On May 10, 2022 Turning Point Therapeutics, Inc. (NASDAQ: TPTX), a clinical-stage precision oncology company designing and developing novel targeted therapies for cancer treatment, reported financial results for the quarter ended March 31, 2022 and provided operational updates (Press release, Turning Point Therapeutics, MAY 10, 2022, View Source [SID1234614081]).

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"We are pleased with our continued progress led by our topline data for repotrectinib and now our third BTD being granted for our lead asset," said Athena Countouriotis, M.D., President and CEO. "We recently expanded our clinical pipeline with our first in-license of TPX-4589, our potential first-in-class ADC targeting Claudin18.2, which was recently granted Orphan Drug designation by the FDA for both gastric and pancreatic cancers. We continue to advance our pipeline including elzovantinib and look forward to a data-rich second half of the year, including updates from our repotrectinib and elzovantinib programs as well as a first look at our discovery program for KRAS G12D."

First quarter and recent operational highlights include:

REPOTRECTINIB, ROS1/TRK INHIBITOR

Announced that the U.S. Food and Drug Administration (FDA) granted an eighth regulatory designation, and third BTD, to lead drug candidate repotrectinib. BTD was granted for the treatment of patients with ROS1-positive metastatic non-small cell lung cancer (NSCLC) who have been previously treated with one ROS1 tyrosine kinase inhibitor and who have not received prior platinum-based chemotherapy (expansion cohort EXP-4 of the TRIDENT-1 study).
Reported positive topline blinded independent central review (BICR) data from all of the ROS1-positive advanced NSCLC cohorts from TRIDENT-1, utilizing a February 11, 2022 data cutoff date. Data in both TKI-naïve and TKI-pretreated cohorts are consistent with a potentially best-in-class drug candidate for patients with ROS1-positive advanced NSCLC.

In the ROS1-positive TKI-naïve advanced NSCLC population (EXP-1: n=71), the cORR was 79% (n=56/71; 95% CI: 68, 88), with 4 patients (6%) achieving a complete response (CR) and 52 patients (73%) achieving a partial response (PR). The cORR does not include one patient in an unconfirmed partial response (uPR) with tumor regression of -38% on the last scan, who remained on treatment awaiting the next scan as of the data cutoff date.

DOR ranged from 1.4+ to 35.1+ months with probability of patients in a response at 6, 9, 12 and 18 months reflected in Table 1 utilizing a Kaplan-Meier analysis, with a median duration of follow-up of 10.2 months.

Patients at Risk: Patients who have reached the specified timepoint without censoring or an event (progression or death).

PFS ranged from 0+ to 40.4+ months with probability of patients remaining progression free at 6, 9, 12 and 18 months reflected in Table 2 utilizing a Kaplan-Meier analysis, with a median duration of follow-up of 10.8 months.

οIn the ROS1-positive advanced NSCLC population pretreated with one prior TKI and prior platinum-based chemotherapy (EXP-2: n=26), the cORR was 42% (95% CI: 23, 63). Duration of response ranged from 3.6 to 18.3+ months.
οIn the ROS1-positive advanced NSCLC population pretreated with two prior TKIs without prior chemotherapy (EXP-3: n=18), the cORR was 28% (95% CI: 10, 54). Duration of response ranged from 1.9+ to 20.3+ months.
οIn the ROS1-positive advanced NSCLC population pretreated with one prior TKI without prior chemotherapy (EXP-4: n=56), the cORR was 36% (95% CI: 23, 50). The cORR does not include two patients with an uPR who both had tumor regressions of -47% on their last scans, both of whom remained on treatment awaiting their next scans as of the data cutoff date. Duration of response ranged from 1.9+ to 17.8 months.
Repotrectinib was generally well tolerated in a total of 380 patients with a safety and tolerability profile that was consistent with previously reported findings.
The TRIDENT-1 study continues to enroll patients globally and enrollment across all six cohorts of the study remains open and continues to progress steadily.
ELZOVANTINIB (TPX-0022), MET/SRC/CSF1R INHIBITOR

Phase 1 dose escalation completed utilizing intermediate dose level of 60 mg QD to 60 mg BID.
Patient enrollment continues in the SHIELD-1 study at 40 mg QD to 40 mg BID in Phase 1 dose expansion.
TPX-0046, RET INHIBITOR

Ongoing characterization of the pharmacokinetics, safety, and efficacy profile in the dose finding portion of the study before determining the recommended Phase 2 dose (RP2D).
TPX-0131, ALK INHIBITOR

Ongoing patient dosing in the Phase 1/2 FORGE-1 study of TPX-0131 in locally advanced or metastatic TKI-pretreated ALK-positive NSCLC.

