On August 8, 2024 Sana Biotechnology, Inc. (NASDAQ: SANA), a company focused on creating and delivering engineered cells as medicines, reported financial results and business highlights for the second quarter 2024 (Press release, Sana Biotechnology, AUG 8, 2024, View Source [SID1234645623]).

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"The hypoimmune platform addresses one of the fundamental challenges with allogeneic cell transplantation and has the opportunity to impact a broad range of diseases," said Steve Harr, Sana’s President and Chief Executive Officer. "We have four ongoing trials and are pleased with the progress and encouraged by our learnings in the clinic to-date. We are accelerating investments to build our pivotal-ready supply chain, decrease our reliance on external manufacturing given ongoing geopolitical uncertainty, and increase our clinical trial site footprint globally. We continue the dose escalation phases of our studies, which makes predicting the numbers of patients and release date for data an inexact science, but we look forward to reporting clinical data in each therapeutic area later this year with the goal of an initial understanding of the safety and efficacy profiles."

Recent Corporate Highlights

Advancing four clinical programs across seven indications, including an allogeneic CAR T program targeting CD19+ cancers, an allogeneic CAR T program for B-cell mediated autoimmune diseases, an allogeneic CAR T program for cancer patients that have failed a CD19-targeted therapy, and a gene-modified primary islet cell therapy in type 1 diabetes:

Oncology – The ARDENT trial evaluates SC291, a hypoimmune (HIP)-modified CD19-directed allogeneic CAR T therapy, in patients with B-cell malignancies. Early SC291 data from the ongoing ARDENT trial suggest the ability to dose safely, the desired immune evasion profile, and early clinical efficacy. The VIVID trial evaluates SC262, a HIP-modified CD22-directed allogeneic CAR T therapy, in patients with relapsed or refractory B-cell malignancies who have received prior CD19-directed CAR T therapy. Sana is enrolling patients in both studies and expects to share data in 2024.

B-cell Mediated Autoimmune Diseases – The GLEAM trial evaluates SC291 in patients with B-cell mediated autoimmune diseases including lupus nephritis, extrarenal lupus, and antineutrophil cytoplasmic antibody (ANCA)-associated vasculitis. Sana is enrolling patients in this study and expects to share initial data in 2024.

Type 1 Diabetes – UP421 is a primary human HIP-modified pancreatic islet cell therapy for patients with type 1 diabetes. The goal of this investigator-sponsored trial (IST) is to understand immune evasion, islet cell survival, and beta cell function, as measured by C-peptide production, of HIP-modified pancreatic islet cells in type 1 diabetics without any immunosuppression. Initial screening took longer than anticipated, and after a protocol amendment to expand the eligible patient pool, multiple patients are now enrolled and awaiting donor availability. Sana awaits initial patient dosing at Uppsala University Hospital and expects to share initial data in 2024.
Published preclinical data in Nature Biotechnology showing that healthy transplanted human glial progenitor cells replaced diseased glial cell population in a preclinical model:

In the study, human glial chimeric mice were used to model the impact of competition between healthy and diseased human glia in vivo. When wild-type (WT) healthy human glial progenitor cells (hGPCs) were transplanted into adult mice that had been neonatally chimerized with mutant Huntingtin (mHTT)-expressing hGPCs, the healthy cells outcompeted and ultimately eliminated diseased glia, repopulating the brain with the healthy cells.
These data establish additional proof-of-concept for the development of SC379, Sana’s pluripotent stem cell (PSC)-derived glial progenitor cell (GPC) product candidate, as a potential therapy to deliver healthy allogeneic GPCs to patients with certain central nervous system disorders.
Second Quarter 2024 Financial Results

GAAP Results

Cash Position: Cash, cash equivalents, and marketable securities as of June 30, 2024 were $251.6 million compared to $205.2 million as of December 31, 2023. The increase of $46.4 million was primarily driven by net proceeds from equity financings of $181.0 million, proceeds from stock option exercises and the employee stock purchase plan of $8.4 million, and proceeds of $7.6 million from a loan to fund tenant improvements for our manufacturing facility in Bothell, Washington during the six months ended June 30, 2024, partially offset by cash used in operations of $124.2 million and cash used for the purchase of property and equipment of $28.9 million.

