A PHASE 1, NON-RANDOMIZED, OPEN-LABEL, PARALLEL-GROUP SINGLE-DOSE STUDY TO ESTIMATE THE EFFECT OF AGE ON THE PHARMACOKINETICS, SAFETY AND TOLERABILITY OF INTRAVENOUS RIVIPANSEL (PF-06460031)

The purpose of this study is to evaluate the effect of age on PK, safety and tolerability of IV rivipansel.

Start Date01 Jan 2017

Sponsor / Collaborator

GlycoMimetics, Inc.

NCT02871570 / CompletedPhase 1

A Phase 1, Non-randomized, Open-label, Parallel-group Single-dose Study To Evaluate The Pharmacokinetics, Safety, And Tolerability Of Intravenous Rivipansel (Pf-06460031) In Subjects With Moderate Hepatic Impairment And In Healthy Subjects With Normal Hepatic Function

The purpose of this study is to determine the effect of hepatic impairment on rivipansel PK and safety.

Start Date01 Sep 2016

Sponsor / Collaborator

GlycoMimetics, Inc.

NCT02813798 / CompletedPhase 1

A PHASE 1, NON-RANDOMIZED, OPEN-LABEL, SINGLE DOSE STUDY TO EVALUATE THE EFFECT OF RENAL IMPAIRMENT ON THE PHARMACOKINETICS, SAFETY AND TOLERABILITY OF RIVIPANSEL (PF-06460031) IN SUBJECTS WITH RENAL IMPAIRMENT AND IN HEALTHY SUBJECTS

The purpose of this study is to determine the effect of renal impairment on rivipansel.

Start Date01 Jun 2016

Sponsor / Collaborator

GlycoMimetics, Inc.

100 Clinical Results associated with Rivipansel Sodium

100 Translational Medicine associated with Rivipansel Sodium

100 Patents (Medical) associated with Rivipansel Sodium

Literatures (Medical) associated with Rivipansel Sodium

01 Jul 2023·Carbohydrate research

Revisiting the structural basis for biological activity of GMI-1070, a sialyl Lewis^x mimetic.

Article

Author: Arend, Lais B. ; Verli, Hugo

When it comes to the treatment of pathologies in which aberrant cell adhesion and extravasation from the bloodstream have been implicated, the selectins represent a central therapeutic target. In this context, the present work investigates the conformational landscape of two prototypes for the design of new antineoplasic and anti-inflammatory agents: the natural selectin ligand sialyl Lewis^x and its mimetic GMI-1070. Accordingly, a series of unbiased molecular dynamics simulations at the microsecond scale using GROMOS 53A6 (GLYC), CHARMM36m and GLYCAM06 force fields were employed, together with ConfID, an analytical method for the characterization of conformational populations of small molecules. Our results for sialyl Lewis^x are in agreement with and expand upon prior work. As for the mimetic, our results indicate that, in spite of its conformational restriction, GMI-1070's behavior in solution deviates from what had been proposed, highlighting thus some features that could be optimized, as the development of sialyl Lewis^x mimetics continues, and new candidates emerge.

12 Jan 2023·Blood

Ups and downs in the treatment of sickle cell disease

Communications

Author: Brousse, Valentine

A review.SCD is a devastating disease with a huge global burden, affecting more than 100 000 patients in the United States as well as in Europe and millions in low-income countries.Its pathophysiol. has emerged as an extremely complex one, involving not only Hb polymerization as the primum movens but also a cascade of interrelated events including hemolysis; activation and adhesion of neutrophils, platelets, and endothelial cells; abnormal coagulation; sterile inflammation; vascular tone impairment; and nitric oxide functional deficit, among other downstream biol. consequences.Despite promising early results, in this issue of Blood, Dampier et al., report the neg. results of rivipansel, a predominantly E-selectin antagonist, in treating hospitalized vaso-occlusive crisis (VOC) in patients with sickle cell disease (SCD).Although these results are disappointing for patients and physicians, important lessons can nevertheless be learned.New drugs that can effectively act on acute vaso-occlusion and/ or synergistically address important facets of the pathophysiol. of SCD are still urgently needed.

