On February 22, 2024 C4 Therapeutics, Inc. (C4T) (Nasdaq: CCCC), a clinical-stage biopharmaceutical company dedicated to advancing targeted protein degradation science, reported financial results for the year ended December 31, 2023, as well as recent business updates (Press release, C4 Therapeutics, FEB 22, 2024, View Source [SID1234640384]).

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"2023 was an important year for C4T as we executed across three clinical trials, entered into two new collaborations, generated positive dose escalation data from our CFT7455 program for patients with relapsed/refractory multiple myeloma, and meaningfully extended our cash runway," said Andrew Hirsch, president and chief executive officer of C4 Therapeutics. "We began 2024 with positive momentum across our portfolio and are looking forward to sharing data from our two lead programs, CFT7455 and CFT1946, in the second half of the year, as well as supporting our partner, Betta Pharmaceuticals, with trial start-up activities for the Phase 1 trial of CFT8919 in Greater China this year. As we continue to advance our portfolio, we are well-positioned with a strong balance sheet to deliver on our goals and execute through and beyond meaningful value inflection points in order to bring new therapeutic options to patients with difficult-to-treat diseases."

FOURTH QUARTER 2023 AND RECENT ACHIEVEMENTS

CFT7455: CFT7455 is an oral degrader of IKZF1/3 for the potential treatment of relapsed/refractory (R/R) multiple myeloma (MM) and R/R non-Hodgkin’s lymphomas (NHL).

Presented Positive Data from the Ongoing Phase 1/2 Trial in R/R MM. In December 2023, presented positive clinical data from the ongoing CFT7455 Phase 1/2 trial in R/R MM showing that the 14 days on/14 days off schedule is optimal. Additionally, the data demonstrated anti-myeloma activity, including International Myeloma Working Group (IMWG) responses in patients who have undergone numerous lines of prior therapy for MM, including BCMA therapies.
Advanced the Phase 1/2 Clinical Trials. The dose escalation portion of the Phase 1/2 trials evaluating CFT7455 in combination with dexamethasone for R/R MM and as a monotherapy for R/R NHL continues to progress. As of February 2024, two dose levels are open for enrollment in the Phase 1/2 trial for R/R MM and one dose level open for enrollment in the Phase 1/2 trial for R/R NHL.
CFT1946: CFT1946 is an oral degrader targeting BRAF V600X mutations for the potential treatment of solid tumors including non-small cell lung cancer (NSCLC), colorectal cancer (CRC) and melanoma.

Shared Encouraging Initial Pharmacokinetic (PK) and Pharmacodynamic (PD) Data. In January 2024, shared PK and PD data from the initial escalation cohorts of the CFT1946 Phase 1/2 trial demonstrating dose proportional exposure and oral bioavailability, which are associated with deep degradation of BRAF V600E, a clinically validated target.
New Preclinical Data Accepted as a Poster at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2024. Accepted to present CFT1946 preclinical data demonstrating differentiated activity in preclinical models of BRAF V600X melanoma, CRC, NSCLC and brain metastasis.
Advanced the Phase 1/2 Clinical Trial. The dose escalation portion of the CFT1946 Phase 1/2 trial for BRAF V600X mutations, including NSCLC, CRC and melanoma, continues to progress. As of February 2024, three escalation cohorts are complete and dose escalation continues with a fourth dose level currently enrolling.
Collaborations:

Merck

In December 2023, C4T and Merck entered into a license and research collaboration to discover and develop degrader antibody conjugates. Under the terms of the agreement, C4T and Merck will collaborate to develop degrader antibody conjugates directed to an initial undisclosed oncology target exclusive to the collaboration; in January 2024, C4T received the $10 million upfront payment for this initial target. C4T is eligible to receive milestone payments totaling approximately $600 million, as well as tiered royalties on future sales, for degrader antibody conjugates directed to this initial target. The agreement also provides Merck with the option to extend the collaboration to include three additional targets that would be exclusive to the collaboration, which could yield option exercise payments as well as potential milestones and royalties. If Merck exercises all of its options to extend the collaboration, C4T would be eligible to receive up to approximately $2.5 billion in potential payments across the entire collaboration.
Betta Pharmaceuticals

In January 2024, the previously announced $25 million stock purchase by a subsidiary of partner Betta Pharmaceuticals was completed.
In December 2023, Betta Pharmaceuticals received approval from the Chinese National Medical Products Administration for the Clinical Trial Application of CFT8919, which is being evaluated in patients with EGFR L858R NSCLC.
Corporate Updates:

