13 Feb New Age Therapeutics Offer Hope to Patients of Multiple Sclerosis
Multiple Sclerosis (MS) is one of the diseases that will be covered under the National Neurological Conditions Surveillance System (NNCSS), a system that is being built by the Centers for Disease Control and Prevention (CDC) as part of the 21st Century Cures Act 2016, which has finally received funding in 2019 after being approved by the Congress. As part of the initiative, it will collate data on areas of biggest impact for neurologic conditions such as incidence, age, race, ethnicity data, as well as geographic data.
A favorable government and regulatory environment, growing incidences of MS along with advancement in technology will propel the growth in the MS treatment Industry. However, delay in diagnosis, prohibitive treatment costs along with adverse side-effects will hamper its growth. The global multiple sclerosis treatment market is expected to grow at a CAGR of 3.5% during forecasted period 2017-2023 according to a report by ResearchAndMarkets.com which also predicts that the global multiple sclerosis drugs market which was valued at US$ 16.13 billion in 2016, is expected to reach US$ 27.38 billion by 2025, expanding at a CAGR of 6.3% from 2017 to 2025.
GlobalData’s report titled ‘Dynamic Market Forecast: Multiple Sclerosis’ projects that the MS market will grow in sales from $19,063 million in 2016 to $25,566 million in 2026 with a compound annual growth rate (CAGR) of 3%.
Multiple Sclerosis affects approximately 1 million individuals in the US and 2.5 million worldwide according to a study. The direct healthcare cost of this illness is estimated to be in excess of $10 billion annually in the U.S alone.
Though the exact cause of this disorder is as yet unknown, some companies are using the rapid advancements in technology to improve diagnosis, understand the cause and alleviate the symptoms that are characteristic of MS;
Here we take a look at some companies that have the potential to disrupt the MS Therapeutic Industry in the near future.
Alkermes (NASDAQ: ALKS)
Market Cap: $4.81B; Current Share Price: 30.92 USD
A biopharmaceutical company that is focused on addressing the needs of patients suffering from debilitating diseases, Alkermes is currently undertaking research on disorders of the Central Nervous System (CNS) such as Multiple Scelorsis, Schizophrenia, depression and addiction.
Alkermes along with Biogen Inc. has submitted a New Drug Application (NDA) to the FDA for diroximel fumarate (BIIB098), an innovative oral fumarate in development for the treatment of relapsing forms of multiple sclerosis. Biogen intends to introduce the product under the brand name VUMERITY™ if approved by the FDA.
The company’s forte is its long-acting technological platform namely Medisorb®, LinkeRx® and NanoCrystal® that enable the gradual release of small and macro molecules at a controlled rate over a monitored period of time which reduces the need for frequent medication and improves patient adherence. Its Oral Controlled Release technology allows it to customize the dosage of a drug to achieve immediate release; oral delayed release, extended release, pulsatile release, chono-timed release or a combination of the above, thereby overcoming the limitations of existing treatment options.
In addition, the company is also developing Nanoparticle Technology platforms called the NanoCrystal® technology and the NanOsmotic® technology that improve the bioavailability of drugs with poor water-solubility, reduce dosage volume and maximize dosage tolerance. The company is also working on drug candidates that target opioid receptors that have the ability to affect addiction, behavior and mood modification, immunology, and pain. It’s proprietary Medifusion™ technology deals with protein and peptides and modifies their circulating half-life to deliver long-acting injectable medications.
The company has dedicated GMP manufacturing facilities in Ohio and Ireland that are FDA / EMA approved.
The company has strategic collaborations with some prominent pharmaceutical companies namely Biogen, AstraZeneca (BYDUREON), Janssen Pharmaceuticals (RISPERDAL CONSTA®, INVEGA SUSTENNA® and INVEGA TRINZA®), Acorda Therapeutics, Inc (ampyra) and Acceleron Pharma.
As per the company’s Q3, 2018 financial results, total revenues for the quarter were $248.7 million as compared to $217.4 million for the same quarter in 2017, an increase of 14%. Non-GAAP net income was $11.6 million for the period. Cash and cash equivalents stood at $578,543 as against $590,716 for the previous year.
Atara Biotherapeutics (NASDAQ: ATRA)
Market Cap: $1.77B; Current Share Price: 38.87 USD
A pioneer in the T-cell immunotherapy space, Atara offers rapid delivery of Off-the-shelf bioengineered allogeneic T cells from donors with healthy immune function for the treatment of cancer, autoimmune and viral diseases. These can target cancerous and disease-causing cells with precision and eliminate them with minimal collateral cell damage. Most importantly there is no need for lymphodepletion before treatment.