12 Jan 2023·Blood

A randomized clinical trial of the efficacy and safety of rivipansel for sickle cell vaso-occlusive crisis

Article

Author: Sivamurthy, Krupa M. ; Desai, Payal ; Telen, Marilyn Jo ; Tseng, Li-Jung ; Taylor, James G. ; Dampier, Carlton D. ; Hassell, Kathryn L. ; Brown, R. Clark ; Rothman, Jennifer ; Readett, David ; Kanter, Julie ; El Rassi, Fuad ; Lozier, Jay Nelson ; Magnani, John L. ; Fuh, Beng ; Tammara, Brinda ; Wun, Ted ; Thackray, Helen ; Pastore, Yves

Abstract:

The efficacy and safety of rivipansel, a predominantly E-selectin antagonist, were studied in a phase 3, randomized, controlled trial for vaso-occlusive crisis (VOC) requiring hospitalization (RESET). A total of 345 subjects (204 adults and 141 children) were randomized and 320 were treated (162 with rivipansel, 158 with placebo) with an IV loading dose, followed by up to 14 additional 12-hourly maintenance doses of rivipansel or placebo, in addition to standard care. Rivipansel was similarly administered during subsequent VOCs in the Open-label Extension (OLE) study. In the full analysis population, the median time to readiness for discharge (TTRFD), the primary end point, was not different between rivipansel and placebo (−5.7 hours, P = .79; hazard ratio, 0.97), nor were differences seen in secondary end points of time to discharge (TTD), time to discontinuation of IV opioids (TTDIVO), and cumulative IV opioid use. Mean soluble E-selectin decreased 61% from baseline after the loading dose in the rivipansel group, while remaining unchanged in the placebo group. In a post hoc analysis, early rivipansel treatment within 26.4 hours of VOC pain onset (earliest quartile of time from VOC onset to treatment) reduced median TTRFD by 56.3 hours, reduced median TTD by 41.5 hours, and reduced median TTDIVO by 50.5 hours, compared with placebo (all P < .05). A similar subgroup analysis comparing OLE early-treatment with early-treatment RESET placebo showed a reduction in TTD of 23.1 hours (P = .062) and in TTDIVO of 30.1 hours (P = .087). Timing of rivipansel administration after pain onset may be critical to achieving accelerated resolution of acute VOC. Trial Registration: Clinicaltrials.gov, NCT02187003 (RESET), NCT02433158 (OLE).

News (Medical) associated with Rivipansel Sodium

09 Sep 2022

·endpts.com

Homology sails with same captains in different seats; Ex-Harpoon chief weathers the Storm at UK biotech

