTEC ODT sales volume and improvements in net price realization due to decreases in sales allowances during the three months ended December 31, 2021, compared to the three months ended December 31, 2020. Sales allowances and accruals mostly consisted of patient affordability programs, distribution fees and rebates.

Research and Development ("R&D") Expenses: R&D expenses, including non-cash share-based compensation costs, were $91.1 million for the three months ended December 31, 2021, compared to $73.5 million for the three months ended December 31, 2020. The increase of $17.6 million was primarily due to an increase in both late-stage product candidates and preclinical research. Non-cash share-based compensation expense was $9.7 million for the three months ended December 31, 2021, an increase of $3.9 million as compared to the same period in 2020.

Selling, General and Administrative ("SG&A") Expenses: SG&A expenses, including non-cash share-based compensation costs, were $219.5 million for the three months ended December 31, 2021, compared to $122.4 million for the three months ended December 31, 2020. The increase of $97.1 million was primarily due to increases in spending to support increased commercial sales of NURTEC ODT for the three months ended December 31, 2021, compared to the three months ended December 31, 2020. Less than half of the SG&A expense was for commercial organization personnel costs, excluding non-cash share-based compensation expense. Non-cash share-based compensation expense was $15.1 million for the three months ended December 31, 2021, an increase of $6.7 million as compared to the same period in 2020.

Net Loss: Biohaven reported a net loss attributable to common shareholders for the three months ended December 31, 2021, of $199.2 million, or $3.01 per share, compared to $217.7 million, or $3.62 per share for the same period in 2020. Non-GAAP adjusted net loss for the three months ended December 31, 2021 was $153.4 million, or $2.32 per share, compared to $161.7 million, or $2.69 per share for the same period in 2020. These non-GAAP adjusted net loss and non-GAAP adjusted net loss per share measures, more fully described below under "Non-GAAP Financial Measures," exclude non-cash share-based compensation charges, non-cash interest expense related to the accounting for mandatorily redeemable preferred shares and liability related to sale of future royalties, changes in the fair value of derivatives, gains or losses from equity method investment, upfront non-cash collaboration and license upfront expenses, and accrued development milestone payments. A reconciliation of the GAAP financial results to non-GAAP financial results is included in the tables below.

Full Year 2021 Financial Highlights

Product Revenue, Net: Net product revenue was $462.5 million for the year ended December 31, 2021, compared to $63.6 million for the year ended December 31, 2020. The increase of $398.9 million in net product revenue was due to both increased NURTEC ODT sales volume and improvements in net price realization due to decreases in sales allowances in 2021, compared to 2020, and a full year of NURTEC ODT sales during 2021 compared to a partial period of NURTEC ODT sales in 2020. Sales allowances and accruals mostly consisted of patient affordability programs, distribution fees and rebates.

R&D Expenses: R&D expenses, including non-cash share-based compensation, were $361.3 million for the year ended December 31, 2021, compared to $229.0 million for the year ended December 31, 2020. The increase of $132.3 million was primarily due to clinical development costs including post-approval commitment studies, expenses from later stage trials in our zavegepant programs and preclinical research costs. Non-cash share-based compensation expense was $52.1 million for the year ended December 31, 2021, an increase of $28.4 million as compared to the same period in 2020.

SG&A Expenses: SG&A expenses, including non-cash share-based compensation costs, were $713.5 million for the year ended December 31, 2021, compared to $462.3 million for the year ended December 31, 2020. The increase of $251.2 million was primarily due to increases in spending to support the commercial launch of NURTEC ODT and non-cash share-based compensation expense. Non-cash share-based compensation expense was $75.0 million for the years ended December 31, 2021, an increase of $41.3 million as compared to the same period in 2020.

Net Loss: The Company reported a net loss attributable to common shareholders for the year ended December 31, 2021 of $846.6 million, or $13.09 per share, compared to $766.8 million, or $13.06 per share for the same period in 2020. Non-GAAP adjusted net loss for the year ended December 31, 2021 was $637.8 million, or $9.86 per share, compared to $605.4 million, or $10.31 per share for the same period in 2020. These non-GAAP adjusted net loss and non-GAAP adjusted net loss per share measures, more fully described below under "Non-GAAP Financial Measures," exclude non-cash share-based compensation charges, non-cash interest expense related to the accounting for mandatorily redeemable preferred shares and liability related to sale of future royalties, changes in the fair value of derivatives, gains or losses from equity method investment, upfront non-cash collaboration and license upfront expenses, and accrued development milestone payments. A reconciliation of the GAAP financial results to non-GAAP financial results is included in the tables below.