Research and Development Expenses: For the three and six months ended June 30, 2024, research and development expenses, inclusive of non-cash expenses, were $60.9 million and $117.3 million, respectively, compared to $73.0 million and $140.2 million for the same periods in 2023. The decreases of $12.1 million and $22.9 million for the three and six months ended June 30, 2024, respectively, compared to the same periods in 2023 were primarily due to lower personnel-related and laboratory costs due to a decrease in headcount and decreased research costs related to the strategic repositioning in the fourth quarter of 2023, lower costs for third-party manufacturing at contract development and manufacturing organizations, and a decline in facility and other allocated costs. These decreases were partially offset by increased clinical development costs and an increase in costs for the acceleration of internal manufacturing capabilities, further de-risking of the supply chain. Research and development expenses include non-cash stock-based compensation of $7.1 million and $13.0 million, respectively, for the three and six months ended June 30, 2024 and $6.7 million and $12.7 million, for the same periods in 2023.
Research and Development Related Success Payments and Contingent Consideration: For the three and six months ended June 30, 2024, Sana recognized a non-cash gain of $27.9 million and a non-cash expense of $10.1 million, respectively, in connection with the change in the estimated fair value of the success payment liabilities and contingent consideration in aggregate, compared to non-cash expenses of $26.7 million and $26.8 million for the same periods in 2023. The value of these potential liabilities fluctuate significantly with changes in Sana’s market capitalization and stock price.

General and Administrative Expenses: General and administrative expenses for the three and six months ended June 30, 2024, inclusive of non-cash expenses, were $16.4 million and $32.7 million, respectively, compared to $16.6 million and $33.3 million for the same periods in 2023. The decrease of $0.2 million for the three months ended June 30, 2024 compared to the same period in 2023 was primarily due to a decrease in costs related to Sana’s previously planned manufacturing facility in Fremont, California, lower personnel-related costs due to a decrease in headcount related to the strategic repositioning in the fourth quarter of 2023, and a decrease in legal fees. These decreases were partially offset by an increase in non-cash stock-based compensation and consulting fees. The decrease of $0.6 million for the six months ended June 30, 2024 compared to the same period in 2023 was primarily due to a decrease in costs related to Sana’s previously planned manufacturing facility in Fremont, California and lower personnel-related costs due to a decrease in headcount related to the strategic repositioning in the fourth quarter of 2023. These decreases were partially offset by an increase in non-cash stock-based compensation, legal fees, and consulting costs.

Net Loss: Net loss for the three and six months ended June 30, 2024 was $50.3 million, or $0.21 per share, and $157.8 million, or $0.70 per share, respectively, compared to $114.0 million, or $0.59 per share, and $196.1 million, or $1.02 per share for the same periods in 2023.

On August 8, 2024 Coherus BioSciences, Inc. (Coherus, Nasdaq: CHRS), reported financial results for the quarter ended June 30, 2024 and recent business highlights (Press release, Coherus Biosciences, AUG 8, 2024, View Source [SID1234645591]):

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RECENT BUSINESS HIGHLIGHTS

UDENYCA RESULTS
UDENYCA net product sales were $50.9 million in Q2 2024, an increase of 19% compared to $42.7 million in Q1 2024 and a 60% increase compared to $31.7 million in Q2 2023.

Total unit demand increased 25% in Q2 2024 compared to Q1 2024, and 138% compared to Q2 2023.
Based on data from IQVIA, UDENYCA franchise market share for Q2 2024 was 29.0%, an increase of 4 market share points from Q1 2024.
LOQTORZI LAUNCH UPDATE

LOQTORZI, the first and only FDA-approved treatment for recurrent, locally advanced or metastatic nasopharyngeal carcinoma (NPC), commercially launched on January 2, 2024.
LOQTORZI net sales in Q2 2024 of $3.8 million, with new patient uptake primarily in relapsed locally advanced and 1L metastatic disease, a potential driver of long-term revenue growth.
LOQTORZI can now be ordered in all 33 National Comprehensive Cancer Network (NCCN) institutions.
The Centers for Medicare and Medicaid Services granted LOQTORZI a product-specific, permanent J Code, which was implemented on July 1, 2024.
ADVANCEMENT OF PROMISING IMMUNO-ONCOLOGY PIPELINE

