On May 6, 2021 Bicycle Therapeutics plc (NASDAQ: BCYC), a biotechnology company pioneering a new and differentiated class of therapeutics based on its proprietary bicyclic peptide (Bicycle) technology, reported financial results for the first quarter ended March 31, 2021 and discussed recent corporate updates (Press release, Bicycle Therapeutics, MAY 6, 2021, View Source [SID1234579374]).

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"We’ve had a great start to 2021, as we advance multiple Bicycle Toxin Conjugates (BTCs) in the clinic, prepare for BT7480, our lead tumor-targeted immune cell agonist, to enter the clinic later this year and continue to expand the use of our platform outside of oncology, while also significantly strengthening our balance sheet," said Kevin Lee, Ph.D., Chief Executive Officer of Bicycle Therapeutics. "We plan to build on this momentum during the year, and our presentation of preclinical data at AACR (Free AACR Whitepaper) serves to highlight the potential therapeutic benefits that Bicycles may offer toward improving the treatment paradigms for people living with cancer and other serious diseases."

First Quarter 2021 and Recent Highlights

Presented at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2021. In April 2021, Bicycle presented new preclinical data describing the discovery of BT7480, a novel Nectin-4/CD137 Bicycle tumor-targeted immune cell agonistTM (Bicycle TICA) during a "New Drugs on the Horizon" session. The Company expects BT7480 to enter the clinic in the second half of 2021. Additionally, the Company presented five posters and one presentation highlighting preclinical data across multiple programs in Bicycle’s oncology pipeline. The posters and presentations are available on the Publications section of bicycletherapeutics.com.

Provided Pipeline Progress Update Across Multiple Therapeutic Programs Beyond Oncology. In March 2021, Bicycle announced progress updates for its Bicycle programs outside of oncology:

Achieved first milestone in the collaboration with Dementia Discovery Fund (DDF) and the University of Oxford’s Alzheimer’s Research UK Oxford Drug Discovery Institute (ODDI): The Company identified and optimized nM affinity Bicycles to transferrin receptor 1 (TfR1), a molecular shuttle. The three parties are collaborating to identify and characterize Bicycles that bind to and activate TREM2, a genetically validated dementia target.

Advanced the platform in multiple anti-infective areas, including antimicrobials and antivirals: Innovate UK’s Biomedical Catalyst (BMC) awarded the Company funding to advance a Bicycle inhibitor for a key cell wall biosynthesis target in Enterobacterales, Penicillin Binding Protein 3 (PBP3). Bicycle, working with investigators at the University of Warwick, intends to progress these PbP3 inhibitors, potentially the first novel class of antibiotics identified in decades, to candidate and initial toxicology testing. Additionally, under a specific Innovate UK program targeting key technologies to rapidly respond to the challenge of the COVID-19 pandemic, Bicycle received funding to support its efforts to discover new healthcare solutions to SARS-CoV-2.

Made significant progress through partnerships: Bicycle successfully discovered and advanced targets outside of oncology through its ongoing collaboration with AstraZeneca, a global biopharmaceutical company, to discover novel agents for the treatment of respiratory and cardiometabolic diseases. Two assets were transitioned to AstraZeneca’s pipeline for further development. Bicycle also identified targets in its collaboration with Bioverativ (acquired by Sanofi) for the treatment of rare hematological diseases. The collaborations successfully identified nM multi-valent inhibitors to P-Selectin, which inhibited neutrophil binding and rolling, with potential applications in sickle cell disease and other inflammatory diseases. The Bioverativ collaboration also identified the first small molecule Factor VIII mimetic for the potential treatment of Hemophilia A. Upon termination of the collaboration in 2019, these "lead stage" assets have been returned to Bicycle.

Appointed Jose-Carlos Gutierrez-Ramos, Ph.D., to its Board of Directors: In March 2021, Bicycle announced the appointment of industry veteran Dr. Gutierrez-Ramos to its Board of Directors. Dr. Gutierrez-Ramos previously served as Chair of Bicycle’s Scientific Advisory Board and has extensive experience in leading biopharmaceutical companies and in academia.