Conference Call Information
As previously announced, the Company will hold a conference call to discuss its fourth quarter and full year 2021 results today at 8:30 a.m. EDT. To access the call, please dial 877-407-9120 (domestic) or 412-902-1009 (international). The conference call webcast and accompanying slide presentation can be accessed through the "Investors" section of Biohaven’s website at www.biohavenpharma.com. To ensure a timely connection, it is recommended that participants register at least 15 minutes prior to the scheduled webcast. A replay of the call will be made available for two weeks following the conference call. To hear a replay of the call, dial 877-660-6853 (domestic) or 201-612-7415 (international) with conference ID 13726591. An archived webcast will be available on Biohaven’s website.

On February 25, 2022 BeiGene, Ltd. (NASDAQ: BGNE; HKEX: 06160; SSE: 688235), a global biotechnology company focused on developing and commercializing innovative medicines worldwide, reported financial results for the fourth quarter and full year of 2021, recent business highlights, and anticipated upcoming milestones (Press release, BeiGene, FEB 25, 2022, View Source [SID1234609052]).

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"Last year was transformative for our company, and we have built strategic competitive advantages that will help BeiGene to achieve our mission of making innovative medicines more readily accessible and affordable for all who need them. We have significantly increased the reach of our medicines, as BRUKINSA is now approved in 45 markets, and we now have 16 approved medicines in China, including our sixth approved indication for tislelizumab and five approved Novartis Oncology products in designated regions of China that we plan to promote following the transition from Novartis," said John V. Oyler, Co-Founder, Chairman and Chief Executive Officer of BeiGene. "With exciting momentum for BRUKINSA including acceptance by the FDA and EMA of our supplemental NDAs for CLL, the most common form of adult leukemia, we are building a strong portfolio with cornerstone assets that will support the development of potential future new therapies. In addition, we have expanded our productive collaboration with Novartis by entering into an option, collaboration and license agreement to accelerate our anti-TIGIT antibody, ociperlimab, which is one of the most advanced assets in its class."
Julia Wang, Chief Financial Officer, commented, "We are strongly positioned financially in an environment where the cost of capital is rising for our industry. In 2021, we added over $4 billion to our balance sheet through our public offering and new listing on the STAR market and the upfront payment from our collaboration with Novartis for tislelizumab, plus an additional $300 million in early 2022 for our TIGIT collaboration, that validates the strength of our research and development. Our commercial team is generating substantial product revenue from our internal and partnered medicines, and we expect revenue to continue to grow meaningfully in the year ahead based on approvals achieved and upcoming catalysts. We remain committed to building our internal clinical development and manufacturing capabilities that offer cost advantages and managing our expenses with discipline."
Fourth Quarter and Full Year 2021 Financial Results
Cash, Cash Equivalents, Restricted Cash and Short-Term Investments were $6.62 billion as of December 31, 2021, compared to $3.92 billion as of September 30, 2021, and $4.66 billion as of December 31, 2020. Cash and cash equivalents as of December 31, 2021 do not include the $300 million upfront payment from Novartis for the ociperlimab option, collaboration and license agreement, which was received in January 2022.
•For the fourth quarter of 2021, total cash and short-term investments increased $2.7 billion; cash used in operating activities was $507.8 million; capital expenditures were $115.0 million; and cash provided from financing activities was $3.4 billion, almost entirely due to the net proceeds from the STAR Market offering on December 15, 2021.
•For the full year 2021, total cash and short-term investments increased $2.0 billion; cash used in operating activities was $1.3 billion; capital expenditures were $262.9 million; and cash provided from financing activities was $3.6 billion, primarily due to the net proceeds from the STAR Market offering on December 15, 2021.
Revenue for the fourth quarter and full year 2021 was $214.0 million and $1.2 billion, respectively, compared to $100.1 million and $308.9 million in the prior year periods. The increase in total revenue in the quarter compared to the prior year is attributable to sales of our internally developed products, sales of in-licensed products from Amgen and BMS, as well as collaboration revenue from the Novartis agreements.
•Product revenues totaled $196.8 million and $634.0 million for the fourth quarter and full year 2021, respectively, compared to $100.1 million and $308.9 million in the prior year periods, and were comprised of:
–Global sales of BRUKINSA of $87.6 million and $218.0 million for the fourth quarter and full year 2021, respectively, compared to $18.3 million and $41.7 million in the prior year periods. Full year 2020 revenue from BRUKINSA reflects sales since its launch in China in June 2020, as well as sales in the United States for the full year;
–Sales of tislelizumab in China of $54.4 million and $255.1 million for the fourth quarter and full year 2021, respectively, compared to $63.5 million and $163.4 million in the prior year periods. Sales of tislelizumab in the fourth quarter were impacted by 2022 National Reimbursement Drug List (NRDL) price reductions, which resulted in a negative adjustment of $25.4 million as a result of the normal process in China of compensating

