// Biotech and Pharma Therapeutics
Pfizer’s Paxlovid access brought into question as WTO set to discuss patent waiver—again
Pfizer’s oral COVID drug Paxlovid has become a standard of care for treating non-severe patients in high-income countries. But advocacy groups remain critical of the drug’s lack of equal access. Low- and middle-income countries are receiving only 26% of Paxlovid courses despite them accounting for 84% of the world’s population, a report by Oxfam and the People’s Vaccine Alliance has found. The organizations reached the finding based on data from health analytics provider Airfinity.
Pfizer’s $1B protein degradation bet yields 3% response rate in Arvinas’ midphase breast cancer trial
Pfizer’s $1 billion bet on Arvinas’ protein degradation technology has delivered phase 2 data. After a publication snafu, Arvinas posted the breast cancer results ahead of schedule—and revealed one partial response among 35 patients at its phase 3 dose. Arvinas planned to present the data Dec. 8. However, the San Antonio Breast Cancer Symposium “incorrectly published the abstract, omitting a key safety data table, and inadvertently released the corresponding full data presentation” on its website on Monday, according to the biotech. In response to the incident, Arvinas released its full-data presentation on Tuesday, two weeks ahead of schedule.
J&J’s nasal spray Spravato bests former AZ drug Seroquel XR in treatment-resistant depression trial
Johnson & Johnson’s esketamine nasal spray continues to build a body of evidence showing it can go toe-to-toe with tough-to-treat depression. Wednesday, J&J’s Janssen unit rolled out data from the phase 3b ESCAPE-TRD trial pitting its esketamine spray Spravato against extended-release quetiapine, formerly marketed by AstraZeneca as Seroquel XR before the British drugmaker pawned off the rights to Cheplapharm in late 2019.
Mirati is once again the subject of Big Pharma takeover talks: report
Bloomberg reported Tuesday that Mirati Therapeutics is once again the subject of takeover talks, reviving similar rumors from a year ago. In fact, Mirati has been considering strategic options “for some time,” according to the latest reports. No formal bids have been proposed, according to Bloomberg, which claimed that the cancer-focused company is working with an advisor and larger pharmas are considering the “merits of a transaction.” Notably, the report said that a deal doesn’t appear to be imminent.
Inovio drops Lassa, MERS vaccines as clinical data fall short, gutting infectious disease pipeline
Inovio’s Lassa fever and Middle East respiratory syndrome (MERS) vaccine candidates have fallen short of the bar set by the biotech’s partner, prompting the collaborators to pull the plug on the programs after getting a look at clinical data. The Coalition for Epidemic Preparedness Innovations (CEPI) partnered with Inovio in 2018, committing up to $56 million to support development of DNA vaccine candidates against Lassa and MERS through to phase 2. Inovio began a phase 2 clinical trial of the MERS candidate, INO-4700, and a phase 1b study of the Lassa prospect, INO-4500, last year. CEPI footed the bill for the studies.
// 4th Industrial Revolution
AI-enabled patient monitoring platform care.ai scores $27M in funding
The Florida-based company’s Smart Care Facility Platform includes a network of sensors spread through a care facility that monitors patients using AI, which allows a facility to collect real-time behavior data for clinical and operational insights. The funds will help the company help grow the company and deliver “ambient intelligence to healthcare.”
One Wearable Camera Captures Full 3D Body Movements
What can 3D imaging tell us about the health of the human body? Lots. The motion capture of one’s gait has the potential to serve as a digital biomarker that identifies disease progression and rehabilitation outcomes, while 3D tracking can identify problematic postures — such as spending a day hunched over a computer — that aggravate things including chronic neck pain. The ability to monitor and measure human movements can aid with detecting and managing a range of musculoskeletal disorders and much more.