The company is leveraging its decade long clinical experience at Memorial Sloan Kettering (MSK) by using cell selection algorithm to identify the most appropriate T-Cell lines by matching key immune characteristics.
ATA188 and autologous ATA190 T-cell immunotherapies, off-the-shelf allogenic cells that use a corresponding targeted antigen recognition technology for specific EBV antigens, are being developed by Atara as a possible treatment option for multiple sclerosis (MS). The company is currently conducting clinical trials of autologous ATA190 in patients with progressive MS, along with a multinational phase I ATA188 clinical study in patients with progressive or relapsing-remitting MS in Australia and the U.S.
The company has created T-Cells to recognize EBV and CMV Viral antigens and the tumor associated antigen, Wilms tumor 1 (WT1) using technology licensed from Memorial Sloan Kettering (MSK) and developed product candidates namely ATA129, ATA230, and ATA520. It has a licensing agreement with QIMR Berghofer for the use of its T-Cell technology that can target selective antigen for specific EBV, CMV, HPV, and BK viral antigens and is developing product candidates ATA188, ATA621, ATA368, and ATA274 on the basis of this technology.
The company is fully focused on CAR T opportunities and is developing numerous next generation and off-shelf technology options that cover a gamut of targets and diseases, overcome resistance and have an improved safety profiles. It has collaboration with Moffitt Cancer Centre and MSK to develop multi-targeted CAR T Immunotherapies.
The company has a state of the art expandable ATOM (Atara T-Cell Operations and Manufacturing) facility in California which is currently completing licensure for clinical production to confirm to global regulatory standards.
Atara has a strong pipeline of product candidates in clinical development stage such as Tab-cel® (tabelecleucel) for treatment of hematologic and solid tumors, Solid Tumors, Acute Myeloid Lukemia, B-Cell Malignancies, Autoimmune disease and Infectious disesase CAR T programs to name a few.
The company has exclusive licensed global rights to a mesothelin-targeted chimeric antigen receptor T-cell (CAR T) immunotherapy for solid tumors from Memorial Sloan Kettering Cancer Center (MSK) as per a company press release.
As per the company’s Q3, 2018 financial results cash, cash equivalents and short-term investments as on September 30, 2018 totalled $364.5 million.
Momenta Pharmaceuticals (NASDAQ: MNTA)
Market Cap: $ 1.24B; Current Share Price: 12.65 USD
The company developed a first-ever FDA approved complex generic product Glatopa® (glatiramer acetate injection), a generic version of daily COPAXONE® 20 mg/ml in collaboration with Sandoz, a novel AP-rated substitutable generic for treatment of patients with relapsing-remitting multiple sclerosis (RRMS) in 2015. In 2018, the company’s collaboration partner Sandoz initiated an Abbreviated New Drug Application for Glatopa (glatiramer acetate injection) 40 mg/mL which was approved by the FDA and was consequently launched in the US.
Momenta is a biotechnology company that is using inventive approaches to understand the structure and biology of complex molecules in order to treat rare immune-related disorders. Its high resolution analytics enable it to understand the structural components of complex molecules to unravel structure-process relationships. By combining an orthogonal approach to studying multiple sets of data, it is able to re-engineer complex molecules. The company’s focus areas are complex generics, biosimilars and novel drugs for autoimmune indications.
The company has entered into various collaborations as part of its growth strategy such as a licensing agreement with Sandoz for the first generic version of LOVENOX® (enoxaparin sodium injection) to be approved by the FDA. It also has a collaboration and licensing agreement with Sandoz for Glatopa® , the first fully substitutable once daily generic equivalent of daily COPAXONE® 20 mg/mL, in 2018 Sandoz received FDA approval of their Abbreviated New Drug Application for a three times weekly COPAXONE® 40mg/mL (glatiramer acetate injection) for treatment of relapsing-remitting multiple sclerosis (RRMS).
In addition, it has a worldwide collaboration with Mylan for its biosimilar candidates, which it proposes to exit owing to corporate restructuring and its need to focus on M710. It has an exclusive research collaboration and global licensing agreement with CSL to develop and commercialize Fc multimer proteins.
In the pipeline are novel therapeutics and proposed biosimilars to Adalimumab (HUMIRA) and Aflicbercept (EYLEA), Anti FcRn Antibody, Recombitant Fc Multimer, Hyper-sialylated IgG
The company received gross proceeds of approximately $230 million on account of its underwritten public offering of 20,000,000 shares of its common stock at the price of $11.50 per share.
The company reported a $13.6 million in product revenues from Sandoz’s sales of Glatopa 20 mg/mL and 40 mg/mL products compared to $10.9 million in profit share from Sandoz sales of Glatopa 20 mg/mL for the same period in 2017, in its Q3 2018 financial report. Total revenues for the third quarter of 2018 were $14.9 million compared to $24.1 million for the same period in 2017.
