TG Therapeutics to Participate in the 42nd Annual J.P. Morgan Healthcare Conference

On January 5, 2024 TG Therapeutics, Inc. (NASDAQ: TGTX), reported that Michael S. Weiss, the Company’s Chairman and Chief Executive Officer, will present at the 42nd Annual J.P. Morgan Healthcare Conference. The presentation is scheduled to take place on Tuesday, January 9, 2024, at 4:30 PM PT (Press release, TG Therapeutics, JAN 5, 2024, View Source [SID1234639022]).

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A live webcast of this presentation will be available on the Events page, located within the Investors & Media section, of the Company’s website at View Source A replay of the webcast will be available on TG’s website following the event.

Curis Reports Inducement Grants Under NASDAQ Listing Rule 5635(c)(4)

On January 5, 2024 Curis, Inc. (NASDAQ: CRIS), a biotechnology company focused on the development of innovative therapeutics for the treatment of cancer, reported that on January 2, 2024, the independent Compensation Committee of the Board of Directors of Curis approved the grant of inducement stock options to purchase a total of 5,800 shares of Curis common stock to a new employee, with a grant date of January 2, 2024 (the "Q1 2024 Inducement Grant") (Press release, Curis, JAN 5, 2024, View Source [SID1234639006]).

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The Q1 2024 Inducement Grant has an exercise price per share equal to the closing price of the Company’s common stock on January 2, 2024. The stock option has a 10 year term and vests over four years, with 25% of the original number of shares underlying the award vesting on the first anniversary of the employee’s date of hire and an additional 6.25% of the original number of shares underlying the award vesting on each successive three-month period thereafter, subject to the employee’s continued service with the Company through the respective vesting dates. The stock option was granted as an inducement equity award outside of the Company’s Fourth Amended and Restated 2010 Stock Incentive Plan and was made as an inducement material to the employee’s acceptance of employment with the Company.

Curing blood cancers through genome and cell engineering

On January 5, 2024 Vor Biopharma presented its corporate presentation (Presentation, Vor BioPharma, JAN 5, 2024, View Source [SID1234639023]).

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Deciphera Pharmaceuticals Announces Nature Medicine Publication of Results from Exploratory ctDNA Analysis from INTRIGUE Phase 3 Study Demonstrating Substantial Clinical Benefit of QINLOCK® in 2L GIST Patients with Mutations in KIT Exon 11 and 17/18

On January 5, 2024 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 Nature Medicine has published results from a circulating tumor DNA (ctDNA) analysis of the INTRIGUE Phase 3 study of QINLOCK (ripretinib) in GIST patients with mutations in KIT exon 11 and 17/18 only previously treated with imatinib (Press release, Deciphera Pharmaceuticals, JAN 5, 2024, View Source [SID1234639007]).

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The article, titled "Ripretinib versus sunitinib in gastrointestinal stromal tumor: ctDNA biomarker analysis of the phase 3 INTRIGUE trial" is now available online and will be published in a future print issue of Nature Medicine.

"The results published in Nature Medicine provide compelling evidence that QINLOCK may provide progression-free and overall survival benefit to second-line (2L) GIST patients in whom a liquid biopsy reveals primary KIT exon 11 mutations plus secondary mutations restricted to KIT exons 17 and 18. It is the first test that measures heterogeneity of resistance and may allow for a more optimized and targeted treatment plan for people living with this disease," said Sebastian Bauer, M.D., Medical Oncologist at the West German Cancer Center in Essen and senior author of the manuscript. "This analysis is leading us to consider a new approach in GIST treatment using sensitive and minimally invasive blood tests to identify the specific secondary mutational profile for individual patients in order to tailor their therapy based on the differential activity of QINLOCK and sunitinib seen in the INTRIGUE subgroup analysis."

"In second-line GIST patients with KIT exon 11 + 17/18 mutations only, treatment with QINLOCK resulted in a 78% reduction in the risk of disease progression and a 66% reduction in the risk of death compared to sunitinib, representing a substantial clinical benefit for these patients," said Matthew L. Sherman, M.D., Chief Medical Officer of Deciphera. "Our ongoing INSIGHT pivotal Phase 3 study is designed to confirm the exceptional efficacy we observed in this exploratory analysis from INTRIGUE. The INSIGHT study is now open at multiple sites and we are committed to enrolling the study as quickly as possible."

INTRIGUE is an international, multi-center study conducted in 122 active sites across 22 countries, where 453 patients in the all patient intent-to-treat population (AP-ITT) with second-line GIST were randomized to receive ripretinib (n=226) or sunitinib (n=227).

