On May 2, 2024 Protara Therapeutics, Inc. (Nasdaq: TARA), a clinical-stage company developing transformative therapies for the treatment of cancer and rare diseases, reported financial results for the first quarter ended March 31, 2024 and provided a business update (Press release, Protara Therapeutics, MAY 2, 2024, View Source [SID1234642593]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

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

                  Schedule Your 30 min Free Demo!

"We have made significant progress thus far in 2024, and with cash resources expected to fund operations into 2026, we are well positioned to execute our programs in oncology and rare disease," said Jesse Shefferman, Chief Executive Officer of Protara Therapeutics. "We are pleased with the positive three-month data announced last month from our clinical program in patients with non-muscle invasive bladder cancer (NMIBC), which support the potential for TARA-002 to play a meaningful role in the treatment landscape. Looking ahead, we are on track to present interim data from our ADVANCED-2 trial of TARA-002 in patients with NMIBC in the second half of this year."

Mr. Shefferman continued, "We also recently aligned with the U.S. Food and Drug Administration (FDA) on a path forward for intravenous (IV) Choline Chloride in patients dependent on parenteral nutrition (PN). We continued to enroll pediatric patients in our Phase 2 STARBORN-1 trial of TARA-002 in lymphatic malformations (LMs), an underserved population with no FDA-approved therapies."

Recent Progress and Highlights

Corporate Update

In April 2024, Protara closed a $45.0 million private placement. The offering was led by RA Capital Management and Acorn Bioventures and included participation from new and existing investors such as Boxer Capital, Woodline Partners LP, Catalio Capital Management, StemPoint Capital, Armistice Capital, Velan Capital and a healthcare fund.
TARA-002 in NMIBC

In April 2024, the Company announced positive data from three-month evaluable carcinoma in situ (CIS) patients treated across its ongoing clinical program of TARA-002 in high-risk NMIBC, including Bacillus Calmette-Guérin (BCG)-unresponsive, BCG-experienced and BCG-naïve patient populations. The overall three-month complete response (CR) rate prior to reinduction for 16 evaluable patients treated across three trials with varying BCG status was 38%, with a CR rate of 63% in CIS-only patients and 13% in patients with CIS +Ta/T1. A 43% CR rate was observed in BCG-Unresponsive/Experienced patients. TARA-002 demonstrated a favorable safety and tolerability profile, with no Grade 3 or greater treatment-related adverse events.
The Company expects to share preliminary results from a pre-planned risk-benefit analysis of the ongoing Phase 2 open-label ADVANCED-2 trial in the second half of 2024. The Phase 2 open-label ADVANCED-2 trial is assessing intravesical TARA-002 in NMIBC patients with CIS (± Ta/T1) who are BCG-Unresponsive (n=75-100) and BCG-Naïve (n=27). The BCG-Unresponsive cohort has been designed to be registrational aligned with the FDA’s 2018 BCG-Unresponsive Non-muscle Invasive Bladder Cancer: Developing Drugs and Biologics for Treatment Guidance for Industry. Trial subjects will receive an induction course of six weekly intravesical instillations, and following mandatory biopsy at three months, will either receive a reinduction course of six weekly intravesical instillations of TARA-002, or the first maintenance course of three weekly installations every three months for an additional 12 months.
In addition to the ADVANCED-2 trial, the Company intends to assess higher dosing at an 80KE1 dose and systemic priming prior to initiation of intravesical administration, as well as the combination of TARA-002 with a checkpoint inhibitor in NMIBC patients with CIS.
IV Choline Chloride for Patients on PN

In April 2024, the Company announced alignment with the FDA on a registrational path forward for IV Choline Chloride in patients dependent on PN. Previously, the Company had been pursuing an indication in intestinal failure-associated liver disease (IFALD) and following feedback from the FDA, is pursuing a broader indication in patients on PN who are or may become unable to synthesize choline from oral or enteral nutrition sources. The Company expects to advance the development of IV Choline Chloride as a source of choline for adult and adolescent patients on long-term PN.
TARA-002 in LMs

Dosing continues to progress in STARBORN-1, a Phase 2 clinical trial of TARA-002 in pediatric patients with macrocystic and mixed-cystic LMs. Including an age de-escalation safety lead-in, the trial will enroll approximately 30 patients who will receive up to four injections of TARA-002 spaced approximately six weeks apart. The primary endpoint of the trial is the proportion of participants with macrocystic and mixed cystic LMs who demonstrate clinical success, defined as having either a CR (90% to 100% reduction from baseline in total LM volume) or substantial response (60% to less than 90% reduction in total LM volume) as measured by axial imaging.
First Quarter 2024 Financial Results