Clinical data from the dose escalation stage of the Phase 1 study of CHS-114, a highly selective cytolytic anti-CCR8 antibody, was presented at the 2024 ASCO (Free ASCO Whitepaper) Annual Meeting in June. These data showed selective depletion of peripheral CCR8+ regulatory T cells, establishing proof of mechanism for CHS-114, and an acceptable safety profile with no dose-limiting toxicities, and a disease control rate of 47% in heavily pretreated patients with solid tumors. Expansion cohorts evaluating CHS-114 as monotherapy and in combination with toripalimab are currently enrolling patients with advanced/metastatic head and neck squamous cell carcinoma (HNSCC).
A Phase 2 study evaluating casdozokitug, an immune regulatory IL-27 antagonizing antibody, in combination with toripalimab and bevacizumab in treatment naïve patients with unresectable locally advanced or metastatic hepatocellular carcinoma is expected to begin enrolling patients in Q4 2024.
An Investigational New Drug (IND) application for CHS-1000, a novel humanized Fc-modified IgG1 monoclonal antibody specifically targeting ILT4 (LILRB2), was filed with the U.S. Food and Drug Administration (FDA) in Q2 2024 and received clearance. Coherus plans to initiate a Phase 1 study in the coming months.
"The first half of 2024 has been a period of disciplined execution of our strategy, which included improving our capital structure, advancing our innovative oncology pipeline and driving increasing sales of our commercial portfolio. We continue to streamline our operations, solidifying our focus on our oncology business," said Denny Lanfear, Coherus’ Chairman and Chief Executive Officer. "Bryan McMichael, our newly appointed Chief Financial Officer, will play a key role on our management team as we further translate this oncology focus into shareholder value."

"I am honored to carry forward my work at Coherus in the role of Chief Financial Officer. The strength of the team, commercial portfolio, and pipeline gives me great confidence in the Company’s future," said Bryan McMichael, Coherus’ Chief Financial Officer. "I look forward to continuing in my partnership with Denny, the Board, and other members of management to execute our strategy and bring value to shareholders."

SECOND QUARTER 2024 FINANCIAL RESULTS

Net revenue was $65.0 million during the three months ended June 30, 2024, and included $50.9 million of net sales of UDENYCA, $3.8 million of net sales of LOQTORZI, which was launched on January 2, 2024, $3.8 million of net sales of YUSIMRY, which was divested to Hong Kong King-Friend Industrial Company Ltd. ("HKF") on June 26, 2024, and other revenue of $6.5 million which included the $6.3 million up-front cash payment received for the outlicense to Apotex, Inc. of the Canadian rights to LOQTORZI on June 27, 2024. Net revenue was $58.7 million during the three months ended June 30, 2023 and included $26.7 million in revenues for CIMERLI, which was divested on March 1, 2024. Net revenue was $142.0 million and $91.2 million for the six months ended June 30, 2024 and 2023, respectively. Total net revenues attributable to the Company’s divested products, CIMERLI and YUSIMRY, during the first half of 2024 and 2023 were $35.9 million and $32.9 million, respectively.

Cost of goods sold (COGS) was $28.4 million and $24.8 million during the three months ended June 30, 2024 and 2023, respectively, and $63.0 million and $41.7 million during the six months ended June 30, 2024 and 2023, respectively. UDENYCA COGS included a mid-single digit royalty on net sales which expired on July 1, 2024. The increases in COGS in both 2024 periods were primarily driven by increased unit volumes, and in the second quarter of 2024, a $4.5 million fee for a contract change with a CMO, partially offset by a $9.7 million decrease in royalty costs in the second quarter of 2024, due to the CIMERLI divestiture on March 1, 2024.

Research and development (R&D) expenses were $22.0 million and $23.3 million for the three months ended June 30, 2024 and 2023, respectively, and $50.4 million and $57.4 million for the six months ended June 30, 2024 and 2023, respectively. The decreases were primarily due to savings from reduced headcount and lower costs related to biosimilar products, partially offset by increased costs for development of casdozokitug and CHS-114.

Selling, general and administrative (SG&A) expenses were $35.2 million and $45.1 million during the three months ended June 30, 2024 and 2023, respectively, and $91.7 million and $94.3 million during the six months ended June 30, 2024 and 2023, respectively. The declines in SG&A compared to the prior year periods were driven primarily by lower headcount. The decrease for the six-month period was partially offset by the net $6.8 million charge in the first quarter of 2024 associated with the full write-off of the outlicense intangible asset and associated release of the CVR liability related to NZV930, obtained in the Surface Oncology, Inc. acquisition.

