On May 8, 2023 Deciphera Pharmaceuticals, Inc. (NASDAQ: DCPH), a biopharmaceutical company focused on discovering, developing, and commercializing important new medicines to improve the lives of people with cancer, reported that members of the management team will participate in a fireside chat at the JMP Securities Life Sciences Conference on Monday, May 15, 2023 at 2:30 PM ET in New York, NY (Press release, Deciphera Pharmaceuticals, MAY 8, 2023, View Source [SID1234631136]).

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A live webcast of the fireside chat will be available on the "Events and Presentations" page in the "Investors" section of the Company’s website at View Source A replay of the webcast will be archived on the Company’s website for 90 days following the presentation.

On May 8, 2023 CRISPR Therapeutics (Nasdaq: CRSP), a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases, reported financial results for the first quarter ended March 31, 2023 (Press release, CRISPR Therapeutics, MAY 8, 2023, View Source [SID1234631135]).

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"In the first quarter of 2023, we continued strong momentum across our portfolio. We and our partner Vertex have now completed regulatory submissions for exa-cel in the United States, European Union and United Kingdom, positioning exa-cel to potentially become the first approved CRISPR-based therapy in the world, a remarkable pace of progress considering the discovery of the CRISPR platform took place a little more than a decade ago," said Samarth Kulkarni, Ph.D., Chief Executive Officer of CRISPR Therapeutics. "In parallel, we continue to drive our portfolio programs, including the initiation of clinical trials for our next-generation CAR T candidates, CTX112 and CTX131. In addition, we expect to advance CTX310, our lead in vivo program targeting ANGPTL3, into clinical trials later this year. We are well-positioned to drive towards our mission of bringing transformative and potentially curative therapies to patients in need."

Recent Highlights and Outlook

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Hemoglobinopathies
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In April, CRISPR Therapeutics and Vertex Pharmaceuticals announced the completion of the rolling submissions of their biologics licensing applications (BLAs) to the U.S. Food and Drug Administration (FDA) for the investigational treatment exagamglogene autotemcel (exa-cel), formerly known as CTX001, for severe sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT). The BLAs include requests for Priority Review, which, if granted, would shorten the FDA’s review of the application to eight months from the time of submission versus a standard review timeline of 12 months. In the U.S., exa-cel has been granted Fast Track, Regenerative Medicine Advanced Therapy (RMAT), Orphan Drug (ODD) and Rare Pediatric Disease designations.
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In December 2022, CRISPR Therapeutics and Vertex Pharmaceuticals completed regulatory submissions for exa-cel with the European Medicines Agency (EMA) and the Medicines and Healthcare products Regulatory Agency (MHRA) in the EU and the U.K., respectively. Both the EMA and the MHRA have validated the Marketing Authorization Applications (MAAs), indicating acceptance of the marketing applications and initiation of the review. Exa-cel has been granted Priority Medicines (PRIME) and ODD in the EU. In the U.K., exa-cel has been granted an Innovation Passport under the Innovative Licensing and Access Pathway (ILAP) from the MHRA.
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The Phase 1/2/3 CLIMB-111 and CLIMB-121 studies and the CLIMB-131 long-term follow-up study are ongoing in patients 12 years of age and older.
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Two additional Phase 3 studies of exa-cel in pediatric patients with TDT and SCD continue to enroll patients.
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CRISPR Therapeutics continues to advance its anti-CD117 (c-Kit) antibody-drug conjugate (ADC), its internal targeted conditioning program, in pre-clinical studies. This targeted conditioning agent has the potential to significantly expand the patient population that can benefit from exa-cel.

