Boston, MA, United States

Rhythm Pharmaceuticals

www.rhythmtx.com
Boston, MA, United States
SEARCH FILTERS
Time filter
Source Type

News Article | May 8, 2017
Site: www.businesswire.com

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Agilis Biotherapeutics, Inc. (Agilis), a biotechnology company advancing innovative gene therapy products for the treatment of rare genetic diseases that affect the central nervous system (CNS), announced today that the Company has expanded its commercial and medical teams, hiring Markus Peters, Ph.D., as Chief Commercial Officer, Kirsten Gruis, M.D. as Chief Medical Officer, and Anne Marie Conway, M.H.A, R.N., as Vice President Clinical Operations. “We are pleased to welcome these three talented individuals to our leadership team. Each brings a wealth of experience to Agilis that is directly aligned with our mission to help patients with rare CNS diseases, advance our clinical pipeline, and lay the foundation for future approval and commercialization of our promising gene therapy product candidates,” said Dr. Mark Pykett, President and CEO of Agilis. Dr. Markus Peters will lead Agilis’ commercial, business development and business analytics activities, and will spearhead market efforts for the company’s aromatic L-amino acid decarboxylase (AADC) deficiency gene therapy globally. He brings a significant background to Agilis in the commercialization of rare disease therapeutics and specialty pharmaceuticals. Most recently, he was an Associate Partner with the consulting group Alacrita. Dr. Peters was previously Vice President, Global Marketing/Commercial with Synageva, where he led the cross-functional global launch team for first-in-class enzyme replacement therapy Kanuma to develop and implement the global strategy and launch plan for the product in ultra-rare LAL deficiency. He was also responsible for the commercial assessment of the Synageva pipeline. Before Synageva, he was Head of Global Marketing Nephrology and Transplant Therapeutic Area at Alexion, leading the global launch of the ultra-orphan Soliris aHUS (atypical hemolytic uremic syndrome) franchise. Dr. Peters previously worked at Merck where he led the global launch of recombinant biologic Elonva, and at Sepracor, Wyeth, Bayer and Boehringer Ingelheim in the US, Japan and Europe in business and commercial roles of increasing responsibility. He holds a Ph.D. in Biochemistry from Heinrich-Heine Universität. Kirsten Gruis, M.D., is an accomplished physician scientist, board certified neurologist, and rare disease specialist, with a broad background in the development of innovative therapeutics. She has worked in Friedreich’s ataxia, Spinal Muscular Atrophy (SMA), Amyotrophic Lateral Sclerosis (ALS) and Duchenne Muscular Dystrophy (DMD), among others, across a range of development stages, including pre-clinical, Phase I, Phase II and Phase III programs. Dr. Gruis was most recently at WAVE Life Sciences leading their clinical development plans in DMD. Previously, she was at Idera Pharmaceuticals where she lead the team to initiate a global, phase II trial in dermatomyositis and, before that, she was at Alnylam Pharmaceuticals as the clinical lead of a global, phase III program of patisiran in the rare disease familial amyloidotic polyneuropathy and, before that, was with Pfizer managing pre-clinical and phase I-II assets in the Rare Disease Research Unit focused on Friedreich’s ataxia, SMA, ALS, and DMD. Dr. Gruis held academic appointments as an Associate Professor of Neurology at both the University of Michigan and SUNY Upstate Medical University. She received her MD from the University of Iowa and did her residency training at the University of Michigan, where she subsequently joined the faculty. While at SUNY Upstate Medical University, she served as the Director of the MDA clinic, Co-Director of the ALS Clinic, and prior to that was Director of the Motor Neuron Disease Center/ALS Clinic at the University of Michigan. Dr. Gruis is a member of the American Academy of Neurology and World Muscle Society, as well as a Fellow American Association of Neuromuscular & Electrodiagnostic Medicine with additional board certification in Neuromuscular Disorders. She has served on multiple NIH Scientific Review Panels for the NINDS and Neurotechnology study groups as well as a principal investigator of several clinical studies for ALS. Anne Marie Conway, M.H.A., R.N., brings extensive experience in clinical operations to Agilis’ clinical development programs. Most recently, she was Principal at AMC Consulting, providing clinical operations services to a range of drug development organizations including Rhythm Pharmaceuticals, bluebird bio and Lantheus Medical Imaging, among others. Before that, she worked at Ziopharm Oncology as head of Clinical Operations and Data Management. While there, she managed the start-up of the gene therapy program for high grade gliomas. Prior to that, she was at Shire Human Genetic Therapies (now integrated into Shire, plc) as Vice President, Development Operations, providing management oversight for the global filing and approval of VPRIV™ for Gaucher disease, running a global Phase III trial and subsequent approval of Firazyr™ for hereditary angioedema and, leading global clinical operations, data management and registry group for eight rare disease pipeline products in Phases I through IV studies. Prior to its acquisition by Shire, Ms. Conway worked at Transkaryotic Therapies and led the integrated development team for the clinical sections of the BLA/MAA filing and subsequent approval in the United States, European Union, and Japan for elaprase™. Before moving into industry, Ms. Conway worked at Tufts Medical Center as a Clinical Trials Manager, Outpatient Nurse Coordinator, and Staff Nurse. She has an M.H.A. from Suffolk University and a B.S. from Boston University, is a licensed nurse, and holds an adjunct faculty position at Suffolk University. Agilis is advancing innovative gene therapies designed to provide long-term efficacy for patients with debilitating, often fatal, rare genetic diseases that affect the central nervous system. Agilis’ gene therapies are engineered to impart sustainable clinical benefits by inducing persistent expression of a therapeutic gene through precise targeting and restoration of lost gene function to achieve long-term efficacy. Agilis’ rare disease programs are focused on gene therapy for AADC deficiency, Friedreich’s ataxia, and Angelman syndrome, all rare genetic diseases that include neurological deficits and result in physically debilitating conditions. We invite you to visit our website at www.agilisbio.com Some of the statements made in this press release are forward-looking statements. These forward-looking statements are based upon our current expectations and projections about future events and generally relate to our plans, objectives and expectations for the development of our business. Although management believes that the plans and objectives reflected in or suggested by these forward-looking statements are reasonable, all forward-looking statements involve risks and uncertainties and actual future results may be materially different from the plans, objectives and expectations expressed in this press release.