Gain on Sale Transactions, Net which included the divestiture of the YUSIMRY franchise, which closed during the three months ended June 30, 2024, was $22.9 million, and reflects total cash proceeds of $40.0 million, net of assets transferred to HKF, liabilities derecognized, and transactions costs of $0.9 million. Gain on Sale Transactions, net for the first half of 2024 was $177.7 million and included a $154.8 million gain on the divestiture of the CIMERLI franchise, which closed during the three months ended March 31, 2024. There was no gain on Sale Transactions in the first half of 2023.

Interest expense was $5.3 million and $9.9 million during the three months ended June 30, 2024 and 2023, respectively, and $16.5 million and $19.7 million during the six months ended June 30, 2024 and 2023, respectively. The declines in both periods were primarily due to prepaying $175.0 million of the principal amount of the senior secured term loan facility that was entered into on January 5, 2022 on April 1, 2024 and prepaying the remaining $75.0 million principal amount on May 8, 2024. This was offset by interest on the $38.7 million principal amount of the new term loan facility and the revenue participation right purchase and sale agreement, both commencing May 8, 2024, as well as an average higher variable rate in the first half of 2024 compared to the first half of 2023.

Net loss for the second quarter of 2024 was $12.9 million, or $(0.11) per share on a diluted basis, compared to a net loss of $42.9 million, or $(0.49) per share on a diluted basis for the same period in 2023. Net income for the first half of 2024 was $90.0 million, or $0.73 per share on a diluted basis, compared to a net loss of $118.6 million, or $(1.42) per share on a diluted basis for the first half of 2023.

Non-GAAP net loss for the second quarter of 2024 was $16.4 million, or $(0.14) per share on a diluted basis, compared to $32.8 million, or $(0.38) per share for the same period in 2023. Non-GAAP net loss for the first half of 2024 was $52.2 million, or $(0.46) per share on a diluted basis, compared to $92.3 million, or $(1.11) per share for the first half of 2023. See "Non-GAAP Financial Measures" below for a discussion on how Coherus calculates non-GAAP net loss and a reconciliation to the most directly comparable GAAP measures.

Cash, cash equivalents and investments in marketable securities were $159.2 million as of June 30, 2024, compared to $117.7 million as of December 31, 2023.

2024 R&D and SG&A Expense Guidance
Coherus projects combined R&D and SG&A expenses for 2024 to be in the range of $250 to $265 million. This guidance includes approximately $40 million of stock-based compensation expense and excludes the effects of acquisitions, collaborations, investments, divestitures including expenses incurred on behalf of and reimbursed by Sandoz and HKF to satisfy Coherus’ obligations under the transition services agreements with those entities, restructuring, the exercise of rights or options related to collaboration programs, and any other transactions or circumstances not yet identified or quantified. This guidance is subject to a number of risks and uncertainties. See Forward-Looking Statements described in the section below.

Conference Call Information

When: Thursday, August 8, 2024, starting at 5:00 p.m. Eastern Daylight Time

To access the conference call, please pre-register through the following link to receive dial-in information and a personal PIN to access the live call: https://register.vevent.com/register/BI9dd3f986eeb7428485a66ce1fb34999f
Please dial in 15 minutes early to ensure a timely connection to the call.

Webcast: View Source

An archived webcast will be available on the "Investors" section of the Coherus website at View Source

On August 8, 2024 Intensity Therapeutics, Inc. ("Intensity" or "the Company") (Nasdaq: INTS), a late-stage clinical biotechnology company focused on the discovery and development of proprietary, novel immune-based intratumoral cancer therapies designed to kill tumors and increase immune system recognition of cancers, reported second quarter 2024 financial results and provides a corporate update (Press release, Intensity Therapeutics, AUG 8, 2024, View Source [SID1234645608]).

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Corporate Update

•In July 2024, the Company initiated and dosed its first patient in a Phase 3 open-label, randomized study (the "INVINCIBLE-3 Study") testing INT230-6, Intensity’s lead drug candidate, as a monotherapy compared to the standard of care ("SOC") drugs in second- and third-line treatment for certain soft tissue sarcoma subtypes. The Company plans to enroll 333 patients with an endpoint of overall survival and has screened and qualified over 50 sites for the INVINCIBLE-3 Study. Contract negotiations are in process to approve and activate these sites, which is estimated to take up to six months per site.

