4 trends witnessed at this year’s DPharm conference
[Image courtesy of iStock Photo]
One of the largest industry conferences, DPharm, was recently held on September 13 to 14 in Boston. Focusing on innovations to modernize clinical research, this year’s event featured speakers ranging from the popular author Malcolm Gladwell to former FDA Commissioner Dr. Scott Gottlieb and BIO CEO Dr. Michelle McMurry-Heath.Here, we provide reflections from several industry experts who attended the event.
1. An urgency to put patients first is gaining ground“There were several topics at this year’s DPharm that truly resonated with attendees,” said Jane Myles, vice president of clinical trials innovation at Curebase, a decentralized clinical trial software platform and clinical service provider. “Malcolm Gladwell emphasized how urgency is the key to driving innovation within the industry,” Myles said. “And Henry Wei referenced a quote from Google: ‘If we focus…
Pfizer shares top-line data from pivotal study of pentavalent meningococcal vaccine in adolescents
Aiming to extend its vaccine empire, Pfizer (NYSE:PFE) has announced that its meningococcal vaccine (MenABCWY) candidate met primary and secondary endpoints in a Phase 3 study.
The NCT04440163 study tested the vaccine in healthy individuals between the ages of 10 and 25.
The vaccine candidate demonstrated non-inferiority to the licensed vaccines Trumenba and Menveo for the five meningococcal serogroups that cause the most invasive meningococcal disease: serogroups A, B, C, W and Y.
If approved, the vaccine could be the first to protect against the five meningococcal serogroups.
Pfizer plans on filing for U.S. approval in the fourth quarter of 2022. The company also plans to seek authorization for the vaccine in other regions.
At present, FDA has approved discrete MenACWY and MenB vaccines.
The NCT04440163 study randomly assigned participants to receive two doses of MenABCWY or two doses of Trumenba plus one dose of Menveo. Pfizer is …
Neuron23 and Qiagen partner on NGS companion diagnostic for LRRK2 inhibitor
The early-stage biotech Neuron23 and Qiagen (NYSE:QGEN) will work together to develop the first next-generation sequencing (NGS) companion diagnostic for Neuron23’s leucine-rich repeat kinase 2 (LRRK2) inhibitor for Parkinson’s disease NEU-723.
In the collaboration, Qiagen will focus on developing and validating a clinical trial assay to detect biomarkers that predict the responsiveness of Parkinson’s disease patients to an LRRK2 inhibitor.
The companion diagnostic is the first to be developed for Parkinson’s, according to Neuron23 CEO Nancy Stagliano. “Qiagen’s blood-based test will help to identify patients with Parkinson’s disease who are likely to respond to Neuron23’s LRRK2 inhibitor,” Stagliano said in a news release. “The development of a companion diagnostic identifying this sub-population of Parkinson’s disease patients will de-risk the clinical development of Neuron23’s LRRK2 inhibitor and help identify individuals who may benefit from this disease-…
Pfizer launches Phase 3 mRNA flu vaccine study
This computer-generated image shows influenza virus hemagglutinin epitopes arrayed on a nanoparticle. [Image courtesy of NIAID]
Big Pharma behemoth Pfizer (NYSE: PFE) has dosed the first participants in a pivotal Phase 3 clinical trial to test its quadrivalent modified RNA (modRNA) influenza vaccine candidate.The mRNA flu vaccine study will enroll roughly 25,000 healthy U.S. adults.
Pfizer is not the only company to develop an mRNA flu vaccine. In December, Moderna (Nasdaq:MRNA) announced positive interim data from a Phase 1 study of its quadrivalent seasonal flu vaccine candidate, mRNA-1010. That data, however, disappointed investors, as mRNA-1010 had a similar performance to Sanofi’s (NSDQ:SNY) Fluzone HD flu vaccine.
Moderna’s (Nasdaq:MRNA) mRNA-1010 flu vaccine is now in a Phase 3 study. That study is fully enrolled with approximately 6,000 participants. Moderna anticipates the va…
U.S. government launches CP-RND, a public-private partnership for rare neurodegenerative diseases
The FDA and NIH have debuted the Critical Path for Rare Neurodegenerative Diseases (CP-RND), a public-private partnership focused on developing new therapies for amyotrophic lateral sclerosis (ALS) and rare neurodegenerative diseases.
One of the goals of the CP-RND is to foster innovation in drug discovery and development as well as clinical testing. In addition, CP-RND will aim to develop a therapy that halts the progression of ALS.
In a similar vein, FDA launched its Action Plan for Rare Neurodegenerative Diseases in June, including amyotrophic lateral sclerosis (ALS).
FDA and NIH have chosen Tucson, Arizona-based Critical Path Institute (C-Path) to help guide the CP-RND initiative.
Founded in 2005 as a public-private partnership, C-Path is an independent, nonprofit organization.
Earlier this year, C-Path established Amsterdam as the head of its European operations.