Research and Markets has announced the addition of the "Breakthrough Therapies: Market Dynamics and Investment Opportunities" report to their offering. The Global Market for Breakthrough Therapy Designation Drugs Should Reach $99.2 Billion by 2022 from $48.8 Billion in 2017 at a CAGR of 15.2% This report highlights the challenges and opportunities of developing breakthrough therapies, it compares and contrasts difference fast track drug development approaches (logistics, criteria, and implications) and the potential risk and rewards of gaining BTD. It analyzes BTD in different therapy areas (cardiovascular, central nervous systems and neurology, rare diseases, oncology and other diseases), leading companies, approved and pipeline products, pricing market access and product revenues. It reviews BTD successes and failures and discusses the different business strategies that companies have adopted in order to maximize the competitive advantage of BTD. It summarizes the current regulatory framework and the potential application of BTD in other regions of the world, and the potential to combine BTD with early access to medicine schemes to improve patient access to medicine to treat rare diseases and address areas of high unmet clinical need. - An overview of the global markets for breakthrough therapies, their dynamics, and investment opportunities - Analyses of global market trends, with data from 2015, 2016, and projections of compound annual growth rates (CAGRs) through 2022 - A look at the materials used in the industry ranging from inorganic clays and concrete to iron and steel, and to commodity and specialty polymers - The challenges and opportunities of developing breakthrough therapies - A compare and contrast of different fast track drug development approaches (logistics, criteria and implications) and the potential risk and rewards of gaining break through designation - Profiles of major players in the industry Key Topics Covered: 1: Introduction - Study Goals And Objectives - Reasons For Doing The Report - Scope Of Report - Intended Audience - Methodology And Information Sources 2: Summary 3: Overview - Defining Breakthrough Therapy Designation 4: Commercial Application Of Breakthrough Therapy Designation - Introduction - Cardiovascular - Central Nervous System And Neurological Disorders - Infectious Diseases - Oncology - Rare Diseases - Other Diseases 5: Implications Of Breakthrough Therapy Designation On Business Strategy - Introduction 6: Regulatory Implications And Breakthrough Therapy Designation - Expedited Programs - Significance Of Btd For The Pharmaceutical And Biotechnology Industries 7: Company Profiles - Abbvie Inc. - Acadia Pharmaceuticals Inc. - Adaptimmune Therapeutics Plc - Aduro Biotech Inc. - Aimmune Therapeutics Inc. - Alexion Pharmaceuticals Inc. - Allergan Plc - Amgen Inc. - Anatara Lifesciences Limited - Ariad Pharmaceuticals Inc. - Astrazeneca Plc - Avexis Inc. - Boehringer Ingelheim Gmbh - Celldex Therapeutics Inc. - Clovis Oncology Inc. - Daiichi Sankyo Company, Limited - DBV Technologies - Eisai Co. Ltd. - Eli Lilly And Company - Exelixis Inc. - F. Hoffmann-La Roche Ltd. - Gilead Sciences Inc. - Glaxosmithkline Plc - Immunomedics Inc. - Individor Plc - Jazz Pharmaceuticals - Johnson & Johnson - Juno Therapeutics Inc. - Kite Pharma Inc. - Merck & Co. Inc. - Merck Kgaa - Novartis AG - Pfizer Inc. - Pharmacyclics Inc. - Progenics Pharmaceuticals Inc. - Regeneron Pharmaceuticals Inc. - Rhythm Pharmaceuticals Inc. - Syndax Pharmaceuticals Inc. - Syneurx International Corporation - Teva Pharmaceuticals Inc. - Trevena Inc. - Vertex Pharmaceuticals Inc. - Vtesse Inc. - Wellstat Therapeutics Corporation 8: References 9: Acronyms 10: Endnotes For more information about this report visit http://www.researchandmarkets.com/research/6369n3/breakthrough


News Article | November 8, 2016
Site: www.newsmaker.com.au

The global diabetic gastroparesis treatment market has been segmented on the basis of product type into drugs and surgical treatment products; on the basis of disease indication into compensated gastroparesis and gastric failure; and on the basis of distribution channel into hospital pharmacies, private clinics, drug stores, retail pharmacies, and e-commerce. Among product types, the drugs segment dominated the market with maximum revenue share of 66% in 2015 followed by the surgical treatment product segment with 34% share. Enhanced development of novel products such as the domperidone drug for the treatment of gastric motility disorder are expected to fuel the growth of the drugs segment over the forecast period. The hospital pharmacies segment is estimated to account for the highest market revenue share of 48.0% in the global diabetic gastroparesis treatment market by the end of 2016. The report covers the global diabetic gastroparesis treatment market across five key regions namely North America, Latin America, Europe, Asia Pacific (APAC), and Middle East & Africa (MEA). North America is expected to remain the dominant market throughout the forecast period, driven by rise in healthcare expenditure, increasing demand for metoclopramide agents in the management of diabetic gastroparesis disease, increasing research on antibodies and other innovative prokinetics drug delivery formats, and technological advancements. Revenue from the diabetic gastroparesis treatment market in North America is expected to register a CAGR of 4.4% over the forecast period. Asia Pacific is expected to remain the second-most lucrative market for diabetic gastroparesis treatment, with an estimated CAGR of 4.5% over the forecast period. Rising prevalence of Type-1 and Type-2 diabetes with high unmet medical needs, growing demand for minimally invasive treatment for refractory gastroparesis, and availability of reimbursement for in-patient hospital stays are driving growth of the global diabetic gastroparesis treatment market. Increasing use of diabetic gastroparesis drugs to control symptoms such as nausea and vomiting is anticipated to fuel growth of the global diabetic gastroparesis treatment market over the forecast period. However, usage of non-prescription drugs to control the symptoms restricts diagnosis of stage 1 and stage 2 gastroparesis. Lack of clinical evidence on improvement offered by prescription drugs and termination of clinical trials are factors expected to hamper growth of the global diabetic gastroparesis treatment market over the forecast period. Some of the top companies profiled in the report are Janssen Global Services, LLC, Salix Pharmaceuticals, Inc., Abbott Laboratories, Medtronic, C. R. Bard, Inc., Kimberly-Clark Corporation, Boston Scientific Corporation, Cardinal Health, Inc., Rhythm Pharmaceuticals, Inc., Evoke Pharma, and Alfa Wassermann SPA. Leading market players are continuously introducing novel products to enhance their product portfolio, increase market share, and expand customer base.