•In May 2024, the Company executed a collaboration agreement with SAKK to conduct a Phase 2 randomized, controlled study (the "INVINCIBLE-4 Study") evaluating clinical and biological effects of INT230-6 followed by SOC vs. SOC alone in early-stage triple-negative breast cancer. The Company plans to enroll 54 to 60 patients in Europe. The INVINCIBLE-4 Study endpoint is the change in the pathological complete response rate for the combination compared to the SOC alone. The Company expects that the data from INVINCIBLE-4 Study will provide data to size a follow-on Phase 3 study. The Company is in the process of screening and qualifying sites for the INVINCIBLE-4 Study, and plans to initiate the study in the third quarter of 2024.

•In May 2024, the Company appointed Thomas Dubin, J.D., MPH, to the Intensity board of directors, increasing the size of the board to five members. Mr. Dubin has extensive pharmaceutical business development, regulatory, and commercialization experience.

"The dosing of the first patient in our randomized controlled Phase 3 sarcoma trial is the most important development milestone Intensity has reached to date," said Lewis H. Bender, Intensity Founder, President and CEO. "A journey of 1,000 miles starts with the first step, and a successful clinical study outcome can only be achieved by initiating sites and enrolling patients. For our Phase 3 study, we have qualified over 50 sites and are in contract and budget negotiations to initiate treatment in multiple countries. Also, our collaboration with SAKK has progressed well during this quarter, and we are also looking forward to enrolling the first patient in the INVINCIBLE-4 study. Finally, I am excited that Tom Dubin joined our Board. Tom is a highly successful and sophisticated biotech executive who has already provided key insights."

Second Quarter 2024 Financial Results

Research and development expenses were $3.6 million for the three months ended June 30, 2024, compared to $0.9 million for the same period in 2023. The increase was primarily due to preliminary work related to the INVINCIBLE-3 Study, and to a lesser extent, costs for manufacturing a new batch of INT230-6 and increased expenses related to salary, benefits, and stock-based compensation.

General and administrative expenses were $1.5 million for the three months ended June 30, 2024, compared to $0.4 million for the same period in 2023. The increase was primarily due to increased expenses related to salary, benefits and stock-based compensation, higher legal, audit, and consulting fees, and higher directors and officers insurance.

Upon the Company’s initial public offering in June 2023, convertible notes outstanding converted to common stock, resulting in a $2.3 million loss on debt conversion. In addition, a preferred stock deemed dividend of $1.3 million was also recognized in June 2023, representing the value that was transferred to Series B and C preferred stockholders upon triggering of anti-dilution provisions concurrent with the initial public offering.

Overall, net loss was $5.0 million for the three months ended June 30, 2024, compared to a net loss of $3.7 million for the three months ended June 30, 2023.

As of June 30, 2024, cash, cash equivalents and marketable debt securities totaled $6.3 million, which the Company expects will be sufficient to fund operations into the first quarter in 2025.

About INT230-6
INT230-6, Intensity’s lead proprietary investigational product candidate, is designed for direct intratumoral injection. INT230-6 was discovered using Intensity’s proprietary DfuseRx℠ technology platform. The drug is comprised of two proven, potent anti-cancer agents, cisplatin and vinblastine, and a penetration enhancer molecule (SHAO) that helps disperse potent cytotoxic drugs throughout tumors for diffusion into cancer cells. These agents remain in the tumor, resulting in a favorable safety profile. In addition to local disease control and direct tumor killing, INT230-6 causes a release of a bolus of neoantigens specific to the malignancy, leading to immune system engagement and systemic anti-tumor effects. Importantly, these effects are mediated without immunosuppression which often occurs with systemic chemotherapy.

On August 8, 2024 Sanofi reported new results from the two-part, double-randomized, German-speaking Myeloma Multicenter Group (GMMG)-HD7 phase 3 study show that Sarclisa (isatuximab) in combination with lenalidomide, bortezomib, and dexamethasone (RVd) during induction therapy in transplant-eligible, newly diagnosed multiple myeloma (NDMM) significantly prolonged progression-free survival (PFS) from first randomization, resulting in a statistically significant and clinically meaningful reduction in disease progression or death, compared to RVd induction regardless of the maintenance regimen (Press release, Sanofi, AUG 8, 2024, View Source [SID1234645624]). Full results will be submitted for presentation at a forthcoming medical meeting.

