Kalvista’s [in]Komplete study gives boost to Orladeyo blockbuster ambitions
Biocryst Pharmaceuticals Inc. caught some industry observers by surprise early in 2022 when it upped its peak sales estimate for Orladeyo (berotralstat), its oral therapy for prevention of hereditary angioedema attacks, from $500 million to $1 billion. But blockbuster status appears more attainable following the latest setback from a potential competitor, as safety concerns prompted Kalvista Pharmaceuticals Inc. management to ax its phase II study testing KVD-824 in prevention of HAE attacks.

Kalvista’s [in]Komplete study gives boost to Orladeyo blockbuster ambitions
Biocryst Pharmaceuticals Inc. caught some industry observers by surprise early in 2022 when it upped its peak sales estimate for Orladeyo (berotralstat), its oral therapy for prevention of hereditary angioedema attacks, from $500 million to $1 billion. But blockbuster status appears more attainable following the latest setback from a potential competitor, as safety concerns prompted Kalvista Pharmaceuticals Inc. management to ax its phase II study testing KVD-824 in prevention of HAE attacks.
Early but tantalizing X4 data in neutropenia sweetens CXCR4’s appeal
X4 Pharmaceuticals Inc.’s recent stock-boosting phase Ib news with lead candidate mavorixafor fueled already-strong interest in finding an alternative therapy for neutropenia and deepened esteem for the drug’s long-known mechanism of action.
Promoting attachments nets 2022’s Nobel Prize in Chemistry
The 2022 Nobel Prize in chemistry was awarded to Carolyn Bertozzi of Stanford University, to Morten Meldal of the University of Copenhagen, and – for the second time – to Barry Sharpless of The Scripps Research Institute “for the development of click chemistry and bioorthogonal chemistry.”

Click chemistry, the Nobel Committee’s Olof Ramström told reporters while announcing the prize, “is almost like it sounds – it’s all about linking different molecules.”

He likened click chemistry to a seatbelt buckle, whose interlocking parts can be attached to many different materials, linking them by snapping the two parts of the buckle together.

“The problem was to find good chemical buckles,” Ramström said – chemicals that “will easily snap together, and importantly, they won’t snap with anything else.”
Today's news in brief
BioWorld briefs for Oct. 5, 2022.
Two decades after seminal paper, Exscientia highlights untapped potential of AI drug discovery
It’s been 20 years since Andrew Hopkins, founder and CEO of artificial intelligence drug research firm Exscientia plc, co-authored the seminal paper “The Druggable Genome,” which laid the foundations for the company and gave insights about how to make research more efficient and less costly. Now Hopkins’ colleagues at Exscientia have taken stock of progress in a new paper, “The druggable genome: Twenty years later,” that summarizes advances in the field and evolution in thinking over the past two decades.
White House blueprint for AI bill of rights hints at need for new enforcement legislation
The Biden administration has released a blueprint for an artificial intelligence bill of rights, which is accompanied by an acknowledgement that these algorithms can be crucial in guiding treatment of cancer patients. While the bill has drawn criticism for lacking enforcement teeth, there is legislation already in the works, such as the Algorithmic Accountability Act (AA Act) of 2022, which would give the FTC oversight and enforcement authority over these algorithms.
Biosimilars: Portrait of a maturing landscape
Although it’s a make-or-break market for many novel drugs, the U.S. is still testing the waters with biosimilars to some extent. That’s expected to change when at least seven biosimilars, including an interchangeable, referencing Abbvie Inc.’s Humira (adalimumab) are set to launch in the U.S. within the first seven months of next year. Next week, BioWorld will look at the significance of that looming competition and how the global biosimilars market is evolving amid a changing landscape of players, policies and pipelines.

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Analysis and data insight

Deaths low, fewer new candidates, but pandemic not over
Vaccines
Although U.S. President Joe Biden suggested in September that the pandemic is over, health officials insist there is still much to do in preparation for an endemic stage of the SARS-CoV-2 virus. The number of weekly COVID-19 confirmed cases both globally and in the U.S. is similar with each of the...
Biopharma stocks up 1.5% as markets continue to struggle
Despite a sharp increase in mid-August, BioWorld’s Biopharmaceutical Index ended that month up a mere 1.4% and rose only slightly in September to 1.5% as of the 26th. It has done significantly better than the Nasdaq Biotechnology Index and the Dow Jones Industrial Average, which are down 23.2% and...
Clinical data slow during summer months
Clinical
The amount of clinical news in 2022 continues to lag last year, partly due to a decline in COVID-19-related data. Through the last week of September, there have been a total of 2,495 phase I, II and III clinical entries, compared with 3,027 during the first three quarters of 2021. This represents a...
Despite slow regulatory activity, NME approvals pick up in September
Regulatory
While U.S. FDA approvals are down by 27% in 2022, the agency was busy throughout the month of September, clearing seven new molecular entities (NMEs), the most for any month this year.

Financings

Cellarity adds $121M in series C to look beyond targets in cell-centric approach
Series C
At first glance, Cellarity Inc. might appear as one more company harnessing the computational power of AI and machine learning to boost drug discovery efforts. A closer look, however, reveals a different approach, one that looks at cells and cellular behavior to address disease rather than the...