Continued to Strengthen the Balance Sheet in 2021. Since January 2021, Bicycle has completed the sale of $75.0 million through its at-the-market (ATM) offering program. Gross proceeds during the first quarter of 2021 totaled $60.6 million, with an additional $14.4 million in gross proceeds recognized in April 2021. Also during the second quarter of 2021, the Company received $2.0 million from Genentech for achieving specified criteria under the collaboration research plan. Cash as of March 31, 2021 does not include the April 2021 ATM proceeds or Genentech proceeds. In addition, in March 2021, the Company drew an additional $15.0 million available under its debt facility with Hercules Capital, Inc. and amended the loan and security agreement to extend the interest-only payment period until the second half of 2023, with the potential to further extend it into 2024, contingent on the satisfaction of performance milestones.
Financial Results

Cash was $195.9 million as of March 31, 2021, compared to $136.0 million as of December 31, 2020. The increase in cash was primarily due to net proceeds of $58.8 million from the ATM offering and net proceeds of $15.0 million from the debt facility with Hercules Capital Inc., offset by cash used in operating activities. Cash at March 31, 2021 does not include net proceeds from the ATM offering received in April 2021. Cash of $195.9 million at March 31, 2021 is expected to provide financial runway through multiple clinical milestones and into 2024.
Research and development expenses were $9.7 million for the three months ended March 31, 2021, compared to $7.8 million for the three months ended March 31, 2020. The increase in expense of $1.9 million for the three months ended March 31, 2021 as compared to the same period in the prior year was primarily due to increased clinical program expenses for BT8009, a second-generation Bicycle Toxin Conjugate (BTC) targeting Nectin-4, and increased other unallocated discovery and platform related expenses due to the timing of development activities, and increased personnel-related expenses, including $0.5 million of incremental non-cash share-based compensation expense.
General and administrative expenses were $8.1 million for the three months ended March 31, 2021, compared to $5.0 million for the three months ended March 31, 2020. The increase of $3.1 million for the three months ended March 31, 2021 as compared to the same period in the prior year was primarily due to an unfavorable effect of foreign exchange rates and an increase in personnel-related costs, including $1.2 million of incremental non-cash share-based compensation expense for the three months ended March 31, 2021.
Net loss was $16.2 million, or $(0.73) basic and diluted net loss per share, for the three months ended March 31, 2021, compared to net loss of $11.3 million, or $(0.63) basic and diluted net loss per share for three months ended March 31, 2020.

On May 6, 2021 Zionexa reported to join GE Healthcare to further develop molecular imaging agents in oncology, all aimed at enabling more precise diagnosis, improved treatment decision-making and ultimately better clinical outcomes for patients (Press release, Zionexa, MAY 6, 2021, View Source [SID1234579390]).​

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Our mission has always been to help physicians personalize treatment to improve patients’ pathway and quality of life. We are delighted to be joining an organization rooted in precision health.

Kevin O’Neill, President and CEO of GE Healthcare Pharmaceutical Diagnostics, said: "Like GE Healthcare, Zionexa’s products are aimed at enabling more precise diagnosis, improved treatment decision-making and ultimately better clinical outcomes for patients. This acquisition further demonstrates our commitment to enabling precision health and providing innovations that support oncologists, nuclear medicine specialists and other physicians throughout a cancer patient’s journey, from initial screening and diagnosis to informing therapy selection and monitoring the effectiveness of treatment."

Olivier Carli, President of Denos, the majority owner of Zionexa, said: "We expect GE Healthcare Pharmaceutical Diagnostics’ acquisition to allow Zionexa to accelerate the development of its promising R&D pipeline as well as its commercial footprint, while providing Zionexa’s team with access to global and complementary expertise

On May 6, 2021 Twist Bioscience Corporation (NASDAQ: TWST), a company enabling customers to succeed through its offering of high-quality synthetic DNA using its silicon platform, reported financial results and business highlights for the second quarter of fiscal 2021 ended March 31, 2021 (Press release, Twist Bioscience, MAY 6, 2021, View Source [SID1234579408]).

