Silence Therapeutics Provides mRNAi GOLD™ Platform and Pipeline Updates at 2021 R&D Day
- Announces plans to initiate a Phase 1 study in polycythaemia vera (PV) with SLN124, the third indication being assessed using endogenous hepcidin modulation
- SLN360 single-ascending dose study on-track for topline data in Q1'22 – independent review committee recommends extending follow-up period to assess potential longer duration of action
- Advancing mRNAi GOLD™ platform programs – poised to deliver 2-3 INDs per year
from 2023
21 October 2021, LONDON and NEW YORK -- Silence Therapeutics plc (AIM:SLN and Nasdaq: SLN), a leader in the discovery, development and delivery of novel short interfering ribonucleic acid (siRNA) therapeutics for the treatment of diseases with significant unmet medical need, announces it is hosting its R&D Day in New York City today.
During the event, Silence plans to showcase its proprietary mRNAi GOLD™ platform and current clinical programs, including SLN360 for cardiovascular disease due to high lipoprotein(a), or Lp(a), and SLN124 for thalassemia and myelodysplastic syndrome (MDS) as well as a newly added program in polycythemia vera (PV). Silence will also provide an update on its growing discovery pipeline and plans to deliver 2-3 INDs per year from 2023.
Mark Rothera, President and Chief Executive Officer of Silence Therapeutics, said: "The vision at Silence has always been to build an exquisite platform that would enable the development of longer-lasting precision medicines to transform patients' lives. We are pleased to report strong progress across both our proprietary and partnered pipelines, including plans to further expand the SLN124 program into a third indication, PV, where endogenous hepcidin modulation can potentially improve outcomes for 3.5 million people worldwide who currently have very limited treatment options. Through our hybrid business model, we are committed to maximizing the substantial opportunity for our mRNAi GOLD™ platform and remain well positioned to deliver 2-3 INDs per year from 2023."
Giles Campion, MD, EVP, Head of R&D and Chief Medical Officer of Silence Therapeutics, said: "We are very encouraged by the outcomes from our recent safety review committee meeting for SLN360. Based on their recommendations, we have now defined our dose range and are ready to start the multiple-ascending dose study. We are also extending the follow-up period in the single-ascending dose study to fully characterize SLN360's duration of action, which may be even longer than we initially anticipated. We look forward to further evaluating SLN360 in the clinic and remain on-track to report topline data in the single-ascending dose study in the first quarter of 2022."
mRNAi GOLD™ Proprietary Program Updates
SLN124 Program
New highlighted updates include:
- Plans to pursue new PV indication and start a phase 1 trial in the second half of 2022.
- FDA acceptance of the US IND in myelodysplastic syndrome (MDS).
- Enrollment continues in the two single-ascending dose studies for thalassemia and MDS. Silence anticipates topline data from both studies in the third quarter of 2022.
- Positive results from the healthy volunteer study reported in May 2021 accepted for poster presentation at the American Society of Hematology (ASH) Annual Meeting being held December 11-14, 2021.
SLN360 Program
New highlighted updates include:
- On-track to report top-line data in the single-ascending dose study in the first quarter of 2022.
- Key outcomes from the independent safety review committee meeting:
- Recommendation to extend follow-up period in the single-ascending dose study from 150 days to 365 days to fully assess the duration of action, which may be longer than initially anticipated based on preclinical modelling.
- The therapeutic dose range has been established based on Cohorts 1-4 (optional Cohort 5 not needed) and study can now proceed to the multiple-ascending dose phase.
- On-track to initiate phase 2 development in the second half of 2022, pending regulatory discussions.
Building Proprietary Pipeline
- Advancing two new undisclosed proprietary programs following the Hansoh collaboration announced last week (read full release here). Hansoh has the option to license China region rights at the end of phase 1.
mRNAi GOLD™ Partnered Program Updates
AstraZeneca Collaboration for Cardiovascular, Renal, Metabolic and Respiratory Diseases
- Continuing work with AstraZeneca to develop siRNAs for two undisclosed targets.
- On-track to initiate work on five targets within the first three years of the collaboration.
Mallinckrodt Collaboration for Complement-Mediated Diseases
- Progressing IND-enabling studies for SLN501 C3-targeting program and expect to initiate a phase 1 study in the first half of 2022.
