Syncona Share Discussion Threads

[chc15] 27/1/2022 10:07

Having a small nibble here on each big drop, will dump if goes back to 2.

[bbonsall] 25/1/2022 17:15

What is happening here after their recent sale and a valuation of more than 220p?

[chc15] 24/1/2022 10:26

Battered, good entry point, or wait..

[robow] 06/1/2022 14:21

tipped in The Telegraph today as their Investment Trust of the year

Questor: this trust just sold a holding for £600m – and its entire market value is only £1.4bn
Questor investment trust bargain: it’s not the first big success for Syncona, the life sciences incubator, and we expect plenty more to come

By
Richard Evans
6 January 2022 • 6:00am


On Wednesday we chose a healthcare company as our stock tip of the year, on the basis not only of our expectations of its own recovery but because of investors’ recent aversion to the sector as a whole. We will double down on that belief today and pick our investment trust of the year from the same arena.

Our choice, Syncona, is an unusual beast: it invests in young life sciences companies with a view to holding them through the various stages of drug discovery, clinical trials, commercialisation and perhaps an eventual stock market listing.

This is not the first time we have tipped the trust; we first did so in 2018 and have reiterated the advice since. Some readers will not thank us as the shares have lost 22.7pc since that first tip. Why then are we backing the fund again?

Simply because we think the market fails to see its potential or perhaps does not have the patience to wait for that potential to become apparent in actual profits. As we said on Wednesday, generating returns from the scientific breakthroughs at which the healthcare sector excels takes time, while many investors have made fortunes over the past couple of years from spotting the immediate bounty on offer from Covid winners such as Microsoft.

Syncona’s portfolio currently consists of 12 companies and it aims to increase that number to between 15 and 20. Several can offer concrete evidence of progress: collectively 12 of their treatments are undergoing clinical trials and three have already floated on the stock market.

Of four companies no longer in the portfolio, two, Blue Earth and Nightstar, were sold for impressive gains of 9.9 and 4.5 times the amount invested respectively, while the other two proved unviable and were wound up. A 50pc success rate is good when you are seeking to build businesses from nothing in a sector that teems with scientific, regulatory and commercial risks.

But it is the fund’s most recent deal that cements our belief that its managers are equal to the challenge and can be expected to produce a regular stream of lucrative winners.

Just before Christmas Syncona announced that it had agreed to sell its 48.5pc stake in one of its holdings, Gyroscope Therapeutics, which makes gene therapies for blindness, for up to £589m. The fund had co-founded Gyroscope in 2016 and funded it since but the really striking thing is how that £589m figure, admittedly the maximum proceeds, compares with the value that the market currently ascribes to the entire trust: just £1.4bn.

Investors seem to be expressing scepticism that any other holdings, current or future, will generate returns along the lines of those achieved by Gyroscope. In view of that 50pc success rate on investments already exited, such a view seems very pessimistic to this column.


Or perhaps the market was spooked by what seemed, before this deal emerged, to be a lofty premium on the fund of 29pc, or a sky-high 69pc implied on the unquoted part of the portfolio. Gyroscope’s sale brings the overall premium down to 7.3pc and that on the unquoted part to 26pc, according to calculations carried out by JP Morgan Cazenove, the broker, on the day the deal was announced.

Questor’s view is simple: the sales of Gyroscope, Blue Earth and Nightstar, along with the flotations of other holdings such as Autolus, show that this fund knows its business. We therefore expect similar success stories in future and the generation of returns that will comfortably exceed its current market value.

The only proviso is that it won’t happen overnight and so readers will have to show more patience than perhaps with other Questor tips. As we have said before, it’s one to buy and tuck away.

Questor says: buy

Ticker: SYNC

[sev22] 06/1/2022 14:13

Freeline Announces FDA Clearance of Investigational New Drug Application for FLT201 for Gaucher Disease Type 1:

FLT201 is the first AAV gene therapy program to enter the clinic for Gaucher disease Type 1.