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"The second quarter of our fiscal year delivered growth across all four areas of our business, with increasing revenue and very strong orders building momentum for the second half of the year," said Emily M. Leproust, Ph.D., CEO and co-founder of Twist Bioscience. "We signed new biopharma partnerships with multiple associated programs, and we continue to drive increasing density for our data storage vertical.

"For the second half of fiscal 2021 we expect continued growth of our NGS and synbio customer base as we build out the Factory of the Future in Oregon. We intend to advance all programs with our biopharma partners while simultaneously generating data to support out-licensing for our proprietary antibodies. In addition, for data storage, we will continue to pursue innovative engineering and collaborate across the DNA Data Storage Alliance to prepare the market for this novel storage medium."

FISCAL 2021 SECOND QUARTER FINANCIAL RESULTS

Orders: Total orders received for the second quarter of fiscal 2021 were $41.7 million, compared to $24.6 million for the same period of fiscal 2020.
Revenue: Total revenues were $31.2 million for the second quarter of fiscal 2021 compared to $19.3 million for the same period of fiscal 2020.
Cost of Revenues: Cost of revenues for the second quarter of fiscal 2021 was $19.0 million compared to $13.6 million for the same period of fiscal 2020.
Research and Development Expenses: Research and development expenses for the second quarter of fiscal 2021 were $15.8 million compared to $10.6 million for the same period of fiscal 2020.
Selling, General and Administrative Expenses: Selling, general and administrative expenses for the second quarter of fiscal 2021 were $34.4 million compared to $27.2 million for the same period of fiscal 2020.
Net Loss: Net loss for the second quarter of fiscal 2021 was $37.9 million, or $0.78 per share, compared to $31.8 million, or $0.85 per share, for the second quarter of fiscal 2020.
Cash Position: As of March 31, 2021, the company had $555.7 million in cash, cash equivalents and short-term investments.
"We increased our margin to 39%, reflecting the impact of scaling our revenue, leveraging our fixed costs and the benefit of higher NGS product mix," commented Jim Thorburn, CFO of Twist. "Importantly, the solid results demonstrate excellent execution across all teams at Twist."

Fiscal Second Quarter 2021 and Recent Highlights

Shipped products to approximately 1,700 customers in the second quarter of fiscal 2021 versus approximately 1,400 in the same period of fiscal 2020.
Exercised our right of first refusal which gives a cost-effective option to double manufacturing capacity at the "Factory of the Future" just outside of Portland, Oregon
Launched the Twist NGS Methylation Detection System, a robust, end-to-end sample preparation and target enrichment solution for identifying methylated regions in the human genome. DNA methylation plays a key role in many biological processes including cancer.
Received Emergency Use Authorization (EUA) from the U.S. Food and Drug Administration (FDA), together with Biotia, for the SARS-CoV-2 Next-Generation Sequencing (NGS) Assay.
Partnered with Watchmaker Genomics to enable innovative research across a wide range of high throughput sequencing applications including tumor profiling, inherited disease detection, liquid biopsy assays and minimal residual disease monitoring.
Added new synthetic RNA controls to develop, validate and verify tests for SARS-CoV-2. The new controls include the B.1.351 (first identified in South Africa) and P.1 (first identified in Brazil) variants of SARS-CoV-2.
Announced three new NGS customers in APAC: Berry Genomics, Victoria Clinical Genetics Services (VCGS) and AcornMed Biotechnology.
Announced three new Twist Biopharma collaborations with Kyowa Kirin, Stanford’s Innovate Medicines Accelerator and Pure Biologics, bringing the total number of partners to 21. Twist Biopharma is now advancing 25 active programs through its partners, 17 of which have milestones and royalties. Additionally, nine programs are now completed.
Published positive preclinical data demonstrating that GLP-1R antibodies identified from Twist Biopharma’s proprietary G-protein coupled receptor (GPCR) libraries showed potent blood glucose control. Glucagon-like peptide-1 receptor (GLP-1R) is a class B GPCR that acts as the receptor for the incretin GLP-1, a peptide released to regulate insulin levels in response to food intake. The data were published online in mAbs.
Exercised our option to purchase all rights to a G-coupled protein receptor (GPCR) library and its proprietary Twist Antibody Optimization software, both developed in collaboration with Distributed Bio.
Named one of Fast Company’s World’s Most Innovative Companies for 2021.
Earned Great Place to Work certification.
Fiscal 2021 Financial Guidance

