// Biotech and Pharma Therapeutics
Bristol Myers taps startup to boost cell therapy production
April 22, 2024 / Cell Therapy / CAR-T Therapies / Bristol Myers Squibb / Partnership
Bristol Myers Squibb has partnered with Cellares, a California-based startup, to boost production of CAR-T cancer cell therapies. The deal, valued at up to $380 million, aims to enhance production efficiency through Cellares’ automated manufacturing platform, the Cell Shuttle.
Ahead of Alnylam’s key heart disease readout, experts say Pfizer rival would be hard to displace
April 22, 2024 / Alnylam / Amvuttra / Pfizer / ATTR
Alnylam Pharmaceuticals’ Amvuttra, an RNA silencer, faces challenges in competing with Pfizer’s tafamidis for treating transthyretin amyloid cardiomyopathy (ATTR-CM). Experts believe tafamidis will retain its lead due to ease of use, safety, and robust clinical data.
Biogen’s Alzheimer’s drug launch on ‘steady pace’ as logistical hurdles recede
April 24, 2024 / Biogen / Alzheimer’s / Skyclarys Launch
Biogen’s first-quarter earnings exceeded Wall Street expectations, driven by reduced expenses and increased sales of its newly launched Alzheimer’s drug, Leqembi, and Skyclarys. These results indicate Biogen’s steady turnaround progress.
Eli Lilly to acquire manufacturing facility from Nexus Pharma
April 22, 2024 / Eli Lilly / Acquisition / Manufacturing / Diabetes / Obesity / GLP-1 / Maunjaro
Eli Lilly is acquiring a manufacturing facility from Nexus Pharmaceuticals to produce injectable medicines. The new plant in Pleasant Prairie, Wisconsin, aims to address supply constraints for diabetes and obesity drugs like Mounjaro and Zepbound, which have faced increased demand.
Does Neurocrine, The No. 1 Biotech Stock, Have The Next Ketamine On Its Hands?
April 24, 2024 / Neurocrine Biosciences / Mental Health / Depression
Neurocrine Biosciences’ experimental drug, NBI-1065845, for major depressive disorder, showed promising results, outperforming ketamine-derived Spravato in symptom improvement. The drug’s novel mechanism may offer ketamine-like benefits with potentially fewer side effects, but safety concerns remain.
// 4th Industrial Revolution
Overheard around the Kaiser nurses’ protest over AI in healthcare
April 23, 2024 / Kaiser / AI / Healthcare / Patient Saf
Kaiser Permanente nurses protested in San Francisco, opposing the use of AI in healthcare. The demonstration, organized by the California Nurses Association, raised concerns about job security and patient safety, with various stakeholders discussing AI’s impact on empathy and patient care.
New codes from the AMA could mean more RPM reimbursement by 2025
April 22, 2024 / Patient Health / AMA CPT / Healthcare
The American Medical Association (AMA) proposes changes to remote patient monitoring (RPM) coding, including new codes for shorter monitoring periods and reduced time requirements for care management. These changes aim to improve RPM reimbursement and expand adoption across healthcare providers.
Patient concerns in healthcare digitalisation: Rising inequalities
April 22, 2024 / Healthcare / EHDS Legislation
Healthcare digitalization can improve processes and expand access to care, but it raises concerns about rising health inequalities, data security risks, and lack of interoperability. Solutions like the European Health Data Space (EHDS) aim to address these issues, but challenges remain.
First-Ever Gene Editing Success in Kissing Bugs
April 24, 2024 / Gene Editing / Disease Control / New Tech
Researchers have achieved the first successful gene editing in kissing bugs, key vectors for Chagas disease, using CRISPR-Cas9 technology. This breakthrough opens new possibilities for controlling the spread of Chagas disease, a major public health concern in Central and South America.
Machine learning helps UK biotech develop liver disease drugs that have led to a $1bn deal
April 23, 2024 / Ochre Bio / Partnership / Liver Disease / Machine Learning / Boehringer Ingelheim
Oxford-based Ochre Bio is partnering with Boehringer Ingelheim to develop liver disease treatments using the liver’s regenerative capacity. Their machine learning-based drug discovery platform aims to find novel regenerative pathways, addressing a critical need for new liver disease therapies.
// Business & Markets
Boehringer Ingelheim and Ochre Bio partner on liver disease in deal worth over $1bn
April 23, 2024 / Boehringer Ingelheim / Ochre Bio / Liver Diease / MASH
Boehringer Ingelheim and Ochre Bio have partnered in a deal exceeding $1 billion to develop novel regenerative treatments for chronic liver disease (CLD), including metabolic dysfunction-associated steatohepatitis (MASH) cirrhosis. The partnership aims to use Ochre’s proprietary platform, leveraging machine learning and human big data, to find innovative regenerative targets to prevent or reverse disease progression.