News Article | February 28, 2017
Site: www.businesswire.com

LONDON--(BUSINESS WIRE)--Technavio’s latest report on the global lifestyle drugs market provides an analysis of the most important trends expected to impact the market outlook from 2017-2021. Technavio defines an emerging trend as a factor that has the potential to significantly impact the market and contribute to its growth or decline. The research study by Technavio on the global lifestyle drugs market for 2017-2021 provides detailed industry analysis based on therapy area (depression, dermatology, sexual dysfunction, and obesity) and geography (the Americas, EMEA, and APAC). The Americas will be the leading regional segment, generating both the highest revenue and maximum incremental growth through 2021. The vendors in this market space are working towards introducing new products to target emerging lifestyle diseases, which will immensely benefit this segment. Technavio’s sample reports are free of charge and contain multiple sections of the report including the market size and forecast, drivers, challenges, trends, and more. The top three emerging trends driving the global lifestyle drugs market according to Technavio healthcare and life sciences research analysts are: Sapna Jha, one of the lead analysts at Technavio for cardiovascular and metabolic disorders, talks about the consolidation of the lifestyle drugs market through mergers and acquisitions (M&A). She says, “Vendors in the market are pursuing inorganic growth strategies to expand their lifestyle drugs portfolio and to improve their global presence. Also, with an increasing number of patent expires in the marketplace, vendors are also looking to enhance their portfolio through the acquisition of generics.” For instance, Concordia Healthcare completed the acquisition of the worldwide rights to four generic products. Similarly, Orexigen Therapeutics entered an agreement with Takeda Pharmaceuticals to acquire the US rights to Contrave for the treatment of obesity. Such initiatives will help vendors to expand their business horizons, thereby driving the market growth. Growing focus on development of drugs for new indications Vendors are increasing their focus on finding drugs to address new indications for which there are no drugs. Pharmaceutical companies are looking to monetize the unmet demands for these new indications to increase their product portfolio, income, and presence in the market. Female sexual dysfunction drugs are currently being researched as there is a huge potential customer base for the disease. Vendors such as Pfizer, Sigma-Tau Pharmaceuticals, VIVUS, Palatin Technologies, and TherapeuticsMD are increasingly looking to gain approval for female dysfunction products. These new drugs will add significant revenue to the market. Changing lifestyles of the general populace are giving rise to diseases related to these changes. Obesity and anti-aging products are two of the most sought after drugs from this sector. Vendors are responding to this demand by developing and launching new drugs targeting these diseases. “Vendors such as Rhythm Pharmaceuticals, Merck, AstraZeneca, Novo Nordisk, and Avolynt are some of the vendors that are trying to tap this growing market segment. Therefore, emerging lifestyle diseases are likely to create substantial growth opportunities for vendors, thereby driving market growth,” says Sapna. Become a Technavio Insights member and access all three of these reports for a fraction of their original cost. As a Technavio Insights member, you will have immediate access to new reports as they’re published in addition to all 6,000+ existing reports covering segments like cardiovascular devices, in-vitro diagnostics, and medical imaging. This subscription nets you thousands in savings, while staying connected to Technavio’s constant transforming research library, helping you make informed business decisions more efficiently. Technavio is a leading global technology research and advisory company. The company develops over 2000 pieces of research every year, covering more than 500 technologies across 80 countries. Technavio has about 300 analysts globally who specialize in customized consulting and business research assignments across the latest leading edge technologies. Technavio analysts employ primary as well as secondary research techniques to ascertain the size and vendor landscape in a range of markets. Analysts obtain information using a combination of bottom-up and top-down approaches, besides using in-house market modeling tools and proprietary databases. They corroborate this data with the data obtained from various market participants and stakeholders across the value chain, including vendors, service providers, distributors, resellers, and end-users. If you are interested in more information, please contact our media team at media@technavio.com.