The following statements are based on Twist’s current expectations for fiscal 2021. The following statements are forward-looking, and actual results could differ materially depending on market conditions and the factors set forth under "Forward-Looking Statements" below. Twist does not plan to update, nor does it undertake any obligation to update, this outlook in the future.

For the full fiscal year 2021, Twist provided the following updated financial guidance:

Increasing revenue guidance in the range of $121 million to $129 million
Revenue from Ginkgo Bioworks expected to be in the range of $11 to $12 million
Synbio revenue excluding Ginkgo Bioworks is expected to be in the range of $43 to $46 million
NGS revenue is estimated to be in the range of $62 to $66 million
Biopharma revenue is estimated to be approximately $5 million
Gross margin is expected to be 36% to 38% for fiscal 2021
Operating expenses including R&D and SG&A are expected to be $192 million for the year
Net loss expected in the range of $144 million to $150 million to reflect our increased investments in our commercial organization and research and development activities
R&D is expected to be approximately $66 million
Stock-based compensation is expected to be approximately $33 million
Depreciation is expected to be $10 million
Capital expenditures are expected to be $40 million, including expansion into "Factory of the Future"
Conference Call Information

The company plans to hold a conference call and live audio webcast for analysts and investors today at 4:30 p.m. Eastern Time to discuss its financial results and provide an update on the company’s business. The call can be accessed by dialing (866) 688-0947 (domestic) or (409) 217-8781 (international) and refer to the conference ID 4647559. A telephonic replay of the conference call will be available beginning approximately four hours after the call through May 20, 2021 and may be accessed by dialing (855) 859-2056 (domestic) or (404) 537-3406 (international). The replay conference ID is 4647559. The webcast replay will be available for two weeks.

Given the circumstances globally, it is recommended to dial-in at most 15 to 20 minutes prior to the call start to reduce waiting times. If a participant will be listen-only, they are encouraged to listen via the webcast on Twist’s investor page.

On May 6, 2021 Kyowa Hakko Kirin reported that Consolidated Financial Summary (IFRS) Fiscal 2020 (Press release, Kyowa Hakko Kirin, MAY 6, 2021, View Source [SID1234579492])

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1. Consolidated Financial Results for the Fiscal Year Ended December 31, 2020 (from January 1, 2020 to December 31, 2020)
2. Dividends
3. Consolidated Earnings Forecasts for the Fiscal Year Ending December 31, 2021 (from January 1, 2021 to December 31, 2021) These financial results reports are exempt from audit conducted by certified public accountants or an audit corporation.

* Notice regarding the appropriate use of the earnings forecasts and other special comments The forward-looking statements, including earnings forecasts, contained in these materials are based on the information currently available to the Company and on certain assumptions deemed to be reasonable by management. As such, they do not constitute guarantees by the Company of future performance. Actual results may differ materially from these projections for a wide variety of reasons.

For more information regarding our suppositions that form the assumptions for the earnings forecasts, please see pages 17 and 18 of the attachment, "

(5) Outlook for Fiscal 2021" in "
1. Summary of Business Performance and Financial Position."1. Summary of Business Performance and Financial Position Responding to the massive changes to the business and social environments occurring as a result of the global spread of the novel coronavirus disease (COVID-19), the Kyowa Kirin Group (the "Group") has been striving to provide stable supply of pharmaceuticals, which is a core mission of a pharmaceutical company, as an utmost priority, and while paying meticulous attention to preventing infection, carrying out activities such as information provision. Furthermore, as this fiscal year is the final year of our FY2016-2020 Mid-term Business Plan, we set our sights on achieving a further leap forward as a global specialty pharmaceutical company through various initiatives including efforts to maximize the value of three global strategic products, strengthen global governance, and research and development for future growth. In addition to changes to healthcare environments and restraints on business activities across the globe due to the COVID-19 pandemic, the Group faced other extremely difficult environments, such as the lowering of drug price standards in Japan.