As of March 31, 2024, cash, cash equivalents and investments in marketable debt securities totaled $55.2 million. The Company expects its cash, cash equivalents, and investments in marketable debt securities, together with approximately $42.0 million in net proceeds from its April 2024 private placement, will be sufficient to fund its planned operations and data milestones into 2026.
Research and development expenses for the first quarter of 2024 increased to $7.7 million from $5.1 million for the prior year period. The increases were primarily due to an increase in expenses related to clinical trial and non-clinical activities for TARA-002 of $1.8 million as well as an increase of $1.1 million in personnel-related expenses, partially offset by a reduction in clinical development activities for Choline of $0.3 million.
General and administrative expenses for the first quarter of 2024 decreased to $4.1 million from $4.6 million for the prior year period. This decrease was primarily due to a reduction of $0.5 million in personnel-related expenses, inclusive of $0.3 million of stock-based compensation.
For the first quarter of 2024, Protara incurred a net loss of $11.1 million, or $0.97 per share, compared with a net loss of $9.0 million, or $0.80 per share, for the same period in 2023. Net loss for the first quarter of 2024 included approximately $1.2 million of stock-based compensation expenses.
About TARA-002

TARA-002 is an investigational cell therapy in development for the treatment of NMIBC and of LMs, for which it has been granted Rare Pediatric Disease Designation by the U.S. Food and Drug Administration. TARA-002 was developed from the same master cell bank of genetically distinct group A Streptococcus pyogenes as OK-432, a broad immunopotentiator marketed as Picibanil in Japan and approved in Taiwan by Chugai Pharmaceutical Co., Ltd. Protara has successfully shown manufacturing comparability between TARA-002 and OK-432.

When TARA-002 is administered, it is hypothesized that innate and adaptive immune cells within the cyst or tumor are activated and produce a pro-inflammatory response with release of cytokines such as tumor necrosis factor (TNF)-alpha, interferon (IFN)-gamma, IL-1b, IL-6, IL-12, granulocyte-macrophage colony-stimulating factor (GM-CSF) and natural killer cells. TARA-002 also directly kills tumor cells and triggers a host immune response by inducing immunogenic cell death, which further enhances the antitumor immune response.

About Non-Muscle Invasive Bladder Cancer (NMIBC)

Bladder cancer is the sixth most common cancer in the United States, with NMIBC representing approximately 80% of bladder cancer diagnoses. Approximately 65,000 patients are diagnosed with NMIBC in the United States each year. NMIBC is cancer found in the tissue that lines the inner surface of the bladder that has not spread into the bladder muscle.

About Lymphatic Malformations (LMs)

LMs are rare, congenital malformations of lymphatic vessels resulting in the failure of these structures to connect or drain into the venous system. Most LMs are present in the head and neck region and are diagnosed in early childhood during the period of active lymphatic growth, with more than 50% detected at birth and 90% diagnosed before the age of three years. The most common morbidities and serious manifestations of the disease include compression of the upper aerodigestive tract, including airway obstruction requiring intubation and possible tracheostomy dependence; intralesional bleeding; impingement on critical structures, including nerves, vessels, lymphatics; recurrent infection, and cosmetic and other functional disabilities.

About IV Choline Chloride

IV Choline Chloride is an investigational, intravenous phospholipid substrate replacement therapy initially in development for patients receiving parenteral nutrition. Choline is a known important substrate for phospholipids that are critical for healthy liver function and also plays an important role in modulating gene expression, cell membrane signaling, brain development and neurotransmission, muscle function, and bone health. PN patients are unable to synthesize choline from enteral nutrition sources, and there are currently no available PN formulations containing choline. Approximately 80 percent of PN-dependent patients are choline-deficient and have some degree of liver damage, which can lead to hepatic failure. There are currently no available PN formulations containing choline. In the U.S. alone, there are approximately 40,000 patients on long-term parenteral nutrition who would benefit from an IV formulation of choline. IV Choline Chloride has the potential to become the first FDA approved IV choline formulation for PN patients. IV Choline Chloride has been granted Orphan Drug Designation by the FDA for the prevention of choline deficiency in PN patients. The Company was issued a U.S. patent claiming a choline composition with a term expiring in 2041.