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Immuno-Oncology
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CRISPR Therapeutics continues to enroll and dose patients in a Phase 2 single-arm potentially registrational clinical trial of CTX110, its wholly-owned allogeneic chimeric antigen receptor T cell (CAR T) investigational therapy targeting CD19+ B-cell malignancies.
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CRISPR Therapeutics continues to enroll and dose patients in the Company’s ongoing Phase 1 COBALT-LYM trial evaluating the safety and efficacy of CTX130, its wholly-owned allogeneic CAR T cell therapy targeting CD70 for the treatment of relapsed or refractory T cell malignancies. Based on preliminary data, CTX130 was granted the RMAT designation by the FDA. Given the encouraging early results, the Company continues to advance CTX130 for these difficult-to-treat T cell lymphomas in its COBALT-LYM trial.
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CRISPR Therapeutics has begun enrolling patients in the CTX112 clinical trial, its next generation CAR T candidate targeting CD19+ B-cell malignancies, following the previously-announced clearance of its Investigational New Drug (IND) application by the FDA. CTX112 incorporates the edits in CTX110 plus additional edits to the genes encoding Regnase-1 and TGFBRII, which have been shown to increase the potency of the CAR T cells in pre-clinical studies. CRISPR Therapeutics has also initiated a clinical trial for CTX131, its next generation CAR T cell candidate targeting CD70, following clearance of its IND application by the FDA in February 2023. CTX131 incorporates the edits in CTX130 plus additional edits to the genes encoding Regnase-1 and TGFBRII, which have been shown to increase the potency of the CAR T cells in pre-clinical studies.
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In April, CRISPR Therapeutics presented an oral presentation of preclinical data at the New Drugs on the Horizon Session (Part 1) in the American Association for Cancer Research (AACR) (Free AACR Whitepaper) 2023 Annual Meeting for CTX112 and CTX131, entitled CTX112 and CTX131: Next-generation CRISPR/Cas9-engineered allogeneic CAR T cells incorporating novel edits that increase potency and efficacy in the treatment of lymphoid and solid tumors.
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Regenerative Medicine and In Vivo
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In March, CRISPR Therapeutics and Vertex and CRISPR Therapeutics and ViaCyte, Inc., which was acquired by Vertex in 2022, entered into agreements relating to the research, development, manufacturing and commercialization of therapeutic products in the diabetes field, including a new non-exclusive licensing agreement for the use of CRISPR Therapeutics’ CRISPR/Cas9 gene editing technology to accelerate the development of Vertex’s hypoimmune cell therapies for type 1 diabetes (T1D). In connection with entering into the agreements with Vertex and ViaCyte, CRISPR Therapeutics received $100 million up-front from Vertex. CRISPR Therapeutics will be eligible for up to an additional $230 million in research and development milestones and receive royalties on any future products resulting from the non-exclusive licensing agreement. CRISPR Therapeutics and ViaCyte continue to collaborate on their existing gene-edited allogeneic stem cell therapies, using ViaCyte cells, for the treatment of diabetes under the terms of their collaboration.
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Based upon ongoing progress with its in vivo approaches for liver gene editing, CRISPR Therapeutics expects to move multiple programs utilizing in vivo approaches into the clinic within the next 12 months. The Company continues to advance its lead in vivo program, CTX310, targeting angiopoietin-related protein 3 (ANGPTL3) into clinical trials this year.
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Other Corporate Matters
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In March, CRISPR Therapeutics announced the appointment of Raju Prasad, Ph.D., as Chief Financial Officer. He joined CRISPR Therapeutics from William Blair & Company, where he served as a Partner and Senior Equity Research Analyst covering cell therapy, gene therapy, and gene editing companies.
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In March, CRISPR Therapeutics announced the departure of Brad Bolzon, Ph.D., Chairman and Managing Director of Versant Ventures, from the Board of Directors after nearly a decade of service.

First Quarter 2023 Financial Results

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Cash Position: Cash, cash equivalents and marketable securities were $1,889.5 million as of March 31, 2023, compared to $1,868.4 million as of December 31, 2022. The increase in cash of $21.1 million was primarily driven by the upfront payment received from Vertex in connection with a non-exclusive license agreement and a benefit from changes in net-working capital, offset by operating expenses.
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Revenue: Total collaboration revenue was $100.0 million for the quarter ended March 31, 2023. Collaboration revenue for the first quarter of 2022 was not material. Collaboration revenue recognized in the first quarter of 2023 was primarily attributable to revenue recognized in connection with the upfront payment from Vertex.
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R&D Expenses: R&D expenses were $99.9 million for the first quarter of 2023, compared to $118.2 million for the first quarter of 2022. The decrease in R&D expense was primarily driven by reduced variable external research and manufacturing costs.
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G&A Expenses: General and administrative expenses were $22.4 million for the first quarter of 2023, compared to $28.0 million for the first quarter of 2022. The decrease in G&A expense was primarily driven by a decrease in external professional costs.
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Collaboration Expense: Collaboration expense, net, was $42.2 million for the first quarter of 2023, compared to $30.6 million for the first quarter of 2022. The increase in collaboration expense, net, was primarily driven by an increase in manufacturing and pre-commercial costs associated with the exa-cel program.
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Net Loss: Net loss was $53.1 million for the first quarter of 2023, compared to a net loss of $179.2 million for the first quarter of 2022.

About exagamglogene autotemcel (exa-cel)

Exa-cel, formerly known as CTX001, is an investigational, autologous, ex vivo CRISPR/Cas9 gene-edited therapy that is being evaluated for patients with TDT or SCD characterized by recurrent vaso-occlusive crises (VOCs), in which a patient’s own hematopoietic stem cells are edited to produce high levels of fetal hemoglobin (HbF; hemoglobin F) in red blood cells. HbF is the form of the oxygen-carrying hemoglobin that is naturally present during fetal development, which then switches to the adult form of hemoglobin after birth. The elevation of HbF by exa-cel has the potential to alleviate transfusion requirements for patients with TDT and reduce painful and debilitating sickle crises for patients with SCD. Earlier results from these ongoing trials were published in The New England Journal of Medicine in January of 2021.