This report studies Anti Obesity Drugs in Global market, especially in North America, Europe, China, Japan, Southeast Asia and India, with production, revenue, consumption, import and export in these regions, from 2011 to 2015, and forecast to 2021. This report focuses on top manufacturers in global market, with production, price, revenue and market share for each manufacturer, covering  Pfizer  Merck  Roche  GlaxoSmithKline  AstraZeneca  Boehringer Ingelheim  Novo Nordisk  Eisai  Norgine  Arena Pharmaceuticals  Orexigen Therapeutics  Vivus  Alizyme  Rhythm Pharmaceuticals  Shionogi  Zafgan By types, the market can be split into  Type I  Type II  Type III By Application, the market can be split into  Application 1  Application 2  Application 3 By Regions, this report covers (we can add the regions/countries as you want)  North America  China  Europe  Southeast Asia  Japan  India 1 Industry Overview of Anti Obesity Drugs  1.1 Definition and Specifications of Anti Obesity Drugs  1.1.1 Definition of Anti Obesity Drugs  1.1.2 Specifications of Anti Obesity Drugs  1.2 Classification of Anti Obesity Drugs  1.2.1 Type I  1.2.2 Type II  1.2.3 Type III  1.3 Applications of Anti Obesity Drugs  1.3.1 Application 1  1.3.2 Application 2  1.3.3 Application 3  1.4 Market Segment by Regions  1.4.1 North America  1.4.2 China  1.4.3 Europe  1.4.4 Southeast Asia  1.4.5 Japan  1.4.6 India 2 Manufacturing Cost Structure Analysis of Anti Obesity Drugs  2.1 Raw Material and Suppliers  2.2 Manufacturing Cost Structure Analysis of Anti Obesity Drugs  2.3 Manufacturing Process Analysis of Anti Obesity Drugs  2.4 Industry Chain Structure of Anti Obesity Drugs 3 Technical Data and Manufacturing Plants Analysis of Anti Obesity Drugs  3.1 Capacity and Commercial Production Date of Global Anti Obesity Drugs Major Manufacturers in 2015  3.2 Manufacturing Plants Distribution of Global Anti Obesity Drugs Major Manufacturers in 2015  3.3 R&D Status and Technology Source of Global Anti Obesity Drugs Major Manufacturers in 2015  3.4 Raw Materials Sources Analysis of Global Anti Obesity Drugs Major Manufacturers in 2015 4 Global Anti Obesity Drugs Overall Market Overview  4.1 2011-2016E Overall Market Analysis  4.2 Capacity Analysis  4.2.1 2011-2016E Global Anti Obesity Drugs Capacity and Growth Rate Analysis  4.2.2 2015 Anti Obesity Drugs Capacity Analysis (Company Segment)  4.3 Sales Analysis  4.3.1 2011-2016E Global Anti Obesity Drugs Sales and Growth Rate Analysis  4.3.2 2015 Anti Obesity Drugs Sales Analysis (Company Segment)  4.4 Sales Price Analysis  4.4.1 2011-2016E Global Anti Obesity Drugs Sales Price  4.4.2 2015 Anti Obesity Drugs Sales Price Analysis (Company Segment) 8 Major Manufacturers Analysis of Anti Obesity Drugs  8.1 Pfizer  8.1.1 Company Profile  8.1.2 Product Picture and Specifications  8.1.2.1 Type I  8.1.2.2 Type II  8.1.2.3 Type III  8.1.3 Pfizer 2015 Anti Obesity Drugs Sales, Ex-factory Price, Revenue, Gross Margin Analysis  8.1.4 Pfizer 2015 Anti Obesity Drugs Business Region Distribution Analysis  8.2 Merck  8.2.1 Company Profile  8.2.2 Product Picture and Specifications  8.2.2.1 Type I  8.2.2.2 Type II  8.2.2.3 Type III  8.2.3 Merck 2015 Anti Obesity Drugs Sales, Ex-factory Price, Revenue, Gross Margin Analysis  8.2.4 Merck 2015 Anti Obesity Drugs Business Region Distribution Analysis  8.3 Roche  8.3.1 Company Profile  8.3.2 Product Picture and Specifications  8.3.2.1 Type I  8.3.2.2 Type II  8.3.2.3 Type III  8.3.3 Roche 2015 Anti Obesity Drugs Sales, Ex-factory Price, Revenue, Gross Margin Analysis  8.3.4 Roche 2015 Anti Obesity Drugs Business Region Distribution Analysis  8.4 GlaxoSmithKline  8.4.1 Company Profile  8.4.2 Product Picture and Specifications  8.4.2.1 Type I  8.4.2.2 Type II  8.4.2.3 Type III