In the AP-ITT population, QINLOCK demonstrated similar efficacy with a median progression-free survival (PFS) of 8.0 months versus 8.3 months for sunitinib (HR 1.05, nominal p=0.72). There were fewer patients with Grade 3-4 drug-related treatment emergent adverse events (TEAE) with QINLOCK (26.5%) compared with sunitinib (55.2%). Based on the primary results from the INTRIGUE study, QINLOCK was included in the National Comprehensive Cancer Network Clinical Practice Guidelines in Oncology (version 1.2023) as the preferred second-line regimen for patients with advanced GIST who are intolerant to sunitinib.

A prespecified exploratory objective in INTRIGUE was to evaluate anti-tumor efficacy of QINLOCK according to baseline KIT primary and secondary mutation status. Baseline peripheral whole blood was analyzed by Guardant360, a 74-gene ctDNA next-generation sequencing liquid biopsy assay in patients for whom evaluable samples were available (n=362) out of whom 280 patients had detectable ctDNA. In patients with a detectable KIT exon 11 primary mutation (n=157), 52 patients also had mutations in KIT exon 17/18 only and 41 had mutations in KIT exon 13/14 only.

Patients with mutations in KIT exon 11 and 17/18 only had improved progression-free survival (PFS), objective response rate (ORR), and overall survival (OS) with QINLOCK versus sunitinib while patients with mutations in KIT exon 11 and 13/14 only had improved PFS, ORR, and OS with sunitinib compared to QINLOCK.

Summary of INTRIGUE Efficacy Results of ctDNA Analysis for Patients with Mutations in KIT Exon 11 and 17/18 Only

Ripretinib

(n=27)

Sunitinib

(n=25)

Hazard
Ratio/Response
Difference
(95% CI)

Median Progression-Free Survival (1)

14.2 months

1.5 months

0.22 (0.11, 0.44), nominal p value <0.0001

Objective Response Rate (1)

44.4%

0%

44.4% (23.0%, 62.7%)

nominal p value = 0.0001

Overall Survival (2)

Not Estimable

17.5 months

0.34 (0.15, 0.76), nominal p value = 0.0061

Notes: (1) Data cutoff as of September 1, 2021; (2) Data cutoff as of September 1, 2022.

The subgroup safety profile was consistent with the primary analysis in the AP-ITT population and demonstrated a more favorable safety profile for QINLOCK compared with sunitinib with fewer patients experiencing Grade 3-4 drug-related TEAEs (KIT exon 11 and 17/18 only: 33.3% for QINLOCK versus 50.0% for sunitinib).

About the INSIGHT Study

The INSIGHT Phase 3 clinical study is a randomized, global, multicenter, open-label study to evaluate the efficacy and safety of QINLOCK compared to sunitinib in patients with GIST previously treated with imatinib with mutations in KIT exon 11 and 17/18 only (excluding patients with mutations in KIT exons 9, 13, or 14). In the study, 54 patients will be randomized 2:1 to either QINLOCK 150 mg once daily or sunitinib 50 mg once daily for four weeks followed by two weeks without sunitinib. The primary endpoint is PFS as determined by independent radiologic review using modified RECIST 1.1 criteria. Secondary endpoints include ORR as determined by independent radiologic review using modified RECIST 1.1 criteria and OS.

About the INTRIGUE Study

The INTRIGUE Phase 3 clinical study is a randomized, global, multicenter, open-label study to evaluate the efficacy and safety of QINLOCK compared to sunitinib in patients with GIST previously treated with imatinib. In the study, 453 patients were randomized 1:1 to either QINLOCK 150 mg once daily or sunitinib 50 mg once daily for four weeks followed by two weeks without sunitinib. As previously reported, the study did not achieve the primary efficacy endpoint of PFS as determined by independent radiologic review using modified RECIST 1.1 criteria. The statistical analysis plan included a hierarchical testing sequence that included testing patients with a KIT exon 11 primary mutation and then in the all patient intent-to-treat (AP-ITT) population. In patients with a KIT exon 11 primary mutation (n=327), QINLOCK demonstrated a median PFS of 8.3 months compared to 7.0 months for the sunitinib arm (HR 0.88, p=0.360). Although not formally tested due to the rules of the hierarchical testing sequence, in the AP-ITT population QINLOCK demonstrated a median PFS of 8.0 months compared to 8.3 months for the sunitinib arm (HR 1.05, nominal p=0.72). QINLOCK was generally well tolerated. Fewer patients in the QINLOCK arm experienced Grade 3-4 treatment-emergent adverse events compared to sunitinib (41.3% vs. 65.6%). Similarly, there were fewer patients with Grade 3-4 drug-related TEAEs with ripretinib (26.5%) compared with sunitinib (55.2%).