Nevertheless, the Group increased its revenues mainly due to the penetration of three global strategic products in US/EU market. In Japan, the Group launched Duvroq, an oral treatment for renal anemia, in August 2020. By utilizing our abundant experience in the field of renal anemia, we carried out activities to provide information on proper use of medication, giving utmost attention to safety.

The Group is seeing steady progress for the three global strategic products. Regarding Crysvita, we obtained approvals for its additional indication for tumor induced osteomalacia in the United States and for extending its indication to include X-linked hypophosphatemia in older adolescents and adult patients in Europe and there was an increase in formulations for self-administration at home in Japan. Regarding the treatment for mycosis fungoides and Sézary syndrome, POTELIGEO, we commenced sales in Europe, beginning with Germany in June 2020.

Furthermore, regarding NOURIANZ (generic name: Istradefylline (product name in Japan: NOURIAST)), which has already been launched in the United States, our application for approval regarding its indication for combination therapy for Parkinson’s disease was accepted in Europe. Concerning the voluntary recall of mitomycin that occurred in 2019, the Group received the report on the investigation from the Group Investigation Committee spearheaded by a third-party in January 2020, and formulated the recurrence prevention measures. As matters of the highest priority for management, the Group has formulated three key management priorities to strengthen our foundation as a global specialty pharmaceutical company: creation of a strong production and quality assurance system, improvement of risk management and reformation of corporate culture. The Group will work on those management priorities continuously and sincerely over the five-year mid-term business plan commencing 2021.

(1) Summary of Business Performance in Fiscal 2020

1) Overview of results The Group now applies the International Financial Reporting Standards ("IFRS") in line with its policy of expanding business globally, and adopts "core operating profit" as a level of profit that shows the recurring profitability from operating activities. Core operating profit is calculated by deducting "selling, general and administrative expenses" and "research and development expenses" from "gross profit," and adding "share of profit (loss) of investments accounted for using equity method" to the amount.