Based on progress in this program to date, exa-cel has been granted Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug, and Rare Pediatric Disease designations from the FDA for both TDT and SCD. Exa-cel has also been granted Orphan Drug Designation from the European Commission, as well as Priority Medicines (PRIME) designation from the European Medicines Agency (EMA), for both TDT and SCD. In the U.K., exa-cel has been granted an Innovation Passport under the Innovative Licensing and Access Pathway (ILAP) from the MHRA.

About CLIMB-111 and CLIMB-121

The ongoing Phase 1/2/3 open-label trials, CLIMB-111 and CLIMB-121, are designed to assess the safety and efficacy of a single dose of exa-cel in patients ages 12 to 35 years with TDT or with SCD, characterized by recurrent VOCs, respectively. The trials are now closed for enrollment. Patients will be followed for approximately two years after exa-cel infusion. Each patient will be asked to participate in CLIMB-131, a long-term follow-up trial.

About CLIMB-131

This is a long-term, open-label trial to evaluate the safety and efficacy of exa-cel in patients who received exa-cel in CLIMB-111, CLIMB-121, CLIMB-141 or CLIMB-151. The trial is designed to follow participants for up to 15 years after exa-cel infusion.

About CLIMB-141 and CLIMB-151

The ongoing Phase 3 open-label trials, CLIMB-141 and CLIMB-151, are designed to assess the safety and efficacy of a single dose of exa-cel in patients ages 2 to 11 years with TDT or with SCD, characterized by recurrent VOCs, respectively. The trials are now open for enrollment and currently enrolling patients ages 5 to 11 years of age and will plan to extend to patients 2 to less than 5 years of age at a later date. Each trial will enroll approximately 12 patients. Patients will be followed for approximately two years after infusion. Each patient will be asked to participate in CLIMB-131, a long-term follow-up- trial.

About the CRISPR-Vertex Collaboration

CRISPR Therapeutics and Vertex Pharmaceuticals entered into a strategic research collaboration in 2015 focused on the use of CRISPR/Cas9 to discover and develop potential new treatments aimed at the underlying genetic causes of human disease. Exa-cel represents the first potential treatment to emerge from the joint research program. Under an amended collaboration agreement, Vertex now leads global development, manufacturing and commercialization of exa-cel and splits program costs and profits worldwide 60/40 with CRISPR Therapeutics.

About CTX110 and CTX112

CTX110, a wholly owned program of CRISPR Therapeutics, is a healthy donor-derived gene-edited allogeneic CAR T investigational therapy targeting cluster of differentiation 19, or CD19. CTX110 is being investigated in the ongoing CARBON clinical trial, which is designed to assess the safety and efficacy of CTX110 in adult patients with relapsed or refractory CD19-positive B-cell malignancies who have received at least two prior lines of therapy. CTX110 has been granted RMAT designation by the FDA. In addition, CTX112, a next-generation allogeneic CAR T cell therapy targeting CD19, is being investigated in a clinical trial. CTX112 includes two additional edits beyond CTX110 that are designed to enhance the potency of the CAR T cells.

About CTX130 and CTX131

CTX130, a wholly owned program of CRISPR Therapeutics, is a healthy donor-derived gene-edited allogeneic CAR T investigational therapy targeting cluster of differentiation 70, or CD70, an antigen expressed on various solid tumors and hematologic malignancies. CTX130 is being investigated for the treatment of relapsed or refractory T-cell hematologic malignancies in the COBALT-LYM trial and for renal cell carcinoma in the COBALT-RCC trial. CTX130 has been granted Orphan Drug designation for the treatment of T cell lymphoma by the FDA and RMAT designation for the treatment of relapsed or refractory Mycosis Fungoides and Sézary Syndrome (MF/SS), types of cutaneous T cell lymphoma (CTCL). In addition, CTX131, a next-generation allogeneic CAR T cell therapy targeting CD70, is being assessed for safety and efficacy in a clinical trial investigating a basket of select solid tumors. CTX131 includes two additional edits beyond CTX130 that are designed to enhance the potency of the CAR T cells.

About VCTX210 and VCTX211

VCTX210 is an investigational, allogeneic, gene-edited, immune-evasive, stem cell-derived investigational therapy for the treatment of T1D. VCTX210 is being developed under a co-development and co-commercialization agreement between CRISPR Therapeutics and ViaCyte, Inc. VCTX211 is an allogeneic, gene-edited, stem cell-derived investigational therapy for the treatment of T1D, which incorporates additional gene edits that aim to further enhance cell fitness. This immune-evasive cell replacement therapy is designed to enable patients to produce their own insulin in response to glucose.

On May 8, 2023 Corvus Pharmaceuticals, Inc. (Corvus or the Company) (Nasdaq: CRVS), a clinical-stage biopharmaceutical company, reported a business update and provided financial results for the quarter ended March 31, 2023 (Press release, Corvus Pharmaceuticals, MAY 8, 2023, View Source [SID1234631134]).

