NTLA - Intellia Therapeutics, Inc. : US

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AnieLee
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NTLA - Intellia Therapeutics, Inc. : US

Posts by AnieLee » Sat Jan 02, 2021 1:35 pm

ขออนุญาตตั้งกระทู้ใหม่ของหุ้นเกี่ยวกับ Gene Edit ครับ

Intellia Therapeutics, Inc. is a preclinical-stage genome editing company focusing on developing novel therapeutics using the genome-editing process known as CRISPR/Cas9, or Clustered, Regularly Interspaced Short Palindromic Repeats. Currently, Intellia is broadly focused on in-vivo, delivering directly to target cells in the body, and ex-vivo, developing a therapy from engineered human cells, with a global clinical focus (three countries in progress).

Founded in 2014, NTLA has since grown to revenues of $43 million (2019) with over 270 employees based in Cambridge, Mass. Although CRISPR/Cas9 genome editing technology is not yet clinically validated for human therapeutic use, the company is making strides in strategic markets to achieve the required approvals and is currently transitioning from pre-clinical to clinical in 2020-2021.

Products: Intellia has a pipeline of four therapies in the post-research phase with three realistically expected to show clinical aspirations before year-end 2021.

1. NTLA-2001 for transthyretin amyloidosis "ATTR": Submitted first CTA to initiate a Phase 1 study; Intend to dose first patient by YE 2020

2. NTLA-5001 for acute myeloid leukemia "AML": Expected to submit IND in 1H 2021 for WT1-directed TCR T-cell therapy

3. NTLA-2002 for hereditary angioedema "HAE": Expected to submit IND in 2H 2021

Customers/market: Currently, Intellia is pre-clinical, but the potential market for patients of each of the above products is as follows. For ATTR alone, the expected patient population is hard to estimate, but rival Akcea has found there to be an estimated 50,000 patients with hereditary ATTR amyloidosis worldwide and 200,000 patients with wild-type ATTR amyloidosis worldwide. Intellia additionally estimates there to be over 120 unique mutations from the traditional ATTR adding a layer of complexity to current therapies, but expanding the patient pool to 250,000-500,000. For AML globally, the expected afflicted population is very roughly estimated to be 450,000 with 20,000 new cases in the USA alone estimated for 2020. For HAE, there are roughly 150,000-250,000 people globally afflicted with this rare deficiency.

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AnieLee
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Re: NTLA - Intellia Therapeutics, Inc. : US

Posts by AnieLee » Sat Jan 02, 2021 1:40 pm


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ความสำเร็จจากการลงทุน ไม่ได้เกิดจาก "การซื้อของดี" แต่มาจาก "การซื้อของได้ดี" ต่างหาก


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AnieLee
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Re: NTLA - Intellia Therapeutics, Inc. : US

Posts by AnieLee » Sat Jan 02, 2021 1:41 pm

Market/Pipeline

NTLA-2001: This is Intellia's first proprietary in vivo development candidate aiming to treat transthyretin amyloidosis -ATTR-, a disease with a target population of both hereditary and wild-type ATTR roughly amounting to 250,000-500,000 globally. Intellia seeks to competitively treat ATTR by reducing the level of transthyretin protein production with a single course of treatment with life-long suppression, a promising first advancement with the use of CRISPR/Cas9 therapy for ATTR. Differentiation to other therapies is based out of significant patient treatment burden reduction over competitors currently only offering chronic treatment options, and potential economic advantages. Intellia is leading the co-development with Regeneron, a long-time Intellia partner.

Intellia announced in Aug 2020 that they submitted a strong first Clinical Trial Application -CTA- in the UK to initiate a small-patient population Phase 1 study with 1st patient dosing expected by Dec. 2020 with other agencies under consideration. Intellia has found good interest in the UK for gene-based medicine and is enthusiastic about clinical efficiency there. Intellia stated at the Baird Conference that they aim to particularly monitor off-target effects with CRISPR and acute toxicities that add a level of risk from the results of mouse/primate tests completed. Data is roughly expected in mid-to-late 2021.

Financially speaking, the global amyloidosis treatment market size (includes types: ATTR, AL, AA, Aβ2M, amongst others) was valued at $4.5 billion (2019) and expected to grow to $5.2 billion (2021) with an expected CAGR of 6.7% until 2025.

NTLA-2002: This is Intellia's development candidate for treating hereditary angioedema -HAE- and second in vivo knockout therapeutic candidate. HAE is a rare genetic disorder affecting roughly 150,000 to 250,000 people globally. Only chronic treatment options are currently available making a knockout edit of the KLKB1 gene particularly effective and competitive over current options. Additionally, NTLA-2002 builds on the ATTR program’s infrastructure, including the modular LNP delivery system.