Ex-Stanford president’s AI biotech startup snares $1 billion
April 24, 2024 / AI / Drug Discovery / Xaira Therapeutics / Startup / Funding / Machine Learning
Xaira Therapeutics, an AI drug discovery startup, has launched with over $1 billion in funding. The San Francisco-based company, co-founded by Marc Tessier-Lavigne, aims to use machine learning and data generation to drive therapeutic product development. Its high-profile board includes industry veterans and former FDA Commissioner Scott Gottlieb.
Novartis raises forecasts as top drug sales beat Wall Street estimates
April 23, 2024 / Novartis / Drug Sales
Novartis reported stronger-than-expected first quarter earnings, with robust sales of Entresto and Cosentyx driving the beat. The Swiss pharmaceutical company raised its 2024 sales guidance to high single to low double digits. Shares rose up to 4.5% following the announcement.
Incyte, fresh off gaining MorphoSys drug, buys Escient for $750M
April 23, 2024 / San Diego / Incyte / Immunology / Inflammation
Incyte is acquiring San Diego-based Escient Pharmaceuticals for $750 million, gaining two immunology and inflammation candidates, EP262 and EP547. The acquisition bolsters Incyte’s dermatology portfolio and complements its JAK1/JAK2 inhibitor cream Opzelura, with additional launch opportunities expected by 2029.
Small medical practices will close because of Change cyberattack, says AMA
April 22, 2024 / Healthcare / Cyberattack
The February 21 Change Healthcare cyberattack’s ongoing impact threatens small medical practices, possibly causing closures that could disrupt patient care, according to an American Medical Association (AMA) survey. Practices with 10 or fewer physicians face significant challenges due to revenue cycle disruptions.
// Legal & Regulatory
Neurocrine scores surprise win with depression drug
April 23, 2024 / Depression / Clinical Trials / FDA / Mental Health
Neurocrine Biosciences achieved a surprise success with its experimental depression drug in a Phase 2 clinical trial, meeting its main goal and key secondary endpoints. The results suggest significant improvement in depression symptoms and may lead to late-stage testing, with potential approval as a first-of-its-kind treatment.
FDA rejects Abeona cell therapy, asks for more manufacturing data
April 23, 2024 / FDA / Abeona therapeutics / Cell Therapy
The FDA rejected Abeona Therapeutics’ application for its cell therapy prademagene zamikeracel, or pz-cel, due to manufacturing concerns. The therapy was intended to treat epidermolysis bullosa, a rare skin condition. Abeona plans to resubmit the application in the third quarter, aiming to address the FDA’s questions on manufacturing and product safety.
FDA Rejects Abeona’s Epidermolysis Bullosa Cell Therapy, Asks for CMC Data
April 23, 2024 / FDA / Abeona Therapeutis / CMC
The FDA rejected Abeona Therapeutics’ application for its gene-corrected cell therapy prademagene zamikeracel (pz-cel), designed to treat recessive dystrophic epidermolysis bullosa (RDEB), citing chemistry, manufacturing, and controls (CMC) issues. Abeona aims to resubmit the application in the third quarter of 2024 after addressing the FDA’s concerns.
ImmunityBio Wins FDA Approval for First IL-15 Superagonist for NMIBC
April 23, 2024 / ImmunityBio / FDA Approval / IL-15 / Anktiva / Bladder Cancer
The FDA approved ImmunityBio’s IL-15 superagonist nogapendekin alfa inbakicept, or Anktiva, for the treatment of non-muscle invasive bladder cancer (NMIBC). Anktiva will be used with Bacillus Calmette-Guérin (BCG) in BCG-unresponsive patients with carcinoma in situ, demonstrating a 62% complete response rate in a Phase II/III trial.
Exo® Launches FDA-Cleared AI on Exo Iris™ to Address Heart Failure
April 23, 2024 / Exo Iris / Ultrasound / AI / FDA / Medical Devices
Exo launched its FDA-cleared cardiac and lung AI applications on Exo Iris, a high-performance handheld ultrasound device. This innovation accelerates heart failure diagnosis and care pathways. The pocket-sized device is especially beneficial in underserved and rural areas, addressing the challenges of costly echocardiography and limited access to medical imaging.
// Research & Development
Sanofi’s $3.7B Principia Buy Starts to Pay Off with Phase III Rilzabrutinib Win
April 23, 2024 / Sanofi / Clinical Trials
Sanofi’s investigational BTK inhibitor rilzabrutinib met the primary endpoint in the Phase III LUNA 3 study, showing a significant durable platelet response in adult patients with immune thrombocytopenia (ITP). Sanofi plans to submit regulatory filings in the U.S. and EU by the end of 2024, with the drug demonstrating potential as a first-in-class oral BTK inhibitor.