News Article | May 6, 2014
Site: www.xconomy.com

Along with all the well-known and frightening effects of diabetes, people with the disease also suffer debilitating gastrointenstinal problems that often send them to the emergency room. Rhythm Pharmaceuticals wants to replace a decades-old drug for diabetic gastroparesis with one of its own, and data released today bring the Boston-based biotech an important step closer. Diabetic gastroparesis is a complication of diabetes in which the stomach doesn’t empty food into the intestine quickly enough, leading to abdominal pain, bloating, and vomiting. In Phase 2 results released today, Rhythm’s relamorelin (also called RM-131) reduced patients’ gastric emptying time—how long it takes for the stomach to flush out food—in a statistically significant way. That was the main goal of the study, and a biomarker for the drug’s effects on the digestion rate of diabetic gastroparesis patients. Rhythm now plans to move the drug into what CEO Keith Gottesdiener says “certainly should be” a pivotal study, though he wouldn’t comment further given the company is currently planning and designing the trial with the FDA. Still, while the biomarker data are evidence the drug might be working, what’s just as important, if not more, is how these digestion effects translate into making people with diabetic gastroparesis feel better. Will it cut down on patients’ nausea and vomiting, keeping them from potential costly hospital stays? Alleviate pain or bloating? While those things weren’t the main goals of Rhythm’s study, they are the types of things Gottesdiener says the company would likely have to show the FDA that the drug can do in a further study. Rhythm still believes relamorelin is proving itself there too, because the drug helped reduce vomiting episodes by 60 percent compared to a placebo—even if it didn’t have the same luck halting some of the other side effects. The company is citing a high placebo effect, for instance, for confounding its numbers measuring the drug’s impact on all side effects, such as nausea, and bloating. “Vomiting is really the cardinal symptom of these people,” says Rhythm co-founder and president Bart Henderson. “It’s what’s drives them to the emergency room, it’s what really drives people to doctors, and it really messes up diabetic control. Of all the symptoms that we wanted to hit, that was the one that was really the most critical to hit and I think we nailed it.” Rhythm, of course, will have to reproduce that kind of the result in a future study powered to gauge side effects before it really knows if it has a potential drug on its hands. That study is expected to get underway by the end of the year, and will look at both side effects, and gastric emptying, according to Gottesdiener. Rhythm was formed four years ago by licensing two peptide drug candidates from Paris-based Ipsen. One of them was relamorelin, an injectable peptide drug derived from ghrelin, a hormone in the stomach that helps food move through the digestive tract. The idea is that the drug might help spur gastrointestinal movement (typically called GI motility), stop the stomach from getting clogged up, and alleviate the pain and vomiting that patients experience. If Rhythm succeeds, there’s a sizeable opportunity awaiting. About 2.3 million of the 24 million or so Americans with type 1 and type 2 diabetes in the U.S. have “moderate to severe” symptoms of gastroparesis and are seeking care, which can lead to costly hospital stays. The last drug specifically approved for the disorder in the past 40 years is the now-generic drug metoclopramide, but Henderson says the drug isn’t commonly used because of safety issues that limit the drug to short courses. Henderson also says metoclopramide isn’t very potent. Solana Beach, CA-based Evoke Pharma (NASDAQ: EVOK) would likely disagree. Evoke is developing a nasal spray version of metoclopramide for gastroparesis, and just began enrolling patients in a Phase 3 trial of the drug a few weeks ago. Rhythm has raised about $73 million from the likes of MPM Capital, Third Rock Ventures, New Enterprise Associates, Pfizer Ventures, and Ipsen to bankroll the development of relamorelin and a second drug candidate, RM-493. Its first clinical data were released in 2012, with a small, 10-person Phase 1 study that showed relamorelin helped reduce gastric emptying rates by about 66 percent. That trial gave Rhythm the confidence to run the drug through the larger Phase 2 trial it’s revealing today. This time around, Rhythm enrolled 204 patients with moderate to severe symptoms of diabetic gastroparesis and slow gastric emptying, according to Gottesdiener. All of those patients were given a placebo treatment for a week, and then broken up into three groups of 67 to 69 patients apiece, randomized, and dosed with either a placebo, a once-daily 10-milligram injection of relamorelin, or a 10 mg injection twice per day, for 28 days. The main goal of the study was to reduce gastric emptying. Rhythm says that the drug succeeded in doing so in a statistically significant way, though the company didn’t provide the full details from the study. Rhythm also put in additional measures to judge a potential change in a combination of various side effects like pain, nausea, and vomiting. Rhythm didn’t hit statistical significance here—meaning had this been a study goal, the drug would’ve failed it. Gottesdiener says this was because an unexpected, strong placebo effect “obscured” the results, so the company added in figures after the study filtering the data. Rhythm looked at a subgroup of people who vomited during that first one-week period when everyone got a placebo, and saw that its drug hit statistical significance. Post-study subgroup analyses like these come off as data spin, of course. But Rhythm executives say that this group of people represented a majority—60 percent—of the folks in its study, and could serve as a way to identify the sicker patients who might better respond to its drug going forward. “That’s a very easy clinical marker for really more severity in these patients,” Gottesdiener says. Now it’s likely on to the deep end of the pool for Rhythm, a make-or-break pivotal trial. Gottesdiener and Henderson were tight-lipped about Rhythm’s strategy in preparation for the study. They wouldn’t comment on whether the company might look to a further round, IPO, or partnership to help bankroll the study, for instance. But Rhythm could get past a Phase 3 study before having those discussions, because that trial will likely be a three-month study that isn’t too pricey for a small company like Rhythm to run, according to Gottesdiener. That being said, assuming the data hold up, a strategic move is coming sooner or later. “Ultimately, we’ll need partners in some form,” Henderson says.