Albert Seymour After a multi-year run as co-workers at Shire Pharmaceuticals , Arthur Tzianabos and Albert Seymour got the band back together in 2016 as two of the early-days employees at Homology Medicines . Tzianabos as CEO and Seymour as science chief of the genetic medicine-focused biotech “was not really ready for prime time,” in the words of Seymour. The plan from the start was to have Seymour assume the chief executive duties once the biotech was fully in the clinic with multiple programs to account for. “When we saw the technology that Homology had to offer with these 15 novel AAV serotypes that were naturally derived from human stem cells, I couldn’t pass up the opportunity to take that next step and give it a try,” Seymour told Endpoints News , reflecting on his 6.5 years at the Bedford, MA, biotech, of which he was the sixth employee. On Sept. 6, the expected management shuffle took place, with Tzianabos moving to the board chair role and Seymour succeeding him at steering the ship, which the new CEO said is “really well-positioned from a cash-flow perspective.” With the leadership moves, existing board chair Kush Parmar is out. The 5AM Ventures managing partner sits on the board of a slate of biotechs like Akouos , Vor Biopharma , Rallybio , Entrada Therapeutics and others. And Homology has announced a CSO successor. “I’d much rather give an opportunity to some of the young talented scientists that we have in the organization,” Seymour said, “to develop perhaps into that role as opposed to going out and hiring someone.” His main priorities in the new post center around clinical development, especially since the biotech prioritized its clinical plans last month, halting enrollment in a gene therapy trial and seeking a partner on another gene therapy so that a gene editing program in PKU can persist. A gene editing program for metachromatic leukodystrophy, or MLD, showed promise in preclinical work, but the asset is still about 18 to 24 months from human testing, Seymour said, so the company is instead searching for somebody to tie up with. “We’re actively talking to a lot of potential partners,” including those with rare disease, lysosomal storage disorders and global experience, Seymour said. — Kyle LaHucik Jerry McMahon → As shares tumbled and its lead prostate cancer program HPN424 underwhelmed at last year’s ASCO conference, Jerry McMahon resigned as CEO of Harpoon Therapeutics and was replaced by Julie Eastland in October 2021. But the skies are getting a little brighter for McMahon, even though the name of his new company doesn’t reflect it. He’s been named chief executive at Storm Therapeutics , an RNA epigenetics player out of Cambridge, UK, that formed a cancer alliance with Exelixis which is going after the RNA editing enzyme ADAR1 first. Incidentally, Harpoon waved the white flag on HPN424 in March, and the staff shakeup continued with the June 1 resignation of CMO Natalie Sacks . Cindy Atwell → Cindy Atwell and Jeff Smith are moving on up at Precision BioSciences under first-year CEO Michael Amoroso , as Atwell gets promoted to CBO and Smith takes on the role of chief research officer. A Precision exec since 2019, Atwell was previously SVP of business development and alliance management and has held business development positions at AbbVie and Halozyme . Smith, the ex-chief technology officer, helped co-found Precision in 2006 with Matt Kane and Derek Jantz . In other Precision developments, Jantz now has a modified title — chief scientific officer & strategy — that allows him to work with Amoroso “in formulating company strategy and managing relationships with external stakeholders, including current and potential collaboration partners.” Because of this change, Jantz will no longer be a member of Precision’s board of directors. Timothy Wright → Georgia-based MiMedx — once embroiled in controversy over CEO Parker “Pete” Petit ’s schemes to boost their sales figures, resulting in a 2020 fraud conviction — is changing chief executives again. With Timothy Wright ’s exit, the allograft maker has tapped Todd Newton as interim CEO while the search gets underway for a permanent replacement. Newton, a board member at MiMedx for three years, is a Deloitte vet who was CEO of Austin, TX medtech Apollo Endosurgery from 2014-21. → AAVantgarde Bio has saddled up Natalia Misciattelli as CEO. Misciattelli makes her way to the Milan-based company from NovalGen , where she served as CBO. Prior to that, Misciattelli was SVP, strategy and operations for Freeline Therapeutics . Peter Pfreundschuh → Al Sandrock is busy assembling his team of execs at Voyager : Peter Pfreundschuh (CFO) has held the same position at a number of stops, including Immunomedics , Sucampo Pharmaceuticals (which he helped sell to Mallinckrodt for $1.2 billion ), UroGen and, until April, Frequency Therapeutics . Todd Carter (CSO), who first jumped on board at Voyager as senior director of