In January 2024, C4T announced 2024 strategic priorities focused on advancing product candidates CFT7455 and CFT1946, delivering on discovery collaborations, and streamlined internal research efforts, which resulted in an approximately 30% reduction in the company’s workforce.
As announced in January 2024, C4T sold approximately 13.7 million shares under the company’s at the market (ATM) offering arrangement, at an average price of $5.42 per share, resulting in approximately $72 million of new equity capital, net of commissions and fees. As of December 31, 2023, 11.2 million of these shares had settled for net proceeds of $57.7 million.
In November 2023, C4T appointed Owen Hughes to its board of directors. Mr. Hughes is an accomplished life sciences executive with nearly three decades of experience in investing, operations and corporate governance.
KEY UPCOMING MILESTONES

CFT7455:

Present updated data from the ongoing Phase 1 dose escalation trial in R/R MM in 2H 2024.
Present data from the ongoing Phase 1 dose escalation trial in R/R NHL in 2H 2024.
Complete Phase 1 dose exploration in R/R MM and R/R NHL by year-end 2024.
CFT1946:

Present preclinical data demonstrating differentiated activity in preclinical models of BRAF V600X NSCLC, CRC, melanoma and brain metastasis at the AACR (Free AACR Whitepaper) Annual Meeting taking place April 5 – 10, 2024 in San Diego, CA.
Present clinical data from the ongoing Phase 1 dose escalation trial in NSCLC, CRC, melanoma and other cancers with BRAF V600X mutations in 2H 2024.
UPCOMING INVESTOR EVENTS

March 4, 2024: Management will present at TD Cowen’s 44th Annual Health Care Conference taking place March 4 – 6, 2024, at the Marriott Copley Place in Boston, MA.
March 11, 2024: Management will participate in a fireside chat at the Leerink Partners Global Biopharma Conference taking place March 11 – 13, 2024, at the Fontainebleau in Miami, FL.
FULL YEAR 2023 FINANCIAL RESULTS

Revenue: Total revenue for the year ended December 31, 2023 was $20.8 million, compared to $31.1 million for the year ended December 31, 2022. The decrease in revenue was primarily due to the collaboration agreement with Calico ending in January 2023 and completion of research activities for a target under the collaboration agreement with Biogen, partially offset by the completion of research activities for select targets under the collaboration agreement with Roche. 2023 revenue reflects amounts recognized under our collaboration agreements with Biogen, Calico, and Roche.

Research and Development (R&D) Expense: R&D expense for the year ended December 31, 2023 was $117.7 million, compared to $117.8 million for the year ended December 31, 2022. R&D expense was relatively flat year over year as preclinical costs decreased and clinical costs increased with the transition of CFT1946 to clinical development.

General and Administrative (G&A) Expense: G&A expense for the year ended December 31, 2023 was $42.1 million, compared to $42.8 million for the year ended December 31, 2022. The decrease in G&A expense was primarily attributable to a decrease in professional fees.

Net Loss and Net Loss per Share: Net loss for the year ended December 31, 2023 was $132.5 million, compared to $128.2 million for the year ended December 31, 2022. Net loss per share for the year ended December 31, 2023 was $2.67, compared to $2.62 for the year ended December 31, 2022.

Cash Position and Financial Guidance: Cash, cash equivalents and marketable securities as of December 31, 2023 were $281.7 million, compared to $337.1 million as of December 31, 2022. Cash and cash equivalents as of December 31, 2023 do not include $25 million in proceeds from the sale of shares of our common stock to a subsidiary of Betta Pharmaceuticals, $14.1 million of proceeds in connection with the settlement of 2,500,601 shares under our ATM program, and the $10.0 million upfront payment related to our collaboration agreement with Merck, all of which were received in January 2024. The company expects that its cash, cash equivalents and marketable securities as of December 31, 2023, together with these amounts received in January 2024, which result in a proforma balance of approximately $330 million, will enable the company to fund its operating plan into 2027.

On February 22, 2024 BridgeBio Pharma, Inc. (Nasdaq: BBIO) (BridgeBio or the Company), a commercial-stage biopharmaceutical company focused on genetic diseases and cancers, reported its financial results for the fourth quarter and full year ended December 31, 2023, and provided an update on the Company’s operations (Press release, BridgeBio, FEB 22, 2024, View Source [SID1234640383]).

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"Our focus this year is executing on the launch of acoramidis for patients with ATTR cardiomyopathy," said Neil Kumar, Ph.D., founder and CEO of BridgeBio. "At the same time, we are also focused on fully enrolling three ongoing Phase 3 clinical trials by the end of 2024. Finally, we hope that reading out potentially exciting data from our Phase 1/2 trial in congenital adrenal hyperplasia later this year will let us take the next step in serving that patient community."