News Article | May 9, 2014
Site: www.xconomy.com

Pharma megadeals once again took center stage this week as more giant divisions were flipped, and the tension around a potential 12-figure, international buyout affecting thousands of workers across the globe escalated even higher. Those stories, and—oh yeah, biotech news—below. —Nothing is more closely watched in the life sciences world right now than the ongoing Pfizer/AstraZeneca saga, and for good reason. First, tensions are rising: Pfizer (NYSE: PFE) publicly boosted its bid to an astronomical $106 billion this week, and AstraZeneca (NYSE: AZN) responded with a presentation detailing why it’s worth way more than that. But also, there are a host of implications should that deal go down, starting with the thousands of workers that would likely lose their jobs, and then the massive portfolio review that will take place—impacting biotechs everywhere. With those issues in mind, I spoke with Francois Nader, the president and CEO of Bedminster, NJ-based NPS Pharmaceuticals (NASDAQ: NPSP) and a veteran of several pharma mergers, about the ins and outs of such deals from an employee perspective, an executive perspective, and that of the nervous biotech looking on. (Separately, NPS lowered its 2014 financial projections for sales of teduglutide (Gattex) to $100 million-$110 million from $110 million-$120 million, sending shares down more than 9 percent.) —Boston-based Rhythm Pharmaceuticals has been trying to replace a decades old, yet flawed treatment for diabetic gastroparesis—a debilitating, digestion-related complication suffered by people with diabetes. While there are still hurdles ahead, the company took a step towards that goal this week when its drug prospect, relamorelin, hit its goal in a Phase 2 clinical trial. Rhythm, a startup backed by the likes of MPM Capital, Third Rock Ventures, and others, plans to move the drug into its next study—likely a pivotal trial—by the end of the year. I spoke with Rhythm CEO Keith Gottesdiener and president Bart Henderson about the trial, and what comes next. —Tarrytown, NY-based Regeneron Pharmaceuticals (NASDAQ: REGN) is looking to gene therapy to produce a potential successor to its blockbuster drug aflibercept (Eylea). The company cut a deal this week with Menlo Park, CA-based startup Avalanche Biotechnologies to co-develop and commercialize gene therapy products for eye diseases, among them potentially the “wet” form of age-related macular degeneration—the disorder aflibercept treats. Avalanche—which just raised a $55 million Series B round in late April—got an unspecified cash payment up front and stands to add another $640 million down the road if the prospects to come out of the tie-up hit various milestones. All told, the deal covers eight therapeutic targets. Regeneron will get worldwide rights to all of the collaboration drugs that move forward, though Avalanche has an option to share the costs and profits of products aimed at two therapeutic targets of its choice. Regeneron also has a time-limited window to negotiate for rights to Avalanche’s wet AMD gene therapy prospect, AVA-101, after it finishes an ongoing Phase 2a study. Separately, Regeneron reported its first quarter earnings. Aflibercept generated $359 million in revenue, missing analysts’ consensus estimates by about $20 million. —Loxo Oncology was formed in New York last year by Aisling, subsequently picked up a preclinical cancer drug from Array BioPharma (NASDAQ: ARRY), and then raised $33 million to help develop it. This week, the biotech, now based in Stamford, CT, fattened up its wallet a bit more, announcing a $24 million Series B round led by New Enterprise Associates. Existing investors Aisling and OrbiMed Advisors also participated in the funding. Sara Nayeem, a principal at NEA and the former vice president of molecular discovery at Epizyme (NASDAQ: EPZM), has joined Loxo’s board. The company’s lead drug, known as LOXO-1, is now in Phase 1 testing. —The shuffling of Big Pharma divisions continued this week, as Whitehouse Station, NJ-based Merck (NYSE: MRK) sold its consumer care business—which includes brands like Dr. Scholl’s, Afrin, and Coppertone—to Bayer for about $14.2 billion. Merck also agreed to pay Bayer up to $2.1 billion as part of a separate deal to co-develop drugs for cardiovascular diseases. In addition, Merck appeared to leapfrog Bristol-Myers Squibb for the lead in the high-stakes cancer immunotherapy race: it filed an application with the FDA to approve MK-3475, its checkpoint inhibitor for cancer. Bristol-Myers has yet to do so with its rival drug, nivolumab. —Shares of Cambridge, MA-based Aegerion Pharmaceuticals (NASDAQ: AEGR) got slammed this week after sales of the company’s rare disease drug lomitapide (Juxtapid) came in well short of analysts’ expectations. Lomitapide generated $27 million in sales in the fourth quarter of 2013, compared with consensus estimates of around $33.6 million. Aegerion also cut its projected 2014 sales for the drug by about $10 million, to $180 million-$200 million for the year, blaming delays on orders for the drug in Brazil. Shares slid more than 22 percent on the news, continuing a precipitous fall for Aegerion during the first year of its drug’s launch. Shares were worth more than $88 apiece in July. They now trade at about $33 per share. —Natick, MA-based Boston Scientific (NYSE: BSX) made its latest acquisition this week, snapping up Cupertino, CA-based IoGyn. Boston Scientific already held a roughly 28 percent stake in the privately-held company, and is paying about $65 million to buy the rest of IoGyn and retire about $8 million worth of note debt it issued to the startup. IoGyn has developed and won FDA clearance of a minimally invasive system surgeons can use to remove intrauterine polyps and fibroids. —Watertown, MA-based Enanta Pharmaceuticals (NASDAQ: ENTA) landed another $20 million check from AbbVie (NYSE: ABBV) this week after the larger company filed an application with regulators in Europe to approve an all-oral regimen for hepatitis C including ABT-450, a drug the two companies co-developed. Enanta got a $20 million payout a couple weeks ago when AbbVie lodged an application with the FDA. —Retiring Biogen Idec (NASDAQ: BIIB) chairman William Young has joined the board of directors of Vertex Pharmaceuticals (NASDAQ: VRTX). Young announced plans to retire as Biogen’s chairman in February. He spent about four years in the role. Young is also the chairman of NanoString Technologies (NASDAQ: NSTG) and a venture partner at Clarus Ventures.


News Article | October 22, 2014
Site: www.xconomy.com

[Updated, 10/23/14, 4:41 pm ET] Rhythm Pharmaceuticals hinted that a strategic move was coming when a drug it’s been developing for diabetic gastroparesis cleared a key clinical hurdle earlier this year. That came to fruition today, because Actavis has stepped and offered Rhythm a financial payday if that drug can make it to the finish line. Actavis (NYSE: ACT), the big Irish drugmaker formerly known as Watson Pharmaceuticals, has nabbed an option to acquire Boston-based Rhythm’s wholly owned subsidiary, Rhythm Health—the unit that houses the company’s gastroparesis drug prospect, relamorelin. Actavis has paid Rhythm $40 million up front, and has the right to buy Rhythm Health and the rights to relamorelin for an unspecified additional sum after a Phase 2b trial. Rhythm expects to begin that study by early 2015. The deal doesn’t include Rhythm Metabolic, the biotech’s other subsidiary, which is developing a drug for obesity. The two companies are establishing a joint development committee to oversee relamorelin’s progress, though Rhythm is managing the drug’s development until Actavis makes a decision on the option. Rhythm was formed in 2010 by licensing two peptide drug prospects from Paris-based Ipsen. Relamorelin was one of them; it’s an injectable peptide drug derived from the hunger-simulating hormone ghrelin. The idea is that the drug might help spur gastrointestinal movement (known as GI motility), and stop the stomach from getting clogged up. That, in turn, is supposed to alleviate diabetic gastroparesis, a complication of diabetes in which the stomach doesn’t empty into the intestine quickly enough, leading to abdominal pain, bloating, and vomiting. The last drug specifically approved for the disorder in the past 40 years is the now-generic drug metoclopramide. Rhythm executives have said previously that the drug isn’t commonly used because of safety concerns. [Updated to add more details from Phase 2b study] Rhythm reported positive, yet somewhat mixed top-line results from a 204-patient, mid-stage trial of relamorelin in May. Though the company didn’t divulge the full details from the study, Rhythm said that the drug hit its main goal—reducing gastric emptying time. The company also hit its goal on a secondary endpoint by significantly reducing patients’ vomiting. It didn’t hit statistical significance on some additional exploratory measures judging the potential change in a variety of side effects associated with diabetic gastroparesis; executives claimed that a strong placebo effect “obscured” the results. Those measures were not established clinical endpoints for the trial, so the drug doesn’t have a failure on its record. But Rhythm will likely have to show in future trials that the drug can hit clinical goals like those it previously missed if it hopes to win the support of the FDA. Co-founder and president Bart Henderson noted in May that Rhythm would likely need partners “in some form” to continue its clinical push with relamorelin. Since then, the company has filed papers to take itself public. Now it’s cut a deal with Actavis. “This venture allows Actavis, in partnership with Rhythm, to advance the development of this molecule in a manner that minimizes risk in early stage development, yet ensures our ability to acquire global rights to the product upon initiation of Phase 3 trials,” said Actavis senior VP of global brands research and development, David Nicholson, in a statement.