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Hartmut Goldschmidt, MD
President of GMMG, Professor of Medicine at the Heidelberg University Hospital (UKHD), Germany and principal investigator of the study
"Successful induction therapy is one of the most critical components to reduce the relapse or recurrence risk in patients with newly diagnosed multiple myeloma. While we observed this investigational combination showed improved minimal residual disease negativity rates in the bone marrow, indicating potentially deeper responses after induction, further follow-up was needed to better understand how this translated to long-term outcomes. These data provide evidence that the Isa-RVd regimen potentially improves progression-free survival in the frontline, transplant-eligible population and supports the potential of this quadruplet to become a new standard-of-care induction regimen in this treatment setting."

GMMG-HD7 is one of six phase 3 studies to report positive results for Sarclisa in patients with multiple myeloma, which includes four positive readouts of a Sarclisa-based quadruplet in the frontline setting. The most recent included results from the IMROZ phase 3 study evaluating the investigational use of Sarclisa with VRd versus VRd for patients with transplant-ineligible NDMM, demonstrating a statistically significant and clinically meaningful improvement in PFS and a higher proportion of patients with minimal residual disease (MRD) negativity.

Dietmar Berger, MD, PhD
Chief Medical Officer and Global Head of Development at Sanofi
"The GMMG-HD7 study was designed to better understand the distinct effect of targeting CD38 with Sarclisa in induction versus maintenance treatment of transplant-eligible patients. These data build upon our belief that Sarclisa has the potential to be a best-in-class CD38 therapy that could improve long-term outcomes versus the standard-of-care for certain patients. We look forward to the full data presentation and continuing our mission of helping make a meaningful difference for people living with multiple myeloma."

In December 2021, Sanofi and GMMG shared the results from part one, which met the primary endpoint of MRD negativity after induction therapy and before transplant in NDMM patients. The GMMG-initiated study is being conducted in close collaboration with Sanofi based on jointly defined research. Sanofi provided financial support to GMMG for this study. The use of Sarclisa in combination with RVd is investigational and has not been evaluated by any regulatory authority.

While considered a rare disease, MM is the second most common hematologic malignancy,1 with more than 180,000 new diagnoses of MM worldwide yearly.2 Despite available treatments, MM remains an incurable malignancy in most patients with an estimated 61% five-year survival rate for newly diagnosed patients.3 Since MM does not have a cure, most patients will relapse. Relapsed MM is the term for when the cancer returns after treatment or a period of remission. Refractory MM refers to when the cancer does not respond or no longer responds to therapy.

About the GMMG-HD7 study
GMMG-HD7 is a pivotal randomized, open-label, multicenter, 2-part phase 3 study evaluating Sarclisa in combination with RVd versus RVd induction followed by post-transplant re-randomization to Sarclisa plus lenalidomide versus lenalidomide maintenance in transplant-eligible NDMM patients. The study enrolled 662 patients with transplant-eligible NDMM across 67 sites in Germany. In the first part of the study, all participants were equally randomized to receive three 42-day cycles of RVd in both arms of the study, while Sarclisa was added to only one study arm. In the second part of the study, patients were re-randomized post-transplant to receive Sarclisa plus lenalidomide or lenalidomide alone as maintenance therapy. During the study, Sarclisa was administered through an intravenous infusion at a dose of 10 mg/kg once weekly for the first four weeks of cycle one, then every other week for the rest of the induction period.

MRD negativity was assessed by next-generation flow cytometry (sensitivity of 1×10-5) after induction. In the latest readout of the study, PFS for both Sarclisa plus RVd as an induction therapy, regardless of maintenance treatment, and Sarclisa plus lenalidomide as a maintenance regimen were measured from first randomization.

GMMG-HD7 protocol defined the primary endpoints of MRD negativity after induction treatment for the first part of the study, and PFS following the second randomization after transplant for part two of the study, in which Sarclisa was added to lenalidomide maintenance, with the latter primary endpoint anticipated to be available at a later date. The key secondary endpoint for the first part of the study was PFS from first randomization. Additional secondary endpoints included rates of complete response after induction, and intensification, overall survival, and safety.

About Sarclisa
Sarclisa (isatuximab) is a monoclonal antibody that binds to a specific epitope on the CD38 receptor on MM cells, inducing distinct antitumor activity. It is designed to work through multiple mechanisms of action including programmed tumor cell death (apoptosis) and immunomodulatory activity. CD38 is highly and uniformly expressed on the surface of MM cells, making it a target for antibody-based therapeutics such as Sarclisa.

