Encaleret sulfate

- Submitted New Drug Application (NDA) to US Food and Drug Administration (FDA) for acoramidis for the treatment of transthyretin amyloid cardiomyopathy (ATTR-CM) based on positive results of Phase 3 ATTRibute-CM trial, which were published in the New England Journal of Medicine; NDA has been accepted for review with a PDUFA date of November 29, 2024; Marketing Authorization Application (MAA) for acoramidis has also been accepted by the European Medicines Agency (EMA) - Presented additional data from ATTRibute-CM at the American Heart Association Scientific Sessions, demonstrating separation at Month 3 of the placebo and acoramidis time-to-first-event Kaplan-Meier curves for a composite of all-cause mortality (ACM) and cardiovascular-related hospitalization (CVH); separation was sustained through Month 30 and represents the most rapid clinical benefit on the composite endpoint of ACM and CVH in ATTR-CM patients to the Company’s knowledge - Shared positive results of single-arm Phase 3 study of acoramidis in Japanese ATTR-CM patients, including no mortality reported over the 30 month acoramidis treatment period - PROPEL 3, the Company's Phase 3 study of infigratinib for achondroplasia continues to enroll with full enrollment expected in 2024; the Company has also announced a partnership granting Kyowa Kirin exclusive license on infigratinib for skeletal dysplasias in Japan in exchange for an upfront payment of $100 million, royalties up to the high twenties percent, and additional milestone-based payments - FORTIFY, the Company's Phase 3 study BBP-418 for limb-girdle muscular dystrophy type 2I (LMGD2I), continues to enroll, with full enrollment of interim analysis population expected in 2024 - CALIBRATE, the Company's Phase 3 study of encaleret for autosomal dominant hypocalcemia type 1 (ADH1) continues to enroll, with full enrollment expected in 2024 and topline data expected in 2025 - Secured up to $1.25 billion of capital from Blue Owl and CPP investments, including $500 million in cash in exchange for a 5% royalty on future global net sales of acoramidis and a $450 million credit facility from Blue Owl that refinanced existing senior secured credit, extending maturity from 2026 to 2029 subject to certain conditions - Ended the quarter with $393 million in cash, cash equivalents, and short-term restricted cash, and $59 million of investments in equity securities PALO ALTO, Calif., Feb. 22, 2024 (GLOBE NEWSWIRE) -- BridgeBio Pharma, Inc. (Nasdaq: BBIO) (BridgeBio or the Company), a commercial-stage biopharmaceutical company focused on genetic diseases and cancers, today reported its financial results for the fourth quarter and full year ended December 31, 2023, and provided an update on the Company’s operations. “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; andAcoramidis 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.” BRIDGEBIO PHARMA, INC.Condensed Consolidated Statements of Operations(in thousands, except shares and per share amounts) Three Months Ended December 31, Year Ended December 31, 2023 2022 2023 2022 (Unaudited) (Unaudited) (1) Revenue $1,745 $1,870 $9,303 $77,648 Operating costs and expenses: Research, development and other expenses 130,824 91,549 458,157 402,896 Selling, general and administrative 47,583 31,862 150,590 143,189 Restructuring, impairment and related charges 754 7,691 7,926 