News Article | October 24, 2014
Site: www.xconomy.com

Deal flow is in full swing on the East Coast. At least three life sciences startups launched out of Boston and New York this week, with a few other firms filling up their bank accounts with either new funding rounds or partnership deals. Those stories and plenty more below: —Boston-based PureTech raised $55 million from a group of investors led by Invesco Perpetual, one of the largest investment fund managers in the U.K. I spoke with PureTech CEO Daphne Zohar about the raise, which should enable PureTech not only to launch a planned three to five startups in the coming year, but to pour a bunch of new cash—on top of that $55 million—into the existing ones in its portfolio. —Cambridge, MA-based Quartet Medicine came out of stealth this week with a $17 million Series A round from Atlas Venture, the venture arms of Pfizer and Novartis, and Partners Innovation Fund. The company aims to exploit new insights into the well-known tetrahydrobioprotein pathway to develop drugs for chronic pain conditions and bridge the gap between animals and the human trials that have doomed so many other pain drugs. —Cambridge-based Unum Therapeutics also launched this week, raising $12 million from Atlas, Fidelity Biosciences, and Sanofi-Genzyme BioVentures. Unum is joining the increasing crowd of companies touting ways to reprogram the immune system to seek out and attack tumors with chimeric antigen receptor T-cell therapy, or CAR-T. Unum’s twist on the concept is cancer treatments with a so-called “antibody-coupled T-cell receptor,” meaning it’s engineering T cells to find tumors with the help of antibodies. —Dublin drug giant Actavis (NYSE: ACT) paid $40 million up front for an option to acquire a drug Boston-based Rhythm Pharmaceuticals is developing to treat diabetic gastroparesis called relamorelin. Actavis can exercise an option to buy the drug, and Rhythm Health, the Rhythm subsidiary that owns it, after a Phase 2b trial that is expected to begin by early 2015. Separately, Rhythm also announced positive results from a Phase 2 trial of relamorelin in chronic constipation. —Waltham, MA-based Proteon Therapeutics (NASDAQ: PRTO) debuted on Nasdaq this week after raising $61 million in an IPO. Proteon had to sell more shares (6.1 million instead of 4.7 million) at a lower price ($10 instead of $12 to $14) than it initially planned to, however. —Cubist Pharmaceuticals (NASDAQ: CBST) said this week that CEO Mike Bonney will step down from his post on Dec. 31 and hand the reins to the company over to COO Robert Perez. Bonney has led Cubist for 12 years, and seen it rise from an antibiotics startup without a marketed product to a mid-cap biotech worth more than $5 billion. —Summit, NJ-based Celgene (NASDAQ: CELG) gave investors a glimpse into why it paid some $710 million up front and potentially $2.6 billion overall to an Irish drugmaker, Nogra Pharma, for the rights to an oral antisense drug for Crohn’s Disease. The company revealed data from a 166-patient Phase 2 study showing the drug, mongerson, helped induce remissions in a broad range of Crohn’s patients. Celgene will begin testing the drug in a Phase 3 trial by the end of the year. As Alex Lash reported, Celgene also cut a deal with Sutro Biopharma giving it an option to acquire the South San Francisco, CA-based company for an undisclosed sum. —In case you missed it, I posted a slideshow this week from our latest local biotech event, “Boston’s Life Science Disruptors,” which featured executives and investors from Zafgen (NASDAQ: ZFGN), Epizyme (NASDAQ: EPZM), and Sage Therapeutics (NASDAQ: SAGE). —Harris & Harris Group announced it’s formed a New York-based startup called Tara Biosystems. The Columbia University spinout is developing organ-on-a-chip technology to help boost the effectiveness of preclinical drug testing. Harris didn’t disclose the size of its investment, but managing director Misti Ushio is serving as the company’s founding CEO. The startup’s first project will be “heart-on-a-chip” tissue models. —New York-based Phreesia got a $30 million investment led by private equity firm LLR Partners, with participation from existing backers HLM Ventures and Ascension Ventures. Phreesia has developed a wireless tablet computer pad that helps patients check in for appointments at the doctor’s office and make payments electronically. —Shares of Cambridge-based Biogen Idec (NASDAQ: BIIB) dropped about 7 percent after the company disclosed that a patient taking its oral multiple sclerosis drug, dimethyl fumarate (Tecfidera) died from a rare brain infection called progressive multifocal leukoencephalopathy. Quarterly sales of the drug also came in slightly lower than consensus analyst estimates. Separately, Biogen hired neuroscientists Christopher Henderson and Richard Ransohoff to help its discovery and development efforts in neurodegenerative diseases. —The FDA this week extended its review of Bedminster, NJ-based NPS Pharmaceuticals’ (NASDAQ: NPSP) engineered version of human parathyroid hormone, known as Natpara, by three months and asked the company to submit a risk evaluation and mitigation strategy for the drug. An FDA advisory panel voted in favor of the drug, 8-5, in September, but the close vote left lingering questions about its commercial potential and what type of label the drug would get, if approved. The FDA will now decide whether to approve the drug by Jan. 24.