TPX-4589, CLAUDIN18.2 ADC

Announced exclusive license agreement with LaNova Medicines to develop and commercialize TPX-4589 (LM-302), a novel, potentially first-in-class, antibody drug conjugate (ADC) targeting Claudin18.2 in the U.S. and rest of the world excluding Greater China and South Korea. Claudin18.2 is a protein expressed in many gastrointestinal cancers, including gastric, gastroesophageal, and pancreatic cancer. TPX-4589 (known in China as LM-302) is currently in Phase 1 clinical trials in both the U.S. and China.
Received two Orphan Drug designations (ODDs) from the FDA in the first quarter of 2022. ODDs were granted to LaNova Medicines for the treatment of pancreatic cancer and for the treatment of gastric cancer, including cancer of the gastroesophageal junction.
DISCOVERY

Continued advancement of internal discovery programs targeting aberrant GTPase signaling known to drive genomically defined cancers with significant unmet medical need. The most advanced programs target KRAS G12D and the p21 activated kinase, or "PAK" family. The company is targeting nomination of two development candidates in the second half of 2022 with a goal to achieve at least one new IND per year beginning in 2023.
Upcoming Milestones

REPOTRECTINIB

Discuss topline BICR data from all the ROS1-positive NSCLC cohorts from TRIDENT-1 with the FDA at a pre-NDA meeting in the second quarter of 2022.
Present detailed study results, including intracranial activity, from the ROS1-positive advanced NSCLC cohorts of the TRIDENT-1 study at a medical conference in the second half of 2022.
Provide a clinical data update from the NTRK+ advanced solid tumor cohorts from TRIDENT-1 in the second half of 2022.
ELZOVANTINIB

Initiate the Phase 1b/2 SHIELD-2 study of elzovantinib in combination with aumolertinib in mid-2022.
Initiate the Phase 2 portion of the SHIELD-1 study in the second half of 2022, pending FDA feedback on data from the intermediate dose level.
Provide a clinical data update from the Phase 1 SHIELD-1 study in the second half of 2022.
TPX-0131

Provide early interim data from initial patients treated in the dose-finding portion of the FORGE-1 study in the fourth quarter of 2022 or early 2023.
TPX-4589

Present preclinical data at a medical conference by early 2023.
Provide additional guidance on clinical development plan by early 2023.
DISCOVERY

Nominate 2 development candidates in the second half of 2022.
Provide details on the other 2 GTPase signaling discovery programs in the second half of 2022.
First Quarter 2022 Financial Results

Revenue: Revenue recognized during the three months ended March 31, 2022 was $0.4 million from the sale of clinical supply to Zai Lab for supporting the TRIDENT-1 Phase 2 clinical trials in the Zai Territory, compared to $25.2 million for the first quarter of 2021, consisting of $25.0 million related to an upfront payment received under the Zai Lab Elzovantinib Agreement and $0.2 million from the sale of clinical supply to Zai for TRIDENT-1.

R&D Expenses: Research and development expenses were $55.1 million for the first quarter compared to $41.3 million for the first quarter of 2021. Primary drivers of the year-over-year increase were investments made to develop repotrectinib, elzovantinib, discovery efforts and personnel expenses.

G&A Expenses: General and administrative expenses were $20.3 million for the first quarter compared to $20.0 million for the first quarter of 2021.

Net Loss: Net loss was $74.4 million for the first quarter compared to net loss of $35.5 million for the first quarter of 2021, which included revenue for $25 million as upfront payment related to Zai Lab collaboration agreement.