Intellia stated in Aug. 2020 that they aim to submit an IND in 2H 2021.

Financially speaking, the global HAE market is estimated globally to be roughly $2.6 billion (2021) and is expected to grow with a CAGR of 8.2% until 2025.

NTLA-5001: This is Intellia's first ex vivo development candidate and Intellia seeks to treat acute myeloid leukemia -AML- by engineering autologous T-cell receptors -TCR- focusing on Wilm's Tumor 1 antigen which is often over-expressed in AML and other cancers (potential lateral therapeutic targets such as ovarian cancer, glioblastoma, lung cancer, and mesothelioma). This affects roughly 450,000 people globally with 20,000 new cases estimated for 2020 in the USA alone.

Intellia stated in Aug 2020 that they aim to submit an IND in 1H 2021.

Financially speaking, the global AML market was valued at $702 million (2018) and is expected to grow to $1.04 billion (2021) with a 14% CAGR until 2024.

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AnieLee
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Re: NTLA - Intellia Therapeutics, Inc. : US

Posts by AnieLee » Sat Jan 02, 2021 2:01 pm

Intellia's Partnership

Intellia has two primary partnerships. A six-year partnership with Regeneron which extends into 2023 and focuses on targets in the liver and a five-year partnership with Novartis which is set to expire in under two weeks. 2019 was quiet on new developments, until the end of the year when Regeneron surprisingly announced it would reduce its share of the ATTR program with Intellia.

Regeneron remains committed to the program, but reduced its share of global development costs and profits from 50% to 25%. While this came as a surprise to many investors, I believe Regeneron remains committed to further collaborations with Intellia. For one thing, no other dynamics of the partnership changed and Regeneron still maintains options to in-license exclusive rights to as many as 10 targets, including ATTR. While I do see this as a potential red flag investors should be aware of and should take under consideration, I believe Regeneron made this move as more of a reflection about the potential ATTR market size. Over the past two years, considerable advancements in ATTR treatment options have been developed by Pfizer (PFE) and Alnylam Pharmaceuticals (ALNY). Just a couple years ago, patients with ATTR had virtually no effective treatment options. Now patients have 2 FDA approved treatment options. Even if Intellia produces the most effective treatment option, it will still face stiff competition for a small patient population. Regeneron likely made this decision based on pure economics and not as a lack of confidence.

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ความสำเร็จจากการลงทุน ไม่ได้เกิดจาก "การซื้อของดี" แต่มาจาก "การซื้อของได้ดี" ต่างหาก


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AnieLee
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Re: NTLA - Intellia Therapeutics, Inc. : US

Posts by AnieLee » Sat Jan 02, 2021 2:05 pm


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ความสำเร็จจากการลงทุน ไม่ได้เกิดจาก "การซื้อของดี" แต่มาจาก "การซื้อของได้ดี" ต่างหาก


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AnieLee
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Re: NTLA - Intellia Therapeutics, Inc. : US

Posts by AnieLee » Sat Jan 02, 2021 3:51 pm


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ความสำเร็จจากการลงทุน ไม่ได้เกิดจาก "การซื้อของดี" แต่มาจาก "การซื้อของได้ดี" ต่างหาก


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AnieLee
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Re: NTLA - Intellia Therapeutics, Inc. : US

Posts by AnieLee » Wed Jan 06, 2021 8:27 am

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ความสำเร็จจากการลงทุน ไม่ได้เกิดจาก "การซื้อของดี" แต่มาจาก "การซื้อของได้ดี" ต่างหาก


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AnieLee
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Re: NTLA - Intellia Therapeutics, Inc. : US

Posts by AnieLee » Wed Jan 06, 2021 8:28 am

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Re: NTLA - Intellia Therapeutics, Inc. : US

Posts by AnieLee » Thu Jan 07, 2021 11:47 pm

Intellia Therapeutics gains after announcing its 2021 priorities
SA NewsToday, 8:55 AM

Genome editing company Intellia Therapeutics (NASDAQ:NTLA) provides 2021 milestones and strategic priorities:
Continued advancement of global Phase 1 study of NTLA-2001, a potentially curative single-course therapy for protein misfolding disorder transthyretin amyloidosis (ATTR).

Expects submitting an IND or IND-equivalent regulatory application for NTLA-5001 for the treatment of acute myeloid leukemia (AML), a rare form of cancer of the blood and bone marrow, in mid-2021.
Expects to submit a similar regulatory application for NTLA-2002 for the treatment of hereditary angioedema (HAE), a swelling disorder, in H2 2021.

Also says, it ended Q4 2020 with about $597M in cash, cash equivalents and marketable securities, and is expected to fund its anticipated operating expenses and capital expenditure requirements for at least the next 24 months.

Intellia shares up ~5% premarket.

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