43,765 Total operating costs and expenses 179,161 131,102 616,673 589,850 Loss from operations (177,416) (129,232) (607,370) (512,202)Other income (expense), net: Interest income 5,578 4,092 18,038 7,542 Interest expense (20,268) (19,990) (81,289) (80,438)Gain from sale of priority review voucher, net — — — 107,946 Other income (expense), net 21,778 4,560 17,370 (7,500)Total other income (expense), net 7,088 (11,338) (45,881) 27,550 Net loss (170,328) (140,570) (653,251) (484,652)Net loss attributable to redeemable convertible noncontrolling interests and noncontrolling interests 2,180 2,979 10,049 3,469 Net loss attributable to common stockholders of BridgeBio $(168,148) $(137,591) $(643,202) $(481,183)Net loss per share, basic and diluted $(0.96) $(0.92) $(3.95) $(3.26)Weighted-average shares used in computing net loss per share, basic and diluted 174,462,332 149,344,380 162,791,511 147,473,076 Three Months Ended December 31, Year Ended December 31, Stock-based Compensation 2023 2022 2023 2022 (Unaudited) (Unaudited) (1) Research, development and others $22,495 $8,941 $61,647 $37,987 Selling, general and administrative 14,638 13,643 53,369 54,669 Restructuring, impairment and related charges — — — 1,172 Total stock-based compensation $37,133 $22,584 $115,016 $93,828 (1)The condensed consolidated financial statements as of and for the year ended December 31, 2022 are derived from the audited consolidated financial statements as of that date. BRIDGEBIO PHARMA, INC.Condensed Consolidated Balance Sheets(In thousands) December 31, December 31, 2023 2022 (Unaudited) (1) Assets Cash, cash equivalents and marketable securities $375,935 $428,269 Investment in equity securities 58,949 43,653 Receivable from licensing and collaboration agreements 1,751 17,079 Short-term restricted cash 16,653 37,930 Prepaid expenses and other current assets 24,305 21,922 Property and equipment, net 11,816 14,569 Operating lease right-of-use assets 8,027 10,678 Intangible assets, net 26,319 28,712 Other assets 22,625 20,224 Total assets $546,380 $623,036 Liabilities, Redeemable Convertible Noncontrolling Interests and Stockholders’ Deficit Accounts payable $10,655 $11,558 Accrued and other liabilities 129,061 106,195 Operating lease liabilities 13,109 15,949 2029 Notes, net 736,905 734,988 2027 Notes, net 543,379 541,634 Term loan, net 446,445 430,993 Other long-term liabilities 9,361 26,643 Redeemable convertible noncontrolling interests 478 (1,589)Total BridgeBio stockholders' deficit (1,354,257) (1,254,617)Noncontrolling interests 11,244 11,282 Total liabilities, redeemable convertible noncontrolling interests and stockholders’ deficit $546,380 $623,036 (1)The condensed consolidated financial statements as of and for the year ended December 31, 2022 are derived from the audited consolidated financial statements as of that date. BRIDGEBIO PHARMA, INC.Condensed Consolidated Statements of Cash Flows(In thousands) Year Ended December 31, 2023 2022 (Unaudited) (1 ) Operating activities: Net loss $(653,251) $(484,652)Adjustments to reconcile net loss to net cash used in operating activities: Stock-based compensation 108,710 91,559 Depreciation and amortization 6,494 6,771 Noncash lease expense 4,032 5,172 Accrual of payment-in-kind interest on term loan 10,207 13,562 Loss on deconsolidation of PellePharm 1,241 — (Gain) loss from investment in equity securities, net (18,314) 8,222 Fair value of shares issued under a license agreement — 4,567 Accretion of debt 8,907 8,570 Fair value adjustment of warrants (984) 1,571 Loss on sale of certain assets — 6,261 Impairment of long-lived assets — 12,720 Gain from sale of priority review voucher, excluding transaction costs — (110,000)Gain from recognition of receivable from licensing and collaboration agreement — (12,500)Other noncash adjustments 181 604 Changes in operating assets and liabilities: Receivable from licensing and collaboration agreements 15,328 15,169 Prepaid expenses and other current assets (2,702) 7,671 Other assets (1,546) 10,971 Accounts payable 2,780 (349)Accrued compensation and benefits 7,802 (2,362)Accrued research and development liabilities (9,855) (4,309)Operating lease liabilities (4,829) (6,245)Deferred revenue (5,438) 15,262 Accrued professional and other liabilities 3,517 (7,729)Net cash used in operating activities (527,720) (419,494)Investing activities: Purchases of marketable securities (29,726) (137,493)Maturities of marketable securities 82,550 479,688 Purchases of investment in equity securities (107,538) (55,562)Sales of investment in equity securities 110,556 52,835 Decrease in cash and cash equivalents resulting from deconsolidation of PellePharm (503) — Payment for intangible asset — (1,500)Proceeds from sale of priority review voucher — 110,000 Proceeds from sale of certain assets — 10,000 Purchases of property and equipment (1,306) (4,821)Net cash provided by investing activities 54,033 453,147 Financing activities: Proceeds from issuance of common stock through Private Placement offering, net 240,796 — Proceeds from issuance of common stock through Follow-on offering, net 144,049 — Proceeds from issuance of common stock through ATM offering, net 64,965 4,852 Transactions with noncontrolling interests (801) — Repayment of term loan — (20,486)Proceeds from BridgeBio common stock issuances under ESPP 3,398 2,558 Repurchase of RSU shares to satisfy tax withholding (6,880) (1,561)Proceeds from stock option exercises, net of repurchases 6,008 666 Other financing activities — 837 Net cash provided by (used in) financing activities 451,535 (13,134)Net increase (decrease) in cash, cash equivalents and restricted cash (22,152) 20,519 Cash, cash equivalents and restricted cash at beginning of period 416,884 396,365 Cash, cash equivalents and restricted cash at end of period $394,732 $416,884 Year Ended December 31, 2023 2022 (Unaudited) (1) Supplemental Disclosure of Cash Flow Information: Cash paid for interest $61,108 $54,443 Supplemental Disclosures of Noncash Investing and Financing Information: Unpaid property and equipment $100 $47 Recognized intangible asset recorded in “Other accrued and other long-term liabilities” $— $11,000 Transfers (to) from noncontrolling interests $(10,534) $(3,512)Payment-in-kind interest added to principal of term loan $— $1,763 Reconciliation of Cash, Cash Equivalents and Restricted Cash: Cash and cash equivalents $375,935 $376,689 Short-term restricted cash 16,653 37,930 Restricted cash — Included in “Other assets” 2,144 2,265 Total cash, cash equivalents and restricted cash at end of periods $394,732 $416,884 (1)The condensed consolidated financial statements as of and for the year ended December 31, 2022 are derived from the audited consolidated financial statements as of that date. About