Cash Position: Cash, cash equivalents and marketable securities as of March 31, 2022 totaled $918.2 million, reflecting a net decrease of approximately $63 million from December 31, 2021. Turning Point projects its cash position is sufficient to fund current operations through the first half of 2024.

On May 10, 2022 Halozyme Therapeutics, Inc. (NASDAQ: HALO) ("Halozyme") reported financial results for the first quarter ended March 31, 2022 and provided an update on its recent corporate activities and outlook (Press release, Halozyme, MAY 10, 2022, View Source [SID1234614080]).

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"We started 2022 strongly, achieving record royalty revenues with significant growth that was in-line with our guidance for 2022, and with the signing of a new ENHANZE collaboration and licensing agreement with Chugai Pharmaceutical," said Dr. Helen Torley, president and chief executive officer of Halozyme. "We were delighted to recently announce our plans to acquire Antares Pharma, Inc., which is expected to increase our top and bottom-line growth for the long term. We look forward to a transformative year as we add three commercial products and explore our combined platform capabilities and build on the strong momentum of our ENHANZE royalty business.

Recent Partner Highlights:

In March 2022, argenx announced positive topline results from its ADAPT-SC study evaluating subcutaneous (SC) efgartigimod (1000mg efgartigimod-PH20) using ENHANZE drug delivery technology for the treatment of generalized myasthenia gravis. The study met its primary endpoint, demonstrating noninferior total IgG reduction at day 29 with subcutaneously administered efgartigimod compared to intravenous (IV) administration. Based on these results, argenx has stated it plans to submit a Biologics License Application to the U.S. Food and Drug Administration by the end of 2022. Efgartigimod SC is on track to be the first of Halozyme’s Wave 3 potential partner launches that are projected to occur between 2023 and 2025.
In March 2022, Halozyme and Chugai Pharmaceutical entered into a global collaboration and license agreement for ENHANZE technology. Halozyme received upfront payments of $25 million from Chugai and is eligible to receive additional future payments of up to $160 million, subject to achievement of specified development, regulatory and sales-based milestones. Halozyme will also be entitled to receive royalties on sales of commercialized medicines using the ENHANZE technology.
In March 2022, ViiV initiated enrollment of a Phase 1 study to evaluate the safety and pharmacokinetics of N6LS, a broadly neutralizing antibody, administered subcutaneously with ENHANZE technology.
Recent Corporate Highlights:

In April 2022, Halozyme announced the pending acquisition of Antares, extending its revenue growth and durability by expanding its drug-delivery capabilities with the addition of an industry-leading auto-injector platform as well as a commercial business with three proprietary products. The proposed acquisition provides compelling financial and strategic benefits including expected revenue and non-GAAP earnings accretion in 2022 and long-term financial upside, accelerating both top and bottom-line growth. Halozyme expects to build on Antares’ existing platform technology and capabilities to drive incremental, durable revenue opportunities with additional intellectual property protections for Antares technology in place beyond 2030. The acquisition is expected to close in the first half of 2022.
In March 2022, Halozyme announced the appointment of Moni Miyashita to its board of directors. Ms. Miyashita is an accomplished executive with over 25 years of global strategic, mergers & acquisitions and business transformation expertise and currently serves as chief strategy officer of Valo Health.
In February 2022, Halozyme announced the appointment of Nicole LaBrosse as Halozyme’s chief financial officer. Nicole brings over 18 years of public accounting and corporate finance experience to Halozyme and most recently served as Halozyme’s vice president of finance and accounting.
First Quarter Financial Highlights