Based on the ICARIA-MM phase 3 study, Sarclisa is approved in more than 50 countries, including the US and the EU, in combination with pomalidomide and dexamethasone for the treatment of patients with relapsed refractory MM (RRMM) who have received ≥2 prior therapies, including lenalidomide and a proteasome inhibitor and who progressed on last therapy. Based on the IKEMA phase 3 study, Sarclisa is also approved in 50 countries in combination with carfilzomib and dexamethasone, including in the US for the treatment of patients with RRMM who have received 1–3 prior lines of therapy and in the EU for patients with MM who have received at least one prior therapy. In the US, the non-proprietary name for Sarclisa is isatuximab-irfc, with irfc as the suffix designated in accordance with nonproprietary naming of biological products guidance for industry issued by the US Food and Drug Administration.

Sarclisa continues to be evaluated in multiple ongoing phase 3 clinical studies in combination with current standard treatments across the MM treatment continuum. It is also under investigation for the treatment of other hematologic malignancies, and its safety and efficacy have not been evaluated by any regulatory authority outside of its approved indication.

Sanofi is committed to pursuing the advancement of Sarclisa through several investigational studies across the MM treatment continuum. Various patient-centric clinical development programs aim to bring Sarclisa to more patients, intercept the disease earlier in the treatment journey, and explore potential new combinations including assessing subcutaneous administration via a proprietary on body device system. The safety and efficacy of Sarclisa has not been evaluated by any regulatory authority outside of its approved indications and methods of delivery.

In striving to become the number one immunoscience company globally, there is a commitment to advancing oncology innovation. The pipeline is being reshaped and prioritized, leveraging expertise in immunoscience to drive progress. Efforts are centered on select hematologic malignancies and solid tumors with critical unmet needs, including multiple myeloma, acute myeloid leukemia, certain types of lymphomas, as well as gastrointestinal and lung cancers.

For more information on Sarclisa clinical studies, please visit www.clinicaltrials.gov.

About the German-speaking Myeloma Multicenter Group (GMMG)
GMMG is the largest study group focusing on MM in Germany, with headquarters based in Heidelberg. Within the last 20+ years, the GMMG study group has performed numerous studies including five randomized, multicenter phase 3 studies with 4,000 patients enrolled from about 90 participating and co-treating centers throughout Germany. The overall goal of GMMG is to generate improved therapies for myeloma patients through the development and testing of novel and personalized, genome- and signaling-driven treatment strategies. The GMMG has set itself the goal of achieving further approvals for effective antibody-based drug combinations for the first-line treatment of myeloma patients, in which antibody-based treatment regimens have been integrated into seven GMMG study concepts (CONCEPT, DANTE, DADA, HD6, HD7, HD8, HD9 and HD10).

On August 8, 2024 Crinetics Pharmaceuticals, Inc. (Nasdaq: CRNX), a clinical stage pharmaceutical company focused on the discovery, development and commercialization of novel therapeutics for endocrine diseases and endocrine-related tumors, reported financial results for the second quarter ended June 30, 2024 (Press release, Crinetics Pharmaceuticals, AUG 8, 2024, View Source [SID1234645592]).

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"The second quarter of 2024 has been yet another successful period of executing our strategy to become the world’s premier endocrine company," said Scott Struthers, Ph.D., Founder and Chief Executive Officer of Crinetics. "Our strong presence at the ENDO 2024 meeting in June showcased the breadth and depth of our pipeline. Our second internally-developed candidate atumelnant* showed profoundly positive initial results in the treatment of both congenital adrenal hyperplasia and Cushing’s disease. We anticipate reporting additional data in both indications later in 2024. Data presentations from the paltusotine program continue to demonstrate clinical benefits for patients, providing even more compelling support for its potential to treat people with acromegaly. We remain on track for our planned paltusotine NDA filing in acromegaly later this year, and multiple workstreams are underway in anticipation of an expected market launch in 2025."

Crinetics today also announced that Marc Wilson will be transitioning from his role as Chief Financial Officer for personal reasons and the Company has initiated a search for a successor. Mr. Wilson will continue to serve at full capacity until a successor is named and will provide all necessary support to ensure a seamless transition.