BridgeBio Pharma, Inc.BridgeBio Pharma, Inc. (BridgeBio) is a commercial-stage biopharmaceutical company founded to discover, create, test, and deliver transformative medicines to treat patients who suffer from genetic diseases and cancers with clear genetic drivers. BridgeBio’s pipeline of development programs ranges from early science to advanced clinical trials. BridgeBio was founded in 2015 and its team of experienced drug discoverers, developers and innovators are committed to applying advances in genetic medicine to help patients as quickly as possible. For more information visit bridgebio.com and follow us on LinkedIn and Twitter. BridgeBio Pharma, Inc. Forward-Looking Statements This press release contains forward-looking statements. Statements in this press release may include statements that are not historical facts and are considered forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended (the Securities Act), and Section 21E of the Securities Exchange Act of 1934, as amended (the Exchange Act), which are usually identified by the use of words such as “anticipates,” “believes,” “continues,” “estimates,” “expects,” “hopes,” “intends,” “may,” “plans,” “projects,” “remains,” “seeks,” “should,” “will,” and variations of such words or similar expressions. We intend these forward-looking statements to be covered by the safe harbor provisions for forward-looking statements contained in Section 27A of the Securities Act and Section 21E of the Exchange Act. These forward-looking statements, including statements relating to the clinical and therapeutic, market potential of our programs and product candidates, including the statements in Dr. Kumar’s and Dr. Stephenson's quotes regarding the potential commercial launch of acoramidis (if approved), continued advancement in our pipeline, including enrollments in clinical trials and anticipated readout, and other benefits resulting from recent financing; the statements related to the FDA's planned actions regarding our NDA for acoramidis for the treatment of ATTR-CM; the potential outcomes of regulatory reviews by the FDA and the EMA; the timing and success of our clinical development programs, including the progress of our clinical development program for acoramidis for patients with ATTR-CM, and our plan for, and the expected timing of, presenting additional detailed results of ATTRibute-CM study at medical meetings; the potential success of our partnership granting Kyowa Kirin an exclusive license on infigratinib for skeletal dysplasias in Japan and the potential payments we may receive under the license; the continuation of PROPEL 3, our Phase 3 study of infigratinib for achondroplasia and the expected timing for full enrollment in the study; our commitment to exploring the potential of infigratinib and the expectation and timing of the initiation of our clinical program for hypochondroplasia; the continuation and progress of FORTIFY, the Phase 3 trial of BBP-418 for LGMD2I, including the ongoing enrollment in the United States, the expectation to enroll in clinical trial sites planned in Europe and Australia, the expectation and timing of full enrollment of the interim analysis population, and the potential to pursue Accelerated Approval for BBP-418 based on recent interactions with the FDA; the continued enrollment in CALIBRATE, the Phase 3 clinical trial of encaleret, and the expectation