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"We continue to focus on advancing CPI-818, our ITK inhibitor, towards a potential registrational Phase 3 randomized trial for T cell lymphoma later this year," said Richard A. Miller, M.D., co-founder, president and chief executive officer of Corvus. "We are generating encouraging clinical data from our ongoing Phase 1/1b trial with meaningful objective responses seen in patients with multiply recurrent T cell lymphomas. The data generated so far, continues to support the use of our recently identified biomarker to enrich for patients most likely to respond to treatment with CPI-818. In addition, we recently presented data at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) annual meeting that highlights CPI-818’s therapeutic potential in solid tumors via a novel immunotherapy mechanism of action. This adds to the broad preclinical and clinical data supporting the potential of ITK inhibition with CPI-818 as a platform opportunity across oncology and immune diseases. For our partner led programs, enrollment is ongoing in clinical trials evaluating ciforadenant and mupadolimab, with the potential for initial ciforadenant data from the Phase 1b/2 trial in patients with metastatic renal cell cancer expected to be released before the end of 2023."

Business Update and Strategy

Prioritized Program: CPI-818 (Corvus’ selective ITK inhibitor)

CPI-818 for T Cell Lymphoma

Corvus continues to enroll patients with relapsed T cell lymphomas (TCL) in a Phase 1/1b trial evaluating single agent therapy with CPI-818. When last reported as of February 23, 2023, 20 patients were enrolled at the optimum 200 mg BID dose, including 13 evaluable for tumor response and seven patients that had not yet been evaluated for tumor response. This data was presented at the 10th Whistler Global Summit on Hematologic Malignancies, which took place March 29 to April 2, 2023 in Whistler British Columbia, Canada. At that meeting Corvus also reported on the use of a recently incorporated biomarker based on peripheral blood absolute lymphocyte count (ALC), which identified patients most likely to respond to therapy with CPI-818. Data presented also showed that this biomarker did not select for more favorable patients based on response to their last treatment regimen prior to receiving CPI-818.
Updated data as of May 1, 2023: A total of 28 patients were enrolled in the Phase 1/1b trial at the optimum 200 mg BID dose, including 19 evaluable for tumor response. There have been 2 complete responses (CR), 1 nodal CR and 3 partial responses (PR). Two of the patients with PRs remain on therapy. A total of nine patients remain on therapy, including five who have not had their initial tumor response evaluation. For patients with ALC above 900 per cubic milliliter of blood, objective responses (CR plus PR) were seen in six of 13 patients with disease control (CR, PR and stable disease) in 11 of 13 patients. No objective responses were seen in six patients (0 for 6) with ALC below 900. The median progression free survival is 19.9 months versus 2.1 months for patients with ALC above 900 and ALC below 900, respectively. Eligible patients for the clinical trial are now required to have ALC above 900.
Based on the current enrollment rate of the Phase 1/1b clinical trial, Corvus believes that the number of patients treated in this clinical trial would provide adequate safety and preliminary efficacy data to inform the design of a potential registrational Phase 3 randomized clinical trial. As recommended by the FDA, Corvus plans to meet with the FDA to discuss such a clinical trial; it is anticipated that this meeting will take place during the third quarter of this year.
CPI-818 Preclinical Data in Solid Tumors

On April 17, 2023, Corvus presented data at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting demonstrating CPI-818’s potential to treat a variety of solid and hematological cancers based on a novel immunotherapy mechanism of action. The data demonstrated that CPI-818 monotherapy (7 days oral administration) provided statistically significant inhibition of growth in established tumors in the following cancer models: CT26 colon cancer, RENCA kidney cancer, B16 melanoma, EL4 TCL and A20 B cell lymphoma. Corvus believes the data supports a novel mechanism of action: CPI-818 modulates T cell differentiation and enhances the immune system via Th1 skewing, increased T cell cytolytic capacity and reduction of T cell exhaustion.
Partner Led Programs: Ciforadenant (adenosine 2a receptor inhibitor) and Mupadolimab (anti-CD73)

The Kidney Cancer Research Consortium (KCRC) is enrolling a Phase 1b/2 clinical trial evaluating ciforadenant as a potential first line therapy for metastatic renal cell cancer (RCC) in combination with ipilimumab (anti-CTLA-4) and nivolumab (anti-PD-1). The clinical trial is expected to enroll up to 60 patients and initial data is anticipated before the end of 2023.
Angel Pharmaceuticals, Corvus’ partner in China, is enrolling patients in a Phase 1/1b clinical trial of mupadolimab in patients with non-small cell lung cancer (NSCLC) and head and neck squamous cell cancers. In this clinical trial, patients will receive mupadolimab monotherapy or in combination with pembrolizumab.
Financial Results
As of March 31, 2023, Corvus had cash, cash equivalents and marketable securities totaling $34.5 million. This compared to cash, cash equivalents and marketable securities of $42.3 million as of December 31, 2022. Corvus expects full year 2023 net cash used in operating activities to be between approximately $19 million and $22 million, resulting in a projected cash balance of between $20 million and $23 million as of December 31, 2023. Based on its current plans, Corvus expects its cash to fund operations into 2024.