Smart Wristband Can Get Stroke Victims Faster Treatment
A stroke can cause irreversible brain damage, long-term disability, and even death. Neuralert Technologies, a spin-out from the University of Pennsylvania, has recently announced that they have reached Minimum Viable Product (MVP) level for their compact, lightweight, non-invasive wristband gadget that facilitates stroke monitoring and alerting. A delay in detecting a stroke reduces the chances of getting immediate treatment, which in turn leads to poorer outcomes. Since many hospital patients are bedridden and/or under the influence of medication, spotting the stroke symptoms can be challenging for the staff.
Turbine boosts AI platform to deliver cancer treatments
Turbine, a company which has developed a cell simulation platform, has completed a €20m financing round. Mercia and MSD – also known as Merck & Co in the US and Canada – Global Health Innovation (GHI) Fund co-led the financing, joined by Day One Capital and existing investors Accel, Delin Ventures and XTX Ventures. Its platform –- The Simulated Cell – is powered by artificial intelligence (AI) and can predict the effectiveness of cancer treatments, will now expand its team and develop new partnerships with biopharmaceutical companies.
New Gene Therapy Biotech Telaria Launches With Rare Skin Disease Focus
Telaria is the latest company unveiled by Replay, which itself is a new gene therapy biotech. Launched earlier this year, Replay operates a “hub-and-spoke” business model in which it develops its own platform technologies and identifies therapeutic areas that can be addressed by one or more of these technologies. Replay, which maintains operations in San Diego and London, then forms companies focused on particular therapeutic areas.
// Business & Markets
Bristol Myers CEO already reassessing portfolio in wake of US pricing law: report
Big Pharma CEOs haven’t been shy in expressing their concerns with Biden’s Inflation Reduction Act (IRA), with the latest broadside coming from Giovanni Caforio. The Bristol Myers Squibb chief took to the pages of the U.K.’s Financial Times over the weekend to warn that the company expects to halt funding to certain drugs as a result of the government’s drug pricing legislation. “I do expect that we will cancel some programs, whether that is, you know, a full-on indication for an existing medicine or a new medicine. We are undergoing a review of our portfolio now,” Caforio is reported to have told the newspaper in an interview published Sunday.
GSK to slash about 150 consumer commercial jobs in India
Amid a wave of recent biopharma layoffs, GSK is adding to the trend by letting go of approximately 150 employees in India. The British drug giant has decided to eliminate its consumer trade channel in the country, The Times of India reports. The employees were responsible for taking orders from chemists and ensuring that the company’s consumer brands were available in stores around the country, according to the publication. These days, that can be achieved digitally.
Novo Nordisk invests $749M to boost API manufacturing for clinical trials
With all the talk of drugmakers trimming back pipelines and laying off staff, it’s good to occasionally report on some more expansionist plans across the sector. For Novo Nordisk, that means investing 5.4 billion Danish kroner ($749 million) to boost R&D manufacturing needed to support its clinical trials. The money has been earmarked to increase the company’s capacity to produce its own active pharmaceutical ingredients (APIs) for its trials around the globe. This means expanding its existing manufacturing site in Bagsværd, Denmark. The project is likely to continue into 2024, with around 160 new roles recruited.
Health tech firm CardieX acquires cardiovascular sensor company Blumio
ASX-listed health tech firm CardieX announced it acquired Silicon Valley-based Blumio, a developer of cardiovascular sensor algorithms and data analytics tools. CardieX focuses on hypertension, cardiovascular health and other arterial health disorders, while Blumio develops wearable-sensor technologies focused on capturing cardiovascular data, including blood pressure measurements. The acquisition follows a three-year collaboration between the companies, which determined Blumio’s sensor technology was compatible with CardieX subsidiary ATCOR’s SphygmoCor arterial technology that measures the central aortic pressure waveform.
Merck & Co. inks $1.4B blood cancer biotech buyout to muscle in on Bristol Myers, Novartis
Merck & Co. has opened its wallet wide to buy a midphase treatment for life-threatening diseases of the bone marrow, paying $1.35 billion to snap up Imago BioSciences for its oral lysine-specific demethylase 1 (LSD1) inhibitor bomedemstat. Imago discovered the small molecule and took it into phase 2 on the strength of evidence that inhibiting LSD1 reduces the hallmark symptoms of myeloproliferative neoplasms (MPNs), a family of bone marrow cancers that can transform into acute myeloid leukemia. If Imago is right, bomedemstat will improve outcomes in the MPNs essential thrombocythemia, myelofibrosis and polycythemia vera.
