Lots of good information in report. TAM for genome medicine est $4.8T-thx for posting. Companies in their coverage are BMRN buy $132 AVXS NR BLUE BUy $308 CRSP Buy $86
Note: this does appear like a first effort for GS as some clear inconsistencies and omissions are apparent. Example for SGMO which is the company I know best: Exhibit 8: Only BMRN and ONCE are highlighted as pursuing Hemophilia cures in the Liver. They did include SGMO is Exh 9 Exhibit 12 cell therapy landscape excludes SGMO completely despite their KITE deal though they do reference this deal in the section on allogeneic cell therapies Exhibit 13 states SGMO is preclinical for gene edit. Factually they are the only one in clinic and the worldwide media attention alone should have caught GS attention one would think Exhibit 22 licensing deals excludes the largest one signed with $3b in milestones Exhibit 24 makes no distinction between editing approaches for off target cuts and concludes CRISPR has an advantages in efficiency up to 80%, ignoring ZFN 99.5% and multiplex publications Several places the discussion of AD completely omits SGMO despite experts commentary such as: "Tau protein plays a critical role in certain devastating neurodegenerative diseases, including dementias such as Alzheimer's disease, and studies point to the potential for tau reduction to prevent neuronal loss and possibly even reverse Alzheimer's disease pathology," said Bradley Hyman, M.D., Ph.D., Director, Massachusetts Alzheimer's Disease Research Center and Alzheimer's Unit Director
Notable section We see a $4.8tn+ potential TAM for genome medicine We see a significant market opportunity for genome medicines that will both disrupt existing biopharma markets as well as create new profit pools (e.g. genetic inherited blindness, last-line cancer). In this section, we size the potential total addressable market of genome medicine in a ‘blue sky’ scenario and identify therapeutic areas with high growth potential, although these could expand on technology advancement. We estimate that the total addressable market for genome medicines could reach $4.8tn+, which reflects a ‘blue sky’ scenario based on various assumptions (see details below) and where genome medicines are successfully commercialized across a wide spectrum of common and rare genetic diseases.
Our calculation of TAM for genome medicines is based on the following methodology and key assumptions:
(1) Pricing for gene therapy/editing at $1mn per treatment (in line with Glybera, first gene therapy approved in the EU, which costs €1mn per treatment; we assume $100k for non-orphan indications) and cell therapy at $375k (based on the current price of GILD/Kite’s recently approved cell therapy Yescarta as a reference), and a one-time upfront full payment for treatment.
100% penetration into all patients — both the incident and prevalent patient pools — given the appeal of a one-shot cure. However, in practice, penetration will be lower in both populations.
TAM only captures US and EU5 patients — therefore the global TAM opportunity exceeds $4.8tn.
Our TAM includes ~$3.6tn derived from the prevalent pool, which we assume will be exhausted over time as patients are “cured”.
Based on our assumptions, the global cumulative TAM for genome medicine across all disease areas based on the current generation of technology platforms (gene therapy, editing and cell therapy) could reach $4.8tn, driven by oncology (>$1tn), neurology (>$1.5tn) and eye disorders (>$0.5tn). This compares with annual global prescription sales of $1.01tn projected in 2022 per independent third party estimates (EvaluatePharma). We see the commercial opportunity driven by both the creation of new profit pools, e.g. orphan disorders, as well as disruption to current therapies/markets, e.g. cancer, heart, neurology and viral infections. We note that a significant proportion of our estimated revenue pool is derived from prevalent patients, i.e. patients who already have the condition ($3.6tn), and once these are treated they are essentially “cured”. Therefore, in the long term, disease incidence, i.e. number of new patients born with or developing the disease, will be the primary driver of recurring sales, with oncology ($1.2tn) as the largest source. In addition, this scenario is also contingent on the continued optimization of gene therapy efficacy/safety, movement to earlier-line settings in cancer, and standardization and successful scaling of manufacturing of viral vectors to meet commercial demand.
We view several current large opportunities in genome medicine: targeted cancer therapy with genetically engineered immune cells and large market disorders with a single-gene defect. Given gene therapy and editing are initially being developed to replace, knock out or modify a single gene / mutation site, we see the near-term therapeutic potential of the technology revolving around orphan disorders (e.g. spinal muscular atrophy, Duchenne muscular dystrophy and sickle cell disease) and blood cancers. However, given the potential for CRISPR/Cas9 multiplexing (modification of several genes / mutations simultaneously) and improving understanding of the various genetic drivers in diseases such as cardiovascular disorders (e.g. stroke) and diabetes, we see room for upside as the technology is optimized - we do not include these diseases in our global TAM estimate of $4.8tn.