As of September 30, 2018, Momenta had $281.6 million in cash, cash equivalents and marketable securities compared to $321.2 million as on June 30, 2018.
TG Therapeutics (NASDAQ: TGTX)
Market Cap: $337.27M; Current Share Price: 4.39 USD
The company seeks to offer novel treatments for B-Cell malignancies and autoimmune diseases and is currently engaged in developing therapeutics for hematological malignancies and autoimmune diseases.
Its novel Ublituximab (TG-1101), a glycoengineered monoclonal antibody that specifically targets unique epitope on the CD20 antigen found on mature B-lymphocytes, is also undergoing Phase 3 clinical trials for treatment of multiple sclerosis. . It is also developing Umbralisib (TGR-1202), a PI3K delta inhibitor for various hematologic malignancies made available in the oral form.
In the pipeline are TG-1501, an anti-PD-L1 monocloncal antibody and TG-1701 its covalently-bound Bruton Tyrosine Kinase (BTK) inhibitor that are currently under Phase 1 development.
In January, the company’s Umbralisib (TGR-1202) received Breakthrough Therapy Designation from the FDA for the treatment of adult patients with marginal zone lymphoma (MZL) who have been administered at least one prior anti-CD20 regimen.
In the words of the company’s CEO and Executive Chairman Michael S. Weiss, “We are pleased by the progress made in the third quarter of 2018, most notably the completion of full enrolment in the current cohorts of the UNITY-NHL trial, as well as in the ULTIMATE Phase 3 program in MS. We now have five Phase 3 or registration directed trials fully enrolled across our three major indications of interest, CLL, NHL and MS, and look forward to significant value creating data releases in 2019 and 2020.” Mr. Weiss, stated that, “This is just the beginning for TG as we solidify the foundation and look towards the future of building proprietary triple combination therapies with our in-house early stage pipeline.”
The company has completed enrollment into the ULTIMATE Phase 3 program in MS. It has a Special Protocol Assessment (SPA) agreement with the FDA for evaluating ublituximab in relapsing form of MS.
TG Therapeutic’s cash, cash equivalents, investment securities, and interest receivables were $97.8 million as on September 30, 2018 as per its Q3 2018 Financial Results.
GW Pharmaceuticals (OTCPK: GWPRF)
Market Cap: $ 338.46M; Current Share Price: 11.90 USD
GW’s aim is to be the world leader in prescription cannbinoid medicines, which it continues to develop and commercialize to meet unfulfilled medical requirements.The company, redefined the plant-based medicine space by introduction of plant-derived cannabinoid therapeutic named “Epidiolex”, a purified cannabinoid to be taken in the oral form for seizures associated with Lennox-Gastaut syndrome or Dravet syndrome. This is the first and only FDA approved cannabinoid medicine. The company’s subsidiary, Greenwich Biosciences Inc., handles its operations and marketing activities in the U.S.
Sativex® (delta-9-tetrahydrocannibinol and cannabidiol in the EU; nabiximols in the US) was the first drug from the company’s stable for the treatment of spasticity, neuropathic pain and bladder control due to multiple sclerosis. It is a prescription only medicine that is available in the form of a Oromucosal spray. This drug is now approved in over 25 countries all over the world and is sold through the company’s marketing partners. It is focused on further research and development on Sativex for use in neuropathic pain and other neurological conditions as it sees a potential to use it for the treatment of multiple indications.
The company’s lead product is the liquid formulation of highly purified CBD. The company is currently undertaking various clinical studies to study the application of CBD in areas such as Autism, Schizophrenia, Rett Syndrome, Glioblastoma and Neonatal Hypoxic-Ischemic Encephalopathy (NHIE).
The company is looking forward to the start of its Phase 3 MS Study to be launched in the second half of 2019.
As per the company’s year-end financial results, cash and cash equivalents as on September 30, 2018 were $354.9 million compared to $322.2 million as on September 30, 2017. Revenue for the year ended September 30, 2018 was $12.7 million compared to $8.6 million for the year ended September 30, 2017.
The company made a public offering on NASDAQ of 1,900,000 American Depositary Shares (“ADSs”) at a price to the public of $158.00 with an option to the underwriters to purchase 285,000 additional ADSs from GW. This equity financing resulted in net proceeds of $324.2 million. The company has also received approval from major coverage policies such as Express Scripts which has 30 million accounts. In fact 4 out of 5 U.S payers have agreed to cover Epidiolex, which suggests that its US$32,500 per annum is not deemed high.
Disclosure: I/we have no positions in any stocks mentioned, and no plans to initiate any positions within the next 72 hours.