// Legal & Regulatory
Apellis’ closely watched eye drug gets new FDA goal date after changes to its filing scared investors
After some investor clamor, Apellis Pharmaceuticals’ blockbuster hopeful eye drug pegcetacoplan is back on the FDA’s review track. The FDA has accepted a voluntary amendment to Apellis’ application for pegcetacoplan as an eye injection for geographic atrophy (GA), an advanced stage of age-related macular degeneration, the company said Friday. The agency has set a new target decision deadline of Feb. 26, 2023.
FDA extends deadline to decide on Gamida Cell’s stem cell transplant therapy by three months
With commercial preparations already underway, Gamida Cell was keenly eyeing the late January deadline for U.S. regulators to decide on the approval application of its stem cell transplant therapy. With commercial preparations already underway, Gamida Cell was keenly eyeing the late January deadline for U.S. regulators to decide on the approval application of its stem cell transplant therapy.
Jazz plays coda to blood cancer approval, securing FDA nod for revised dosing schedule
Jazz Pharmaceuticals has won FDA approval for a revised dosing schedule for its blood cancer therapy Rylaze, giving physicians the option to prescribe intramuscular doses of the drug on Monday, Wednesday and Friday. Last year, the FDA approved Rylaze, a form of asparaginase used in multi-agent chemotherapeutic regimens, for administration to adults and children with acute lymphoblastic leukemia or lymphoblastic lymphoma every 48 hours.
Lilly wades deeper into copycat insulin market, scoring interchangeability tag for Lantus biosim
On the heels of Viatris and Biocon Biologics’ landmark diabetes copycat Semglee, another interchangeable biosimilar is crashing the infamously pricey insulin market. Wednesday, the FDA blessed Eli Lilly’s approved insulin simulacrum Rezvoglar with an interchangeability tag, meaning the product can be swapped out for its brand-name counterpart right at the pharmacy counter. Biosimilar interchangeability has been touted for its potential to improve drug access to high-cost biologics by offering alternatives at competitive prices.
Teva, AbbVie’s Allergan lock in $6.6B deal to settle thousands of US opioid lawsuits
The companies this week locked in a combined $6.6 billion in settlements to resolve allegations that they helped fuel the U.S. opioid epidemic by overstating the painkillers’ benefits, downplaying the risk of addiction and failing to maintain controls to prevent opioid misuse. With Teva and Allergan’s agreements finalized, state and local governments will now get a chance to opt in to the deals, Teva explained Tuesday. Under an agreement floated in July, Teva is on the hook to pay $4.25 billion—a sum that will be paid out over 13 years and includes up to $1.2 billion worth of Teva’s generic version of the overdose reversal drug Narcan.
// Research & Development
Lysogene’s lead gene therapy flunks phase 2/3 trial almost a year after Sarepta walked out
With cash running out and a number of strategic options under consideration to stay afloat, Lysogene was banking on a clinical home run from its lead asset to woo investors. But new data presented by the company are anything but a splash. The company’s top pipeline candidate, LYS-SAF302, failed its phase 2/3 trial among patients with mucopolysaccharidosis type IIIA (also known as Sanfilippo syndrome), a neurodegenerative disease that can spur a wide range of bodily dysfunction, including infections, dementia, sleep disturbances and propensity to chew on objects.
After FDA setback, MEI, Kyowa confirm phase 2 efficacy with Japanese blood cancer data drop
Kyowa Kirin and MEI Pharma have posted more midphase data on their oncology candidate zandelisib, confirming the efficacy seen in an earlier trial of the PI3K delta inhibitor. But with the FDA raising the bar for the asset, the companies need an ongoing phase 3 to deliver to get the prospect to the U.S. market. The latest data come from the phase 2 MIRAGE clinical trial, which enrolled 61 patients with relapsed or refractory indolent B-cell non-Hodgkin lymphoma in Japan to receive two cycles of zandelisib.