Revenue for the first quarter was $117.3 million compared to $89.0 million for the first quarter of 2021. The 32% year-over-year increase was primarily driven by an increase in royalty revenue primarily attributable to subcutaneous DARZALEX (daratumumab), partially offset by a decrease in revenues under collaborative agreements. Revenue for the quarter included $69.6 million in royalties, an increase of 89% compared to $36.9 million in the prior year period.
Cost of product sales for the first quarter was $15.9 million, compared to $18.2 million for the first quarter of 2021. The year-over-year decrease, despite an increase in product sales, was primarily driven by the timing of manufacturing overhead costs in the prior year period.
Research and development expenses for the first quarter were $11.9 million, compared to $9.0 million for the first quarter of 2021. The increase is primarily due to an increase in compensation expense, including share-based compensation, for personnel in support of investment in ENHANZE.
Selling, general and administrative expenses for the first quarter were $13.8 million, compared to $11.1 million for the first quarter of 2021. The increase was primarily due to an increase in compensation expense and costs associated with M&A and diligence activities.
Operating Income: On a GAAP basis in the first quarter of 2022, operating income was $75.7 million, compared to an operating income of $50.7 million in the first quarter of 2021.
Net Income: On a GAAP basis in the first quarter of 2022, net income was $60.1 million, compared with net income of $27.9 million in the first quarter of 2021. Non-GAAP net income was $66.1 million in the first quarter of 2022, compared with non-GAAP net income of $54.3 million in the first quarter of 2021.1
Earnings per Share: On a GAAP basis in the first quarter of 2022, diluted earnings per share was $0.43, compared with $0.19 in the first quarter of 2021. On a non-GAAP basis, diluted earnings per share was $0.47, compared with diluted earnings per share of $0.37 in the first quarter of 2021.1
Cash, cash equivalents and marketable securities were $786.1 million on March 31, 2022, compared to $740.9 million on December 31, 2021.
Financial Outlook for 2022

The Company is reiterating its financial guidance for 2022 which was first provided on January 10, 2022. For the full year 2022, the Company expects:

Total revenue of $530 million to $560 million, representing growth of 20%-26% over 2021 total revenue. The Company expects revenue from royalties to increase approximately 50% over revenue from royalties in 2021 to approximately $300 million.
GAAP operating income of $350 million to $380 million, representing growth of 27%-38% over 2021 GAAP operating income, resulting in operating margins greater than 65%.
GAAP net income of $270 million to $295 million; and non-GAAP net income of $290 million to $315 million.1 The Company notes that 2022 will be the first full fiscal year in which Halozyme will record income tax expense as part of its income statement.
GAAP diluted earnings per share of $1.90 to $2.05, inclusive of the first full year of income tax expense, projected to be $0.55-$0.60 per share. In comparison, in 2021 the Company recorded a one-time non-cash income tax benefit of $154.2 million or $1.05 per share, related to the release of its tax valuation allowance.
Non-GAAP diluted earnings per share are expected to be $2.05 to $2.20,1 reflective of the first full year in which the company will record income tax expense, projected to be $0.55-$0.60 per share.
The Company’s financial guidance does not consider the impact of the potential Antares acquisition, and the Company’s earnings per share guidance does not consider the impact of potential future share repurchases.

Halozyme will webcast its Quarterly Update Conference Call for the first quarter of 2022 today, Tuesday, May 10, 2022 at 4:30 p.m. ET/1:30 p.m. PT. Dr. Torley will lead the call, which will be webcast live through the "Investors" section of Halozyme’s corporate website, and a replay will be available following the close of the call. To register for this conference call, please use this link: View Source After registering, you will receive an email confirmation that includes dial in details and unique conference call codes for entry. Registration is open through the live call. However, to ensure you are connected for the full call, please register a day in advance or at minimum 10 minutes before the start of the call.

On May 10, 2022 Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR) reported financial results for its fiscal second quarter ended March 31, 2022 (Press release, Arrowhead Research Corporation, MAY 10, 2022, View Source [SID1234614078]). The company is hosting a conference call today, May 10, 2022, at 4:30 p.m. ET to discuss the results.

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Conference Call and Webcast Details

Investors may access a live audio webcast on the Company’s website at View Source For analysts that wish to participate in the conference call, please dial 855-215-6159 or 315-625-6887 and provide Conference ID 3791265.

A replay of the webcast will be available on the company’s website approximately two hours after the conclusion of the call and will remain available for 90 days. An audio replay will also be available approximately two hours after the conclusion of the call and will be available for 3 days. To access the audio replay, dial 855-859-2056 or 404-537-3406 and provide Conference ID 3791265.