BridgeBio’s key programs:

Acoramidis (AG10) – Transthyretin (TTR) stabilizer for transthyretin amyloid cardiomyopathy (ATTR-CM):
The Company filed an NDA for acoramidis for the treatment of ATTR-CM with the US FDA; the NDA was accepted for review with a PDUFA date of November 29, 2024. The Company has also filed a Marketing Authorization Application for acoramidis with the EMA, which has been accepted for review.
The regulatory filings were based on data from the Phase 3 ATTRibute-CM study, which met its primary endpoint (Win Ratio of 1.8) with a highly statistically significant p-value (p<0.0001). Additional results from ATTRibute-CM include:
An 81% survival rate on acoramidis, which approaches the survival rate in the age-matched U.S. database (~85%), and a 0.29 mean annual CVH rate on acoramidis, which approaches the annual hospitalization rate observed in the broader U.S. Medicare population (~0.26);
Improvements from baseline observed for a large proportion of participants treated with acoramidis on laboratory and functional measures including n-terminal prohormone of brain natriuiretic peptide (NT-proBNP) and 6-minute walk distance;
Rapid clinical benefit on the composite endpoint of ACM and CVH in participants treated with acoramidis, demonstrated by placebo and acoramidis time-to-first event Kaplan-Meier curves for a composite of ACM and CVH that separated at Month 3 and continued to diverge steadily through Month 30 as presented at the American Heart Association Scientific Sessions in November 2023; and
Acoramidis was well-tolerated with no safety signals of potential clinical concern identified.
The Company also shared positive results of an open-label, single-arm Phase 3 study conducted in Japan by licensing partner Alexion, AstraZeneca Rare Disease, including that no mortality was reported over the 30 month acoramidis treatment period.
Additional detailed results of ATTRibute-CM are planned for presentation at 2024 medical meetings.
Low-dose infigratinib – FGFR1-3 inhibitor for achondroplasia and hypochondroplasia:
In December 2023, the Company announced the dosing of the first child in PROPEL 3, its global Phase 3 registrational study of infigratinib in achondroplasia.
In February 2024, the Company announced a partnership with Kyowa Kirin wherein the Company grants Kyowa Kirin an exclusive license to develop and commercialize infigratinib for achondroplasia, hypochondroplasia, and other skeletal dysplasias in Japan; in exchange, the Company will receive an upfront payment of $100 million as well as royalties up to the high-twenties percent on sales of infigratinib in Japan, with the potential for additional milestone-based payments.
The Company is committed to exploring the potential of infigratinib on the wider medical and functional impacts of achondroplasia, hypochondroplasia and other skeletal dysplasias, and anticipates initiating its clinical program for hypochondroplasia in 2024.
BBP-418 – Glycosylation substrate for limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9):
FORTIFY, the global Phase 3 registrational trial of BBP-418, continues to enroll in the U.S. with clinical trial sites planned for Europe and Australia. Full enrollment of the interim analysis population is expected in 2024. The Company believes there is potential to pursue Accelerated Approval for BBP-418 based on recent interactions with the FDA on the use of glycosylated αDG levels as a surrogate endpoint.
Encaleret – Calcium-sensing receptor (CaSR) inhibitor for autosomal dominant hypocalcemia type 1 (ADH1):
CALIBRATE, the Phase 3 clinical trial of encaleret, continues to enroll; the Company anticipates sharing topline data from CALIBRATE in 2025.
Recent Corporate Updates:

·Secured up to $1.25 billion of capital from Blue Owl and CPP Investments: The raise includes $500 million in cash from Blue Owl and CPP Investments available upon FDA approval of acoramidis in exchange for a 5% royalty on future global net sales of acoramidis, as well as a $450 million credit facility from Blue Owl that refinanced existing senior secured credit, extending maturity from 2026 to 2029 subject to certain conditions.

Fourth Quarter and Full Year 2023 Financial Results:

Cash, Cash Equivalents, Marketable Securities and Short-term Restricted Cash

Cash, cash equivalents and short-term restricted cash, totaled $392.6 million as of December 31, 2023, compared to cash, cash equivalents, marketable securities and short-term restricted cash of $466.2 million as of December 31, 2022. The net decrease of $73.6 million in cash, cash equivalents, marketable securities and short-term restricted cash was primarily attributable to net cash used in operating activities of $527.7 million and $6.9 million in repurchase of shares to satisfy tax withholdings, primarily offset by net proceeds received of $449.8 million from various equity financings, $6.0 million from stock option exercises, and $3.4 million from common stock issuances under our employee stock purchase plan during the year ended December 31, 2023.

Revenue

Revenue for the three months and year ended December 31, 2023 were $1.7 million and $9.3 million, respectively, as compared to $1.9 million and $77.6 million for the same periods in the prior year, respectively. The net decreases of $0.2 million and $68.3 million for the three months and year ended December 31, 2023, respectively, compared to the same periods in the prior year, were primarily due to license revenue recognized in 2022 upon the transfer of the license in accordance with the Navire-BMS License Agreement which was entered into in May 2022.