Sanofi pulls off phase 3 LUNA landing, sparking race to regulators for BTK inhibitor
April 23, 2024 / Sanofi / FDA Approval / Clinical Trials
Sanofi’s $3.7 billion acquisition of Principia Biopharma delivered a phase 3 success with rilzabrutinib, an oral, reversible BTK inhibitor, in a study for immune thrombocytopenia (ITP). Sanofi plans to file for approval in the U.S. and Europe by the end of 2024, positioning rilzabrutinib as a potential multi-indication blockbuster.
Krystal Biotech doses first subject in lung cancer drug trial
April 23, 2024 / Krystal Biotech / Lung Cancer / Cytokine Therapy / Lung Cancer / FDA / Oncology
Krystal Biotech has initiated a Phase I trial for its inhaled oncology candidate, KB707, to treat patients with advanced or metastatic lung cancer. KB707 is a modified HSV-1 vector that delivers human interleukins IL-12 and IL-2 to the lungs. The trial will assess the drug’s safety and tolerability, aiming to find the optimal dose through a dose escalation and expansion phase.
AbbVie-bound Cerevel reports phase 3 Parkinson’s victory in early win for $8.7B deal
April 18, 2024 / Parkinson’s / Clinical Trials / AbbVie / Tria; Success
Cerevel Therapeutics, set to be acquired by AbbVie, announced the success of its phase 3 Parkinson’s disease trial with tavapadon, a dopamine D1/D5 receptor partial agonist. The trial, involving 507 adults with Parkinson’s and motor fluctuations, demonstrated that patients on tavapadon spent significantly more time without troublesome uncontrolled, involuntary movements compared to placebo, with a favorable safety profile. This development adds value to AbbVie’s $8.7 billion acquisition deal with Cerevel.
China’s investigator trials accelerate competitive CAR T development
April 22, 2024 / China / CAR T / Cell Therapy
China’s CAR T-cell therapy market is projected to grow from $72 million in 2022 to $342 million by 2032. With over 400 CAR T therapies in the pipeline, the market is driven largely by specialized Chinese biotechs. Strategic partnerships between multinational corporations and domestic companies, such as Johnson & Johnson and Nanjing Legend Biotech, play a significant role in the rapid development and commercialization of these therapies.
// Politics
Supreme Court Denies Vanda’s Petition in Hetlioz Patent Case Against Teva
April 23, 2024 / Supreme Court / Vanda Pharmaceuticals / Hetlioz / Teva
The U.S. Supreme Court has declined to review Vanda Pharmaceuticals’ patent litigation against Teva Pharmaceuticals concerning the sleep-wake disorder drug Hetlioz. This decision follows lower court rulings that invalidated several of Vanda’s patents for the drug.
Trump surrogates hint at how he could reshape U.S. health care policy
April 23, 2024 / Trump / Healthcare / Affordable Care Act / Drug Pricing / Medicare
Former President Donald Trump’s health care policies for his potential second term remain vague, with uncertainty surrounding his stance on federal abortion bans, repealing the Affordable Care Act (ACA), and strategies to reduce drug costs. His messaging on these topics has been inconsistent, yet there are indications that he may shape drug pricing policies and ACA-related strategies in a different direction from President Biden’s approach. Although Trump’s health policy agenda is still in development, he is expected to focus on reshaping Medicare’s drug pricing policies and may offer alternative options to the ACA rather than completely repealing it.
Supreme Court will not hear D.C. biotech’s patent infringement appeal
April 23, 2024 / Supreme Court / Revenue Loss
The U.S. Supreme Court has declined to hear the appeal of a D.C.-based biotechnology company’s patent infringement case, impacting its ability to protect a key revenue source. This outcome could lead to revenue loss related to one of the three drugs the company has on the market.
Health care lobbying giants spent big as little got done in Congress
April 23, 2024 / Healthcare / Congress / PBM Regulation / Pharmaceutical Care Management Association
Health care lobbying groups, particularly the Pharmaceutical Care Management Association, significantly increased their spending in Washington to prevent new regulations on pharmacy benefit managers (PBMs). Despite notable reform efforts in Congress, the PBM industry lobbied heavily to maintain the status quo, with lobbying spending increasing by 71% compared to the same period in 2023.
America’s moves against Chinese biotech will hurt patients at home
April 18, 2024 / China / Biosecure Act / Trade
The U.S. is extending its crackdown on Chinese trade to the biotechnology sector. The proposed Biosecure Act, which has bipartisan support in Congress, aims to end government contracts with American firms that work with Chinese biotech companies. This shift, part of broader efforts to restrict China’s access to sensitive technology, could affect American consumers by disrupting the supply of genomic sequencing equipment and large-molecule drugs, like weight-loss injectables.