neuroscience in 2016, earns this latest promotion after six months as SVP of research. And Trista Morrison (SVP, corporate affairs) first appeared in this space two years ago when she became Saniona ’s chief communications officer. The Sobi and Ironwood vet had been elevated to chief corporate affairs officer in March, but she left the next month to start her own consulting firm. All three appointments take effect on Sept. 19. Andy Schmeltz → In case you missed it last Friday, Pfizer ’s Andy Schmeltz is starting a new gig as SVP, commercial strategy & innovation, while Suneet Varma — the global president of rare disease since 2020 — has replaced Schmeltz as global oncology & US president. And in this particular game of musical chairs, Kevin Sullivan will take over for Varma. That leads us to GlycoMimetics , where the one-time Pfizer sickle cell partner has appointed GSK and Otsuka alum Edwin Rock as CMO. Formerly the medical chief at Partner Therapeutics , Rock was VP, clinical research at MacroGenics from 2017-20. Pfizer is looking to get it right in the sickle cell space with its $5.4 billion acquisition of Global Blood Therapeutics after the pharma giant and GlycoMimetics stumbled in Phase III with rivipansel in 2019. Jessica Stitt → Jeff Bluestone has found a new finance chief at Sonoma Biotherapeutics a little more than a year removed from the Treg biotech’s prodigious $265 million Series B . Jessica Stitt was on our radar when she became CFO of Gyroscope in March 2021, and she didn’t hang around Bristol Myers Squibb for long after the $13 billion buyout of MyoKardia , where she was VP of finance and operations. Earlier, she wrapped up nine years with Theravance Biopharma as VP of finance and investor relations. Sonoma announced in late August that its manufacturing and R&D presence in Seattle will encompass 83,000 square feet and will be located at Unison Elliott Bay. Jonathan Biller → Winning an expanded approval with CF med Orkambi for children between 1 and 2 years of age, Vertex has installed Jonathan Biller as chief legal officer. Vertex poaches Biller from Agios , a biotech undergoing a leadership change at the top as Jackie Fouse entrusted the CEO job to Brian Goff on Aug. 8. Biller was hired at Agios as chief legal officer in late 2019, and the ex- Celgene general counsel then rose to CFO and head of corporate affairs. Biren Amin → Former Jefferies analyst Biren Amin is already on to his next challenge, joining Pyramid Biosciences as its new chief financial and strategy officer. Amin left Jefferies to take the CFO job at Immuneering in April 2021, and earlier in his career, he was with Prudential Equity Group and Aventis Pharmaceuticals . Pyramid Biosciences, an unheralded cancer biotech developing a pair of TRK inhibitors, is led by Bristol Myers alum Brian Lestini . Jörn Drappa → Viela Bio co-founder Jörn Drappa has left Ventyx Biosciences after an eight-month stint to become CMO of Alumis , a Foresite biotech now run by ex- Principia chief Martin Babler that began as Esker Therapeutics and pulled in a $200 million Series B to start 2022. After Horizon bought the AstraZeneca spinout for $3 billion , Drappa stayed as R&D chief for just a bit until Ventyx came calling. Elsewhere at Alumis, Genentech alum Roman Rubio has been named SVP, clinical development and translational medicine after six years as an exec at Revance , which crossed the FDA goal line with its Botox rival Daxxify on Thursday. Caroline Rowland → Much-hyped AI outfit Exscientia has named Caroline Rowland as chief people officer. Rowland had held a series of positions at UK software design company Arm since 2015 and was VP of talent before her departure. Exscientia made a splash on Nasdaq with a $304.7 million IPO on top of multiple nine-figure raises last year, and teamed up with Sanofi in January on an AI pact worth $100 million upfront. Last month, Exscientia and Bayer “ mutually agreed to end ” a partnership that dates back to January 2020 after hitting a “proof of concept milestone supporting advancement of the program into late discovery.” Tim Moore → Tim Moore didn’t survive PACT Pharma ’s wave of layoffs as the cell therapy biotech scrapped its lone clinical trial in late August, but he has since resurfaced as COO of Dallas TIL player Instil Bio . Moore, the president and COO of PACT Pharma for two years until his dismissal, had a 12-year run at Genentech and then flew into Kite as EVP, technical operations from 2016-19. Since its 2021 IPO , Instil Bio ’s stock price $TIL has plummeted nearly 80%, an oft-told tale during this bear market. Gustavo Pesquin → Ready for the FDA to comb through its latest formulation of the Parkinson’s combo carbidopa / levodopa , Amneal Pharmaceuticals has brought in Gustavo Pesquin as chief commercial officer, specialty. So ends an 11-year association with Sanofi in a range of roles, including North America head for general medicines and global head of the