Operating Costs and Expenses

Operating costs and expenses for the three months and year ended December 31, 2023 were $179.2 million and $616.7 million, respectively, compared to $131.1 million and $589.9 million, for the same periods in the prior year, respectively.

The overall increase of $48.1 million in operating costs and expenses for the three months ended December 31, 2023, compared to the same period in the prior year, was primarily due to an increase of $39.3 million in research and development and other expenses (R&D) to advance the Company’s pipeline of development programs, an increase of $15.7 million in selling, general and administrative (SG&A) expenses to support commercialization readiness efforts, offset by a decrease of $6.9 million in restructuring, impairment and related charges given that the majority of the restructuring initiatives occurred in the prior year.

The overall increase of $26.8 million in operating costs and expenses for the year ended December 31, 2023 , compared to the same period in the prior year, was primarily due to an increase of $55.2 million in R&D expenses to advance the Company’s pipeline of development programs, an increase of $7.4 million in SG&A expenses to support commercialization readiness efforts, offset by a decrease of $35.8 million in restructuring, impairment and related charges given that the majority of the restructuring initiatives occurred in the prior year.

Restructuring, impairment and related charges for the three months and year ended December 31, 2023, amounted to $0.8 million and $7.9 million, respectively. These charges primarily consisted of winding down, exit costs, and severance and employee-related costs. Restructuring, impairment and related charges for the same periods in the prior year were $7.7 million and $43.8 million, respectively. These charges primarily consisted of impairments and write-offs of long-lived assets, severance and employee-related costs, and exit and other related costs.

Stock-based compensation expenses included in operating costs and expenses for the three months ended December 31, 2023 were $37.1 million, of which $22.5 million is included in R&D expenses, and $14.6 million is included in SG&A expenses. Stock-based compensation expenses included in operating costs and expenses for the same period in the prior year were $22.6 million, of which $8.9 million is included in R&D expenses, and $13.6 million is included in SG&A expenses.

Stock-based compensation expenses included in operating costs and expenses for the year ended December 31, 2023 were $115.0 million, of which $61.6 million is included in R&D expenses, and $53.4 million is included in SG&A expenses. Stock-based compensation expenses included in operating costs and expenses for the same period in the prior year were $93.8 million, of which $38.0 million is included in R&D expenses, $54.7 million is included in SG&A expenses, and $1.2 million is included in restructuring, impairment and related charges.

"Coming off of our recent royalty financing, we find ourselves well capitalized to launch acoramidis this year alongside strong new partners who share our confidence in acoramidis’ potential in the ATTR-CM market," said Brian Stephenson, Ph.D., CFA, Chief Financial Officer of BridgeBio. "We are excited for this launch, as well as for the continued advancement of our late stage pipeline, which we hope will allow us to serve patients with genetic diseases both directly with the advancement of those medicines towards the market as well as by diversifying our top line revenue and enabling reinvestment into the R&D and business development opportunities that will allow us to be sustainable in the long term."

On February 22, 2024 Morphic Therapeutic (Nasdaq: MORF), a biopharmaceutical company developing a new generation of oral integrin therapies for the treatment of serious chronic diseases, reported corporate highlights and financial results for the full year 2023 (Filing, 3 mnth, DEC 31, Morphic Therapeutic, 2024, FEB 22, 2024, View Source [SID1234640378]).

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"Today, our conviction in MORF-057 as a potential oral, well tolerated, and efficacious treatment for inflammatory bowel disease (IBD) is stronger than ever, based on the clear success of the EMERALD-1 trial in UC. Looking forward in 2024, we will work to translate this momentum into further progress with the GARNET phase 2 study in patients with moderately to severely active Crohn’s disease running in parallel with the EMERALD-2 phase 2b study in UC," commented Praveen Tipirneni, Chief Executive Officer of Morphic. "Further, we are working to expand the pipeline of candidates generated by Morphic’s MInT Platform, most notably with our α5β1 program for pulmonary hypertensive diseases and additional new projects against both integrin and non-integrin targets
"On a personal note, I am thankful to return to Morphic after an acute medical event and grateful for the immense support from friends and peers within the biotechnology community and especially the Morphic team," Tipirneni continued. "This experience has crystallized, for me, that Morphic’s opportunity to fundamentally improve patients’ lives is immense and that there is no time to waste."
2023 and Recent Corporate Highlights
EMERALD-1 Phase 2a trial of MORF-057 in UC:
•In the EMERALD-1 phase 2a trial of MORF-057 in ulcerative colitis (UC), topline data and additional data presented at UEGW 2023 indicated that in a moderately-to-severely-active UC population with severe disease burden, MORF-057:
◦Was generally well tolerated with no safety signal observed
◦Achieved the study’s primary endpoint with statistical significance in reduction of Robarts Histopathology Index (RHI) Score from baseline to week 12 of 6.4 points (p=0.002)
◦Showed consistent clinical improvement across key measures at week 12, including modified Mayo Clinic Score (mMCS) remission of 25.7% and mMCS response of 45.7%
◦Demonstrated RHI change ≥7 points in 48.6% of patients and RHI remission in 22.9% of patients
◦Led to clinical improvement in mMCS within the 12-week induction period for 77% of patients
◦Pharmacokinetic and pharmacodynamic results confirmed the results seen in healthy volunteer studies
▪Median α4β7 RO >99% and sustained saturation at week 12
▪α4β1 inhibition below the limit of quantification, in line with the design of MORF-057
▪Predicted lymphocyte subset changes observed, consistent with engagement of α4β7
◦Demonstrated deepening of clinical effect beyond the 12-week induction period, with symptomatic remission rates continuing to increase out to 44 weeks in both advanced treatment-naïve and advanced treatment-experienced patients
•Announced completion of enrollment in the exploratory cohort of the EMERALD-1 study comprised of UC patients who have previously failed treatment with vedolizumab
•Continued the 40-week maintenance phase of the EMERALD-1 study as planned