On May 2, 2024 Novartis reported that it has entered into an agreement to acquire Mariana Oncology, a preclinical-stage biotechnology company based in Watertown, Massachusetts focused on developing novel radioligand therapies (RLTs) to treat cancers with high unmet patient need (Press release, Novartis, MAY 2, 2024, View Source [SID1234642592]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

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

                  Schedule Your 30 min Free Demo!

The transaction bolsters the Novartis RLT pipeline and expands the company’s research infrastructure and clinical supply capabilities, supporting Novartis strategic priorities in oncology and RLT platform innovation.
The acquisition encompasses a robust portfolio of RLT programs spanning lead optimization to early development across a range of solid tumor indications such as breast, prostate and lung cancer – including development candidate MC-339, an actinium-based RLT being investigated in small cell lung cancer.

"The acquisition of Mariana Oncology reflects our commitment to radioligand therapy as one of our company’s key technology platforms and strengthens our leadership in this field," said Fiona Marshall, President of Biomedical Research at Novartis. "We are excited to work with the Mariana team to bring forward next-generation RLTs for patients living with cancer and together shape the future of RLT as a pillar for oncology treatment."

RLTs, or radiopharmaceuticals, are a form of precision medicine that combines a tumor-targeting molecule (ligand) with a therapeutic radioisotope (a radioactive particle). RLTs bind to specific receptors expressed on the surface of certain types of tumors. Once bound to a target cell, emissions from the therapeutic radioisotope cause DNA damage that can inhibit cell growth and replication and potentially trigger cell death. This targeted approach enables the delivery of radiation to the tumor, while limiting damage to the surrounding cells.

"As pioneers in radioligand therapies, we are dedicated to building on our scientific leadership and expanding the breadth of these potentially transformative treatments to a broader range of cancer types," said Shiva Malek, Global Head of Oncology for Biomedical Research at Novartis. "This acquisition brings to Novartis phenomenal talent and new capabilities in RLT research that complement our wide-ranging internal efforts to explore novel isotopes, combinations, disease areas, and more."

As of today, Novartis has two approved RLTs for certain patients with metastatic castration-resistant prostate cancer and for certain types of gastroenteropancreatic neuroendocrine tumors. The company’s early and late pipeline has several programs in or entering the clinic, including a spectrum of studies and assets for prostate cancer, as well as other preclinical and discovery programs to identify the next wave of novel RLTs. Novartis is actively exploring new isotopes and new combinations with complementary mechanisms of action, as well as looking at new disease areas for RLT.

Under the terms of the agreement, Novartis will make an upfront payment of USD 1 billion and additional USD 750 million in payments upon completion of pre-specified milestones.

The transaction is subject to customary closing conditions.

On May 2, 2024 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 2024 financial results and provided a corporate update (Press release, Kura Oncology, MAY 2, 2024, View Source [SID1234642591]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

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

                  Schedule Your 30 min Free Demo!

"Ziftomenib continues to demonstrate a best-in-class safety and efficacy profile as well as optimal pharmaceutical properties, which we believe will enable it to become a cornerstone of therapy in acute leukemias and beyond," said Troy Wilson, Ph.D., J.D., President and Chief Executive Officer of Kura Oncology. "This belief is backed by increasing investigator enthusiasm, as evidenced by rapid enrollment across our ongoing ziftomenib studies, and further supported by the FDA’s decision to grant Breakthrough Therapy Designation (BTD) to ziftomenib, making it the first investigational treatment to be granted BTD for the treatment of NPM1-mutant acute myeloid leukemia (AML). In the near term, we look forward to completing enrollment in our registration-directed trial of ziftomenib in NPM1-mutant AML and working closely with FDA to bring this potentially innovative medicine to patients in urgent need of effective treatments."

Recent Highlights

Breakthrough Therapy Designation for ziftomenib in NPM1-mutant AML – Last month, the U.S. Food and Drug Administration (FDA) granted BTD to ziftomenib for the treatment of relapsed/refractory (R/R) NPM1-mutant AML. FDA granted BTD based on data from Kura’s ongoing KOMET-001 trial of ziftomenib in patients with R/R NPM1-mutant AML. BTD is awarded for a drug that treats a serious or life-threatening condition and may demonstrate substantial improvement on one or more clinically significant endpoints over available therapies.