News Article | August 14, 2015
Site: www.xconomy.com

Investors in the broader biotech indices may have been losing money this week, but East Coast startups were finding it fairly easy to come by. A gene editing startup corralled $120 million and a portfolio of big-name investors. A scientist working for decades in the RNA drug space emerged with funding for his latest startup. And a former Teva CEO laid the foundation to take his new startup public. We’ve got the details below. —Cambridge, MA-based Editas Medicine was the first of a group of startups formed to exploit the gene editing technology CRISPR-Cas9 for human therapeutics. And this week, it became the first of those companies to land a big financing round backed by a slew of crossover investors. The $120 million round also included participation from Bill Gates and a vehicle set up by his science and technology advisor, Boris Nikolic. I spoke with CEO Katrine Bosley about the financing, how Editas intends to use CRISPR-Cas9, and the technical hurdles that have to be overcome. —RNA therapeutics veteran and former Sarepta Therapeutics (NASDAQ: SRPT) chief scientific officer Art Krieg reemerged this week as the CEO of a new Cambridge-based startup called Checkmate Pharmaceuticals, which raised $20 million from venBio and Sofinnova Ventures. The startup has licensed a drug from struggling Swiss biotech Cytos Technology—another company from the venBio portolio—and plans to combine it with checkpoint inhibiting cancer drugs to boost their effectiveness.  It’s also gained access to Cytos’s method for creating “virus-like particles,” which look like viruses and are supposed to trick the immune system into responding. —It’s taken former Teva Pharmaceutical Industries CEO Jeremy Levin about a year to form a biotech startup, New York-based Ovid Therapeutics, and assemble a group of crossover investors to fund it. Ovid raised a $75 million Series B this week to continue developing a group of drugs for rare brain diseases. Its lead drug, as I’ve written previously, is OV101, or gaboxadol—a drug once tested by Merck and Lundbeck as a sleeping pill. Ovid will begin two Phase 2 trials next year testing gaboxadol in patients with Angelman’s syndrome and Fragile X syndrome. —Hedge fund boss Martin Shkreli led a $90 million funding round for his new startup, Switzerland and New York-based Turing Pharmaceuticals. Shkreli founded Turing last year and stocked it with a group of assets he bought from his former company, Retrophin (NASDAQ: RTRX)—from which he was ousted in a messy split. Turing put some of that cash to work immediately by paying Impax Laboratories (NASDAQ: IPXL) $55 million for the U.S. rights to a drug called pyrimethamine. —Cambridge-based Eleven Biotherapeutics’s (NASDAQ: EBIO) shares were routed earlier this year when its lead drug, EBI-005, flunked a Phase 3 test in dry eye disease. But the stock rebounded a bit this week after Eleven started enrolling patients in a new Phase 3 trial testing the drug for severe allergic conjunctivitis (Eleven had previously disclosed plans in April to begin this trial in the second half of the year). The Boston Business Journal and FierceBiotech have more on Eleven’s plans for the study. —Shares of Newton, MA-based Karyopharm Therapeutics (NASDAQ: KPTI) plummeted more than 40 percent this week after the company, in its latest earnings release, disclosed cases of sepsis in acute myeloid leukemia patients taking its experimental drug, selinexor, in a Phase 2 trial. Karyopharm amended the study protocol to dial down the dose of its drug. —Hampton, NJ, and Needham, MA-based Celldex Therapeutics (NASDAQ: CLDX) also saw its stock fall this week. Shares slumped 20 percent after Celldex disclosed that based on discussions with regulators, it’ll likely have to go the normal approval route with its brain cancer vaccine rindopepimut (Rintega) and finish a full Phase 3 trial before filing papers with the FDA. Celldex officials had spoken publicly, albeit cautiously, of plans to talk to the FDA about an accelerated approval for rindopepimut off of its small Phase 2 trial. (Executives addressed their interactions with the FDA on a conference call this week—here’s the transcript). The company’s late-stage study is already underway. —Boston-based Rhythm raised $40 million from OrbiMed, Deerfield Management and others that’ll be used to fund one of its subsidiaries, Rhythm Metabolic, and a drug that unit is developing for genetic obesity-related disorders like Prader-Willi Syndrome. Rhythm’s other subsidiary, Rhythm Pharmaceuticals, is developing a drug for diabetic gastroparesis; Actavis nabbed an option to buy that program outright last year after a Phase 2b trial. —Huntington, NY-based startup Envisagenics won a $225,000 Small Business Innovation Research Award from the National Institutes of Health to help develop a technology it’s calling “SpliceCore,” a cloud-based software program to help researchers analyze and interpret genomic data. You can read more here about the startup, the most recent graduate of Accelerate Long Island. —Kenilworth, NJ-based Merck (NYSE: MRK) is teaming up with Texas’ MD Anderson Cancer Center to test its cancer immunotherapy drug pembrolizumab (Keytruda) in tandem with a variety of other treatments, such as chemotherapy, radiation, or other anti-cancer drugs.  The three-year collaboration will lead to trials in pancreatic, liver, and gastroesophageal cancers, the first of which will begin this year. —New York-based Bristol-Myers Squibb (NYSE: BMY) paid roughly $53 million to Uniqure (NASDAQ: QURE) as part of the collaboration between the two to develop gene therapies for cardiovascular diseases. Bristol shelled out $15 million in cash and bought $38 million worth of Uniqure stock, which together with previous purchases brought its stake in the Netherlands-based biotech to 9.9 percent. Aerial photo of Coney Island Peninsula courtesy of a Creative Commons license via user Psychocadet.

Loading Rhythm Pharmaceuticals collaborators
Loading Rhythm Pharmaceuticals collaborators