- Developing siRNAs for two other undisclosed complement-mediated disease targets.
Hansoh Collaboration
- In addition to the two proprietary programs outlined above, Silence will work with Hansoh on a third undisclosed program which Hansoh has the option to license worldwide at the point of IND filing.
- As part of the Hansoh agreement, Hansoh made a $16 million upfront cash payment and Silence has the potential for up to $1.3 billion in additional milestones plus royalties tiered from low double-digit to mid-teens on Hansoh net product sales.
Targeting 2-3 INDs per year from 2023
- Silence remains on-track to deliver 2-3 INDs per year from 2023 through both its proprietary and partnered mRNAi GOLD™ platform programs. Key highlighted updates include:
- Expanded translational genomics efforts, contributing both to finding new, genetically validated disease-causing genes and continuously fine-tuning the siRNA design algorithm using machine learning
- A fine-tuned drug discovery process
- Aiming for a mixed-risk portfolio of validated and novel targets with projects at different stages of drug discovery ensuring multiple shots on goal
The Company's R&D Day will be held today, October 21, 2021 from 9:00 am to 11:30 am ET at the Convene, 530 Fifth Avenue, in New York City and will include a live video stream.
Webcast Link: https://silence-therapeutics-rd-day.convene.com
Enquiries:
Silence Therapeutics plc
Gem Hopkins, Head of IR and Corporate Communications
ir@silence-therapeutics.com
| Tel: +1 (646) 637-3208 |
Investec Bank plc (Nominated Adviser and Broker)
Daniel Adams/Gary Clarence
| Tel: +44 (0) 20 7597 5970 |
European PR
Consilium Strategic Communications
Mary-Jane Elliott/Chris Welsh/Angela Gray
silencetherapeutics@consilium-comms.com
US Media Relations
MKC Strategies, LLC
Mary Conway
MConway@MKCStrategies.com
| Tel: +44 (0) 20 3709 5700
Tel: +1 (516) 606-6545
|
About Silence Therapeutics
Silence Therapeutics is developing a new generation of medicines by harnessing the body's natural mechanism of RNA interference, or RNAi, to inhibit the expression of specific target genes thought to play a role in the pathology of diseases with significant unmet need. Silence's proprietary mRNAi GOLD™ platform can be used to create siRNAs (short interfering RNAs) that precisely target and silence disease-associated genes in the liver, which represents a substantial opportunity. Silence's wholly owned product candidates include SLN360 designed to address the high and prevalent unmet medical need in reducing cardiovascular risk in people born with high levels of lipoprotein(a) and SLN124 designed to address iron-loading anemia conditions. Silence also maintains ongoing research and development collaborations with AstraZeneca, Mallinckrodt Pharmaceuticals, and Takeda, among others. For more information, please visit https://www.silence-therapeutics.com/.
Forward-Looking Statements
Certain statements made in this announcement are forward-looking statements within the meaning of the U.S. Private Securities Litigation Reform Act of 1995 and other securities laws, including with respect to the Company's clinical and commercial prospects and the anticipated timing of data reports from the Company's clinical trials. These forward-looking statements are not historical facts but rather are based on the Company's current expectations, estimates, and projections about its industry; its beliefs; and assumptions. Words such as 'anticipates,' 'expects,' 'intends,' 'plans,' 'believes,' 'seeks,' 'estimates,' and similar expressions are intended to identify forward-looking statements. These statements are not guarantees of future performance and are subject to known and unknown risks, uncertainties, and other factors, some of which are beyond the Company's control, are difficult to predict, and could cause actual results to differ materially from those expressed or forecasted in the forward-looking statements, including those risks identified in the Company's most recent Admission Document and its amended Annual Report on Form 20-F filed with the U.S. Securities and Exchange Commission on April 29, 2021. The Company cautions security holders and prospective security holders not to place undue reliance on these forward-looking statements, which reflect the view of the Company only as of the date of this announcement. The forward-looking statements made in this announcement relate only to events as of the date on which the statements are made. The Company will not undertake any obligation to release publicly any revisions or updates to these forward-looking statements to reflect events, circumstances, or unanticipated events occurring after the date of this announcement except as required by law or by any appropriate regulatory authority.