FLT201 Phase 1/2 trial for Gaucher disease Type 1 on track for patient dosing in the first half of 2022.

LONDON, January 6, 2022 – Freeline Therapeutics Holdings plc (Nasdaq: FRLN) (the “Company”; or “Freeline̶1;), a clinical-stage biotechnology company developing transformative AAV-mediated gene therapies for patients suffering from inherited systemic debilitating diseases, today announced that the U.S. Food and Drug Administration (FDA) has cleared its Investigational New Drug (IND) application for FLT201 as an investigational gene therapy for the treatment of Gaucher disease Type 1.

“The FDA clearance of this IND is an important milestone for FLT201, which is the first AAV-mediated gene therapy for patients with Gaucher disease Type 1 in the clinic,” said Michael Parini, Chief Executive Officer of Freeline. “Our FLT201 program harnesses our unique scientific platform capabilities – our highly potent, proprietary AAVS3 capsid, robust CMC and pre-clinical data across all our programs, and our advanced protein engineering capabilities – to develop a potentially transformative treatment for patients suffering from Gaucher disease. The entry of our third program into the clinic is important validation of the advantage of Freeline’s portfolio approach to development.”

“This IND clearance is an important step toward bringing FLT201 to patients and is made possible by the extended safety database across our proprietary platform,” said Dr. Pamela Foulds, Chief Medical Officer of Freeline. “Our extensive preclinical proof-of-concept studies suggest FLT201 has the potential to reach difficult to treat tissues, such as bone marrow and lung, which are not sufficiently addressed by standard-of-care enzyme replacement therapy. These data, along with the promise of sustained, endogenous production of GCase following a one-time intravenous infusion, suggest that treatment with FLT201 may be a transformative treatment that can significantly reduce or eliminate the need for enzyme or substrate replacement therapy.”

FLT201 consists of a potent, rationally designed AAV capsid (AAVS3) containing an expression cassette that encodes for a novel glucocerebrosidase variant (GCasevar85) under the control of a liver-specific promoter. The GCasevar85 contains two novel amino acid substitutions to the wild-type human GCase, which results in a 20-fold increase in GCase half-life at lysosomal pH conditions, but similar catalytic parameters to those of wild-type GCase and enzyme replacement therapy (ERT).

Freeline initiated the Phase 1/2 dose-finding trial of FLT201 at the end of 2021 in Europe and expects to dose two patients in the first dose cohort in the first half of 2022, with initial safety and biomarker data from the first cohort expected in Q3 2022. The Company expects to report data on all dosed patients, including those dosed in Q3, prior to year-end 2022.

About Freeline Therapeutics

Freeline is a clinical-stage biotechnology company developing transformative adeno-associated virus (AAV) vector-mediated systemic gene therapies. The Company is dedicated to improving patient lives through innovative, one-time treatments that provide functional cures for inherited systemic debilitating diseases. Freeline uses its proprietary, rationally designed AAV vector, along with novel promoters and transgenes, to deliver a functional copy of a therapeutic gene into human liver cells, thereby expressing a persistent functional level of the missing or dysfunctional protein into the patient’s bloodstream. The Company’s integrated gene therapy platform includes in-house capabilities in research, clinical development, manufacturing, and commercialization. The Company has clinical programs in hemophilia B, Fabry disease, and Gaucher disease Type 1. Freeline is headquartered in the UK and has operations in Germany and the US.

About Gaucher Disease

Gaucher disease is a genetic disorder in which a fatty substance called glucosylceramide accumulates in macrophages in certain organs due to the lack of functional glucocerebrosidase (GCase). The disorder is hereditary and presents in various subtypes. Freeline is currently focused on Gaucher disease Type 1, the most common type, which impacts the health of many organs of the body including the spleen, liver, blood system and bones. The current standard of care is intravenous infusion of ERT every two weeks, which is a significant treatment burden on the patient.