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Exhibit 99.1

distributors for products previously sold at the 2021 price during the quarter that remained in the distribution channel. Full year 2021 sales of tislelizumab included two negative adjustments totaling $45.6 million for distributor channel inventory compensation as a result of inclusion in the March 2021 and January 2022 NRDL lists. Full year 2020 revenue from tislelizumab reflects sales since its launch in China in March 2020;
–Sales of BMS in-licensed products in China of $29.9 million and $89.7 million for the fourth quarter and full year 2021, respectively, compared to $12.6 million and $95.1 million in the prior year periods, respectively; and
–Sales of Amgen in-licensed products in China of $20.3 million and $58.8 million for the fourth quarter and full year 2021, respectively, compared to $5.4 million and $8.5 million in the prior year periods. We began selling Amgen’s XGEVA (denosumab) and BLINCYTO (blinatumomab) in July 2020 and August 2021, respectively.
•Collaboration revenue totaled $17.2 million and $542.3 million for the fourth quarter and full year 2021, resulting from the partial recognition of the upfront payments from Novartis of $650.0 million related to the tislelizumab agreement and $300.0 million related to the ociperlimab agreement, which were entered into in the first and fourth quarters of 2021, respectively. We had no collaboration revenue in 2020.
Expenses for the fourth quarter and full year 2021 were $785.7 million and $2.6 billion, respectively, compared to $585.0 million and $2.0 billion in the prior year periods.
•Cost of Sales for the fourth quarter and full year 2021 were $48.5 million and $164.9 million, respectively, compared to $21.1 million and $70.7 million in the prior year periods. Cost of sales increased primarily due to increased product sales of tislelizumab, BRUKINSA and XGEVA, as well as sales of BLINCYTO, which commenced in August 2021.
•R&D Expenses for the fourth quarter and full year 2021 were $430.5 million and $1.5 billion, respectively, compared to $355.5 million and $1.3 billion in the prior year periods. The increase in R&D expenses was primarily attributable to increases in headcount and costs related to investment in our discovery and development activities, including our continued efforts to internalize research and clinical development activities, partially offset by lower fees paid to external CROs on clinical trials for BRUKINSA, tislelizumab and pamiparib, as well as decreased expense related to upfront fees for in-process R&D. Upfront fees related to in-process R&D for in-licensed assets totaled $30.0 million and $83.5 million in the fourth quarter and full year 2021, respectively, compared to nil and $109.5 million in the prior year periods. Employee share-based compensation expense also contributed to the overall increase in R&D expenses and was $30.6 million and $114.4 million for the fourth quarter and full year 2021, respectively, compared to $23.5 million and $93.0 million in the prior year periods, due to increased headcount and a higher share price.
•SG&A Expenses for the fourth quarter and full year 2021 were $306.5 million and $990.1 million, respectively, compared to $208.2 million and $600.2 million in the prior year periods. The increase in SG&A expenses was primarily attributable to increased headcount, largely related to continued expansion of our commercial teams, higher professional service fees and higher external commercial expenses, including selling and marketing, market access studies and promotional activities. The overall increase in SG&A expenses was also attributable to higher SG&A-related share-based compensation expense, which was $32.4 million and $126.4 million for the fourth quarter and full year 2021, respectively, compared to $26.0 million and $90.5 million for the prior year periods, due to increased headcount and a higher share price.
•Net Loss for the fourth quarter and full year 2021 was $585.7 million and $1.4 billion, or $0.47 and $1.17 per share, respectively, or $6.16 and $15.23 per ADS, respectively, compared to $472.7 million and $1.6 billion, or $0.40 and $1.47 per share, or $5.20 and $19.13 per ADS, respectively, in the prior year periods.
Recent Business Highlights
Commercial Operations
•Product sales increased 96.6% and 105.3% in the fourth quarter and full year of 2021 compared to the prior year periods, primarily due to increased sales of our internally developed products and in-licensed products from Amgen;
•Global sales of BRUKINSA totaled $87.6 million and $218.0 million in the fourth quarter and full year 2021, representing 378% and 423% increases compared to the prior year periods; U.S. sales of BRUKINSA totaled $55.9 million and $115.7 million in the fourth quarter and full year 2021, representing growth of 539% and 535%, compared to the prior year periods. U.S. sales continued to accelerate in the quarter, driven by continued uptake in mantle cell lymphoma (MCL) and the recent FDA approvals in Waldenström’s macroglobulinemia (WM) and marginal zone lymphoma (MZL). BRUKINSA sales in China totaled $30.6 million and $101.2 million in the fourth