"Marc has been an invaluable member of the Crinetics team over the last six years and we thank him for his outstanding contributions," said Scott Struthers, Ph.D., Founder and Chief Executive Officer of Crinetics. "His leadership has been instrumental in the success of our capital markets activities, the build-out of our finance and corporate affairs functions, and the maturation of our organization and culture. During his tenure, we have successfully raised a significant amount of capital to fund development of our differentiated pipeline, our rapidly growing clinical and research programs, and our anticipated commercial launch."

"I am proud of what Crinetics has accomplished, and it has been a privilege to work with such a talented and dedicated team over the last six years," said Mr. Wilson. "The company is in a strong financial position to invest across its deep pipeline, and I have unwavering confidence in the team’s ability to continue discovering new, meaningful therapies and advance them through the clinic to reach as many patients as possible. I will continue to support the company through this transition and look forward to following the company’s continued success."

Second Quarter 2024 and Recent Highlights:
Presented positive initial results from atumelnant studies at the Endocrine Society’s Annual Meeting (ENDO 2024). In June, Crinetics presented positive initial results from its ongoing, open-label studies of atumelnant for the treatment of ACTH-dependent Cushing’s syndrome and congenital adrenal hyperplasia (CAH) at ENDO 2024 in Boston.
Presented data from paltusotine development program at ENDO 2024. In June, Crinetics presented findings from its paltusotine development program in acromegaly. Data presented included results of the Phase 3 PATHFNDR-2 trial, a new analysis of patient reported outcome data from the Phase 3 PATHFNDR-1 trial, and interim long-term efficacy and safety results at 42 months from the open-label ACROBAT Advance extension study.
Selected development candidates in two programs. Crinetics has identified an oral parathyroid hormone antagonist development candidate for the treatment of hyperparathyroidism and IND-enabling studies have commenced. In addition, a development candidate in the SST3 agonist program was selected for the treatment of autosomal dominant polycystic kidney disease.
Scott Struthers, Ph.D., founder and chief executive officer of Crinetics, was named an Entrepreneur of The Year 2024 Pacific Southwest Award winner.
Strengthened scientific leadership team. In April, Crinetics appointed Lise Kjems, M.D., Ph.D. as Senior Vice President of Endocrinology Clinical Research, and in May, Crinetics appointed Robert M. Cuddihy, M.D., as Senior Vice President of Medical Affairs.
Key Upcoming Milestones
Topline data and additional data from the ongoing Phase 2 studies of atumelnant in CAH and ACTH-dependent Cushing’s syndrome, respectively, are anticipated by the end of 2024.
Submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) seeking regulatory approval of paltusotine for the treatment of acromegaly is on track for the second half of 2024.
Initiation of a Phase 3 program of paltusotine for carcinoid syndrome is expected by the end of 2024, following consultation with the FDA.
Additional research pipeline updates are expected by the end of 2024.
Second Quarter 2024 Financial Results
Research and development expenses were $58.3 million for the three months ended June 30, 2024, compared to $40.6 million for the same period in 2023. The increase was primarily attributable to higher personnel costs and manufacturing and development activities, both of which were driven by the advancement of our clinical programs and the expansion of our preclinical portfolio.
General and administrative expenses were $24.8 million for the three months June 30, 2024, compared to $13.3 million for the same period in 2023. The increase was primarily driven by higher personnel and commercial planning costs.
Net loss for the three months ended June 30, 2024, was $74.1 million, compared to a net loss of $51.0 million for the same period in 2023.
Revenues were $0.4 million for the three months ended June 30, 2024, compared to $1.0 million for the same period in 2023. Revenues during the current year’s quarter were derived from the paltusotine licensing arrangement with our Japanese partner, Sanwa Kagaku Kenkyusho.
Unrestricted cash, cash equivalents, and investments totaled $863.0 million as of June 30, 2024, compared to $558.6 million as of December 31, 2023. Based on its current projections, Crinetics expects that its cash, cash equivalents and short-term investments will be sufficient to fund its current operating plan into 2028.
Conference Call and Webcast Details
Management will hold a live conference call and webcast today, Thursday, August 8, 2024 at 4:30 p.m. ET. To participate, please dial 1-800-267-6316 (domestic) or 1-203-518-9783 (international) and refer to Conference ID CRNXQ2. To access the webcast, click here. Following the live event, a replay of the call will be available on the Investors section of the Company’s website.

*Proposed international nonproprietary name under review.