[robow] 22/12/2021 14:40

thanks SEV22

[sev22] 20/12/2021 14:26

Achilles Therapeutics Added to the NASDAQ Biotechnology Index.

LONDON, Dec. 20, 2021 -- Achilles Therapeutics plc (NASDAQ: ACHL), a clinical-stage biopharmaceutical company developing precision T cell therapies to treat solid tumors, today announced that the Company has been added to the NASDAQ Biotech Index (Nasdaq: NBI), effective prior to market open today, Monday, December 20, 2021.

The NASDAQ Biotechnology Index is designed to track the performance of a set of securities listed on The Nasdaq Stock Market® (Nasdaq®) that are classified as either biotechnology or pharmaceutical according to the Industry Classification Benchmark (ICB). The NASDAQ Biotechnology Index is calculated under a modified capitalization-weighted methodology. Companies in the NASDAQ Biotechnology Index must meet eligibility requirements, including minimum market capitalization, average daily trading volume and seasoning as a public company, among other criteria. NASDAQ selects constituents once annually in December.

[sev22] 09/12/2021 15:07

Published today:

Achilles Therapeutics Presents Positive Data at ESMO I-O Congress 2021 on High-Dose Manufacturing Process for Precision T Cell Therapies Targeting Clonal Neoantigens.

Over 10-fold dose increase at GMP scale with VELOS™ Process 2 manufacturing.

cNeT maintain CD4+ and CD8+ T cell subsets with a highly potent polyclonal phenotype.

Dosing of patients with high-dose cNeT expected in 1H 2022.

LONDON, Dec. 09, 2021 (GLOBE NEWSWIRE) -- Achilles Therapeutics plc (NASDAQ: ACHL), a clinical-stage biopharmaceutical company developing precision T cell therapies to treat solid tumors, today presented positive data at the ESMO Immuno-Oncology Congress 2021 (ESMO I-O) that further demonstrate that Achilles’ VELOS™ Process 2 manufacturing increases clonal neoantigen-reactive T cell (cNeT) doses by more than 10-fold over Process 1 at GMP scale and maintains a highly potent polyclonal phenotype. These data add to the pilot scale proof-of-concept study recently reported at the 2021 Society for Immunotherapy of Cancer (SITC) Annual Meeting.

“We are extremely pleased to have demonstrated the ability to generate a significant boost in cNeT dose over Process 1 in clinical-scale runs and to have successfully completed the technology transfer of Process 2 into clinical manufacture,” said Dr Ed Samuel, SVP Technical Operations of Achilles. “These Process 2 GMP data validate our previously reported R&D data and confirm retention of critical phenotypic characteristics and the ability to identify the active drug component of our products without adding to end-to-end manufacturing time. We anticipate dosing patients with high-dose Process 2 cNeT in the first half of 2022, with 6-week clinical and translational science data available in the second half 2022.”

Key highlights from the presentation entitled “Achilles VELOS Process 2 generates a >10-fold improvement in cNeT dose over Process 1 with a highly potent polyclonal phenotype and has been successfully validated at GMP scale for clinical use in solid cancer,” include:

Reporting that GMP validation of VELOS Process 2 has successfully been completed and transferred into clinical manufacture in Achilles’ ongoing Phase I/IIa CHIRON and THETIS clinical trials.

Showing that VELOS Process 2 generates a cell product that retains both CD4+ and CD8+ T cell subsets and maintains polyclonal cNeT reactivity with a favorable cell fitness phenotype.

Demonstrating the quantification of the active cNeT drug component with Achilles’ proprietary potency assay which further underlines the strength of the Achilles platform.