Selected Recent Events

Initiated the PALISADE Phase 3 clinical study to evaluate the efficacy and safety of ARO-APOC3, Arrowhead’s investigational RNA interference (RNAi) therapeutic designed to inhibit the production of apolipoprotein C-III (APOC3), a key regulator of triglyceride metabolism, in adults with familial chylomicronemia syndrome
Completed enrollment of 204 patients in the Phase 2b ARCHES-2 clinical study of ARO-ANG3, our investigational medicine designed to reduce production of angiopoietin-like protein 3 ANGPTL3 as a potential treatment for patients with mixed dyslipidemia
ARCHES-2 is expected to be complete around the end of 2022 and topline data are anticipated to be available in the first half of 2023
Initiated the Phase 2 GATEWAY clinical study of ARO-ANG3, Arrowhead’s investigational medicine designed to silence the hepatic expression of angiopoietin-like protein 3 (ANGPTL3), in patients with homozygous familial hypercholesterolemia (HoFH)
Initiated a Phase 1/2 study of ARO-C3, Arrowhead’s investigational medicine designed to reduce production of complement component 3 (C3) as a potential therapy for various complement mediated diseases, in up to 24 adult healthy volunteers, up to 24 adult patients with paroxysmal nocturnal hemoglobinuria (PNH), and up to 14 adult patients with complement-mediated renal disease
Filed Clinical Trial Applications (CTA) requesting regulatory clearance to begin clinical studies for two new investigational medicines designed to treat various muco-obstructive and inflammatory pulmonary conditions
ARO-MUC5AC, an investigational RNAi therapeutic designed to inhibit the production of mucin 5AC (MUC5AC)
ARO-RAGE, an investigational RNAi therapeutic designed to inhibit the production of Receptor for Advanced Glycation End products (RAGE)
Formed a joint venture, Visirna Therapeutics, with Vivo Capital to expand the reach of innovative medicines in Greater China
Broke ground on construction of a new drug manufacturing facility and announced awards of up to $18.5 million in tax incentives from the city of Verona and the Wisconsin Economic Development Corporation

On May 10, 2022 Lyell Immunopharma, Inc., (Lyell) (Nasdaq: LYEL), a T-cell reprogramming company dedicated to the mastery of T cells to cure patients with solid tumors, reported financial results for the first quarter of 2022 (Press release, Lyell Immunopharma, MAY 10, 2022, View Source [SID1234614077]).

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"We continue to progress our mission to develop T-cell therapies that can outlast and eradicate solid tumors," said Liz Homans, CEO of Lyell Immunopharma. "Our multi-modal pipeline has advanced two products into Phase 1 clinical development, and we remain on track to submit an IND in the second half of this year for LYL845, our wholly owned TIL product candidate, and with our collaborators at GSK we remain on track to submit an IND for LYL331, a next-generation NY-ESO-1 T-cell receptor product candidate in late 2022 – early 2023. We remain focused on executing towards clinical data, and our strong financial position enables us to see our current pipeline through key milestones in evaluating T-cell exhaustion and lack of durable stemness as key barriers to successful cell therapy in patients with solid tumor cancers."

Recent Business Highlights

Announced two upcoming abstract presentations at the 25th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT) (Free ASGCT Whitepaper), scheduled for May 16 – 19, 2022, in Washington, DC. The presentations will highlight preclinical data characterizing two investigational products in Phase 1 clinical development, LYL797 and LYL132, that incorporate Lyell reprogramming technologies designed to address major barriers to successful adoptive cell therapy.
Presented preclinical data characterizing LYL797 at the Association for Cancer Research (AACR) (Free AACR Whitepaper) 2022 Annual Meeting. LYL797 is a receptor tyrosine kinase-like orphan receptor 1 (ROR1)-targeted CAR T-cell therapy that incorporates Lyell’s genetic and epigenetic reprogramming technologies, Gen-R and Epi-R, designed to overcome T-cell exhaustion and promote durable stemness.
Initiated screening for the Phase 1 clinical trial for LYL797. The trial is designed to be an open label dose escalation and expansion trial that initially enrolls patients with relapsed/refractory triple‑negative breast cancer or non-small cell lung cancer who have failed at least two lines of therapy. Initial data is expected in 2023.
Announced FDA clearance of the IND for LYL132, a next-generation T-cell receptor (TCR) therapy for patients with solid tumors expressing New York esophageal squamous cell carcinoma 1 (NY-ESO-1) that incorporates Epi-R.
Expanded executive management team with the appointment of veteran biotech leader Gary Lee, Ph.D. as Chief Scientific Officer. With more than a decade of experience heading translational cell and gene therapy programs, Dr. Lee sets and oversees the company’s research strategy and pipeline.
First Quarter 2022 Financial Results