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Exhibit 99.1

quarter and full year 2021, representing growth of 219% and 331% compared to the prior year periods, driven by a significant increase in all approved indications, including chronic lymphocytic leukemia (CLL). Fourth quarter and full year 2021 sales of BRUKINSA in China were negatively impacted by accrued compensation to distributors of $4.4 million and $7.9 million, respectively, due to price changes related to BRUKINSA’s inclusion in the March 2021 and January 2022 NRDL lists;
•Sales of tislelizumab in China totaled $54.4 million and $255.1 million in the fourth quarter and full year 2021, representing a decline of 14% and growth of 56% compared to the prior year periods. In the fourth quarter, new patient demand from broader reimbursement and further expansion of our salesforce and hospital listings continued to drive increased market penetration and market share for tislelizumab;
•Continued to build commercial capabilities in designated regions of China referred to as Broad Markets to accommodate increasing medical demand. Our new agreement with Novartis granting us rights to market and promote five Novartis approved and nationally reimbursed oncology products expands our portfolio and enables our commercial team to reach patients in the Broad Markets with these medicines. These products include: TAFINLAR (dabrafenib), MEKINIST (trametinib), VOTRIENT (pazopanib), AFINITOR (everolimus), and ZYKADIA (ceritinib);
•Secured NRDL inclusion in China for our eligible medicines, including tislelizumab in first-line non-squamous non-small cell lung cancer (NSCLC), first-line squamous NSCLC and second- or third-line hepatocellular carcinoma (HCC), BRUKINSA in WM, and pamiparib in germline in patients with BRCA (gBRCA) mutation-associated recurrent advanced ovarian, fallopian tube, or primary peritoneal cancer who have been treated with two or more lines of chemotherapy; and
•Our commercial organization in China continued to demonstrate its ability to bring new products to market, launching QARZIBA and POBEVCY in the fourth quarter.
Development Programs
BRUKINSA (zanubrutinib), a small molecule inhibitor of Bruton’s tyrosine kinase (BTK) designed to maximize BTK occupancy and minimize off-target effects, approved in 45 markets including the U.S., China, European Union (EU), Great Britain, Canada, and Australia in selected indications and under development for additional approvals globally. The global BRUKINSA development program includes nearly 4,000 subjects enrolled to-date in more than 25 countries and regions.
•Announced acceptance by the U.S. FDA of a supplemental new drug application for the treatment of adult patients with CLL or small lymphocytic lymphoma (SLL) based on data from two pivotal randomized Phase 3 studies ALPINE (NCT03734016) and SEQUOIA (NCT03336333) presented at the 26th European Hematology Association (EHA) (Free EHA Whitepaper) (EHA2021) Virtual Congress in June 2021 and at the 63rd American Society for Hematology (ASH) (Free ASH Whitepaper) Annual Meeting in December 2021, respectively. The Prescription Drug User Fee Act (PDUFA) target action date is October 22, 2022;
•Announced acceptance by the European Medicines Agency of two marketing authorization applications for the treatment of patients with CLL and MZL;
•Received approval in South Korea for the treatment of adult patients with MCL who have received at least one prior therapy, and for the treatment of adult patients with WM who have received at least one prior therapy;
•Received approval in the EU for the treatment of adult patients with WM who have received at least one prior therapy or for the first-line treatment of patients unsuitable for chemo-immunotherapy. The approval is applicable to all 27 EU member states, plus Iceland, Liechtenstein and Norway. BeiGene has begun launching BRUKINSA in the EU;
•Received approvals for WM in Great Britain and Switzerland, and new approvals for MCL in Saudi Arabia and Ecuador, as well as new indication approval for MZL in Canada, and national reimbursement coverage for patients with WM in Israel. There are currently more than 40 marketing authorization applications in multiple indications under review around the world;
•Included in the National Comprehensive Cancer Network (NCCN) Clinical Practice Guidelines in Oncology (NCCN Guidelines version 2.2022) for patients with both CLL/SLL as a Category 2A preferred treatment option in the first- and second-line, for patients with and without del(17p)/TP53 mutation. BRUKINSA is not approved in CLL/SLL outside of China; and