As gene therapy / editing matures, we see the technology expanding into largermarkets including potentially gene augmentation, in utero gene editing for fatal congenital diseases or even modification of germline cells (sperm and egg) to eliminate a disease-causing mutation in a population. We also view infectious diseases, particularly HIV, as amenable to gene editing as these viruses express specific DNA sequences that can be targeted by CRISPR/Cas9. Below, we conduct a deep dive on the genome medicine opportunities across multiple therapeutic areas.
Three healthcare stocks driving the gene therapy momentum These companies' pipeline of gene therapy products could create a long runway of revenue and profitability growth. By Vikram Barhat | 18/04/18 The recent announcement by Swiss pharmaceutical giant Novartis that it would acquire U.S.-based AveXis Inc. for US$8.7 billion brought gene therapy into sharp focus. Gene therapy, a rapidly growing slice of the overall healthcare market, uses human genes to treat or prevent disease. Simply put, it is a technique to fix or forestall an intractable genetic problem at its very source.
About the AuthorVikram Barhat is a Toronto-based financial writer specializing in investing, personal finance and small business. His experience working in various editorial capacities in digital and print media spans 15 years across three continents. He is also a former content editor of Advisor's Edge and Advisor.ca. He can be reached at firstname.lastname@example.org.
In the future, it's expected, this technique could replace drugs or surgery by treating a genetic disorder by inserting DNA directly into a patient's cells, according to Genetics Home Reference, a service of the National Library of Medicine (NLM), part of the U.S. Department of Health and Human Services.
Currently a handful of companies are working to develop drugs that modify human genes in a market that is projected to skyrocket from US$584 million in 2016 to about US$4.5 billion by 2023, growing at 33.3% annually, according to a global market study from Allied Market Research.
Spurred by the recent U.S. FDA nod to the therapy, these companies are pushing hard to stay ahead of the curve by cranking up product innovation -- through in-house research, collaborations and acquisitions -- to develop cures for a range of life threatening medical conditions including cancer, haemophilia, muscular degeneration and potentially to help astronauts survive extreme levels of radiation in space.
As gene therapy moves to the forefront of medicine, these companies are well positioned to benefit from a pipeline of existing and new gene therapy products that could create a long runway of revenue and profitability growth.
Pfizer Inc. Ticker: PFE Current yield: 3.58% Forward P/E: 12.4 Price: US$36.56 Fair value: US$43.50 Value: 16% discount Data as of April 16, 2018 Pfizer (PFE) is one of the world's largest pharmaceutical firms with over US$50 billion in annual sales, the majority of which are in prescription drugs and vaccines. Pfizer's products, which include top sellers Prevnar 13 -- a vaccine for meningitis -- and Lyrica for epilepsy and neuropathic pain, are sold globally, with international sales representing close to half of total sales.
Pfizer aims to build an industry-leading gene therapy portfolio through in-house capabilities, strategic tie ups, and merger and acquisitions. As part of that vision, the U.S. pharma behemoth recently collaborated with Sangamo Therapeutics to develop a gene therapy to treat amyotrophic lateral sclerosis (ALS), a disease that affects nerve cells in the brain and the spinal cord. Another partnership with Spark Therapeutics seeks to develop a gene therapy for haemophilia B, a rare blood disorder. Pfizer has been pouring millions of dollars into expanding its gene therapy research and development capabilities.
These initiatives are supported by strong cash flows generated from a basket of diverse drugs. The wide-moat drugmaker's financial resources and established research power "confer significant competitive advantages in developing new drugs," says a Morningstar equity report. "This unmatched heft, combined with a broad portfolio of patent-protected drugs, has helped Pfizer build a wide economic moat around its business."
The Viagra maker's portfolio includes several potential blockbusters in cancer, heart disease and immunology. "Pfizer's patent-protected drugs carry strong pricing power that enables the firm to generate returns on invested capital in excess of its cost of capital," says Morningstar sector director Damien Conover, who recently raised the stock's fair value from US$38 to US$43.50. "Further, the patents give the company time to develop the next generation of drugs before generic competition arises."