GAAP and Non-GAAP Operating Results

Lyell reported a net loss of $68.1 million for the first quarter ended March 31, 2022, compared to a net loss of $55.0 million for the same period in 2021. Non-GAAP net loss, which excludes non-cash stock-based compensation and non-cash expenses related to the change in the estimated fair value of success payment liabilities, was $50.0 million for the first quarter ended March 31, 2022 compared to $32.3 million for the same period in 2021.
Research and development (R&D) expenses were $35.8 million for the first quarter ended March 31, 2022, compared to $41.5 million for the same period in 2021. The decrease in R&D expense was primarily driven by a reduction in the success payment liability balance, which offset increases in infrastructure and personnel costs to support the expansion of our R&D and manufacturing capabilities. Non‑GAAP R&D expenses, which exclude non-cash stock-based compensation and non-cash expenses related to the change in the estimated fair value of success payment liabilities for the first quarter ended March 31, 2022, were $35.9 million, compared to $26.7 million for the same period in 2021.
General and administrative (G&A) expenses were $34.4 million for the first quarter ended March 31, 2022, compared to $16.8 million for the same period in 2021. The increase in G&A expense was primarily due to a $10.4 million increase in stock-based compensation expense, primarily related to award modifications and new awards granted. Non‑GAAP G&A expenses, which exclude non-cash stock-based compensation, for the first quarter ended March 31, 2022 were $16.2 million, compared to $9.0 million for the same period in 2021. The increase in non-GAAP G&A expenses was driven by litigation-related expenses and public company operating costs.
A discussion of these non-GAAP financial measures, including reconciliations of the most comparable GAAP measures to non-GAAP financial measures, is presented below under "Non-GAAP Financial Measures."

Cash, cash equivalents and marketable securities

Cash, cash equivalents and marketable securities as of March 31, 2022 were $838.0 million, compared to $898.3 million as of December 31, 2021. Lyell believes that its cash, cash equivalents and marketable securities balances will be sufficient to meet working capital and capital expenditure needs into 2025.

On May 10, 2022 Sana Biotechnology, Inc. (NASDAQ: SANA), a company focused on creating and delivering engineered cells as medicines, reported financial results and business highlights for the first quarter 2022 (Press release, Sana Biotechnology, MAY 10, 2022, View Source [SID1234614076]).

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"In the first quarter, we made significant progress in moving toward clinical trials for programs across our multiple platforms including the ex vivo hypoimmune allogeneic CAR T (SC291) and in vivo fusogen CAR T (SG295) programs, and we remain on track to file INDs for both of these programs this year," said Steve Harr, Sana’s President and Chief Executive Officer. "In addition to advancing these programs, we continue to make progress across our earlier pipeline, including SC451, our hypoimmune stem-cell derived pancreatic islet cell therapy for patients with type 1 diabetes, and multiple product candidates in our CAR T portfolio. Our people, broad set of technologies, and strong balance sheet enable us to pursue this ambitious pipeline."

Continued progress in building Sana’s hypoimmune ex vivo platform and in vivo fusogen platform with presentations at multiple conferences

Hypoimmune ex vivo platform: Presented preclinical data demonstrating that hypoimmune CAR T cells were able to evade both the innate and adaptive arms of the immune system in animal models while retaining their antitumor activity at the 2022 American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting.