Silence Therapeutics Provides mRNAi GOLD™ Platform and Pipeline Updates at 2021 R&D Day
- Announces plans to initiate a Phase 1 study in polycythaemia vera (PV) with SLN124, the third indication being assessed using endogenous hepcidin modulation
- SLN360 single-ascending dose study on-track for topline data in Q1'22 – independent review committee recommends extending follow-up period to assess potential longer duration of action
- Advancing mRNAi GOLD™ platform programs – poised to deliver 2-3 INDs per year
from 2023
21 October 2021, LONDON and NEW YORK -- Silence Therapeutics plc (AIM:SLN and Nasdaq: SLN), a leader in the discovery, development and delivery of novel short interfering ribonucleic acid (siRNA) therapeutics for the treatment of diseases with significant unmet medical need, announces it is hosting its R&D Day in New York City today.
During the event, Silence plans to showcase its proprietary mRNAi GOLD™ platform and current clinical programs, including SLN360 for cardiovascular disease due to high lipoprotein(a), or Lp(a), and SLN124 for thalassemia and myelodysplastic syndrome (MDS) as well as a newly added program in polycythemia vera (PV). Silence will also provide an update on its growing discovery pipeline and plans to deliver 2-3 INDs per year from 2023.
Mark Rothera, President and Chief Executive Officer of Silence Therapeutics, said: "The vision at Silence has always been to build an exquisite platform that would enable the development of longer-lasting precision medicines to transform patients' lives. We are pleased to report strong progress across both our proprietary and partnered pipelines, including plans to further expand the SLN124 program into a third indication, PV, where endogenous hepcidin modulation can potentially improve outcomes for 3.5 million people worldwide who currently have very limited treatment options. Through our hybrid business model, we are committed to maximizing the substantial opportunity for our mRNAi GOLD™ platform and remain well positioned to deliver 2-3 INDs per year from 2023."
Giles Campion, MD, EVP, Head of R&D and Chief Medical Officer of Silence Therapeutics, said: "We are very encouraged by the outcomes from our recent safety review committee meeting for SLN360. Based on their recommendations, we have now defined our dose range and are ready to start the multiple-ascending dose study. We are also extending the follow-up period in the single-ascending dose study to fully characterize SLN360's duration of action, which may be even longer than we initially anticipated. We look forward to further evaluating SLN360 in the clinic and remain on-track to report topline data in the single-ascending dose study in the first quarter of 2022."
mRNAi GOLD™ Proprietary Program Updates
SLN124 Program
New highlighted updates include:
- Plans to pursue new PV indication and start a phase 1 trial in the second half of 2022.
- Enrollment continues in the two single-ascending dose studies for thalassemia and MDS. Silence anticipates topline data from both studies in the third quarter of 2022.
- Positive results from the healthy volunteer study reported in May 2021 accepted for poster presentation at the American Society of Hematology (ASH) Annual Meeting being held December 11-14, 2021.
SLN360 Program
New highlighted updates include:
- On-track to report top-line data in the single-ascending dose study in the first quarter of 2022.
- Key outcomes from the independent safety review committee meeting:
- Recommendation to extend follow-up period in the single-ascending dose study from 150 days to 365 days to fully assess the duration of action, which may be longer than initially anticipated based on preclinical modelling.
- The therapeutic dose range has been established based on Cohorts 1-4 (optional Cohort 5 not needed) and study can now proceed to the multiple-ascending dose phase.
- On-track to initiate phase 2 development in the second half of 2022, pending regulatory discussions.
Building Proprietary Pipeline
- Advancing two new undisclosed proprietary programs following the Hansoh collaboration announced last week (read full release here). Hansoh has the option to license China region rights at the end of phase 1.
mRNAi GOLD™ Partnered Program Updates
AstraZeneca Collaboration for Cardiovascular, Renal, Metabolic and Respiratory Diseases
- Continuing work with AstraZeneca to develop siRNAs for two undisclosed targets.
- On-track to initiate work on five targets within the first three years of the collaboration.
Mallinckrodt Collaboration for Complement-Mediated Diseases
- Progressing IND-enabling studies for SLN501 C3-targeting program and expect to initiate a phase 1 study in the first half of 2022.
- Developing siRNAs for two other undisclosed complement-mediated disease targets.