About Achilles Therapeutics
Achilles is a clinical-stage biopharmaceutical company developing precision T cell therapies targeting clonal neoantigens: protein markers unique to the individual that are expressed on the surface of every cancer cell. The Company has two ongoing Phase I/IIa trials, the CHIRON trial in patients with unresectable locally advanced and metastatic non-small cell lung cancer (NSCLC) and the THETIS trial in patients with recurrent or metastatic melanoma. Achilles uses DNA sequencing data from each patient, together with its proprietary PELEUS™ bioinformatics platform, to identify clonal neoantigens specific to that patient, and then develop precision T cell-based product candidates specifically targeting those clonal neoantigens.

[chc15] 09/12/2021 09:14

What happened here, rose for no reason, then plummeted.

[sev22] 14/11/2021 16:12

The Syncona Investment Trust, a member of Interactive Investor’s ACE 40 rated list of ethical investments, has reported its latest half-yearly results (11th November 2021):

Syncona Ord (SYNC) investment trust, which invests in life science companies, reported a decline in net asset value of 11.4% for the first six months of its financial year – from the end of March to the end of September.

Over this period, the trust reported that its net assets sat at £1.15 billion, down from £1.3 billion at the end of March 2021.

Figures from FE Analytics show that over this period its share price total return was a loss of 33.6%, suffering from its high premium notably declining.

The decline, the report notes, was driven predominantly by the decline in the share price of two of the trust’s listed holdings, Freeline Therapeutics Holdings ADR FRLN
and Achilles Therapeutics ADR ACHL.

Freeline’s poor performance is put down to “operational challenges” owing to the Covid-19 pandemic. Syncona says these concerns have now been addressed.

Achilles’ share price decline was driven by “market sentiment towards cell and gene therapies”. Syncona says that it believes the business is performing well and in line with expectations.

The trust’s strategy is to establish, build and fund companies to turn exceptional science into a dynamic portfolio of global leaders in life sciences. The aim is for the companies to deliver their product to market. The trust has a long-term target of owning between 15 and 20 companies. It currently has 10.

Martin Murphy, chief executive of Syncona Investment Management Limited, said: “While we are disappointed by the decline in NAV during the period, we are continuing to build a diverse portfolio across the development cycle and therapeutic areas and remain confident in our companies' potential. The substantial capital that a number of our companies have accessed so far this year validate the significant opportunity ahead for them.”

The trust also said that it planned to deploy another £100 million to £175 million into both existing companies and new opportunities this year.

Murphy said: “With clinical data the key driver of value and risk for Syncona, we believe our companies are well positioned and on track to further validate our model and strategy in the next 12 months with the potential for a rich seam of data.”

The trust is a member of Interactive Investor’s ACE 40 rated list of ethical investments. It was placed under formal review in the summer due to the volatility of its share price, but it kept its place on the list.

Dzmitry Lipski, head of funds research at Interactive Investor, notes: “We remain positive on the trust outlook and see it as a strong choice for adventurous investors prepared to tolerate high volatility in the short term, but potentially reap rewards over the longer term.

[sev22] 08/11/2021 19:36

This was before today's two positive announcements.

[the shuffle man] 08/11/2021 13:59

Two well received announcements today and increased news flow over the last few weeks has certainly moved this off the bottom. Great track record here worth backing IMO.

[sev22] 08/11/2021 10:03

Peel Hunt Limited have a new price target of £2.79.

[chc15] 29/10/2021 15:44

Pretty decent recovery here, must be strong institutional support.

[sev22] 22/10/2021 15:01

Achilles Therapeutics Presents Data at the 2021 European Society for Gene and Cell Therapy (ESGCT) Congress Demonstrating its Proprietary Manufacturing Process Can Generate Potent, Personalized Anti-Cancer Cell Therapy Candidates in Multiple Tumor Types (22nd October 2021).