Research and development expenses for the three months ended March 31, 2023 totaled $4.6 million compared to $5.1 million for the same period in 2022. The decrease of $0.5 million was primarily due to lower clinical trial and manufacturing costs associated with the development of mupadolimab.

The net loss for the three months ended March 31, 2023 was $7.9 million compared to a net loss of $8.3 million for the same period in 2022. Total stock compensation expense for the three months ended March 31, 2023 was $0.5 million compared to $0.7 million for the same period in 2022 and the non-cash loss from Corvus’ equity method investment in Angel Pharmaceuticals was $1.7 million for the three months ended March 31, 2023 compared to $1.0 million in the same period in 2022.

On May 8, 2023 Coherus BioSciences, Inc. ("Coherus", "the Company", Nasdaq: CHRS), reported financial results for the quarter ended March 31, 2023 and recent business highlights (Press release, Coherus Biosciences, MAY 8, 2023, View Source [SID1234631133]):

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RECENT BUSINESS HIGHLIGHTS

CIMERLI

On April 1st, the permanent, product-specific Q-code assigned to CIMERLI (ranibizumab-eqrn) by the U.S. Centers for Medicare & Medicaid Services (CMS) became active, enabling more efficient, electronic billing processes and reducing time to reimbursement for providers. Demand increased sharply as expected in April, with over 7,000 units of CIMERLI shipped, exceeding in one month 70% of Q1 unit sales.
UDENYCA

The FDA approved a single-dose, prefilled autoinjector (AI) presentation of UDENYCA (pegfilgrastim-cbqv) on March 3, 2023, which represents the first presentation innovation in the pegfilgrastim space in eight years and highlights Coherus’ commitment to developing innovative treatments that expand access and address the needs of patients undergoing cancer treatment. Coherus plans to launch UDENYCA AI later this month.
The FDA review of the prior approval supplement for Coherus’ third pegfilgrastim presentation, the UDENYCA on-body injector (OBI), is ongoing, and Coherus plans to launch UDENYCA OBI directly upon potential approval later this year.
Toripalimab

The U.S. Food and Drug Administration (FDA) has notified the Company that it plans to conduct the required inspection of the toripalimab manufacturing facility in China later in May 2023. The inspections, previously hindered by COVID-related travel restrictions, are part of the FDA’s review of the biologics license application (BLA) for toripalimab, a PD-1 inhibitor for the treatment of nasopharyngeal carcinoma (NPC). Coherus anticipates potential FDA approval and commercial launch of toripalimab in the U.S. in Q3 2023.
Positive toripalimab clinical data will be presented at the upcoming 2023 ASCO (Free ASCO Whitepaper) Annual Meeting including the final overall survival analysis for JUPITER-02 in NPC, updated overall survival analysis for CHOICE-01 in advanced non-small cell lung cancer (NSCLC), interim analysis of event-free survival for NEOTORCH in Stage II/III NSCLC, and clinical data for TORCHLIGHT in advanced triple-negative breast cancer.
YUSIMRY

Preparations are underway for commercial launch in Q3 2023 of YUSIMRY, a Humira biosimilar with a citrate-free and sting-free formulation delivered via a state-of-the-art autoinjector. Coherus has invested in robust, large-scale manufacturing capabilities to ensure ample supply upon launch.
The FDA recently approved YUSIMRY prior approval supplements for the autoinjector presentation and for large-scale drug supply manufacturing.
Novel Immuno-oncology Pipeline

Patient recruitment is underway in the U.S.-based Phase 1/2 clinical trial of CHS-006, a TIGIT-targeted antibody in combination with toripalimab in patients with advanced solid tumors (NCT05757492).
Coherus expects to file an IND by year end for CHS-1000, a novel ILT4-targeted antibody.
"With the approval of the UDENYCA autoinjector, the activation of the CMS-assigned Q-code and the other product launches planned across our diversified portfolio, we are well positioned for accelerated revenue growth for the remainder of 2023 and beyond. We continue to sharply focus on commercial execution, and are already beginning to see the impact of the Q-code on CIMERLI sales at the start of the second quarter," said Denny Lanfear, Coherus’ Chairman and Chief Executive Officer. "Innovative presentations offering differentiated value, such as the UDENYCA autoinjector launching this month, as well as the UDENYCA on-body injector presentation, with anticipated approval and launch later in the year, will offer patients and physicians unprecedented choice in their treatment options, driving market share gains and long-term value for the franchise."

Mr. Lanfear continued, "Inspections of the toripalimab manufacturing facilities are scheduled for later this month, with clinical site inspections to follow. NPC patients currently have no FDA approved therapies, and toripalimab has the potential to be the new standard of care for across multiple lines of treatment, if approved. We are planning for approval and launch in the third quarter."