Hansoh Collaboration
- In addition to the two proprietary programs outlined above, Silence will work with Hansoh on a third undisclosed program which Hansoh has the option to license worldwide at the point of IND filing.
- As part of the Hansoh agreement, Hansoh made a $16 million upfront cash payment and Silence has the potential for up to $1.3 billion in additional milestones plus royalties tiered from low double-digit to mid-teens on Hansoh net product sales.
Delivering on 2-3 INDs per year from 2023
- Silence remains on-track to deliver 2-3 INDs per year from 2023 through both its proprietary and partnered mRNAi GOLD™ platform programs. Key highlighted updates include:
- Expanded translational genomics efforts, contributing both to finding new, genetically validated disease-causing genes and continuously fine-tuning the siRNA design algorithm using machine learning
- A fine-tuned drug discovery process
- Aiming for a mixed-risk portfolio of validated and novel targets with projects at different stages of drug discovery ensuring multiple shots on goal
The Company's R&D Day will be held today, October 21, 2021 from 9:00 am to 11:30 am ET at the Convene, 530 Fifth Avenue, in New York City and will include a live video stream.
Webcast Link: https://silence-therapeutics-rd-day.convene.com
Enquiries:
Silence Therapeutics plc
Gem Hopkins, Head of IR and Corporate Communications
ir@silence-therapeutics.com
| Tel: +1 (646) 637-3208 |
Investec Bank plc (Nominated Adviser and Broker)
Daniel Adams/Gary Clarence
| Tel: +44 (0) 20 7597 5970 |
European PR
Consilium Strategic Communications
Mary-Jane Elliott/Chris Welsh/Angela Gray
silencetherapeutics@consilium-comms.com
US Media Relations
MKC Strategies, LLC
Mary Conway
MConway@MKCStrategies.com
| Tel: +44 (0) 20 3709 5700
Tel: +1 (516) 606-6545
|
About Silence Therapeutics
Silence Therapeutics is developing a new generation of medicines by harnessing the body's natural mechanism of RNA interference, or RNAi, to inhibit the expression of specific target genes thought to play a role in the pathology of diseases with significant unmet need. Silence's proprietary mRNAi GOLD™ platform can be used to create siRNAs (short interfering RNAs) that precisely target and silence disease-associated genes in the liver, which represents a substantial opportunity. Silence's wholly owned product candidates include SLN360 designed to address the high and prevalent unmet medical need in reducing cardiovascular risk in people born with high levels of lipoprotein(a) and SLN124 designed to address iron-loading anemia conditions. Silence also maintains ongoing research and development collaborations with AstraZeneca, Mallinckrodt Pharmaceuticals, and Takeda, among others. For more information, please visit https://www.silence-therapeutics.com/.
Forward-Looking Statements
Certain statements made in this announcement are forward-looking statements within the meaning of the U.S. Private Securities Litigation Reform Act of 1995 and other securities laws, including with respect to the Company's clinical and commercial prospects and the anticipated timing of data reports from the Company's clinical trials. These forward-looking statements are not historical facts but rather are based on the Company's current expectations, estimates, and projections about its industry; its beliefs; and assumptions. Words such as 'anticipates,' 'expects,' 'intends,' 'plans,' 'believes,' 'seeks,' 'estimates,' and similar expressions are intended to identify forward-looking statements. These statements are not guarantees of future performance and are subject to known and unknown risks, uncertainties, and other factors, some of which are beyond the Company's control, are difficult to predict, and could cause actual results to differ materially from those expressed or forecasted in the forward-looking statements, including those risks identified in the Company's most recent Admission Document and its amended Annual Report on Form 20-F filed with the U.S. Securities and Exchange Commission on April 29, 2021. The Company cautions security holders and prospective security holders not to place undue reliance on these forward-looking statements, which reflect the view of the Company only as of the date of this announcement. The forward-looking statements made in this announcement relate only to events as of the date on which the statements are made. The Company will not undertake any obligation to release publicly any revisions or updates to these forward-looking statements to reflect events, circumstances, or unanticipated events occurring after the date of this announcement except as required by law or by any appropriate regulatory authority.