For the fiscal year ended December 31, 2020, revenue was ¥318.4 billion (up 4.1% compared to the previous fiscal year) and core operating profit was ¥60.0 billion (up 1.0%). Profit attributable to owners of parent was ¥47.0 billion (down 29.9%).
 The increase in revenue was the result of steady growth of global strategic products in North America and EMEA and strong sales in Asia, mainly in China, despite the impact of lower revenue in Japan from the reduction in drug price standards and the switching to Darbepoetin Alfa Injection Syringe [KKF], an authorized generic of NESP, a renal anemia treatment drug, among others. The negative effect on revenue from foreign exchange was ¥2.9 billion.
 The increase in core operating profit was the result of an increase in gross profit due to an increase in overseas revenue, despite an increase in selling, general and administrative expenses, and a decrease in share of profit (loss) of investments accounted for using equity method. The negative effect on core operating profit from foreign exchange was ¥1.3 billion.
 Profit attributable to owners of parent decreased as a result of the absence of the profit from discontinued operations recorded in the previous fiscal year, despite lower business restructuring expenses and impairment losses in addition to an increase in core operating profit.
 Revenue in Japan decreased year on year because of the significant impact of switching to Darbepoetin Alfa Injection Syringe [KKF], an authorized generic of NESP, a renal anemia treatment drug whose patent has expired, in addition to the impact of the reductions in drug price standards implemented in October 2019 and April 2020, despite the growth in sales of new product groups.
 Darbepoetin Alfa Injection Syringe [KKF] achieved rapid progress in switching from NESP, a renal anemia treatment drug.
 Duvroq, an oral treatment for renal anemia, was launched in August 2020, and it is penetrating the market favorably.  Revenue from Patanol, anti-allergy eye drops, and ALLELOCK, an anti-allergy agent, decreased as a result of smaller pollen counts and the impact of the suppression of examinations, etc. due to COVID-19.
 Revenue from ORKEDIA, a treatment for secondary hyperparathyroidism, increased. Meanwhile, revenue from REGPARA, a treatment for secondary hyperparathyroidism, decreased due to factors such as switching to ORKEDIA and the impact of rival products.
 Revenue from ROMIPLATE, a treatment for chronic idiopathic thrombocytopenic purpura, increased as a result of receiving approval of its indication for treatment of patients with aplastic anemia who have had an inadequate response to conventional therapy, in June 2019.
 Firm growth in revenue was realized for G-Lasta, an agent for decreasing the incidence of febrile neutropenia, and Rituximab BS [KHK], an anticancer agent.
 In December 2019, Crysvita, a treatment for FGF23-related diseases, and HARUROPI, a Parkinson’s disease treatment patch, were launched and they have been penetrating the market favorably.
 Revenue in North America increased year year due to the steady growth of global strategic products.
 Revenue from Crysvita, a treatment for X-linked hypophosphatemia, has been growing steadily since its launch in 2018. Approval for additional indication for treatment of tumor induced osteomalacia was acquired in June 2020.
 Revenue from POTELIGEO, an anticancer agent, stayed at the same level as in the previous fiscal year, due to the impact of the COVID-19 pandemic.
NOURIANZ (product name in Japan: NOURIAST), an antiparkinsonian agent which was launched in October 2019, has been penetrating the market favorably.
 Revenue in EMEA increased year on year due to the steady growth of global strategic products.
 Revenue from Crysvita, a treatment for X-linked hypophosphatemia, has been growing steadily as the number of countries where it has been released has been increasing since its launch in 2018. Approval for sale with the extended indication for older adolescents and adults was acquired in September 2020.
 In Germany, sales of POTELIGEO an anticancer agent, was launched in June 2020, and it has been penetrating the market favorably as the number of countries where it has been released has been increasing.
 Revenue in Asia/Oceania increased year on year, reflecting strong sales particularly in China.
 Revenue from REGPARA, a treatment for secondary hyperparathyroidism, increased year on year due to market expansion in China.
 Revenue from Others decreased year on year.
 Revenue decreased year on year due to a decline in other income such as original equipment manufacturing despite an increase in technology out-licensing such as royalties revenue from AstraZeneca in relation to benralizumab.
 Core operating profit increased year on year due to an increase in overseas revenue mainly from global strategic products, despite a lower gross profit due to a decrease in revenue in Japan, and an increase in selling, general and administrative expenses associated with sales of global strategic products.

On May 6, 2021 Kura Oncology, Inc. (Nasdaq: KURA), a clinical-stage biopharmaceutical company committed to realizing the promise of precision medicines for the treatment of cancer, reported first quarter 2021 financial results and provided a corporate update (Press release, Kura Oncology, MAY 6, 2021, View Source [SID1234579342]).

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"We believe KO-539 is well-positioned as a potentially best-in-class and first-in-class menin inhibitor," said Troy Wilson, Ph.D., J.D., President and Chief Executive Officer of Kura Oncology. "This confidence is supported by a growing body of clinical data, including compelling activity, a favorable safety and tolerability profile and a wide therapeutic window. As such, we intend to conduct a comprehensive clinical development strategy for KO-539, both as a monotherapy and in combination, aimed at providing the greatest benefit to patients with acute leukemia, and we are well funded to execute on this strategy."

"A critical component of our KO-539 development plan is the determination of an optimal Phase 2 dose," continued Dr Wilson. "Given the wide therapeutic window of KO-539, we have amended our KOMET-001 trial to include two genetically enriched Phase 1b expansion cohorts. This should enable us to maximize the benefit-risk for KO-539 in our target patient populations and better inform an optimal dose for Phase 2 and beyond. Enrollment in these Phase 1b expansion cohorts is expected to begin mid-year, and we look forward to sharing our progress as we work to bring this important therapeutic option to patients."