Registration-directed trial of ziftomenib in NPM1-mutant AML nearing completion – Kura remains on track to complete enrollment of 85 patients in its KOMET-001 registration-directed trial of ziftomenib in NPM1-mutant R/R AML by mid-2024. In the Phase 1 trial, ziftomenib demonstrated a 35% CR rate and 45% overall response rate in 20 heavily pretreated patients with NPM1-mutant AML treated at the recommended Phase 2 dose. NPM1-mutant AML accounts for approximately 30% of new AML cases annually and represents a disease of significant unmet need for which no approved targeted therapy exists.

Positive preliminary combination data for ziftomenib in NPM1-mutant and KMT2A-rearranged AML – In January 2024, Kura reported preliminary data from the KOMET-007 dose-escalation trial of ziftomenib in combination with venetoclax/azacitidine or cytarabine/daunorubicin (7+3) in patients with NPM1-mutant or KMT2A-rearranged AML. As of the data cutoff on January 11, 2024, all five newly diagnosed patients treated with ziftomenib and 7+3 achieved a complete remission (CR) with full count recovery, for a CR rate of 100%. The overall response rate among the 15 R/R patients treated with ziftomenib and venetoclax/azacitidine was 53%. Continuous daily dosing of ziftomenib at 200 mg was well tolerated. No differentiation syndrome events of any grade were reported, and no dose-limiting toxicities, evidence of QTc prolongation, drug-drug interactions or additive myelosuppression were observed. As of the data cutoff, 16 of the first 20 patients remained on trial, including all 11 NPM1-mutant patients.

Dose escalation continues in KOMET-007 combination trial of ziftomenib – To date, the 400 mg dose of ziftomenib has been cleared in three of the four cohorts in the KOMET-007 trial: 1) in combination with venetoclax/azacitidine in R/R NPM1-mutant AML, 2) in combination with venetoclax/azacitidine in R/R KMT2A-rearranged AML and 3) in combination with 7+3 in newly diagnosed adverse risk NPM1-mutant AML. Enrollment at the 600 mg dose is ongoing in all three cohorts. Enrollment continues at the 400 mg dose in combination with 7+3 in newly diagnosed adverse risk KMT2A-rearranged AML.

First patients dosed in KOMET-008 combination trial of ziftomenib – In February, Kura began dosing patients in its KOMET-008 trial of ziftomenib in combination with additional standards of care, including the FLT3 inhibitor gilteritinib, FLAG-IDA or LDAC, for the treatment of R/R NPM1-mutant or KMT2A-rearranged AML. Preclinical data for ziftomenib in combination with FLT3 inhibitors demonstrate strong synergistic effects compared to either single agent alone. Roughly half of patients with R/R NPM1-mutant AML have co-occurring FLT3 mutations, and the prognosis for these patients is poor.

First patients dosed with KO-2806 and cabozantinib in renal cell carcinoma – In March, Kura announced dosing of the first patient with KO-2806, the Company’s next-generation farnesyl transferase inhibitor (FTI), in combination with cabozantinib in clear cell renal cell carcinoma, just four months after dosing the first patients with KO-2806 as a monotherapy in the FIT-001 dose-escalation trial. The Company remains on track to dose the first patient in combination with adagrasib in KRASG12C-mutated non-small cell lung cancer by the middle of this year, as dose escalation of KO-2806 as a monotherapy continues in parallel.
Financial Results

Research and development expenses for the first quarter of 2024 were $36.3 million, compared to $25.2 million for the first quarter of 2023.

General and administrative expenses for the first quarter of 2024 were $18.2 million, compared to $11.4 million for the first quarter of 2023.

Net loss for the first quarter of 2024 was $49.5 million, compared to a net loss of $34.1 million for the first quarter of 2023. This included non-cash share-based compensation expense of $8.5 million, compared to $6.8 million for the same period in 2023.

As of March 31, 2024, Kura had cash, cash equivalents and short-term investments of $527 million, compared to $424 million as of December 31, 2023. This includes net proceeds of approximately $145.8 million from the Company’s private placement in January 2024.

Based on its operating plan, management expects that cash, cash equivalents and short-term investments will fund current operations into 2027.
Forecasted Milestones

Complete enrollment of 85 patients in the KOMET-001 registration-directed trial of ziftomenib in NPM1-mutant R/R AML by mid-2024.