沉默治療公司在2021年研發日提供mRNAi黃金™平臺和管道更新
- 宣佈計劃啟動的第一階段研究 紅細胞增多症維拉(PV)使用SLN124第三個跡象是評估使用內源性海普西丁調節
- SLN360單次遞增劑量研究在……的軌道上 背線數據輸入Q1'22 – 獨立審查委員會建議延展隨訪期評估潛力訴訟期限更長
- 前進 MRNAi金牌™p格式程序 – 蓄勢待發，準備每年交付2-3個IND
從2023年開始
亞洲網倫敦和紐約10月21日電發現、開發和交付新型短幹擾核糖核酸(SiRNA)療法的領先企業Silence Treeutics Plc(AIM：SLN和Nasdaq：SLN)宣佈，該公司今天將在紐約市舉辦研發日活動。
在活動期間，思萊斯公司計劃展示其專有的mRNAi GOLD™平臺和當前的臨牀項目，包括針對高脂蛋白(A)引起的心血管疾病的SLN360，針對地中海貧血和骨髓增生異常綜合徵的SLN124，以及新增加的針對真性紅細胞增多症(PV)的項目。Silent還將提供其不斷增長的發現管道的最新情況，並計劃從2023年起每年交付2-3個IND。
沉默治療公司總裁兼首席執行官馬克·羅瑟拉説：沉默公司的願景一直是建立一個精緻的平臺，使更持久的精確藥物的開發能夠改變患者的生活。我們很高興地報告，我們的專有和合作渠道都取得了強勁的進展，包括計劃進一步將SLN124計劃擴大到第三種適應症PV，在這種情況下，內源性海普西丁調製可能會改善全世界目前治療選擇非常有限的350萬人的結果。通過我們的混合業務模式，我們致力於最大限度地利用我們的mRNAi Gold™平臺的實質性機會，並保持良好的地位。
Silence Treeutics的研發主管兼首席醫療官、醫學博士、執行副總裁賈爾斯·坎皮恩(Giles Campion)説：“我們對最近召開的SLN360安全審查委員會會議的結果感到非常鼓舞。根據他們的建議，我們現在已經確定了我們的劑量範圍，並準備開始多次遞增劑量研究。我們還延長了單次遞增劑量研究的跟蹤期，以充分描述SLN360的作用時間，這可能比我們最初預期的更長。我們期待着在臨牀上進一步評估SLN360，並繼續按計劃在第一季度報告單次遞增劑量研究中的背線數據。”
MRNAi 黃金™ 專有程序更新
SLN124計劃
新h亮點更新包括：
- 計劃尋求新的PV適應症，並在2022年下半年開始一期試驗。
- FDA接受美國IND治療骨髓增生異常綜合徵(MDS)。
- 針對地中海貧血和MDS的兩項單劑量遞增劑量研究的登記仍在繼續。Silent預計2022年第三季度這兩項研究的背線數據。
- 2021年5月報道的健康志願者研究的陽性結果接受了2021年12月11日至14日舉行的美國血液學會(ASH)年會的海報展示。
SLN360 計劃
N電子戰突出顯示更新包括：
- 在2022年第一季度報告單次上升劑量研究的一線數據。
- 獨立安全審查委員會會議的主要結果：
- 建議將單次遞增劑量研究的隨訪期從150天延長到365天，以全面評估作用持續時間，這可能比基於臨牀前建模的最初預期要長。
- 治療劑量範圍已經建立在1-4組(不需要選擇5組)的基礎上，研究現在可以進行到多個遞增劑量階段。
- 正在按部就班地在2022年下半年啟動第二階段開發，等待監管討論。
建設專有管道
- 繼上週宣佈的Hansoh合作之後，推進了兩個新的未披露的專有項目(點擊此處閲讀完整版本)。Hansoh有權在第一階段結束時許可中國地區權利。
MRNAi黃金™ 合作計劃更新
阿斯利康合作心血管、腎臟、代謝和呼吸系統疾病
- 繼續與阿斯利康合作，為兩個未披露的目標開發siRNA。
- 在協作的前三年內啟動五個目標的工作。
Mallinckrodt協作針對補體介導的疾病
- SLN501 C3目標計劃的啟用IND的研究正在進行中，預計將在2022年上半年啟動第一階段研究。
- 為另外兩個未公開的補體介導的疾病靶點開發siRNA。
漢索(Hansoh)協作