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Exhibit 99.1

•Announced acceptance of supplemental new drug applications (sNDA) in China as a treatment for adult patients with WM, and as a treatment for adult patients with CLL or SLL with breakthrough therapy designation (BTD).
Tislelizumab, a humanized IgG4 anti-PD-1 monoclonal antibody specifically designed to minimize binding to FcγR on macrophages; approved in China in selected indications and under development for additional approvals globally. The global tislelizumab clinical development program includes more than 9,000 subjects enrolled to-date in more than 35 countries and regions.
•Received approval in China in a sixth indication, for second- or third-line treatment of locally advanced or metastatic NSCLC;
•Announced positive findings from the global Phase 3 RATIONALE 305 trial (NCT03777657) versus placebo in combination with chemotherapy as a first-line treatment for patients with locally advanced, unresectable or metastatic gastric or gastroesophageal junction (G/GEJ) adenocarcinoma with PD-L1 expression. At the interim analysis, tislelizumab in combination with chemotherapy met the primary endpoint of overall survival (OS) in patients with PD-L1 expression, with additional follow-up needed to assess OS benefits in the intention-to-treat (ITT) population. The safety profile of tislelizumab was consistent with that observed in previous trials, with no new safety signals identified with the addition of chemotherapy; and
•Presented results from the Phase 3 RATIONALE 309 trial (NCT03924986) of tislelizumab in first-line patients with nasopharyngeal cancer at the European Society for Medical Oncology Immuno-Oncology Congress 2021. The trial significantly prolonged progression-free survival for patients and demonstrated a safety profile consistent with known risks of each treatment agent.
Ociperlimab (BGB-A1217), an investigational anti-TIGIT monoclonal antibody with competent Fc function. The global ociperlimab development program includes approximately 800 subjects enrolled to-date in 25 countries and regions.
•Announced an option, collaboration, and license agreement with Novartis to develop, manufacture and commercialize ociperlimab in North America, Europe, and Japan. BeiGene received an upfront payment of $300 million and granted Novartis an exclusive time-based option until late 2023 under which BeiGene would receive an additional payment of $600 or $700 million upon exercise of the option, subject to receipt of required antitrust approval. During the option period, Novartis will conduct and fund additional global clinical trials of ociperlimab in combination with tislelizumab in selected tumor types; and
•Initiated patient enrollment in BeiGene’s global Phase 2 AdvanTIG-205 trial (NCT05014815) in frontline stage IV NSCLC.
Early Stage Programs
•Continued to advance our early stage clinical pipeline of internally-developed product candidates at dose escalation stage, including BGB-A445 (an investigational non-ligand competing OX40 monoclonal antibody as monotherapy or in combination with tislelizumab in solid tumors), BGB-15025 (an investigational hematopoietic progenitor kinase 1 (HPK1) inhibitor as monotherapy or in combination with tislelizumab in solid tumors), and BGB-10188 (an investigational PI3Kδ inhibitor as monotherapy or in combination with BRUKINSA in hematology malignancies, or in combination with tislelizumab in solid tumors); and
•Initiated the first Phase 1 clinical trial (NCT05093270) of BGB-23339, a potent, allosteric investigational tyrosine kinase 2 (TYK2) inhibitor being developed for inflammation and immunology.
Collaboration with Amgen
•Launched KYPROLIS (carfilzomib) for injection, a next-generation proteasome inhibitor, in China for patients with R/R multiple myeloma.
Other Collaboration Programs
•Received approval and launched POBEVCY (a biosimilar to bevacizumab) in China, licensed from Bio-Thera Solutions, Ltd., for the treatment of patients with advanced, metastatic or recurrent NSCLC and metastatic colorectal cancer;