Identify the recommended Phase 2 dose of ziftomenib in combination with venetoclax and azacitidine by mid-2024.

Identify the recommended Phase 2 dose of ziftomenib in combination with 7+3 by mid-2024.

Initiate Phase 1b expansion study of ziftomenib in combination with standards of care, including venetoclax/azacitidine in newly diagnosed NPM1-mutant or KMT2A-rearranged AML, in the second half of 2024.

Submit an investigational new drug application for ziftomenib in a solid tumor indication and present preclinical data at a medical meeting in the second half of 2024.

Dose the first patients with KO-2806 and adagrasib in KRASG12C-mutated non-small cell lung cancer by mid-2024.

Complete enrollment of two expansion cohorts in KURRENT-HN and identify the optimal biologically active dose of tipifarnib and alpelisib by the end of 2024.

Present data from the KURRENT-HN trial of tipifarnib in combination with alpelisib in PIK3CA-dependent head and neck squamous cell carcinoma (HNSCC) in the first half of 2025.
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 2, 2024, to discuss the financial results for the first quarter 2024 and to provide a corporate update. The live call may be accessed by dialing (888) 886-7786 for domestic callers and (416) 764-8658 for international callers and entering the conference ID: 20226736. 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.

On May 2, 2024 ImmunityBio, Inc. (NASDAQ: IBRX) reported that it has signed an exclusive global arrangement with the Serum Institute of India, the world’s largest manufacturer of vaccines by number of doses produced, to supply ImmunityBio with Bacillus Calmette-Guerin (BCG) (Press release, ImmunityBio, MAY 2, 2024, View Source [SID1234642589]). The agreement covers the manufacturing of standard BCG (sBCG) that is currently approved for use outside the U.S., as well as a next-generation recombinant BCG (iBCG) undergoing testing, intended for use in combination with ImmunityBio’s ANKTIVA (nogapendekin alfa inbakicept-pmln) for currently approved and potential future indications, subject to regulatory approvals.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

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

                  Schedule Your 30 min Free Demo!

"We are pleased to partner with the Serum Institute of India so that the power of its large-scale, world-class, GMP manufacturing capacity can be used to address the issue of BCG shortage, which affects thousands of bladder cancer patients annually," said Patrick Soon-Shiong, M.D., Executive Chairman and Global Chief Scientific and Medical Officer at ImmunityBio. "We are especially proud to be partnering with Dr. Cyrus Poonawalla, the Institute’s Chairman and founder and someone who has made such a positive impact on global health."

The arrangement will result in additional supplies of the current standard sBCG immediately for trials. At the same time, the two companies will work to accelerate the ongoing Phase 2 clinical trials of iBCG currently being conducted in Europe which has so far demonstrated safety advantageous over standard BCG as well as enhanced immunogenicity in driving both CD8+ and CD4 T cells.

The collaboration between SII and ImmunityBio comes on the heels of the FDA’s approval of ANKTIVA for the treatment of non-muscle invasive bladder cancer with carcinoma in situ (CIS). Increasing the available supply of BCG is intended to address shortages for the combination therapy with ANKTIVA.

"The collaboration between Serum Institute of India and ImmunityBio will undoubtedly transform the way we approach cancer treatment. It will improve global access to BCG and at the same time—the unique therapy is the key to achieve a complete solution for bladder cancer. We are truly excited to witness the incredible impact this collaboration will have on improving patient outcomes and saving countless lives," said Mr. Adar C. Poonawalla, CEO, Serum Institute of India.

Originally used as a tuberculosis vaccine, BCG administered via intravesical instillation (delivery to the bladder via a catheter) has been the standard of care for patients with non-muscle invasive bladder cancer since 1977. BCG is a benign bacterium that induces an immune response in the bladder in proximity to the cancer cells, leading to clearance of the cancer in many patients.

BCG is one of the most widely used vaccines worldwide and has been given to more than 4 billion individuals with astonishing safety records. However, because BCG is a biologic drug that uses benign bacteria it is more complicated to make than many other types of drugs. SII is the largest manufacturer of BCG vaccine across the world, while Merck & Co. based in New Jersey currently is the only manufacturer of BCG (TICE BCG) in the U.S.

"The scale and quality of vaccines that the Serum Institute manufacturers is unparalleled and we are honored to partner with Dr. Poonawalla and his leadership team on this important initiative," said Richard Adcock, President and CEO of ImmunityBio. "By providing another option for BCG, we believe more NMIBC patients will be able to benefit from this proven treatment as both a monotherapy and a combination therapy with ANKTIVA."