EMERALD-2 Phase 2b trial of MORF-057 in UC:
•Continued to enroll the EMERALD-2 phase 2b study of MORF-057 in patients with moderately-to-severely active UC
◦EMERALD-2 is a global phase 2b randomized, double-blind, placebo-controlled trial of MORF-057 in patients with moderate-to-severe UC
◦The primary endpoint of EMERALD-2 is clinical remission rate as measured by mMCS at 12 weeks and is expected to report in the first half of 2025
GARNET Phase 2 trial of MORF-057 in Crohn’s Disease:
•Announced that launch activities are underway for the randomized placebo-controlled GARNET Phase 2 study of MORF-057 in CD and that the study is anticipated to enroll its first patients in the first half of 2024
◦The GARNET study will evaluate 210 patients across three cohorts, each comprising 70 patients: 70 patients receiving MORF-057 200 mg BID (twice daily), 70 patients receiving MORF-057 100 mg BID and 70 patients receiving placebo
◦The primary endpoint of the GARNET study is the proportion of participants in endoscopic response (>=50% reduction) at week 14 as determined using Simple Endoscopic Score for Crohn’s Disease (SES-CD)
MORF-057 Preclinical and Phase 1 Studies:
•Presented new biomarker data at DDW 2023, demonstrating increases in circulating plasmablasts, consistent with the increased antibody activity expected with anti-inflammatory mechanism of α4β7 inhibition, supporting MORF-057 program in UC
•Presented preclinical data on rational selection of combination therapy for IBD treatment using an established clinical mode at UEGW 2023
◦This study explored preclinical combination models in UC and preliminarily examined the potential utility and rationale of combining anti-inflammatory mechanisms with α4β7 integrin inhibition in IBD
Pipeline Programs:
•Announced α5β1 as the integrin target of Morphic’s small molecule integrin inhibitor program in pulmonary hypertensive diseases
◦The inhibition of fibronectin integrins, including α5β1, suppresses pulmonary arterial smooth muscle cell proliferation and data to date indicate that inhibition of α5β1 contributes to improved cardiac output and the reversal of vascular remodeling
•Initiated discovery efforts that expand the Company’s scope in the immunology space with oral programs targeting the IL-23 and TL1A pathways, among others
Financial Results for the Full Year 2023
•Net loss for the year ended December 31, 2023, was $152.1 million or $3.59 per share compared to a net loss of $59.0 million or $1.55 per share for the year ended December 31, 2022
•Revenue was $0.5 million for the year ended December 31, 2023, compared to $70.8 million for the year ended December 31, 2022. The change was primarily due to recognition of revenue due to the conclusion of the AbbVie collaboration in 2022 and to the amounts due at the conclusion of the Janssen collaboration in 2023
•Research and development expenses were $140.4 million for the year ended December 31, 2023, as compared to $102.1 million for the year ended December 31, 2022. The increase was primarily attributable to higher development costs along with increased clinical trial costs to support phase 2 clinical studies and development activities for MORF-057, as well as other research costs to support early development candidates
•General and administrative expenses were $38.8 million for the year ended December 31, 2023, compared to $32.1 million for the year ended December 31, 2022. The increase was due to increased personnel related costs and non-cash equity-based compensation, partially offset by decreases in consulting and insurance expenses
•Morphic raised approximately $444 million, net, through equity financings in 2023 comprised of approximately $100 million in proceeds from a PIPE offering, approximately $259 million in a public offering and approximately $85 million though use of the ATM facility
•As of December 31, 2023, Morphic had cash, cash equivalents and marketable securities of $704.3 million, compared to $348.2 million as of December 31, 2022. We believe that our cash, cash equivalents and marketable securities of $704.3 million as of December 31, 2023, will enable us to fund our operating expenses and capital expenditure requirements into the second half of 2027
About MORF-057
Morphic is developing MORF-057 as a selective, oral small molecule inhibitor of the α4β7 integrin for patients with inflammatory bowel disease (IBD). α4β7 has been clinically validated as a target for the treatment of IBD by the success of the approved injectable antibody therapeutic vedolizumab. MORF-057, like vedolizumab, is designed to block the interactions between α4β7 on the surface of lymphocytes and the mucosal endothelial cell ligand MAdCAM-1, substantially reducing lymphocyte migration from the bloodstream into intestinal mucosal tissues and avoiding inflammation that is associated with IBD.
About the EMERALD-1 Study
EMERALD-1 (MORF-057-201) is an open-label multi-center phase 2a trial designed to evaluate the efficacy, safety, and tolerability of MORF-057 in adults with moderate to severe ulcerative colitis. The primary endpoint of EMERALD-1, change in Robarts Histopathology Index (RHI) from baseline at twelve weeks, was achieved with statistical significance. RHI is a validated instrument that measures histological disease activity in ulcerative colitis. Patients were eligible to continue for an additional 40 weeks of maintenance therapy followed by a 52-week assessment as well as an open-label extension period. Secondary and additional outcome measures in the EMERALD-1 study include change in the modified Mayo clinic score, safety, pharmacokinetic parameters and key pharmacodynamic measures including α4β7 receptor occupancy and lymphocyte subset trafficking.
About the EMERALD-2 Study
EMERALD-2 (MORF-057-202) is a global phase 2b randomized, double-blind, placebo-controlled trial of MORF-057 that is currently enrolling patients with moderate-to-severe ulcerative colitis. The primary endpoint of EMERALD-2 is clinical remission rate as measured by the Modified Mayo Clinic Score (mMCS) at 12 weeks. EMERALD-2 will also measure several secondary and exploratory endpoints based on the mMCS as well as histologic, pharmacokinetic and pharmacodynamic measures, and safety parameters. Patients in the EMERALD-2 study will be randomized to receive either 200 mg BID (twice daily) MORF-057, 100 mg BID MORF-057, a QD (once daily) dose of MORF-057, or a placebo dose. Following the 12-week induction phase, all patients will receive MORF-057 for 40 weeks of maintenance dosing. For more information about the EMERALD clinical trials of MORF-057, please click here.
About the GARNET Study
GARNET (MORF-057-203) is a global Phase 2b randomized, double-blind, placebo-controlled trial of MORF-057 in Crohn’s disease. The primary endpoint of GARNET is the proportion of participants in endoscopic response (>=50% reduction) at week 14 as determined using Simple Endoscopic Score for Crohn’s Disease, or SES-CD. The secondary endpoints will include the change in Crohn’s Disease Activity Index, or CDAI, measures, as well as safety parameters. Patients enrolled in the GARNET study will be randomized to receive one of two active doses or a placebo: 200 mg BID (twice daily), 100 mg BID or a placebo that will cross over to MORF-057 after the 14-week induction phase. Following the 14-week induction phase, patients will move to a 38-week maintenance phase.