- 除了上述兩個專有項目外，Silence還將與Hansoh合作開發第三個未披露的項目，Hansoh有權在提交IND申請時在全球範圍內許可該項目。
- 作為Hansoh協議的一部分，Hansoh預付了1600萬美元的現金，Silence有可能獲得高達13億美元的額外里程碑，外加Hansoh淨產品銷售額從低兩位數到十幾歲的特許權使用費。
瞄準從2023年開始，每年2-3個IND
- Silent仍將繼續通過其專有和合作的mRNAi Gold™平臺計劃，從2023年開始每年提供2-3個IND。重點介紹的主要更新包括：
- 擴大翻譯基因組學的努力，既有助於發現新的、經基因驗證的致病基因，也有助於利用機器學習不斷微調siRNA設計算法
- 微調的藥物發現過程
- 針對已驗證和新目標的混合風險組合，在藥物發現的不同階段進行項目，確保目標的多次命中
該公司的研發日將於今天(2021年10月21日)美國東部時間上午9：00至11：30在紐約市第五大道530號的會議中心舉行，並將包括現場視頻流。
網絡直播鏈接: Https://silence-therapeutics-rd-day.convene.com
查詢：
沉默治療公司
吉姆·霍普金斯(Gem Hopkins)，投資者關係和企業公關主管
電子郵箱：ir@Silence-Treateutics.com
| 電話：+1(646)637-3208 |
天達銀行(Investec Bank Plc) (指定顧問及經紀)
丹尼爾·亞當斯/加里·克拉倫斯
| 電話：+44(0)207597570 |
歐洲人PR
Consilium戰略傳播
瑪麗-簡·埃利奧特/克里斯·威爾士/安吉拉·格雷
郵箱：silenceTreateutics@conconlium-comms.com
美國媒體關係
MKC Strategy，LLC
瑪麗·康威
郵箱：mconway@MKCStrategies.com
| 電話：+44(0)2037095700
電話：+1(516)606-6545
|
關於沉默療法
沉默治療公司正在開發新一代藥物，方法是利用人體自然的RNA幹擾(RNAi)機制，抑制特定靶基因的表達，這些基因被認為在有重大未得到滿足的需求的疾病的病理中發揮作用。Silent公司專有的mRNAi金™平臺可以用來創建siRNA(短幹擾RNA)，它可以精確地針對肝臟中的疾病相關基因並使其沉默，這代表着一個巨大的機會。SILE公司全資擁有的候選產品包括SLN360和SLN124。SLN360旨在滿足在降低天生高水平脂蛋白(A)人羣心血管風險方面的高和普遍的未得到滿足的醫療需求，SLN124旨在解決鐵負荷貧血癥問題。沉默公司還與阿斯利康、Mallinckrodt製藥公司和武田等公司保持着持續的研發合作。欲瞭解更多信息，請訪問網址：https://www.silence-therapeutics.com/.。
前瞻性陳述
本公告中的某些陳述屬於“1995年美國私人證券訴訟改革法”和其他證券法所指的前瞻性陳述，包括與公司的臨牀和商業前景以及公司臨牀試驗數據報告的預期時間有關的陳述。這些前瞻性陳述不是歷史事實，而是基於該公司目前對其行業的預期、估計和預測、其信念和假設。諸如“預期”、“期望”、“打算”、“計劃”、“相信”、“尋求”、“估計”等詞彙以及類似的表達方式都是為了識別前瞻性陳述。這些陳述不是對未來業績的保證，會受到已知和未知的風險、不確定性和其他因素的影響，其中一些是公司無法控制的，很難預測，可能會導致實際結果與前瞻性陳述中表達或預測的結果大不相同，包括公司最近的承認文件及其於2021年4月29日提交給美國證券交易委員會的修訂的20-F表格年度報告中確定的風險。公司告誡證券持有人和潛在的證券持有人不要過度依賴這些前瞻性陳述，這些陳述僅反映公司截至本公告日期的觀點。本公告中所作的前瞻性陳述僅涉及截至該陳述發表之日的事件。除非法律或任何適當的監管機構要求，否則公司不承擔任何義務公開發布對這些前瞻性陳述的任何修訂或更新，以反映本公告發布之日之後發生的事件、情況或意想不到的事件。