In vivo fusogen platform: Scheduled to present preclinical data regarding hypoimmune pancreatic islet cells, generation of hypoimmune allogeneic regulatory T cells, retargeted fusosomes for in vivo delivery to T cells, fusosome-targeted gene transfer to human hepatocytes, and a novel vector copy number assay at the 2022 American Society of Gene & Cell Therapy meeting later in May.
Expanded Sana’s CAR T capability to potentially develop best-in-class, broadly accessible CAR T cell therapies

Entered into an exclusive agreement with the National Institutes of Health (NIH) for worldwide commercial rights to the NIH’s CD22 chimeric antigen receptor with a fully-human binder. This CAR construct has shown efficacy in several clinical studies, including in CD19 CAR T cell therapy failures. Targeting both CD19 and CD22 with an "off-the-shelf" product, whether in combination with Sana’s hypoimmune platform or fusogen platform, offers the potential of higher and more durable complete response rates in earlier-stage patients as well as in patients that have previously failed an autologous CD19 CAR T cell therapy.

Entered into a non-exclusive agreement with IASO Biotherapeutics and Innovent Biologics for commercial rights to a clinically validated fully-human B cell maturation antigen (BCMA) CAR construct, which Sana intends to incorporate into both the company’s ex vivo hypoimmune allogeneic and in vivo fusogen platforms for the treatment of multiple myeloma.
First Quarter 2022 Financial Results

GAAP Results

Cash Position: Cash, cash equivalents, and marketable securities as of March 31, 2022 were $657.4 million compared to $746.9 million as of December 31, 2021. The decrease of $89.5 million was primarily driven by cash used in operations of $77.7 million and cash used for the purchase of property and equipment of $7.5 million. Cash used in operations includes $6.2 million of upfront license payments related to licensing CD22 and BCMA as well as multiple cash payments that will not recur this year.

Research and Development Expenses: For the three months ended March 31, 2022, research and development expenses, inclusive of non-cash expenses, was $72.7 million compared to $41.9 million for the same period in 2021. The increase of $30.8 million was due to an increase in personnel expenses related to increased headcount to expand Sana’s research and development capabilities, increased costs for third-party manufacturing, laboratory supplies, facility and other allocated costs, and costs to acquire technology complementary to our own. Research and development expenses include non-cash stock-based compensation of $5.7 million and $2.7 million for the three months ended March 31, 2022 and 2021, respectively.

Research and Development Related Success Payments and Contingent Consideration: For the three months ended March 31, 2022, we recognized a non-cash gain of $55.4 million in connection with the change in the estimated fair value of the success payment liabilities and contingent consideration in aggregate, compared to expenses of $127.1 million for the same period in 2021. The value of these potential liabilities can fluctuate significantly with changes in our market capitalization and stock price.

General and Administrative Expenses: General and administrative expenses for the three months ended March 31, 2022, inclusive of non-cash expenses, were $14.4 million compared to $11.8 million for the same period in 2021. The increase of $2.6 million was primarily due to increased personnel-related expenses attributable to an increase in headcount to support our continued research and development activities. General and administrative expenses include stock-based compensation of $2.0 million and $1.5 million for the three months ended March 31, 2022 and 2021, respectively.

Net Loss: Net loss for the three months ended March 31, 2022 was $31.4 million, or $0.17 per share, compared to $180.6 million, or $1.52 per share, for the same period in 2021.
Non-GAAP Measures

Non-GAAP Operating Cash Burn: Non-GAAP operating cash burn for the three months ended March 31, 2022 was $82.0 million compared to $48.9 million for the same period in 2021. Non-GAAP operating cash burn is the decrease in cash, cash equivalents, and marketable securities, excluding cash inflows from financing activities, cash outflows from business development activities, and the purchase of property and equipment.

Non-GAAP Net Loss: Non-GAAP net loss for the three months ended March 31, 2022 was $86.9 million, or $0.47 per share, compared to $53.6 million, or $0.45 per share, for the same period in 2021. Non-GAAP net loss excludes certain one-time costs to acquire technology and non-cash expenses related to the change in the estimated fair value of contingent consideration and success payment liabilities.
A discussion of non-GAAP measures, including a reconciliation of GAAP and non-GAAP measures, is presented below under "Non-GAAP Financial Measures."