On February 22, 2024 Relay Therapeutics, Inc. (Nasdaq: RLAY), a clinical-stage precision medicine company transforming the drug discovery process by combining leading-edge computational and experimental technologies, reported fourth quarter and full year 2023 financial results and corporate highlights (Filing, 3 mnth, DEC 31, Relay Therapeutics, 2024, FEB 22, 2024, View Source [SID1234640377]).

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"We made important progress across our portfolio during 2023, advancing multiple clinical programs and continuing to invest significantly in our research engine – the Dynamo platform," said Sanjiv Patel, M.D., President and Chief Executive Officer of Relay Therapeutics. "We are very pleased with the RLY-2608 data disclosed to-date and how its clinical profile continued to mature throughout last year. Our clinical team is focused on advancing this program in the near term in both a doublet and a triplet combination, and we look forward to sharing additional data in the second half of 2024. Our Dynamo platform continues to demonstrate precision and productivity with each target we’ve chosen to-date, and we are excited to disclose at least one new program that has come out of it later this year, which is being designed to have first-in-class potential."

Recent Corporate Highlights

PI3Kα

•
RLY-2608 doublet
o
Completed enrollment in initial dose expansion cohort of RLY-2608 600mg BID + fulvestrant in patients with PI3Kα-mutant, HR+, HER2- locally advanced or metastatic breast cancer
o
Initiated two additional dose expansion cohorts of RLY-2608 in combination with fulvestrant – a second 600mg BID cohort as well as one at 400mg BID
o
Published RLY-2608 preclinical profile and clinical proof-of-concept in Cancer Discovery (Discovery and Clinical Proof-of-Concept of RLY-2608, a First-in-Class Mutant-Selective Allosteric PI3Kα Inhibitor That Decouples Antitumor Activity from Hyperinsulinemia) with vignettes from two patients with advanced HR+ breast cancer with kinase or helical mutations, with no observed wildtype PI3Kα-related toxicities
•
RLY-2608 triplet

o
Initiated RLY-2608 + fulvestrant + ribociclib triplet combination in patients with PI3Kα-mutant, HR+, HER2- locally advanced or metastatic breast cancer
•
RLY-5836
o
Deprioritized further clinical development
Lirafugratinib (RLY-4008)

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Presented initial clinical data in patients with FGFR2-altered solid tumors at the 2023 AACR (Free AACR Whitepaper)-NCI-EORTC AACR-NCI-EORTC (Free AACR-NCI-EORTC Whitepaper) International Conference on Molecular Targets and Cancer Therapeutics (EORTC-NCI-AACR) (Free ASGCT Whitepaper) (Free EORTC-NCI-AACR Whitepaper)
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As previously disclosed, the company will minimize resource allocation in 2024 to allow data to mature and inform future clinical development decisions
Anticipated 2024 Milestones

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RLY-2608
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RLY-2608 + fulvestrant data update in the second half of 2024
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RLY-2608 + fulvestrant + ribociclib initial safety data in the second half of 2024
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Lirafugratinib: tumor agnostic data and regulatory update in the second half of 2024
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Pre-clinical: disclose new program(s) in 2024

Fourth Quarter and Full Year 2023 Financial Results

Cash, Cash Equivalents and Investments: As of December 31, 2023, cash, cash equivalents and investments totaled $750.1 million compared to approximately $1 billion as of December 31, 2022. The company expects its current cash, cash equivalents and investments will be sufficient to fund its current operating plan into the second half of 2026.