沉默治療公司在2021年研發日提供mRNAi黃金™平臺和管道更新
- 宣佈計劃啟動的第一階段研究 紅細胞增多症維拉(PV)使用SLN124第三個跡象是評估使用內源性海普西丁調節
- SLN360單次遞增劑量研究在……的軌道上 背線數據輸入Q1'22 – 獨立審查委員會建議延展隨訪期評估潛力訴訟期限更長
- 前進 MRNAi金™p格式程序 – 蓄勢待發，準備每年交付2-3個IND
從2023年開始
亞洲網倫敦和紐約10月21日電發現、開發和交付新型短幹擾核糖核酸(SiRNA)療法的領先企業Silence Treeutics Plc(AIM：SLN和Nasdaq：SLN)宣佈，該公司今天將在紐約市舉辦研發日活動。
在活動期間，思萊斯公司計劃展示其專有的mRNAi GOLD™平臺和當前的臨牀項目，包括針對高脂蛋白(A)引起的心血管疾病的SLN360，針對地中海貧血和骨髓增生異常綜合徵的SLN124，以及新增加的針對真性紅細胞增多症(PV)的項目。Silent還將提供其不斷增長的發現管道的最新情況，並計劃從2023年起每年交付2-3個IND。
沉默治療公司總裁兼首席執行官馬克·羅瑟拉説：沉默公司的願景一直是建立一個精緻的平臺，使更持久的精確藥物的開發能夠改變患者的生活。我們很高興地報告，我們的專有和合作渠道都取得了強勁的進展，包括計劃進一步將SLN124計劃擴大到第三種適應症PV，在這種情況下，內源性海普西丁調製可能會改善全世界目前治療選擇非常有限的350萬人的結果。通過我們的混合業務模式，我們致力於最大限度地利用我們的mRNAi Gold™平臺的實質性機會，並保持良好的地位。
Silence Treeutics的研發主管兼首席醫療官、醫學博士、執行副總裁賈爾斯·坎皮恩(Giles Campion)説：“我們對最近召開的SLN360安全審查委員會會議的結果感到非常鼓舞。根據他們的建議，我們現在已經確定了我們的劑量範圍，並準備開始多次遞增劑量研究。我們還延長了單次遞增劑量研究的跟蹤期，以充分描述SLN360的作用時間，這可能比我們最初預期的更長。我們期待着在臨牀上進一步評估SLN360，並繼續按計劃在第一季度報告單次遞增劑量研究中的背線數據。”
MRNAi黃金™ 專有程序更新
SLN124計劃
新h亮點更新包括：
- 計劃尋求新的PV適應症，並在2022年下半年開始一期試驗。
- 針對地中海貧血和MDS的兩項單劑量遞增劑量研究的登記仍在繼續。Silent預計2022年第三季度這兩項研究的背線數據。
- 2021年5月報道的健康志願者研究的陽性結果接受了2021年12月11日至14日舉行的美國血液學會(ASH)年會的海報展示。
SLN360 計劃
N電子戰突出顯示更新包括：
- 在2022年第一季度報告單次上升劑量研究的一線數據。
- 獨立安全審查委員會會議的主要結果：
- 建議將單次遞增劑量研究的隨訪期從150天延長到365天，以全面評估作用持續時間，這可能比基於臨牀前建模的最初預期要長。
- 治療劑量範圍已經建立在1-4組(不需要選擇5組)的基礎上，研究現在可以進行到多個遞增劑量階段。
- 正在按部就班地在2022年下半年啟動第二階段開發，等待監管討論。
建設專有管道
- 繼上週宣佈的Hansoh合作之後，推進了兩個新的未披露的專有項目(點擊此處閲讀完整版本)。Hansoh有權在第一階段結束時許可中國地區權利。
MRNAi金™ 合作計劃更新
阿斯利康合作心血管、腎臟、代謝和呼吸系統疾病
- 繼續與阿斯利康合作，為兩個未披露的目標開發siRNA。
- 在協作的前三年內啟動五個目標的工作。
Mallinckrodt協作針對補體介導的疾病
- SLN501 C3目標計劃的啟用IND的研究正在進行中，預計將在2022年上半年啟動第一階段研究。
- 為另外兩個未公開的補體介導的疾病靶點開發siRNA。
漢索(Hansoh)協作