Achilles Therapeutics plc (NASDAQ: ACHL), a clinical-stage biopharmaceutical company developing precision T cell therapies to treat solid tumors, today delivered an oral presentation (OR54) at the 2021 European Society for Gene and Cell Therapy (ESGCT) Congress. In the presentation entitled ‘Multicentre, prospective research protocol for development of a clonal neoantigen-reactive T cell (cNeT) therapy pipeline across multiple tumour types,’ Dr. Michael Grant, Associate Medical Director at Achilles, reviewed initial data from the Company’s Material Acquisition Platform (MAP)​ showing that Achilles’ proprietary VELOS™ manufacturing process is able to extract tumor infiltrating lymphocytes (TIL) and generate potent clonal neoantigen-reactive T cells (cNeT) across a range of solid tumor types. cNeT target clonal neoantigens, which are unique proteins expressed on every cancer cell within a patient but not on healthy tissue.

MAP is a unique prospective study that facilitates the procurement of patient material across a range of solid tumor types, enabling a comprehensive evaluation of indications prior to clinical development. MAP was developed to enable the detailed genomic and cellular characterization of different cancer tissue types and assess the ability to produce significant doses of potent cNeT. The study is currently collecting patient material at eight sites in the United Kingdom, European Union, and United States from patients with a range of cancers including lung, melanoma, head and neck, renal, bladder and breast cancer.

“This important research shows that our bioinformatics and manufacturing processes together produce fit, potent and neoantigen-specific cells and supports the potential use of our cNeT in a broad range of solid tumor indications beyond our current clinical trials in non-small cell lung cancer (NSCLC) and metastatic melanoma, and into indications including head and neck cancer,” said Dr Sergio Quezada, Chief Scientific Officer of Achilles. “MAP showcases the analytical strength of the Achilles platform, delivers vital translational science insights, and highlights our organizational and supply chain expertise. It has been extremely successful in accumulating a broad set of tumour-related material and continues to expand into new countries and indications.”

Data presented show that the VELOS manufacturing process delivers higher neoantigen-specificity and potency, in both CD4+ and CD8+ T cell activity, relative to traditionally manufactured TIL products from the same tumor source material. These enhancements can be seen in specificity and potency assays in which IFN-γ and TNF-α cytokine secretion and production of inflammatory cytokines is triggered in response to clonal neoantigen presentation. Additionally, analysis of CD4+ and CD8+ cells in cNeT products shows favorable cell phenotypes consistent with high cell fitness and reduced cell exhaustion. At the time of submission, 74 patient samples had been processed across five solid tumor indications. The proprietary PELEUSTM platform identified a median of 107 clonal neoantigens in NSCLC samples, 156 in melanoma samples, and 71 in head and neck squamous cell carcinoma samples, consistent with publicly available data sets, illustrating the accuracy of the platform. Additional data for renal and bladder tumor samples were still in process at the time of submission.

The presentation delivered by Dr. Grant is available in the Events & Presentations section of the Achilles Therapeutics website.

About Achilles Therapeutics
Achilles is a clinical-stage biopharmaceutical company developing precision T cell therapies targeting clonal neoantigens: protein markers unique to the individual that are expressed on the surface of every cancer cell. The Company has two ongoing Phase I/IIa trials, the CHIRON trial in patients with unresectable locally advanced and metastatic non-small cell lung cancer (NSCLC) and the THETIS trial in patients with recurrent or metastatic melanoma. Achilles uses DNA sequencing data from each patient, together with its proprietary PELEUS™ bioinformatics platform, to identify clonal neoantigens specific to that patient, and then develop precision T cell-based product candidates specifically targeting those clonal neoantigens.

[acuere] 22/10/2021 10:52

Return of interest here for last couple of weeks. I was looking for 160p, so not tempted at this level to buy back though. The last reported results gave NAV of 178p as at 30 Jun. The share price was about 200p so trading at a 13% premium.
At that time Freeline was trading at $8.15 and Autolus was $6.64. They are currently $3.30 and $5.79 respectively. Both are 12-14% down in the last month. So we are likely to see a NAV to end Sept of around 160p. So, by my reckoning it’s currently trading at a 17% premium to NAV.
Not justified imho. I’ll wait to see results and look for Autolus and Freeline to reverse their downward trends before buying again.