FIRST QUARTER 2023 FINANCIAL RESULTS

Net revenue was $32.4 million during the three months ended March 31, 2023 and included $26.2 million of net sales of UDENYCA and $6.2 million of net sales of CIMERLI, which was launched in October 2022. Net sales of UDENYCA for the first quarter of 2023 were reduced by a $1.7 million charge for a contingent liability related to resolving a dispute regarding certain sales from October 2020 through December 2021. Net revenue was $60.1 million during the three months ended March 31, 2022. The decline was primarily due to a decrease in the number of units of UDENYCA sold as well as a lower net realized price due to increased competition.

Cost of goods sold (COGS) was $16.9 million and $9.4 million during the three months ended March 31, 2023 and 2022, respectively. UDENYCA COGS includes a mid-single digit royalty on net sales payable through the first half of 2024, and CIMERLI COGS includes a low to mid 50% royalty on gross profits. COGS for the first quarter of 2023 also includes $3.0 million in contract modification fees with one of our manufacturers and $2.7 million in write-offs of inventory that was damaged during processing at one of our manufacturers.

Research and development (R&D) expense for the three months ended March 31, 2023 was $34.2 million. R&D expense for the three months ended March 31, 2022 was $82.9 million, which included a $35 million option exercise fee paid to Junshi Biosciences to license CHS-006, a clinical-stage TIGIT-targeted antibody, as well as development and manufacturing costs for clinical and preclinical pipeline programs.

Selling, general and administrative (SG&A) expense for the three months ended March 31, 2023 was $49.2 million compared to $48.8 million for the same period in 2022. The increase was primarily due to $1.3 million in restructuring charges from our reduction in force that occurred in the first quarter of 2023.

Net loss for the first quarter of 2023 was $75.7 million, or $(0.96) per share on a diluted basis, compared to a net loss of $96.1 million, or $(1.24) per share on a diluted basis for the same period in 2022.

Non-GAAP net loss for the first quarter of 2023 was $59.5 million, or $(0.75) per share on a diluted basis, compared to non-GAAP net loss of $77.0 million, or $(1.00) per share on a diluted basis for the same period in 2022. 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 $128.1 million as of March 31, 2023, compared to $191.7 million at December 31, 2022.

2023 Revenue and R&D and SG&A Expense Guidance
Coherus expects its 2023 net product revenue will exceed $275 million, including at least $100 million of CIMERLI net revenue.

Coherus projects combined R&D and SG&A expenses for 2023 to be in the range of $315 to $335 million. This guidance includes approximately $50 million of stock-based compensation expense and excludes any potential collaboration upfront payments to Klinge Pharma for the in-license of its Eylea biosimilar program or milestones payments to Junshi Biosciences due upon U.S. approval of toripalimab.

This financial guidance also excludes the effects of any potential future strategic acquisitions, collaborations or investments, 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: Monday, May 8th, 2023, starting at 5:00 p.m. Eastern 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/BI12e6d284dae8440e91891f2cef4f2097

Please dial-in 15 minutes early to ensure a timely connection to the call.

Webcast Link: View Source
A replay of the webcast will be archived on the "Investors" section of the Coherus website at View Source

On May 8, 2023 BioXcel Therapeutics, Inc. (Nasdaq: BTAI), a biopharmaceutical company utilizing artificial intelligence approaches to develop transformative medicines in neuroscience and immuno-oncology, reported its financial results for the first quarter ended March 31, 2023, and provided an update on key strategic initiatives (Press release, BioXcel Therapeutics, MAY 8, 2023, View Source [SID1234631132]).

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"The first quarter marked a strong start to the year with numerous advancements in our clinical programs and continued commercial focus building the agitation market for our new therapeutic option in a historically underdiagnosed and underserved medical condition," said Vimal Mehta, Ph.D., CEO of BioXcel Therapeutics. "We are gearing up to announce top-line data readouts in agitation from two Phase 3 pivotal trials as well as BXCL501 potential as an adjunctive treatment for chronic use in our MDD program. In addition, IGALMI’s launch momentum is expanding our reach into addressable market opportunities. We believe the second quarter of 2023 represents a defining moment for the Company as we expand the full potential of BXCL501 in agitation for at-home use and long-term care settings, and in depression. These upcoming catalysts may have a transformational impact for patients in need and all our stakeholders."

Company Highlights

Neuroscience Franchise

IGALMI (dexmedetomidine) sublingual film

IGALMI is approved by the U.S. Food and Drug Administration (FDA) for the acute treatment of agitation associated with schizophrenia or bipolar I or II disorder in adults.1 Up to an estimated 16 million institutional episodes occur annually within these two patient populations in the U.S.2-4*

Key Commercial Parameters

· IGALMI launch is accelerating with continued deployment of integrated commercial team in 2023:

o Secured more than 130 hospital formulary wins to date, which have doubled in the last two months; equates to $55 million of addressable market with Integrated Delivery Network (IDN) formulary approvals.

o Six hundred additional Pharmacy & Therapeutics (P&T) Committee votes scheduled and approximately 25% of target IDN beds representing an additional $255 million of addressable market scheduled to vote.

o More than half of all ordering hospitals have reordered; repeat orders demonstrate real-world utility and growing health care provider (HCP) interest.

o Sales team has reached over 75% of 1,700 targeted hospitals with focus on deepening advocacy and driving demand.

o Group Purchasing Organization (GPO) process largely complete with nearly 80% of targeted beds under contract.

o Amplifying IGALMI awareness through multi-channel approach including digital and print media, peer influence initiatives, and Free Trial Program.