and timing of full enrollment and sharing topline data from CALIBRATE; the Company’s financial performance, capitalization status, strategy, business plans and goals reflect our current views about our plans, intentions, expectations and strategies, which are based on the information currently available to us and on assumptions we have made. Although we believe that our plans, intentions, expectations and strategies as reflected in or suggested by those forward-looking statements are reasonable, we can give no assurance that the plans, intentions, expectations or strategies will be attained or achieved. Furthermore, actual results may differ materially from those described in the forward-looking statements and will be affected by a number of risks, uncertainties and assumptions, including, but not limited to, initial and ongoing data from our preclinical studies and clinical trials not being indicative of final data, the potential size of the target patient populations our product candidates are designed to treat not being as large as anticipated, the design and success of ongoing and planned clinical trials, future regulatory filings, approvals and/or sales, despite having ongoing and future interactions with the FDA or other regulatory agencies to discuss potential paths to registration for our product candidates, the FDA or such other regulatory agencies not agreeing with our regulatory approval strategies, components of our filings, such as clinical trial designs, conduct and methodologies, or the sufficiency of data submitted, the continuing success of our collaborations, the Company’s ability to obtain additional funding, including through less dilutive sources of capital than equity financings, potential volatility in our share price, uncertainty regarding any impacts due to global health emergencies such as COVID-19, including delays in regulatory review, manufacturing and supply chain interruptions, adverse effects on healthcare systems and disruption of the global economy, the impacts of current macroeconomic and geopolitical events, including changing conditions from hostilities in Ukraine and in Israel and the Gaza Strip, increasing rates of inflation and rising interest rates, on business operations and expectations, as well as those risks set forth in the Risk Factors section of our most recent Annual Report on Form 10-K and our other filings with the U.S. Securities and Exchange Commission. Moreover, we operate in a very competitive and rapidly changing environment in which new risks emerge from time to time. These forward-looking statements are based upon the current expectations and beliefs of our management as of the date of this press release, and are subject to certain risks and uncertainties that could cause actual results to differ materially from those described in the forward-looking statements. Except as required by applicable law, we assume no obligation to update publicly any forward-looking statements, whether as a result of new information, future events or otherwise. BridgeBio Contact: Vikram Bali contact@bridgebio.com (650)-789-8220