- 除了上述兩個專有項目外，Silence還將與Hansoh合作開發第三個未披露的項目，Hansoh有權在提交IND申請時在全球範圍內許可該項目。
- 作為Hansoh協議的一部分，Hansoh預付了1600萬美元的現金，Silence有可能獲得高達13億美元的額外里程碑，外加Hansoh淨產品銷售額從低兩位數到十幾歲的特許權使用費。
從2023年開始，每年交付2-3個IND
- Silent仍將繼續通過其專有和合作的mRNAi Gold™平臺計劃，從2023年開始每年提供2-3個IND。重點介紹的主要更新包括：
- 擴大翻譯基因組學的努力，既有助於發現新的、經基因驗證的致病基因，也有助於利用機器學習不斷微調siRNA設計算法
- 微調的藥物發現過程
- 針對已驗證和新目標的混合風險組合，在藥物發現的不同階段進行項目，確保目標的多次命中
該公司的研發日將於今天(2021年10月21日)美國東部時間上午9：00至11：30在紐約市第五大道530號的會議中心舉行，並將包括現場視頻流。
網絡直播鏈接: Https://silence-therapeutics-rd-day.convene.com
查詢：
沉默治療公司
吉姆·霍普金斯(Gem Hopkins)，投資者關係和企業公關主管
電子郵箱：ir@Silence-Treateutics.com
| 電話：+1(646)637-3208 |
天達銀行(Investec Bank Plc) (指定顧問及經紀)
丹尼爾·亞當斯/加里·克拉倫斯
| 電話：+44(0)207597570 |
歐洲人PR
Consilium戰略傳播
瑪麗-簡·埃利奧特/克里斯·威爾士/安吉拉·格雷
郵箱：silenceTreateutics@conconlium-comms.com
美國媒體關係
MKC Strategy，LLC
瑪麗·康威
郵箱：mconway@MKCStrategies.com
| 電話：+44(0)2037095700
電話：+1(516)606-6545
|
關於沉默療法
沉默治療公司正在開發新一代藥物，方法是利用人體自然的RNA幹擾(RNAi)機制，抑制特定靶基因的表達，這些基因被認為在有重大未得到滿足的需求的疾病的病理中發揮作用。Silent公司專有的mRNAi金™平臺可以用來創建siRNA(短幹擾RNA)，它可以精確地針對肝臟中的疾病相關基因並使其沉默，這代表着一個巨大的機會。SILE公司全資擁有的候選產品包括SLN360和SLN124。SLN360旨在滿足在降低天生高水平脂蛋白(A)人羣心血管風險方面的高和普遍的未得到滿足的醫療需求，SLN124旨在解決鐵負荷貧血癥問題。沉默公司還與阿斯利康、Mallinckrodt製藥公司和武田等公司保持着持續的研發合作。欲瞭解更多信息，請訪問網址：https://www.silence-therapeutics.com/.。
前瞻性陳述
本公告中的某些陳述屬於“1995年美國私人證券訴訟改革法”和其他證券法所指的前瞻性陳述，包括與公司的臨牀和商業前景以及公司臨牀試驗數據報告的預期時間有關的陳述。這些前瞻性陳述不是歷史事實，而是基於該公司目前對其行業的預期、估計和預測、其信念和假設。諸如“預期”、“期望”、“打算”、“計劃”、“相信”、“尋求”、“估計”等詞彙以及類似的表達方式都是為了識別前瞻性陳述。這些陳述不是對未來業績的保證，會受到已知和未知的風險、不確定性和其他因素的影響，其中一些是公司無法控制的，很難預測，可能會導致實際結果與前瞻性陳述中表達或預測的結果大不相同，包括公司最近的承認文件及其於2021年4月29日提交給美國證券交易委員會的修訂的20-F表格年度報告中確定的風險。公司告誡證券持有人和潛在的證券持有人不要過度依賴這些前瞻性陳述，這些陳述僅反映公司截至本公告日期的觀點。本公告中所作的前瞻性陳述僅涉及截至該陳述發表之日的事件。除非法律或任何適當的監管機構要求，否則公司不承擔任何義務公開發布對這些前瞻性陳述的任何修訂或更新，以反映本公告發布之日之後發生的事件、情況或意想不到的事件。