BioMarin Pharmaceutical Inc. Ticker: BMRN Current yield: - Forward P/E: 119.0 Price: US$82.94 Fair value: US$107 Value: 22.5% discount Data as of April 16, 2018 With a focus on cures for rare diseases, Biomarin (BMRN) markets therapies independently or through joint ventures.
The company, which has potential new therapies in various phases of clinical trials, is pushing to take things to a whole new level with gene therapy. Results of the recent clinical trials from BioMarin, and the prospect of a possible one-off cure for rare genetic conditions like haemophilia, has generated widespread hope and excitement in the medical community. Known as valoctocogene roxaparvovec, the treatment was granted Breakthrough Therapy status by the FDA, a program intended to speed up the development and review of new drugs.
"BioMarin is amassing a portfolio of genetic-disease therapeutics," says a Morningstar equity report, noting that while "commercialization and research and development expenses have kept BioMarin in the red, we're confident in the profit-generating power of its rare-disease treatments."
A deep in-house pipeline and the ability to supplement growth with strategic acquisitions place the company in a strong position. "BioMarin's life-saving therapies may serve only a few thousand patients globally, but with six-figure price tags on most products and high barriers to entry, we see this as a very attractive marketplace," says Morningstar sector strategist Karen Andersen, who pegs the stock's fair value at US$107.
Although only 3,000 people in the developed world are afflicted with the life-threatening disease MPS I, BioMarin's first drug for its treatment, Aldurazyme, carries a price tag of US$200,000 and is now a US$200 million product thanks to market monopoly. BioMarin is also well-positioned to treat the entire spectrum of patients with PKU, a metabolic disorder, for which no alternative drug therapies exist, says Andersen, who projects 20% average annual sales growth over the next five years, driven by international growth and ongoing and upcoming drug launches.
Novartis AG ADR Ticker: NVS Current yield: 3.67% Forward P/E: 15.3 Price: US$81.02 Fair value: US$87 Value: 6.9% discount Data as of April 16, 2018 Novartis (NVS) develops and manufactures branded drugs, generic pharmaceuticals, eye care products and consumer products. The company has a global footprint with the U.S. accounting for nearly a third of total sales.
The firm is betting big on the fast-evolving field of medical treatment through gene manipulation. It recently agreed to make a US$8.7 billion acquisition of gene-therapy company AveXis Inc. The deal comes close on the heels of another in which it agreed to pay Spark Therapeutics US$105 million upfront and $65 million in milestone payments for rights outside the U.S. to the latter's one-off gene therapy, Luxturna, to cure blindness. The firm's CEO, Vas Narasimhan, recently expressed his keenness to build a growing pipeline of gene therapies across therapeutic areas.
With entrenched position in multiple key healthcare businesses, the Swiss pharmaceuticals heavyweight is well-placed for steady long-term growth, says a Morningstar equity report. "Strong intellectual property supporting multibillion-dollar products, combined with an abundance of late-pipeline products, creates a wide economic moat," the report notes.
The wide-moat firm's pharmaceutical segment is poised for long-term growth fuelled by new pipeline products and existing drugs. "Novartis differentiates itself because of its sheer number of blockbusters, including Gilenya for multiple sclerosis, and Afinitor and Tasigna for cancer," says Conover, who appraised the stock's fair value to be US$87. As well, the company has generated a strong late-stage pipeline with recent launches of heart failure drug Entresto and immunology drug Cosentyx.
Novartis is positioned well to make a major acquisition, Conover notes, adding that "given the firm's lagging position in immuno-oncology, Bristol Myers represents an attractive target for the firm."
Gene Therapy Market Size Worth $39.54 Million by 2026 | CAGR 19.1%: Grand View Research, Inc. PRESS RELEASE PR Newswire Jul. 2, 2018, 05:15 AM SAN FRANCISCO, July 2, 2018 /PRNewswire/ --
The global gene therapy market size is expected to reach USD 39.54 million by 2026. Rising competition among manufacturers and high number of molecules in pipeline are supporting the growth of the market.
Gene therapy development is aimed to cure rare diseases and even some hereditary diseases, which are caused by a mutated or faulty gene. Moreover, ever-increasing need for new cures for orphan diseases and rising incidence of cancer caused due to mutations in genes are likely to stir up the demand for gene therapy.