Recent Highlights

Enrollment in KOMET-001 Phase 1b expansion cohorts to begin shortly – KO-539 continues to demonstrate a wide therapeutic window and compelling single-agent activity in an all-comer population of patients with relapsed or refractory acute myeloid leukemia (AML), including patients with NPM1 mutations and KMT2A rearrangements. In order to better inform an optimal Phase 2 dose, Kura has amended its KOMET-001 trial of KO-539 to include two Phase 1b expansion cohorts. Both cohorts will be enriched with NPM1-mutant and KMT2A-rearranged relapsed/refractory AML patients. The Company expects to enroll at least 12 patients in each of the Phase 1b expansion cohorts and assess those patients for safety and tolerability, pharmacokinetics/pharmacodynamics and efficacy in order to determine the recommended Phase 2 dose. In addition, the amended Phase 1b protocol gives the Company flexibility to enroll up to 18 additional patients per cohort, as appropriate. Kura believes the patients enrolled in the cohort selected as the recommended Phase 2 dose have the potential to be included in the subsequent, registration-directed portion of the KOMET-001 trial. Patient enrollment in the genetically enriched Phase 1b expansion cohorts is expected to begin at existing and new clinical sites in mid-2021.

Multiple expansion opportunities in acute leukemias – Pending determination of an optimal Phase 2 dose, Kura is preparing to conduct a comprehensive clinical development plan for KO-539, both as a monotherapy and in combination, aimed at broadening the opportunity in acute leukemias. Additional opportunities include front line combination studies, additional genetic subtypes, a pediatric development strategy and other indications, such as acute lymphocytic leukemia and myelodysplastic syndrome.

Publication of tipifarnib Phase 2 data in Journal of Clinical Oncology – Data from Kura’s Phase 2 clinical trial (RUN-HN) of tipifarnib were recently published in the Journal of Clinical Oncology. These data formed the basis of the Breakthrough Therapy Designation granted by the U.S. Food and Drug Administration (FDA) earlier this year for the treatment of patients with recurrent or metastatic HRAS mutant head and neck squamous cell carcinoma (HNSCC). Tipifarnib is currently being evaluated in an ongoing registration-directed clinical trial (AIM-HN) in this indication of high unmet need.

Breakthrough Device Designation for HRAS companion diagnostic – The FDA has granted Breakthrough Device Designation (BDD) to Illumina for a companion diagnostic to detect HRAS mutations in HNSCC in support of Kura’s tipifarnib program, as the device provides for more effective treatment of a life-threatening disease. The next-generation sequencing-based companion diagnostic is being developed in collaboration with Illumina leveraging the content of TruSight Oncology 500. The BDD enables frequent interactions with the FDA and prioritized review on regulatory submissions.
Financial Results

Research and development expenses for the first quarter of 2021 were $20.3 million, compared to $12.6 million for the first quarter of 2020.

General and administrative expenses for the first quarter of 2021 were $10.6 million, compared to $7.6 million for the first quarter of 2020.

Net loss for the first quarter of 2021 was $30.7 million, compared to a net loss of $19.2 million for the first quarter of 2020. This included non-cash share-based compensation expense of $5.1 million, compared to $3.2 million for the same period in 2020.

Cash, cash equivalents and short-term investments totaled $603.9 million as of March 31, 2021, compared with $633.3 million as of December 31, 2020. Management expects that current cash, cash equivalents and short-term investments will be sufficient to fund current operations into 2024.
Upcoming Milestones

Initiation of genetically enriched Phase 1b expansion cohorts in KOMET-001 in mid-2021

Additional Phase 1 data from KOMET-001 in the second half of 2021

Initiation of a Phase 1/2 proof-of-concept study of tipifarnib in combination with a PI3Kα inhibitor in the second half of 2021

Nomination of a next-generation farnesyl transferase inhibitor Development Candidate in mid-2021
Conference Call and Webcast

Kura’s management will host a webcast and conference call at 4:30 p.m. ET / 1:30 p.m. PT today, May 6, 2021, to discuss the financial results for the first quarter 2021 and provide a corporate update. The live call may be accessed by dialing (888) 771-4371 for domestic callers and (847) 585-4405 for international callers and entering the conference code: 50156205. A live webcast and archive of the call will be available online from the investor relations section of the company website at www.kuraoncology.com.