「  本文共：15条资讯，阅读时长约：3分钟 」今‍日‍头条开拓SMO抑制剂获批IPF临床。开拓药业Hedgehog/SMO抑制剂GT1708F新增适应症获国家药监局临床许可，即将开展用于治疗特发性肺纤维化(IPF) 的II期临床试验。GT1708F可通过抑制SMO蛋白的活性来影响Hedgehog通路的活性及其下游相关蛋白的表达，从而达到治疗IPF的目的。此前GT1708F已在恶性血液疾病的中国I期临床获得积极初步结果，并显示出良好的安全性和耐受性。‍‍‍国‍‍‍‍‍‍‍‍‍内‍‍‍‍药‍‍讯‍‍‍‍1.百济神州Bcl-2抑制剂上III期临床。百济神州高选择性Bcl-2抑制剂Sonrotoclax（BGB-11417）在clinicaltrials.gov网站上登记一项III期研究（NCT06073821），旨在评估与BTK抑制剂泽布替尼联用对比Bcl-2抑制剂维奈克拉（Venetoclax）与CD20单抗奥妥珠单抗（Obinutuzumab）联用，治疗慢性淋巴细胞白血病（CLL）的疗效与安全性。在临床前研究和肿瘤模型中，Sonrotoclax均显示出较维奈克拉更高的效力和靶点选择性，并有可能克服耐药性。2.信达PCSK9单抗见刊柳叶刀子刊。信达生物PCSK9单抗信必乐（托莱西单抗注射液，IBI306）在《柳叶刀》子刊上发表其用于治疗中国非家族性高胆固醇血症（non-FH）患者的Ⅲ期临床（CREDIT-1）研究成果。结果显示，托莱西单抗（450mg Q4W和600mg Q6W）较安慰剂均可显著降低LDL-C水平，LDL-C水平较基线降幅分别为65.0%和57.3%；两个治疗组LDL-C水平降低50%以上的患者比例为87.8%和71.8%。研究期间托莱西单抗总体安全性良好。3.北京锤特溶瘤病毒获批实体瘤临床。北京锤特生物重组人nsIL12溶瘤腺病毒注射液（BioTTT001）获国家药监局临床试验默示许可，拟开发用于恶性实体肿瘤的治疗。BioTTT001特定靶向带有常见Rb基因和抗凋亡基因异常的人肿瘤细胞，可诱导机体产生肿瘤治疗作用，杀伤远处转移肿瘤细胞和防止肿瘤复发。在临床前人实体瘤细胞系研究中，BioTTT001已显示出较强的杀伤能力。4.岸迈与Almirall联合开发双抗药物。岸迈生物与Almirall公司将利用前者专有的FIT-Ig®技术平台，就最多3对未披露的靶点，合作开发用于皮肤病学领域的双特异性抗体。FIT-Ig®技术平台可以只用单抗的基本结构部件生成双特异性抗体。根据协议，Almirall将拥有这些双特异性抗体的全球独家权益；岸迈生物将获得最多合计2.1亿美元的里程碑付款，以及产品的净销售收入的特许权使用费。5.苏州宜联HER3 ADC授权BioNTech。苏州宜联生物宣布与BioNTech达成合作许可协议，授予后者一款ADC产品在全球范围内（不包括中国内地、香港和澳门）开发、制造和商业化的独家权利，双方将合作开发HER3 ADC。根据协议，宜联生物将获得7000万美元首付款，以及总金额超过10亿美元的开发、监管和商业化里程碑潜在付款。目前全球范围内尚未有靶向HER3的疗法上市。‍‍‍‍国‍‍‍‍‍‍‍‍‍‍际‍‍‍‍药‍讯‍‍1.低浓度阿托品预防儿童近视长期疗效积极。Vyluma公司低浓度阿托品（atropine）眼药水NVK002用于预防儿童近视的Ⅲ期临床CHAMP最新结果积极。治疗第4年结果显示，NVK002（0.01%和0.02%）均具有良好的安全性，治疗相关不良事件发生率仅为8%。此外，NVK002（0.01%）的疗效与历史对照组相比继续扩大，未发现疗效减弱的迹象。2.MSD个体化肿瘤疫苗启动第二项III期临床。默沙东与Moderna开发的肿瘤新抗原mRNA疫苗V940（mRNA-4157）登记启动第二项Ⅲ期临床，评估联合帕博利珠单抗（Keytruda）对比安慰剂联合K药辅助治疗完全切除II-IIIB期非小细胞肺癌（NSCLC）患者的有效性和安全性。今年7月，该新药联合Keytruda一线治疗经手术切除高风险黑色素瘤（IIB-IV期）患者的Ⅲ期临床（V940-001）首批患者已在澳大利亚入组。3.OSA口服复方Ⅱ期临床见刊。Apnimed公司潜在“first-in-class”口服复方AD109（托莫西汀/aroxybutynin）在《美国呼吸与重症监护医学杂志》上发表其治疗阻塞性睡眠呼吸暂停（OSA）的Ⅱb期临床MARIPOSA完整数据。与安慰剂相比，AD109治疗使患者的呼吸暂停低通气指数（AHI4）降低45%；AD109（2.5/75 mg）组患者在PROMIS疲劳量表上表现出统计学显著改善；药物耐受性良好。4.Incyte公司JAK1抑制剂白癜风IIb期临床积极。Incyte公司口服JAK1抑制剂Povorcitinib（INCB54707）治疗广泛性非节段性白癜风的IIb期研究最新结果公布于欧洲皮肤病和性病学会大会上。52周数据显示，与安慰剂相比，povorcitinib（15mg、45mg和75mg）治疗组患者的总白癜风面积评分指数（T-VASI）降幅更大（40.7%、42.7%、41.3%vs18.1%），面部的F-VASI指数分别降低了63.6%、63.8%、64.4%和54.8%。最常见的治疗期间不良事件（TEAE）是COVID-19（36.1%）。5.CaSR拮抗剂Ⅱb期临床见刊NEJM。BridgeBio公司小分子CaSR拮抗剂encaleret在NEJM上发表用于治疗常染色体显性低钙血症（ADH1）的Ⅱb期临床积极结果。24周随访数据显示，encaleret可治疗低钙血症并减少高钙尿症的发生，有77%（10/13）患者可观察到正常尿钙排出的现象。此前，Encaleret已获得FDA授予的快速通道资格，以及FDA与欧盟的孤儿药资格。6.PPAR⍺拮抗剂联合标准治疗肝癌早期临床积极。Tempest公司PPAR⍺拮抗剂TPST-1120一线治疗无法切除或转移性肝细胞癌（HCC）的Ⅰb/Ⅱ期试验结果积极。与阿替利珠单抗和贝伐珠单抗标准治疗方案相比，TPST-1120联合标准治疗提高患者的客观缓解率（cORR：30%vs13.3%）和无进展生存期（中位PFS：7个月vs4.27个月）；TPST-1120联合治疗耐受性良好，两组的安全性数据相当。‍‍‍‍‍‍‍‍‍医‍‍‍‍‍‍药‍‍‍‍‍‍‍‍‍‍‍‍‍热‍‍‍‍‍‍‍点‍。1.红肉摄入或增加糖尿病、心脏病风险。近日，香港大学李嘉诚医学院的研究人员在《欧洲心脏杂志》上发表的最新研究论文显示，大量食用加工、未加工的红肉，会增加患心血管疾病和糖尿病的风险。每天多吃50克加工红肉，患心血管疾病风险增加26%，患糖尿病风险增加44%。每天多吃100克未加工红肉，患心血管疾病风险增加11%，患糖尿病风险增加27%。 2.湖南省新增和修订药学类医疗服务价格项目。为稳妥有序探索和完善药学类医疗服务价格政策，推进医疗机构临床药学服务模式转变，湖南省医保局日前印发《新增和修订药学类医疗服务价格项目的通知》。《通知》统一药学服务价格项目编码，并新增4项、修订11项药学类医疗服务价格项目，“药学门诊诊查费”“全胃肠外营养药物配置”“细胞毒性药物配置”“用药指导的基因检测”等9个项目集中归类为“药学服务”项目。3.山东省立医院菏泽医院揭牌。10月11日，国家区域医疗中心项目——山东省立医院菏泽医院揭牌仪式在菏泽举行。该项目以山东第一医科大学附属省立医院（山东省立医院）为输出医院，菏泽市立医院为依托医院，以菏泽市立医院“改扩新”综合楼为建设基础。项目总占地面积约10.7万㎡，2024年底预计全部竣工投入使用，届时医院总建筑面积约37万平方米，开放床位预计达到2200张。‍‍‍‍‍‍‍评‍‍‍‍‍审‍‍‍‍动‍‍态‍‍‍‍‍‍‍  1. CDE新药受理情况（10月12日) 2. FDA新药获批情况（北美10月09日）股市资讯上个交易日 A 股医药板块 +0.35%涨幅前三     跌幅前三海辰药业 +20.01%  *ST 吉药 -3.91%翰宇药业 +12.08%  华兰疫苗 -3.41%科源制药 +11.84%  康为世纪 -3.38%[汇宇制药]化学创新药HYP-2090PTSA胶囊收到国家药品监督管理局核准签发的药物临床试验批准通知书，用于治疗KRASG12C突变的晚期实体瘤(如非小细胞肺癌、结直肠癌、胰腺癌等)。[贝达药业]:甲磺酸贝福替尼胶囊一线治疗适应症获得药品注册证书，适用于具有EGFR外显子19缺失或外显子21(L858R)置换突变的局部晚期或转移性NSCLC成人患者的一线治疗。[海思科]:创新药HSK39297片获得IND申请受理通知书，用于治疗原发性或继发性肾小球疾病。- The End -戳“阅读原文”，了解更多医药研发及股市资讯。