ImmunityBio plans to conduct clinical trials to study recombinant BCG (iBCG) and sBCG manufactured by Serum Institute in combination with ANKTIVA for the treatment of different types of bladder and other cancers. Supply of BCG is expected to be available once the protocol for the trial has been authorized by the FDA. ImmunityBio plans to submit the protocol to the FDA and to global regulatory bodies in the next 30 days.

"The opportunity to initiate a trial of an immunogenic recombinant BCG, which has already demonstrated enhanced safety compared to standard BCG in Phase 1/2 studies, is exciting. We look forward to exploring ANKTIVA in combination with BCG in non-muscle invasive bladder cancer (NMIBC) and across other tumor types. With our ability to overcome immune evasion of the tumor to BCG when BCG is given alone, and by converting a MHC- negative cold tumor to a MHC+ positive hot tumor with the combination of ANKTIVA with BCG, we will now further expand the development of our therapeutic cancer vaccine with BCG," said Dr. Soon-Shiong.

For investigators interested in participating in clinical trials or to learn more information, please email [email protected]

On May 2, 2024 Illumina, Inc. (Nasdaq: ILMN) ("Illumina" or the "company") reported its financial results for the first quarter of fiscal year 2024, which include the consolidated financial results for GRAIL (Press release, Illumina, MAY 2, 2024, View Source [SID1234642587]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

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

                  Schedule Your 30 min Free Demo!

"The Illumina team delivered results ahead of our expectations by supporting our customers with innovative solutions that enable the next wave of progress in genomics and multiomics," said Jacob Thaysen, Chief Executive Officer. "Our customers are leveraging the NovaSeq X to drive increased sequencing activity, even in a persistently challenging global macroeconomic environment. Additionally, our team’s strong execution and continued focus on operational excellence enabled us to deliver year-over-year improvement in both gross margin and non-GAAP operating margin."

First quarter consolidated results
GAAP Non-GAAP (a)
Dollars in millions, except per share amounts
Q1 2024
Q1 2023
Q1 2024
Q1 2023
Revenue $ 1,076 $ 1,087 $ 1,076 $ 1,087
Gross margin 62.0 % 60.3 % 66.5 % 64.7 %
Research and development ("R&D") expense $ 339 $ 341 $ 335 $ 339
Selling, general and administrative ("SG&A") expense $ 439 $ 378 $ 349 $ 343
Operating (loss) profit $ (111) $ (64) $ 33 $ 21
Operating margin (10.3) % (5.7) % 3.1 % 1.9 %
Tax provision (benefit) $ 17 $ (81) $ 13 $ 5
Tax rate (15.3) % 103.9 % 46.4 % 27.3 %
Net (loss) income $ (126) $ 3 $ 14 $ 13
Diluted (loss) earnings per share $ (0.79) $ 0.02 $ 0.09 $ 0.08

(a) See the tables included in the "Results of Operations – Non-GAAP" section below for reconciliations of these GAAP and non-GAAP financial measures.
1

Capital expenditures for free cash flow purposes were $36 million for Q1 2024. Cash flow provided by operations was $77 million, compared to cash flow provided by operations of $10 million in the prior year period. Free cash flow (cash flow provided by operations less capital expenditures) was $41 million for the quarter, compared to $(42) million in the prior year period. Depreciation and amortization expenses were $108 million for Q1 2024. At the close of the quarter, the company held $1,115 million in cash, cash equivalents and short-term investments.

First quarter segment results
Illumina has two reportable segments, Core Illumina and GRAIL.

Core Illumina
GAAP Non-GAAP (a)
Dollars in millions Q1 2024 Q1 2023 Q1 2024 Q1 2023
Revenue (b)
$ 1,056 $ 1,076 $ 1,056 $ 1,076
Gross margin (c)
65.7 % 63.8 % 67.1 % 65.2 %
R&D expense $ 241 $ 259 $ 237 $ 257
SG&A expense $ 336 $ 286 $ 254 $ 257
Operating profit
$ 116 $ 142 $ 218 $ 187
Operating margin 11.0 % 13.2 % 20.6 % 17.4 %

(a) See Table 3 included in the "Results of Operations – Non-GAAP" section below for reconciliations of these GAAP and non-GAAP financial measures.
(b) Core Illumina revenue for Q1 2024 was down 2% as compared to Q1 2023 and down 2% on a constant currency basis. Amounts for Q1 2024 and Q1 2023 included intercompany revenue of $7 million and $9 million, respectively, which is eliminated in consolidation.
(c) The year-over-year increase in gross margin was primarily driven by a more favorable mix of sequencing consumables and execution of our operational excellence priorities that delivered cost savings, including freight and improved productivity. This was partially offset by certain strategic partnership revenue that is lower margin and increased warranty and field service costs.