Revenue: There was no material revenue for the fourth quarter of 2023 or 2022. Revenue was $25.5 million for the full year 2023, as compared to $1.4 million for the full year 2022. The increase was primarily due to the recognition of previously received milestone payments under the company’s Collaboration and License Agreement with Genentech, Inc.

R&D Expenses: Research and development expenses were $77.5 million for the fourth quarter of 2023, as compared to $67.3 million for the fourth quarter of 2022. The increase was primarily due to additional clinical trial expenses and employee-related costs, which were offset by a decrease in other external research costs. Research and development expenses were $330.0 million for the full year 2023, as compared to $246.4 million for the full year 2022. The increase was primarily due to $50.0 million of additional external costs in connection with our clinical trials and $32.4 million of additional employee costs from increased headcount in our research and development functions, which includes $17.7 million of additional stock compensation expense.

G&A Expenses: General and administrative expenses were $16.8 million for the fourth quarter of 2023, as compared to $16.4 million for the fourth quarter of 2022. The increase was primarily due to additional stock compensation expense. General and administrative expenses were $75.0 million for the full year 2023, as compared to $66.0 million for the full year 2022. The increase was primarily due additional stock compensation expense.

Net Loss: Net loss was $83.5 million for the fourth quarter of 2023, or a net loss per share of $0.67, as compared to a net loss of $67.5 million for the fourth quarter of 2022, or a net loss per share of $0.56.

Net loss was $342.0 million for the full year 2023, or a net loss per share of $2.79, as compared to a net loss of $290.5 million for the full year 2022, or a net loss per share of $2.59.

On February 22, 2024 Veracyte, Inc. (Nasdaq: VCYT) reported financial results for the fourth quarter and full year ended December 31, 2023 (Press release, Veracyte, FEB 22, 2024, View Source [SID1234640376]).

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"We closed 2023 with another quarter of excellent results, driven by our Afirma and Decipher businesses," said Marc Stapley, Veracyte’s chief executive officer. "Looking to 2024 and beyond, we will leverage our Veracyte Diagnostics Platform to continue to drive near- and long-term revenue. With multiple growth catalysts over the coming years and with our strong financial discipline, we expect to achieve positive cash flow for the third consecutive year and going forward."

Key Business Highlights

Grew total test volume to 33,836 in the fourth quarter and 126,977 for the full year 2023, an increase of 21% and 24%, respectively, compared to the prior year periods.
Enhanced our Endocrinology offering in 2023 with the launch of TERT promoter mutation testing for Afirma, the introduction of our Afirma GRID research tool, and enhancements to our online physician ordering portal.
Continued to strengthen the body of evidence for Decipher Prostate in 2023, adding 8 publications and 16 abstracts to the clinical-evidence library, reinforcing Decipher’s status as the only molecular test to receive Level 1 evidence designation in the National Comprehensive Cancer Network’s (NCCN) prostate cancer guidelines.
Acquired C2i Genomics Inc., adding whole-genome minimal residual disease (MRD) capabilities to our novel Veracyte Diagnostics Platform and expanding our ability to serve patients across the cancer care continuum.
Signed an agreement with Illumina to develop some of our tests as in vitro diagnostics (IVDs) for use on their NextSeq 550Dx next-generation sequencing (NGS) instrument, as part of our multi-platform strategy to accelerate global expansion of our tests as IVDs.
Published the clinical validation study for our Percepta Nasal Swab test in CHEST.
For the full year, generated $44 million of cash from operations and ended the year with $216 million of cash and cash equivalents.
Fourth Quarter 2023 Financial Results