BioCryst Announces Preliminary Full Year 2023 ORLADEYO® (berotralstat) Net Revenue of $325 Million, Provides 2024 Guidance and Accelerated Path to Profitability

On January 5, 2024 BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) reported preliminary, unaudited ORLADEYO (berotralstat) net revenue for the fourth quarter and full year 2023 (Press release, BioCryst Pharmaceuticals, JAN 5, 2024, View Source [SID1234639024]). The company also provided guidance for full year 2024 ORLADEYO net revenue, full year 2024 operating expenses, expected peak ORLADEYO sales and an accelerated path to profitability.

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"After three years on the market, ORLADEYO continues on a steady growth trajectory to achieve $1 billion at peak. This commercial success, alongside our proven discovery platform that is producing additional first-in-class or best-in-class molecules, uniquely positions BioCryst to achieve financial independence from the capital markets and accelerate our path to profitability," said Jon Stonehouse, president and chief executive officer of BioCryst.

The company also announced that, if its ongoing proof-of-concept trial produces best-in-class data, it plans to out-license late-stage development and commercialization of BCX10013, its potential once-daily, oral Factor D inhibitor, to a partner that can drive the speed and breadth of investment required to accelerate BCX10013 for patients across multiple complement-mediated diseases and maximize the commercial potential of the program. As a result, the company has reduced the size of its R&D organization and accelerated its timeline to profitability.

Preliminary Fourth Quarter and Full Year 2023 ORLADEYO Revenue and 2024 ORLADEYO Outlook
Preliminary, unaudited ORLADEYO net revenue in the fourth quarter of 2023 was $89.9 million (+27 percent y-o-y). Preliminary, unaudited ORLADEYO net revenue for full year 2023 was $325 million (+29 percent y-o-y).

The company expects full year 2024 global net ORLADEYO revenue to be between $380 million and $400 million. The general pattern of revenue throughout 2024 is expected to be similar to past years, with the seasonal impact of prescription reauthorizations and the potential impact of the Inflation Reduction Act in the first quarter driving a quarter-over-quarter revenue decline in the first quarter, followed by a strong return to growth in the second quarter.

"ORLADEYO growth remained strong in the fourth quarter of 2023 as hereditary angioedema patients gain the excellent attack control they expect. Comparing U.S. patient trends year over year (y-o-y), we had more new patient prescriptions and a lower average rate of monthly discontinuations in 2023 compared to 2022. U.S. performance combined with continued global expansion keep ORLADEYO on track for $1 billion in peak sales," said Charlie Gayer, chief commercial officer of BioCryst.

Operating Expense and Profitability Outlook
The company expects full year 2024 operating expenses to be between $365 million and $375 million, flat to expected full year 2023 operating expenses. The company now expects that R&D expenses in 2024 will be reduced by $20 million versus 2023. This represents a $45 million to $55 million reduction from the 2024 R&D expense guidance it provided at its R&D Day in November 2023, and reflects both the R&D restructuring and the postponement of previously planned capital expenditures at its Discovery Center in Alabama. SG&A expenses are expected to increase by $20 million in 2024, primarily to support the continued U.S. and global growth of ORLADEYO to $1 billion in peak sales.

This operating expense outlook does not reflect non-cash stock compensation expense, or one-time expenses related to the reduction of 59 jobs (10 percent of total organization) in the first quarter of 2024.

Based on the company’s disciplined approach to capital allocation, and the revenue expected from ORLADEYO, the company expects to achieve a full-year operating profit in 2024 (not including non-cash stock compensation), be approaching quarterly positive earnings per share (EPS) and positive cash flow in the second half of 2025 (not including non-cash stock compensation), and be profitable on an EPS basis, with positive cash flow, for full year 2026. The company expects it can achieve these financial milestones without raising additional funds and does not intend to draw the additional $150 million of debt available to it from Pharmakon.

Presentation Tuesday at 42nd Annual J.P. Morgan Healthcare Conference
On Tuesday, January 9, 2024 at 6:00 p.m. ET, the company will present at the 42nd Annual J.P. Morgan Healthcare Conference in San Francisco. Links to a live audio webcast and replay of the presentation may be accessed in the Investors section of BioCryst’s website at https://www.biocryst.com/.