As of early 2016, there were more than 1000 molecules in the pipeline in various clinical phases. However, around 76.0% of the molecules are in the developmental or preclinical stages and expected to hit the market in late 2020's.
A great number of large pharma/biotech players are estimated to acquire small firms as many have been trying to develop in-house expertise and build their own pipelines. This trend is anticipated to help the market gain tremendous traction over the coming years. Combination of gene therapy with small molecules or protein therapy is said to have lesser side effects and better efficacy as compared to gene therapy alone.
Cancer held the dominant share in the market in 2017 owing to relatively high adoption of gene therapies for cancer treatment. Continual rise in new cancer cases and related mortality per year triggers the need for development of robust treatment options. Ongoing developments in cancer gene studies have provided significant information about cancer-related molecular signatures, which in turn, is projected to support ongoing clinical trials for cancer therapeutics.
Browse full research report with TOC on"Gene Therapy Market Size, Share & Trends Analysis Report By Indication (Cancer, Genetic Disorders, Infectious Diseases, CVD, Neuro Disorders), By Vector Type (Viral, Non-Viral), By Region, and Segment Forecasts, 2018 - 2026"at:https://www.grandviewresearch.com/industry-analysis/gene-therapy-market
Further Key Findings From the Report Suggest:
More than 60.0% of the market is occupied by cancer research owing to a large pipeline. Adenoviral vectors are the most used in pipeline development, closely followed by retroviral vectors. Europe and the U.S. are poised to occupy the largest cumulative share in the market throughout the forecast period. Asia Pacific is set to post noteworthy CAGR during the forecast period, owing to growing pipeline molecules and a large number of companies competing in the market. Some of the key players in this market are UniQure N.V, Spark Therapeutics LLC, Bluebird Bio, Juno Therapeutics, GlaxoSmithKline, Celgene Corporation, Shire Plc, Sangamo Biosciences, Dimension Therapeutics, Voyager Therapeutics, Human Stem Cell Institute, Bristol Myer's Squibb, and Chiesi Farmaceutici S.p.A.
The diversity of the previous two market sizing examples seems pretty amazing. I don't have access to the reports so any explanation is pure speculation. That said, my guess is the first one was including milestone and upfronts being paid for collaborations signed. The later appears to be more tied to actual drug deliveries. Neither is particularly useful without further info.
Under: Been on the sidelines for a bit holding (building) cash. Now that "BIGLEY" has rolled out the tax plan its time to jump in.
Dec 21, 2017 19:06:02 GMT -6
martyc: Looks like you are buying Msft again!
Dec 15, 2017 11:23:29 GMT -6
martyc: The news that Trump called Rupert to congratulate him sure seems to indicate that this is heading to approval
Dec 15, 2017 11:22:23 GMT -6
Under: DIS finally getting some traction.?
Dec 14, 2017 17:08:45 GMT -6
martyc: I took an entry level position in DIS. Will add eventually to overweight when it becomes clearer that the deal will go thru. Can't believe how well positioned they will be. 60% Hulu. 20% of content watched on NFLX they can pull. More in thread
Dec 14, 2017 11:05:16 GMT -6
Under: Great posts on $DIS
Dec 13, 2017 17:50:49 GMT -6
Under: $ROKU Citron on a war path.
Nov 28, 2017 15:11:20 GMT -6
Under: $HAS takeover bid for $MAT?
Nov 10, 2017 16:16:07 GMT -6
martyc: Not looking like the market will provide any discounted opp for SGMO. Call was just too professional and all signs indicate they are on a great path for commercialization. Happy with core but wish I had some trading shs
Nov 10, 2017 9:04:05 GMT -6
martyc: For anyone looking to find an entry point into SGMO, I'm almost hoping is sells off in next few days so I can add more. They are really clicking but the fact they haven't signed new deals might cause some to exit. Watching as I have room for trading shs
Nov 9, 2017 18:28:09 GMT -6
martyc: Been an interesting ride so far. I figured the Bears would be about this good but hoped the O wouldn't look so lame. Another building yr but still possible to get to 8-8 IMO
Nov 9, 2017 18:26:08 GMT -6
Under: whats up with your Bears this year Marty?
Nov 9, 2017 17:35:25 GMT -6
martyc: Hope you were long ROKU. I wanted to see Q first so missed out
Nov 9, 2017 7:08:53 GMT -6