CSL’s Gene Therapy Approval Marks First for Hemophilia B and New High in Pricing
The FDA has approved the first hemophilia B gene therapy, a one-time treatment that addresses a root cause of this inherited disorder and potentially eliminates the need for regular infusions that patients currently receive to prevent bleeding episodes. Removing that economic burden from patients and the healthcare system the comes with a premium upfront price: $3.5 million for a single infusion. The regulatory approval announced late Tuesday for the CSL Behring therapy covers adults who receive prophylactic infusions or have a history of life-threatening bleeding, as well as those who have repeated and serious spontaneous bleeding episodes.
Infex begins study of pseudomonas aeruginosa candidate RESP-X
The treatment is a new anti-virulence therapy to treat pseudomonas aeruginosa (Pa) infections in non-cystic fibrosis bronchiectasis (NCFB) patients. Pa is a critical factor in this respiratory disease, leading to recurring episodes of life-threatening infection – currently there are no approved preventative treatments. Research involves a 32-subject, first-in-human, double-blind, placebo-controlled, ascending dose study, delivered intravenously to groups of healthy volunteers.
Study brings personalised immunotherapy prescriptions closer
With a focus on advancing personalised cancer treatment, scientists have demonstrated that biomarkers are not enough to tell which patients are likely to respond best to immunotherapy. According to a study from University of Bath’s Centre for Therapeutic Innovation (CTI-Bath), clinicians also need to understand how immune cells and tumours are interacting within a patient, rather than relying on the levels of associated proteins present when prescribing the optimum treatment.
Conservative group sues FDA in bid to overturn decades-old approval for abortion pill
For nearly five decades, women in the U.S. had the constitutional right to abortion, until it was revoked earlier this year by the Supreme Court. Now, emboldened by the ruling, anti-abortion medical organizations are taking aim at pills that induce miscarriage and have been on the market in the U.S. for more than two decades. Friday, the conservative Alliance Defending Freedom filed (PDF) suit in Amarillo, Texas, against the FDA, challenging the agency’s 2000 approval of Mifeprex.
PDS Biotech Executives to Present an Overview of Oncology and Infectious Disease Programs at the World Vaccine & Immunotherapy Congress
PDS Biotechnology Corporation (Nasdaq: PDSB), a clinical-stage immunotherapy company developing a growing pipeline of targeted immunotherapies for cancer and infectious disease, today announced two executives will be giving presentations at the World Vaccine & Immunotherapy Congress, which will take place from November 28 through December 1, 2022, in San Diego. More than 800 attendees are expected to attend the World Vaccine & Immunotherapy Congress to examine challenges around scientific, commercial, public health and policy issues in manufacturing, clinical trials, regulation, immune profiling, biomarkers, platform technologies, and additional topics.
Programmable Biology Puts Biotech on the Geopolitical Agenda
In September, Jake Sullivan—President Biden’s national security adviser—announced that the U.S. government expects biotechnology to play an “outsized importance over the coming decade” in the context of geopolitical competition, because of the ability to “read, write, and edit genetic code, which has rendered biology programmable.” Sullivan’s remarks came just days after senior security, economic, and science and technology officials gathered at the White House for a summit on biotechnology and biomanufacturing.
Senator aims to cut biosimilar ‘red tape’ with proposal to eliminate interchangeable status
In August, when the FDA approved Coherus’ Cimerli, a biosimilar version of Roche’s eye disease medicine Lucentis, the agency also took the unusual step of granting the copycat “interchangeable” status without requiring a switching study. While that move was unexpected at the time, it could become standard practice for biosimilars if one senator gets his way. Last week, Sen. Mike Lee (R-Utah) introduced a proposal in Congress that would put an end switching studies and, in effect, make all approved biosimilars interchangeable. The goal of the Biosimilar Elimination Red Tape Act is to speed access to biosimilars, the senator said in a statement.