Total revenue for the fourth quarter of 2023 was $98.2 million, an increase of 22% compared to $80.3 million reported in the fourth quarter of 2022. Testing revenue was $90.4 million, an increase of 29% compared to $70.3 million in the fourth quarter of 2022, driven primarily by the strong performance of our Decipher Prostate and Afirma tests. Product revenue was $3.7 million, an increase of 13% compared to $3.2 million in the fourth quarter of 2022. Biopharmaceutical and other revenue was $4.1 million, a decrease of 39% compared to $6.8 million in the fourth quarter of 2022.

Total gross margin for the fourth quarter of 2023, including the amortization of acquired intangible assets, was 66%, compared to 61% in the fourth quarter of 2022. Non-GAAP gross margin, excluding the amortization of acquired intangible assets and other acquisition related expenses was 70%, compared to 67% in the fourth quarter of 2022.

Operating expenses, excluding cost of revenue, were $100.3 million, which included an impairment charge of $32.0 million associated with the impairment of HalioDx developed biopharmaceutical services technology, customer relationships and customer backlog finite-lived intangible assets, compared to $54.1 million in the fourth quarter of 2022. Non-GAAP operating expenses, which excludes cost of revenue, amortization of acquired intangible assets, impairment charges, other acquisition related expenses, and other restructuring costs, grew 28% to $65.6 million, including a technology access fee of $3.5 million to develop our IVD kitted tests on an NGS platform, compared to $51.1 million in the fourth quarter of 2022.

Net loss for the fourth quarter of 2023 was $28.3 million, and basic and diluted net loss per common share was $0.39, which includes the aforementioned $32.0 million impairment charge. Net cash provided by operating activities in the fourth quarter of 2023 was $15.6 million, an improvement of $5.8 million compared to the same period in 2022.

A reconciliation of GAAP to non-GAAP financial measures has been provided in the tables included in this press release. An explanation of these measures is also included below under the heading "Note Regarding Use of Non-GAAP Financial Measures."

Full Year 2023 Financial Results

Total revenue for 2023 was $361.1 million, an increase of 22% compared to $296.5 million in 2022. Testing revenue was $326.5 million, an increase of 30% compared to $250.5 million in 2022, driven primarily by the strong performance of our Decipher and Afirma tests. Product revenue was $15.6 million, an increase of 23% compared to $12.6 million in 2022. Biopharmaceutical and other revenue was $18.9 million, a decrease of 43% compared to $33.4 million in 2022, driven by the reduction of customer projects given overall spending constraints across the industry.

Total gross margin for the full year 2023, including the amortization of acquired intangible assets, was 64%, compared to 59% in 2022. Non-GAAP gross margin, excluding the amortization of acquired intangible assets and other acquisition related expenses was 69%, compared to 66% in 2022.

Operating expenses, excluding cost of revenue, were $315.5 million, an increase of 46% compared to $216.8 million in 2022. Non-GAAP operating expenses, which excludes cost of revenue, amortization of acquired intangible assets, impairment charges, other acquisition related expenses and other restructuring costs, grew 20% to $240.7 million compared to $200.3 million in 2022.

Net loss for the full year 2023 was $74.4 million and basic and diluted net loss per common share was $1.02, which includes impairment charges of $68.3 million in the year. Net cash provided by operating activities in 2023 was $44.2 million, an increase of $36.7 million compared to 2022.

A reconciliation of GAAP to non-GAAP financial measures has been provided in the tables included in this press release. An explanation of these measures is also included below under the heading "Note Regarding Use of Non-GAAP Financial Measures."

2024 Financial Outlook

The company is maintaining full-year 2024 total revenue guidance of $394 million to $402 million and, consistent with prior guidance, expects cash, cash equivalents and short-term investments at the end of the year to be $230 million to $234 million.

Conference Call and Webcast Details

Veracyte will host a conference call and webcast today at 4:30 p.m. Eastern Time to discuss the company’s financial results and provide a general business update. The conference call will be webcast live from the company’s website and will be available via the following link: View Source The webcast should be accessed 10 minutes prior to the conference call start time. A replay of the webcast will be available for one year following the conclusion of the live broadcast and will be accessible on the company’s website at View Source

The conference call dial-in can be accessed by registering at the following link: https://register.vevent.com/register/BI38de228ea0a04e0ab786cfe56be5ca1b