Medical Affairs

· Medical Science Liaison and Medical Managed Care teams engaging in scientific dialogue with HCPs ahead of IDN and hospital formulary reviews.

o Observed a 73% increase in medical community requests for IGALMI clinical information over prior quarter.

o Conducted a real-world survey of early-adopter HCPs; results expected to be presented at the American Society of Health-System Pharmacists Annual Meeting in June 2023.

o Continuing extensive medical education initiatives through multiple clinical and scientific publications and presentations.

Development Pipeline

BXCL501, an investigational proprietary, sublingual film formulation of dexmedetomidine, has received Breakthrough Therapy and Fast Track designation for the acute treatment of agitation associated with dementia.

• Alzheimer’s Disease-related Agitation: TRANQUILITY program is designed to evaluate BXCL501 for the acute treatment of Alzheimer’s-related agitation; up to 100 million Alzheimer’s-related agitation episodes are estimated to occur in the U.S. annually.2*

o TRANQUILITY II: Trial is fully enrolled, and all patients have completed the study in assisted living facilities (ALFs) and residential care settings.

• Data cleaning and verification in progress.

• Top-line data from pivotal trial expected in June 2023.

o TRANQUILITY III: Continuing enrollment of patients with moderate to severe dementia in long-term-care facilities.

• Bipolar or Schizophrenia-related Agitation (At-Home Use): SERENITY III program is designed to evaluate BXCL501 for at-home use, where up to 23 million bipolar or schizophrenia-related agitation episodes are estimated to occur in the U.S. annually.2-4*

SERENITY III consists of two parts:

o Part 1: Assessing the efficacy and safety of 60mcg dose in acute treatment of agitated patients with bipolar I or II disorder or schizophrenia.

• Data cleaning and verification in progress.

• Top-line efficacy data from pivotal trial expected in May 2023.

o Part 2: Evaluating the safety of 60 mcg dose, and a second 60 mcg dose is allowed if required, at-home.

• Expect to initiate Part 2 in Q2 2023.

• Adjunctive Treatment for Major Depressive Disorder (MDD) for At-Home Use: Phase 1b Multiple Ascending Dose (MAD) trial was designed to test safety and tolerability of daily dosing of BXCL501 for seven days in healthy volunteers to inform proof-of-concept (POC) trial dose selection. In Phase 2 program, treatment in MDD patients will be evaluated with BXCL501 in combination with selective serotonin- or serotonin-norepinephrine reuptake inhibitors (SSRIs or SNRIs, respectively) to assess rapid antidepressant response. Over 300 million antidepressant prescriptions are filled annually in the U.S.,5* and current treatments are limited by slow onset of action and incomplete responses.

o Data cleaning and verification in progress.

o Top-line results are expected in May 2023.

OnkosXcel Therapeutics

OnkosXcel Therapeutics is a subsidiary of BioXcel Therapeutics focused on the sustained growth of the Company’s immuno-oncology (I-O) franchise, including BXCL701, its most advanced I-O program. BXCL701 is an investigational, oral innate immune activator in development for the treatment of aggressive forms of prostate cancer and other solid and liquid tumors.

• Continuing to actively evaluate strategic options for OnkosXcel Therapeutics:

o Small Cell Neuroendocrine Prostate Cancer (SCNC) Program: In 2023, it is estimated there will be 288,3006 new prostate cancer patients in the U.S., with approximately 11,500 patients progressing to SCNC.7

• Planned Phase 2b potential pivotal study for BXCL701 monotherapy and in combination with KEYTRUDA (pembrolizumab) in SCNC expected to initiate in 2H 2023, subject to further discussions with FDA.

o Predictive Biomarker for BXCL701: Additional findings on DPP9 overexpression, a potential actionable biomarker for BXCL701 response, expected to be presented at an upcoming medical meeting.

o Investigator-Sponsored Trials at Top Academic Centers: BXCL701 to be studied in combination with KEYTRUDA (pembrolizumab) at Georgetown Lombardi Cancer Center and Dana-Farber Cancer Institute in pancreatic cancer and AML, respectively.

First Quarter 2023 Financial Results

Net Revenue: Net revenue was approximately $206,000 for the quarter, in line with the fourth quarter. The Company expects to see a notable uptick in revenues in the second half of the year in connection with additional formulary approvals.

Research and Development (R&D) Expenses: R&D expenses were $27.8 million for the first quarter of 2023, compared to $18.6 million for the same period in 2022. The increased expenses were primarily attributable to multiple clinical trials and CMC costs related to the upcoming three data readouts.