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Exhibit 99.1

•Received approval in China of SYLVANT (siltuximab for injection), licensed from EUSA Pharma (UK), Ltd., for the treatment of adult patients with multicentric Castleman disease (MCD) who are human immunodeficiency virus (HIV) negative and human herpes virus-8 (HHV-8) negative, also known as idiopathic MCD (iMCD);
•Launched QARZIBA (dinutuximab beta) in China, a targeted immunotherapy licensed from EUSA Pharma, for the treatment of high-risk neuroblastoma in patients aged 12 months and above who have previously received induction chemotherapy and achieved at least a partial response, followed by myeloablative therapy and stem cell transplantation, as well as patients with a history of R/R neuroblastoma with or without residual disease; and
•Entered a worldwide license and collaboration agreement with Nanjing Leads Biolabs, Co., Ltd. (Leads Biolabs), for research, development and manufacturing rights and exclusive commercialization rights outside of China to LBL-007, a novel investigational antibody targeting the LAG-3 pathway.
Zanidatamab, an investigational bispecific antibody targeting HER2 in late-stage clinical development with Zymeworks Inc.
•Received Breakthrough Therapy Designation from the Center for Drug Evaluation (CDE) of China’s National Medical Products Administration (NMPA) for treating patients with biliary tract cancer who have failed prior systemic therapies; and
•Initiated the global Phase 3 HERIZON‑GEA‑01 clinical trial (NCT05152147) of zanidatamab plus chemotherapy, with or without tislelizumab, versus standard of care (trastuzumab plus chemotherapy), for the first-line treatment of metastatic HER2‑positive gastroesophageal adenocarcinoma (GEA).
Corporate Developments
•Completed the public offering and initial listing of our ordinary shares on the Science and Technology Innovation Board (STAR Market) of the Shanghai Stock Exchange under the stock code "688235" with net proceeds of approximately US$3.4 billion;
•Held a grand opening ceremony for the BeiGene Bioisland Innovation Center (BIC) in Guangzhou, China to support scientists and entrepreneurs in accelerating the development of highly differentiated, cutting-edge medical innovations. The BIC is an innovator-centric incubator built on BeiGene’s goal of supporting exploration of new paths to meet patient needs around the world;
•Announced the appointment of Drs. Margaret Dugan and Alessandro Riva to our Board of Directors and the resignation of Sam Su from our Board after almost four years of service; and
•Expanded our Executive Committee with the following new leaders:
–Mark Lanasa, M.D., Senior Vice President, Chief Medical Officer, Solid Tumor;
–Kyu-Sung ("Q") Lee, Senior Vice President, Global Head of Technical Operations and Manufacturing
–Kevin Mannix, Senior Vice President, Investor Relations;
–Jurij Petrin, Head of New Market Development; and
–Eva Yin, Chief Commercial Officer, Greater China.
Manufacturing Operations
•Closed on the acquisition of a 42-acre site at the Princeton West Innovation Campus in Hopewell, N.J., which BeiGene plans to develop as a new commercial-stage manufacturing and clinical R&D campus. Construction of the initial phase is expected to commence in 2022. In addition, the property has more than one million square feet of developable real estate for potential future expansion;
•Continued construction on the new small molecule manufacturing campus in Suzhou, China. Phase 1 of construction will bring over 52,000 square meters and 600M solid oral dosage capacity and is expected to be completed in 2023. Once completed, qualified, and approved, the total production capacity is expected to increase BeiGene’s small molecule manufacturing capability in China by up to six times the current capacity; and