diabetes and cardiovascular franchise. Fabio Cataldi → Almost three months into Greg Oakes ’ tenure as the CEO of Landos Biopharma , the Xontogeny biotech and LianBio IBD partner has recruited Fabio Cataldi as CMO. Like Doug Manion and other execs before him, Cataldi is turning the page after Pfizer’s $6.7 billion purchase of Arena Pharmaceuticals . Before he was Arena’s VP and therapeutic area head of gastroenterology, Cataldi was team lead of gastroenterology and immunology in global pharmaceutical R&D at AbbVie, and he’s also held leadership roles at Shire , Pfizer, Biogen and Novartis . Carolyn Bertozzi → Stanford chemistry luminary Carolyn Bertozzi has been appointed to the scientific advisory board of Rondo Therapeutics , a Bay Area I/O player that got the ball rolling in March with a $67 million Series A . Bertozzi has founded a number of biotechs — including Redwood Bioscience (which Catalent bought in 2014), Palleon Pharma and Lycia Therapeutics — and she was one of 15 LGBTQ biopharma leaders that Endpoints News featured in June . Simon Lowry → Simon Lowry has signed on as CMO of Zenas BioPharma , Lonnie Moulder ’s autoimmune and rare disease play that picked up Xencor ’s obexelimab last November. Lowry, the former head of immunology R&D at Roivant , has held CMO posts at Kinevant and Sun Pharma North America . At Novartis, Lowry was VP, global medical affairs franchise head, immunology & dermatology, and to close out his six years at Pfizer, he was VP, oncology medical affairs group leader. Georgia Erbez → Rusty Williams ’ B cell joint Walking Fish Therapeutics has welcomed Georgia Erbez as COO, almost a year to the day after its $50 million Series A . Erbez spent the last four years as CFO of Harpoon Therapeutics and she’s also been finance chief with Zosano Pharma and Revolution Medicines . Concurrent with her appointment, Walking Fish also announced the opening of its 22,000 square-foot headquarters in South San Francisco. Howard Bernstein → On Oct. 31, Howard Bernstein will step aside as CSO of SQZ Biotechnologies and will join the board of directors, chaired by Polaris ’ Amy Schulman . CMC SVP Ipsita Roymoulik , VP of translational research Scott Loughhead and head of engineering Maisam Dadgar will split CSO duties. “While it is an emotional experience to step away from my full-time role, I am very proud of my technical leadership team and look forward to seeing them demonstrate their potential,” Bernstein said in a statement. Garrett Nichols → Paul Peter Tak has filled two key positions at oncolytic virus biotech Candel Therapeutics , which has competition from San Diego-based IconOVir Bio and Germany’s Abalos Therapeutics in a field that’s hit into a lot of hazards in the past. New CFO Jason Amello comes from Saniona, where he took on the same role for nearly two years, and the Genzyme vet was SVP, CFO and treasurer with Akebia Therapeutics from 2013-20. Peer Review recently mentioned that Garrett Nichols would fill in temporarily as CMO at ContraFect , and he’s heading to Candel in the same capacity after two years as the medical chief at Istari Oncology . Roger Sawhney → Garuda Therapeutics has welcomed aboard Roger Sawhney as CFO. This isn’t Sawhney’s first time donning the CFO hat, having served in the same role at Omega Therapeutics . In addition to his role as CFO at Omega, Sawhney also served as the company’s CBO. Earlier in his career, Sawhney had gigs at Bain Capital , Boston Consulting Group and Novartis. → London-based Purespring Therapeutics has found itself a CSO in Alice Brown . Brown brings with her experience from her time at Takeda , GammaDelta Therapeutics (VP research and VP gene engineering) and PsiOxus (director of biology). → Lytix Biopharma has reeled in Stephen Worsley as CBO. Worsley hails from Redwood Bioscience/Catalent, where he served as VP strategic business development. Prior to that, Worsley was with Sutro Biopharma (CBO), IndiMolecular (SVP business development), and Peregrine Pharmaceutical (VP of business development). Trier Bryant → Alloy Therapeutics —the conglomerate of biotech services, technologies and spinouts backed by billionaire investor Peter Thiel — has named Trier Bryant president of 82VS , Alloy’s affiliated venture studio. Bryant comes aboard from aerospace startup Astra , where she served as chief people officer. Bryant also has experience under her belt from stints at Twitter , Goldman Sachs , and formerly served in the US Air Force. Lorna Neilson → Jeremiah Hanes jumped on board to lead R&D at Ansa Biotechnologies a couple weeks ago, and the DNA synthesis upstart has quickly followed that up with the hire of Lorna Neilson as CBO. Neilson had spent a decade with Takara Bio USA and was VP of corporate development. → Backed by ARCH and Takeda in a $24 million Series A round , tRNA startup hC Bioscience has tapped Yosef Landesman as CSO and Gautam Goel as chief data science officer. Landesman was SVP of research