Selling, General and Administrative (SG&A) Expenses: SG&A expenses were $23.6 million for the first quarter of 2023, compared to $12.9 million for the same period in 2022. The increased expenses were primarily attributable to personnel and sales, market access, and marketing costs associated with the commercialization of IGALMI in the U.S.

Net Loss: BioXcel Therapeutics had a net loss of $52.8 million for the first quarter of 2023, compared to a net loss of $31.5 million for the same period in 2022.

Cash and cash equivalents totaled $165.5 million as of March 31, 2023. BioXcel Therapeutics believes that full execution of its strategic financing with Oaktree and Qatar Investment Authority, and IGALMI revenues, would result in a cash runway into 2025.

Anticipated Milestones

• Top-line Clinical Trial Data Readouts

o Pivotal SERENITY III Phase 3 trial (Part 1): May 2023

o Phase 1b MAD trial for MDD program: May 2023

o Pivotal TRANQUILITY II Phase 3 trial: June 2023

• Expected Clinical Trial Initiations

o Pivotal SERENITY III Phase 3 trial (Part 2): Q2 2023

o Phase 2b potential pivotal study of BXCL701 in SCNC: 2H 2023

Conference Call

BioXcel Therapeutics will host a conference call and webcast on May 8 at 8:00 a.m. ET to discuss its first quarter 2023 financial results and provide an update on recent operational highlights. To access the call, please dial 877-407-5795 (domestic) and 201-689-8722 (international). A live webcast will be available on the Investors section of the corporate website, bioxceltherapeutics.com, and a replay will be available through August 8, 2023.

BioXcel Therapeutics may use its website as a distribution channel of material information about the Company. Financial and other important information regarding the Company is routinely posted on and accessible through the Investors sections of its website at www.bioxceltherapeutics.com. In addition, you may automatically receive email alerts and other information about the Company when you enroll your email address by visiting the "Email Alerts" option under the News/Events menu of the Investors & Media section of its website.

About IGALMI (dexmedetomidine) sublingual film

INDICATION

IGALMI (dexmedetomidine) sublingual film is a prescription medicine, administered under the supervision of a health care provider, that is placed under the tongue or behind the lower lip and is used for the acute treatment of agitation associated with schizophrenia and bipolar disorder I or II in adults. The safety and effectiveness of IGALMI has not been studied beyond 24 hours from the first dose. It is not known if IGALMI is safe and effective in children.

IMPORTANT SAFETY INFORMATION

IGALMI can cause serious side effects, including:

• Decreased blood pressure, low blood pressure upon standing, and slower than normal heart rate, which may be more likely in patients with low blood volume, diabetes, chronic high blood pressure, and older patients. IGALMI is taken under the supervision of a healthcare provider who will monitor vital signs (like blood pressure and heart rate) and alertness after IGALMI is administered to help prevent falling or fainting. Patients should be adequately hydrated and sit or lie down after taking IGALMI and instructed to tell their healthcare provider if they feel dizzy, lightheaded, or faint.

• Heart rhythm changes (QT interval prolongation). IGALMI should not be given to patients with an abnormal heart rhythm, a history of an irregular heartbeat, slow heart rate, low potassium, low magnesium, or taking other drugs that could affect heart rhythm. Taking IGALMI with a history of abnormal heart rhythm can increase the risk of torsades de pointes and sudden death. Patients should be instructed to tell their healthcare provider immediately if they feel faint or have heart palpitations.

• Sleepiness/drowsiness. Patients should not perform activities requiring mental alertness, such as driving or operating hazardous machinery, for at least 8 hours after taking IGALMI.

• Withdrawal reactions, tolerance, and decreased response/efficacy. IGALMI was not studied for longer than 24 hours after the first dose. Physical dependence, withdrawal symptoms (e.g., nausea, vomiting, agitation), and decreased response to IGALMI may occur if IGALMI is used longer than 24 hours.

The most common side effects of IGALMI in clinical studies were sleepiness or drowsiness, a prickling or tingling sensation or numbness of the mouth, dizziness, dry mouth, low blood pressure, and low blood pressure upon standing.

These are not all the possible side effects of IGALMI. Patients should speak with their healthcare provider for medical advice about side effects.

Patients should tell their healthcare provider about their medical history, including if they suffer from any known heart problems, low potassium, low magnesium, low blood pressure, low heart rate, diabetes, high blood pressure, history of fainting, or liver impairment. They should also tell their healthcare provider if they are pregnant or breastfeeding or take any medicines, including prescription and over-the-counter medicines, vitamins, and herbal supplements. Patients should especially tell their healthcare provider if they take any drugs that lower blood pressure, change heart rate, or take anesthetics, sedatives, hypnotics, and opioids.

Everyone is encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch or call 1-800-FDA-1088. You can also contact BioXcel Therapeutics, Inc. at 1-833-201-1088 or [email protected].