In its work on the CP-RND project, C-Path will gather experts in r…
Women in Pharma: Insmed’s CFO on what is most fulfilling about working in life sciences
Sara Bonstein [Image courtesy of Insmed]
In January 2020, rare disease biopharma Insmed (Nasdaq: INSM) hired Sara Bonstein as its chief financial officer. At that point, Bonstein had more than 15 years of operational and financial experience in the life sciences industry.In the following interview highlighting her role as a female pharma leader, Bonstein shares what initially attracted her to drug discovery and development and which projects at Insmed she finds most rewarding. She also provides perspective on leadership skills and what the industry can do to promote greater participation of young women in the pharma industry — especially in senior finance roles.
What first drew you to drug discovery and development? When did you first know you wanted to be in the industry?Bonstein: During my freshman and sophomore years of college, Johnson & Johnson was recruiting on campus and I went to the informa…
Mindset Pharma bolsters IP portfolio with three novel families of next-generation psychedelics
Focused on developing therapies for neuropsychiatric and neurological disorders, Mindset Pharma (CSE:MSET/FSE:9DF) has identified three families of next-generation psychedelics.
Families 6, 7 and 8 are chemically distinct, small molecule non-tryptamine scaffolds. Tryptamine hallucinogens include LSD, DMT and psilocybin. Mindset Pharma has filed provisional patent applications for the three families.
The Toronto-based company had already announced it had four families of investigational psychedelic compounds. The first includes a psilocybin-like conjugate known as MSP-1014.
The second family includes high-potency, short-acting psilocybin-like compounds, while the third includes a low-potency, long-duration compound for micro-dosing.
To learn more about the new compounds and Mindset Pharma in general, we reached out to Chief Scientific Officer Joseph Araujo. He provided an overview of what sets Mindset apart from the competition in the therapeutic p…
Inhaled COVID-19 vaccines making inroads in China, India and Russia
This colorized scanning electron microscope image shows SARS-CoV-2 (round blue objects), the virus that causes COVID-19, emerging from the surface of cells cultured in the lab. [Image courtesy of National Institute of Allergy and Infectious Diseases]
The idea of inhalable COVID-19 vaccines may not be new, but, until recently, no country had approved a COVID-19 vaccine with an alternative delivery route.That changed on March 31, 2022, when Russia greenlit the Galaleya Institute’s Sputnik V inhaled vaccine. The country authorized a similar nasal-drop-based vaccine known as Salnavac on July 4, 2022.
To authorize those vaccines, Russian regulators weighed data from the formerly approved Sputnik V vaccine in conjunction with Phase 1/2 safety and immunogenicity data from the intranasal versions of that vaccine.
More recently, China authorized the inhalable Convidecia Air COVID-19 vaccine from CanS…
Niraparib promoted long-term progression-free survival benefit in Phase 3 advanced ovarian cancer study
Brentford, UK–headquartered GSK (LSE/NYSE:GSK) reported long-term data from the phase 3 PRIMA study indicating that the PARP inhibitor Zejula (niraparib) promoted a sustained and clinically meaningful progression-free survival (PFS) benefit in ovarian cancer patients.
The survival benefit was evident across biomarker subgroups, including BRCAm, HRd and HRp.
Patients in the HRd subgroup had a 48% reduction of progression or death compared to placebo.
The estimated probability of no progressive disease or death at four years in the broader population was 24% for niraparib compared to 14% for placebo.
GSK plans to present the updated efficacy analysis data on September 11 at the (European Society for Medical Oncology) ESMO Annual Meeting in Paris.
GSK noted in a news release that the overall survival data are “not yet mature based on the prespecified analysis plan.”
The study tested niraparib as maintenance therapy in first-line ovarian…
Lilly’s lebrikizumab yielded durable skin clearance in Phase 3 atopic dermatitis studies
Eli Lilly and Company’s (NYSE:LLY) Phase 3 monotherapy ADvocate trials in atopic dermatitis (AD) found that the investigational interleukin 13 inhibitor lebrikizumab supported durable skin clearance and reduced itching in a subset of patients with atopic dermatitis.
The Indianapolis–based company defined responders as those with at least 75% improvement in the Eczema Area and Severity Index from baseline (EASI-75) or an Investigator Global Assessment of 0 or 1 (“clear” or “almost clear”) with a 2-point improvement at week 16 without rescue medication. The clinical trial re-randomized responders to receive lebrikizumab 250 mg every two weeks or four weeks or a placebo for 36 more weeks.
Lebrikizumab performed similarly in responders when dosed at a two- or four-week interval after week 16.
“Despite available treatment options, many patients with atopic dermatitis experience distressing symptoms every day over the course…
Why Medipost is upbeat about its stem cell-based treatment of osteoarthritis
Medipost has announced that its allogeneic human umbilical cord blood-derived mesenchymal stem cell product for knee osteoarthritis was featured in the peer-reviewed Orthopaedic Journal of Sports Medicine.
The Seoul, Korea–headquartered company specializes in allogeneic umbilical cord blood-derived mesenchymal stem cells for various therapeutic applications.
Medipost’s Phase 3 study found that 97% of recipients of its Cartistem cellular therapeutic agent showed significant improvement in cartilage restoration after 48 weeks.
Participants in the study underwent a five-year observational follow-up.
More than a decade of Cartistem historySouth Korean regulatory authorities approved Cartistem in January 2012, making it one of the world’s first companies to obtain approval for cell therapy.
Medipost is optimistic about the potential to expand the use of Cartistem, given the rising rates of knee osteoarthritis. “People are gett…