and translational medicine during an 11-year stay at Karyopharm , and he also worked on such drugs as Onpattro while he was a senior scientist at Alnylam . Goel is a translational computational biologist who logged four years with ImmusanT as director of precision medicine. Jenny Holt → San Francisco eye disease biotech Ray Therapeutic s has enlisted Peter Francis as chief scientific & medical officer and Jenny Holt as SVP, program portfolio management. Francis climbed through the ranks at 4D Molecular Therapeutics , becoming CMO and then CSO at the tail end of his seven-year tenure with the company. Holt is a Novartis vet and also comes from 4DMT, where she was head of program management. → Remus Vezan is joining the team at CytoImmune Therapeutics and taking up a role he’s familiar with as CMO. Vezan previously served as CMO of CERo Therapeutics and was the former executive director of clinical development at Kite, overseeing drugs like Yescarta and Tecartus . → Plasma-focused Evolve Biologics is bringing aboard Chris Carr as CFO. Carr’s career spans roles at Aeon Biopharma (CFO), Dendreon (EVP of finance and IT), Abbott Labs , PerkinElmer and Genzyme. Andrea van Elsas → Andrea van Elsas has been promoted to partner at Third Rock Ventures , which raised $1.1 billion for its sixth fund in June. A venture partner at Third Rock since 2020, van Elsas is the interim CSO at Abata Therapeutics and is a board member at Alexis Borisy -chaired Celsius Therapeutics . → Led by Nurix and Five Prime alum Pierre Beaurang , Nitrase Therapeutics — the crew previously named Nitrome Biosciences — has selected Arun Kashyap as SVP of oncology. Kashyap takes on this new role after two years as Arch Oncology ’s VP of research, and he had a brief tenure with Dren Bio as senior director, protein science. Catherine Mathis → Once named Horama and fueled by a Series B extension from July 2021, French biotech Coave Therapeutics has picked up Catherine Mathis as VP, regulatory affairs and Julien Berger as head of legal affairs. Mathis is a 20-year Transgene vet who comes to Coave from Enterome , where she was chief development officer, while Berger had a 16-year career with Genzyme/Sanofi Genzyme until he joined Galapagos in 2020 as senior legal counsel director. → Immunomic Therapeutics has brought on Frances Harrison as SVP, regulatory affairs. Harrison is taking over the reins from Louise Peltier , who retired. Harrison makes her way to the Maryland-based company from BTG International (now Boston Scientific ), where she served as SVP, global regulatory affairs and compliance. Her other stints include roles at Life Cell Corporation ( Allergan ), Covidien ( Medtronic ) and Lumenis , among others. → Medical AI company Owkin has brought on Jean-Philippe Vert as chief research and development officer. Vert formerly served as a professor at Mines Paris PSL University and a research scientist at Google Brain . Ian Read → Ex-Pfizer chief Ian Read has turned up as chairman of the board at Areteia Therapeutics . In July, the asthma biotech made a $350 million debut with contributions from Bain, GV , ARCH, Sanofi and the VC where Read is a partner, Clive Meanwell ’s Population Health Partners . → Efgartigimod developer argenx has made room for Camilla Sylvest on the board of directors. Sylvest has been with Novo Nordisk for 26 years and is the Danish pharma’s EVP, commercial strategy & corporate affairs. Neha Krishnamohan → Riding high with its FDA-approved plaque psoriasis drug Zoryve and its buyout of Ducentis BioTherapeutics , Arcutis has elected Neha Krishnamohan to the board of directors. After 13 years with Goldman Sachs , Krishnamohan became Kinnate Biopharma ’s CFO and EVP of corporate development in June 2021. → Oncolytics Biotech has lassoed in Jonathan Rigby to its board of directors. Currently, Rigby serves as group CEO of Revelo Biotherapeutics and also sits on the boards of Revelo, ImmunoMolecular Therapeutics and BIOS (chairman). Rigby’s career also spans roles at SteadyMed (CEO), Zogenix (co-founder), Merck, Bristol Myers and Profile Therapeutics (now Phillips Medical ). Nicole Paulk → Nicole Paulk has joined the scientific advisory board at GRO Biosciences , which is chaired by co-founder George Church . Paulk, an assistant professor of AAV gene therapy at UCSF, is also on the SABs of Excision BioTherapeutics and Dyno Therapeutics . → Societal CDMO , formerly known as Recro before its rebrand in March, has pulled up a chair for Elena Cant on its board of directors. Cant currently serves as COO at TwinStrand Biosciences and formerly served in roles at Takeda, Hospira , Mead Johnson Nutrition , and McKinsey & Company . → Leap Therapeutics is adding a new face to its board of directors with the appointment of Richard Schilsky . Schilsky is the former CMO and EVP of ASCO and served as its president from 2008-2009. Additionally, Schilsky served in chair roles for the NCI and the FDA’s ODAC.