GRAIL
GAAP Non-GAAP (a)
In millions Q1 2024 Q1 2023 Q1 2024 Q1 2023
Revenue $ 27 $ 20 $ 27 $ 20
Gross (loss) profit $ (22) $ (25) $ 12 $ 9
R&D expense $ 101 $ 86 $ 101 $ 86
SG&A expense $ 104 $ 93 $ 96 $ 87
Operating loss $ (227) $ (204) $ (185) $ (164)

(a) See Table 3 included in the "Results of Operations – Non-GAAP" section below for reconciliations of these GAAP and non-GAAP financial measures.

Key announcements by Illumina since Illumina’s last earnings release
•Released XLEAP-SBS chemistry on NextSeq 1000 and NextSeq 2000 Systems, our fastest, highest quality, and most robust sequencing by synthesis (SBS) chemistry to date
•Launched Illumina Complete Long Reads (ICLR) with Enrichment, a flexible, cost-effective solution to enhance coverage of known challenging-to-map regions with targeted long reads where they provide the most value
•A recent literature review, published in the Nature journal Genomic Medicine, showed that short-read genomic sequencing (GS) reduces the time it takes to diagnose and treat pediatric patients who may have a rare genetic condition
•Appointed Ankur Dhingra as Chief Financial Officer and Jakob Wedel as Chief Strategy and Corporate Development Officer
•Appointed Jenny Zheng as Head of Region, Greater China
2

A full list of recent Illumina announcements can be found in the company’s News Center.

Key announcements by GRAIL since Illumina’s last earnings release
•Presented new data at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting that:
•Demonstrated the first real-world evaluation of repeat multi-cancer early detection (MCED) / Galleri testing showing the potential value of adding repeat MCED screening
•Exhibited 4-year overall survival follow-up demonstrating the prognostic significance of detecting cancer with a methylation-based cfDNA platform like Galleri
•Illustrated data demonstrating the power of Galleri to preferentially detect high grade, clinically significant prostate cancer over indolent cases.
•Announced a collaboration with AstraZeneca in which participants from Japan will have their samples tested using GRAIL’s novel risk classification test on its Methylation Platform

A full list of recent GRAIL announcements can be found in GRAIL’s Newsroom.

Financial outlook and guidance
For fiscal year 2024, the company continues to expect Core Illumina revenue to be approximately flat compared to fiscal year 2023 and Core Illumina non-GAAP operating margin to be approximately 20%. While Illumina continues to move as quickly as possible to resolve GRAIL, the company is focusing its financial outlook on Core Illumina, as the specific timing and impact of the GRAIL divestment remains uncertain.

The company provides forward-looking guidance on a non-GAAP basis. The company is unable to provide a reconciliation of forward-looking non-GAAP financial measures to the most directly comparable GAAP reported financial measures because it is unable to predict with reasonable certainty the financial impact of items such as acquisition-related expenses, gains and losses from our strategic investments, fair value adjustments related to contingent consideration and contingent value rights, potential future asset impairments, restructuring activities, and the ultimate outcome of pending litigation without unreasonable effort. These items are uncertain, inherently difficult to predict, depend on various factors, and could have a material impact on GAAP reported results for the guidance period. For the same reasons, the company is unable to address the significance of the unavailable information, which could be material to future results.

Conference call information
The conference call will begin at 2 p.m. Pacific Time (5 p.m. Eastern Time) on Thursday, May 2, 2024. Interested parties may access the live teleconference through the Investor Info section of Illumina’s website at investor.illumina.com. Alternatively, individuals can access the call by dialing 866.400.0049 or +1.323.701.0225 outside North America, both using conference ID 9170634. To ensure timely connection, please dial in at least ten minutes before the scheduled start of the call.

A replay of the conference call will be posted on Illumina’s website after the event and will be available for at least 30 days following.