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Exhibit 99.1

•Continued construction on the state-of-the-art biologics facility in Guangzhou, China, which currently is approved for 8,000 liters of biologics capacity, with an additional phase of construction to bring total capacity to 64,000 liters expected to be completed and GMP-ready by the end of 2022.
Expected Milestones
BRUKINSA
•Announce updated topline results for the Phase 3 ALPINE trial (NCT03734016) in R/R CLL/SLL in the second quarter of 2022;
•Announce clinical data from the global Phase 2 ROSEWOOD trial (NCT03332017) in R/R follicular lymphoma in 2022;
•Continue to support ongoing FDA review of the sNDA submission for CLL/SLL, which has a PDUFA target action date of October 22, 2022;
•Continue to support the EMA review of new indication applications for CLL and MZL; and
•Continue to expand BRUKINSA’s registration program globally in new geographies and indications, including potential launches in 2022 in more than 10 markets.
Tislelizumab
•Receive approvals in China for the three sBLAs currently under review in first-line NPC, second-line ESCC, and second- or third-line MSI-High solid tumors in 2022;
•In collaboration with Novartis, continue to support the ongoing FDA review of the BLA submission in second-line ESCC, which has a PDUFA target action date of July 12, 2022;
•Continue to support additional planned BLA filings by Novartis in first-line NPC in the U.S. and in NSCLC in the U.S. and EU in 2022; and
•Announce topline results from the global Phase 3 clinical trial (NCT03412773) as first-line treatment for patients with HCC in 2022.
Ociperlimab
•Initiate additional pivotal clinical trials in 2022; and
•Announce data from Phase 1 trial (NCT04047862) cohorts in various solid tumor types in the second half of 2022.
Pamiparib
•Report topline results from the Phase 3 trial (NCT03519230) in China of pamiparib as a maintenance treatment in patients with platinum-sensitive recurrent ovarian cancer, in the first half of 2022.
BGB-11417 (BCL-2)
•Activate pivotal trials in 2022.
Early Stage Programs
•Initiate dose expansion in the Phase 1 clinical trial (NCT04215978) of BGB-A445 (OX-40) in patients with advanced solid tumors in the first half of 2022.
COVID-19 Impact and Response
•The Company expects that the worldwide health crisis of COVID-19 will continue to have a negative impact on its operations, including commercial sales, regulatory interactions, inspections, filings, manufacturing, and clinical trial recruitment, participation, and data read outs. There remains uncertainty regarding the future impact of the pandemic globally. The Company is striving to minimize delays and disruptions, and continues to execute on its commercial, regulatory, manufacturing, and clinical development goals globally.

On February 25, 2022 Lexicon Pharmaceuticals, Inc. (Nasdaq: LXRX), reported that it will release its fourth quarter 2021 financial results on Monday, February 28, 2022 before the markets open (Press release, Lexicon Pharmaceuticals, FEB 25, 2022, View Source [SID1234609080]). Management will conduct a conference call and live webcast at 8:00 a.m. ET (7:00 a.m. CT) that day to discuss its financial and operating results and to provide a general business update.

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The dial-in replay will be available for 14 days following the call. An audio webcast will be available online at www.lexpharma.com/events, with a webcast replay accessible for 14 days after the call.

On February 25, 2022 Ascendis Pharma A/S (Nasdaq: ASND) reported that the Company will hold a conference call and live webcast on Wednesday, March 2, 2022 at 4:30 p.m. Eastern Time (ET) to review its 2021 financial results and provide a business update (Press release, Ascendis Pharma, FEB 25, 2022, View Source [SID1234609058]).

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Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

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Conference Call Details

A live webcast of the conference call will be available on the Investors and News section of the Ascendis Pharma website at www.ascendispharma.com. A webcast replay will be available on this website shortly after conclusion of the event for 30 days.

On February 25, 2022 Molecular Partners AG (SIX: MOLN; NASDAQ: MOLN), a clinical-stage biotech company developing a new class of custom-built protein drugs known as DARPin therapeutics, reported that its Chief Executive Officer, Patrick Amstutz, Ph.D., will participate in two upcoming healthcare investor events in March, 2022 (Press release, Molecular Partners, FEB 25, 2022, View Source [SID1234609082]). In addition, the Company will publish its full year 2021 annual report on March 15 and hold an investor conference call on March 16, 2021.

Schedule your 30 min Free 1stOncology Demo!
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Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

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Presentation Details:

Cowen Health Care Conference – Fireside chat

Wednesday, March 9, 2022 at 2:10-2:40pm ET
Conference Participation Details:

Credit Suisse London Global Healthcare Conference

Tuesday, March 1, 2022 to Wednesday, March 2, 2022
Webcasted presentations will be made available through the Molecular Partners website.