Gene TherapyCell TherapyExecutive ChangeAcquisitionASCO

08 Aug 2022

·medcitynews.com

Pfizer to buy sickle cell disease biotech Global Blood Therapeutics in $5.4B deal

Pfizer has reached a $5.4 billion deal to buy Global Blood Therapeutics, a company with one commercialized therapy for sickle cell disease and a pipeline of additional drugs in various stages of development for the blood disorder. According to financial terms announced Monday, Pfizer is paying $68.50 for each share of Global Blood, which represents a 7.2% premium to Friday’s closing price and a more than 100% premium to the biotech’s stock price a week ago, before speculation about Pfizer acquisition started circulating. The Wall Street Journal reported last Friday that Pfizer was in advanced talks to buy South San Francisco-based Global Blood. Global Blood’s drug Oxbryta, is one of the few FDA-approved treatments for sickle cell disease, and the only one that addresses a root cause of the disorder. The inherited condition leads hemoglobin, the oxygen-carrying protein in red blood cells, to become rigid and misshapen, which in turn makes the normally round and flexible red blood cell rigid, taking on a sickle shape. These rigid blood cells clog and impede blood flow and the delivery of oxygen to tissues. Complications include vaso-occlusive crisis (VOC), in which the blocked blood flow and oxygen deprivation sets off an inflammatory response from the body. Oxbryta is a small molecule designed to bind to hemoglobin and block polymerization, the process in which hemoglobin inside red blood cells clump together leading the cell to take on a sickle shape. The FDA approved the drug in 2019. It accounted for $194.7 million in revenue in 2021, according to Global Blood’s annual report. In the first half of 2022, the company reported $126.7 million in Oxbryta sales, a 46.3% increase compared to the first half of last year. Oxbryta, a once-daily pill, is a chronic treatment taken potentially for the patient’s entire life. The drug could eventually face competition from one-time treatments in the form of genetic medicines in clinical development by Bluebird Bio, Sangamo Therapeutics, and Vertex Pharmaceuticals. But in the meantime, Global Blood is developing a next-generation version of Oxbryta, code-named GBT601. This small molecule, which comes from Global Blood’s labs, is designed to work the same way as Oxbryta, but with potentially greater efficacy at a much lower dose. Global Blood has advanced GBT601 to the Phase 2 part of a Phase 2/3 clinical trial. The most advanced program in the pipeline is a polymerization-blocking drug called inclacumab. This monoclonal antibody is designed to target P-selectin, a protein that is known to reduce the incidence of VOC. That Global Blood drug, licensed from Roche in 2018, is currently in Phase 3 testing. Dealmaking has brought Global Blood additional drug candidates for sickle cell disease. A 2019 partnership with Syros Pharmaceutical covers the development of small molecule drugs that boost the production of fetal hemoglobin. This approach, which is still preclinical, is intended to dilute the concentration of the form of hemoglobin that leads to sickle cell disease, offering a potential cure. Last year, the company committed up to $353 million for the rights to two Sanofi small molecules in preclinical development. Though sickle cell disease is relatively rare in the developed world, Pfizer estimates that there are about 4.5 million people globally who have the disorder and more than 45 million people who carry the sickle cell trait. The disorder disproportionately affects those with sub-Saharan African ancestry, though it can also occur in other ethnic groups. In addition to its U.S. approval, Oxbryta has regulatory nods in the European Union, United Arab Emirates, Oman, and Great Britain. Pfizer said it plans to use its global footprint to accelerate distribution of Global Blood’s drug to parts of the world most affected by the disorder. If the other drugs in Global Blood’s pipeline reach the market, Pfizer estimates that combined with Oxbryta, they could achieve peak sales topping $3 billion. “The deep market knowledge and scientific and clinical capabilities we have built over three decades in rare hematology will enable us to accelerate innovation for the sickle cell disease community and bring these treatments to patients as quickly as possible,” Albert Bourla, Pfizer’s chairman and CEO, said in a prepared statement. Pfizer’s Global Blood acquisition agreement follows misses in the pharmaceutical giant’s earlier efforts to address sickle cell disease. Two years ago, Pfizer ended a partnership with GlycoMimetics on the drug rivipansel. That drug, which was in development for treating the VOC complication of sickle cell disease, failed its Phase 3 study. Early this year, Pfizer terminated a Phase 1 test of PF-07059013, another small molecule that was in development for the disorder. The pharma giant said that the drug did not demonstrate sufficient pharmacological effect. The Pfizer and Global Blood boards of directors have both approved the acquisition, which the Wall Street Journal reported was a competitive process that drew interest from other potential buyers. The merger agreement bars Global Blood from seeking another bid, but permits the company to discuss and negotiate with a party that approaches with a higher offer. If Global Blood decides to take that offer, it would owe Pfizer a $217 million termination fee. But if the merger agreement is terminated for certain antitrust reasons, Pfizer must pay Global Blood a “reverse termination fee” of $326 million. In a research note sent to investors, William Blair analyst Raju Prasad wrote that his firm does not expect the Federal Trade Commission will have any major issues with the acquisition given the rarity of sickle cell disease and its lack of treatment options.

CollaborateAntibodySmall molecular drugAcquisition

100 Deals associated with Rivipansel Sodium

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R&D Status

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Pain	Phase 3	US	GlycoMimetics, Inc.	16 Dec 2015
Pain	Phase 3	CA	GlycoMimetics, Inc.	16 Dec 2015
Anemia, Sickle Cell	Phase 3	US	GlycoMimetics, Inc.	17 Jun 2015
Anemia, Sickle Cell	Phase 3	CA	GlycoMimetics, Inc.	17 Jun 2015
Vaso-occlusive crisis	Phase 3	US	GlycoMimetics, Inc.	17 Jun 2015
Vaso-occlusive crisis	Phase 3	CA	GlycoMimetics, Inc.	17 Jun 2015
liver function failure	Phase 1	US	GlycoMimetics, Inc.	01 Sep 2016
Renal Insufficiency	Phase 1	US	GlycoMimetics, Inc.	01 Jun 2016
Hematologic Neoplasms	Preclinical	US	GlycoMimetics, Inc.	-

Clinical Result

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT02187003 \| NCT02433158 (OLE \| RESET, Pubmed) Manual	Phase 3	Vaso-occlusive crisis	320	Rivipansel	htctwwamow(itpmjfxcqr) = not different between rivipansel and placebo ivjysaginq (jbsvgqefnr ) View more	Positive	18 Aug 2022
				Placebo
NCT02871570 (CTgov)	Phase 1	liver function failure	16	rivipansel (Moderate Hepatic Impairment Group)	rgfixercfd(zbmyobxygw) = bwpbtsvril pojizcppzd (kqgfkmtrag, jhnszomhxy - kfnrtjxknc) View more	-	28 Nov 2018
				rivipansel (Normal Hepatic Function Group)	rgfixercfd(zbmyobxygw) = yfkmghcfwa pojizcppzd (kqgfkmtrag, dbjnkhefax - awfktteiza) View more
NCT01119833 (Pubmed \| Circulation) Manual	Phase 2	Anemia, Sickle Cell	76	rivipansel	rvskeapxpw(wxcfjuoemz) = jgpexgvvzt aaibipgdbw (yamtsyyvqo )	Positive	23 Apr 2015
				Placebo	rvskeapxpw(wxcfjuoemz) = esvxjunwrl aaibipgdbw (yamtsyyvqo )
NCT00911495 (CTgov)	Phase 1/2	Anemia, Sickle Cell	15	GMI-1070	glmnarzopp(spovkbiidq) = ifyjhzaoay vnbnanwjga (pfejdcplmx, brdhjjcmdp - vilucmysee) View more	-	03 May 2013

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