Science

Current TMB estimation methods fall short in minority populations
Biomarkers
Tumor mutational burden (TMB), a biomarker used to assess whether a patient will respond to immunotherapy, needs to be recalculated in order to be useful for patients of Asian or African descent. Scientists at the Dana-Farber Cancer Institute found a significant bias in the estimated TMB values...

From ancient DNA, a Nobel Prize, and perhaps modern drug targets
Genetic/Congenital
The Nobel Prize in Physiology or Medicine 2022 was awarded to Svante Pääbo today "for his discoveries concerning the genomes of extinct hominins and human evolution." Pääbo, who is currently the director of the Max Planck Institute for Evolutionary Anthropology in Leipzig, Germany, and his...
Laskers go for integrins, prenatal testing, COVID-19 dashboard
Coronavirus
The 2022 Albert Lasker Basic Medical Research Award has been awarded to Richard Hynes, of the Massachusetts Institute of Technology, Erkki Ruoslahti, of the Sanford Burnham Prebys Medical Discovery Institute, and Timothy Springer, of Harvard Medical School “for discoveries concerning the integrins,...

Newco news

UK’s Stratosvir aims to unlock the potential of oncolytic viruses
Cancer
Stratosvir Ltd. aims to overcome the drawbacks experienced with oncolytic viruses, using technology based around vaccinia viruses that allow for a systemic injection and with a larger payload capacity. Stratosvir was started up by CEO Chris Ullman and co-founder Antonio Postigo in 2020 with funding...
Avelos raises $8M series A funding to develop biomarker-driven oncology pipeline
Cancer
Startup Avelos Therapeutics Inc. raised $8 million in series A funding that will launch the company’s biomarker-driven cancer therapy pipeline using its synthetic lethality platform. Participating in the series A funding were SV Investment, UTC Investment, Quad Investment Management, Timepolio...
E-nitiate Biopharma raises $14.4M to develop drugs for dermatosis
Dermatologic
E-nitiate Biopharmaceuticals Co. Ltd. has raised ¥100 million (US$14.4 million) in a series A round to speed up the clinical trials of its lead assets, QY-201 and QY-101, and expand the pipeline. “Our strategy is to focus on the ‘blue ocean’ of the dermatosis market,” said Shi Jun, chief medical...
Neobe plans bacterial assault on tumor microenvironment
Cancer
Over the past decade cancer immunotherapy has redefined standard care in many kinds of tumor, but low response rates remain a problem and there have been some shock trial failures where checkpoint inhibitors have failed to work as expected. To help, Neobe Therapeutics Ltd. is attempting to tackle...
Nodus looks to deleted ‘passenger genes’ in anticancer drive
Cancer
Nodus Oncology Ltd. is set to explore new avenues of DNA damage response by targeting the chromosome next-door neighbors of tumor suppressor genes that are damaged when tumor suppressor genes are inactivated via homozygous deletion. These collaterally deleted ‘passenger genes’ play diverse...

U.S.

US Medicare beneficiaries to get small relief in Part B premiums
Regulatory
The U.S. Centers for Medicare & Medicaid Services is finally making a long-expected, and requested, adjustment to Medicare Part B premiums, which were raised nearly 15% for 2022 in the wake of Biogen Inc.’s initial $56,000 annual price tag for its Alzheimer’s drug, Aduhelm (aducanumab).

Europe

UK pharma facing ‘perfect storm’ of economic turmoil and looming 30% rebate hike
Biosimilar
The U.K. could be downgraded as a place to research and launch new medicines because of economic shocks and a looming rebate of 30% or more on sales of branded products, according to industry sources.

Antibiotics development incentive scheme makes economic sense, EFPIA says
Regulatory
To jumpstart the development of much-needed antibiotics, the European Federation of Pharmaceutical Industries and Associations (EFPIA) released a new report Sept. 28 demonstrating the economic benefit of granting additional exclusivity for another drug as a way of incentivizing antibiotic R&D.
EMA calls for biosimilar interchangeability across the EU
Regulatory
In an effort to get drug regulators in the various EU member states on the same page, the EMA issued a Sept. 19 statement confirming that all biosimilars approved in the EU are interchangeable with other approved biosimilars referencing the same biologic, as well as the reference biologic itself.

Clinical

Top-line results suggest Paradigm’s injectable Zilosul could be disease-modifying in osteoarthritis
Inflammatory
Paradigm Biopharmaceuticals Ltd. reported top-line data from its phase II trial of Zilosul (pentosan polysulfate/PPS) that suggest the non-opioid treatment for knee osteoarthritis is effective in navigating pain management but also has the...
Space robust as Calithera, Kronos, others anchor in Syk bay
Cancer
Calithera Biosciences Inc.’s in-licensing deal to take ownership of a pair of oncology assets from Takeda Pharmaceutical Co. Ltd. may be on its way to paying off, perhaps especially with regard to the oral Syk/FLT3 inhibitor mivavotinib, formerly...
Trefoil’s TTHX-1114 gets the all-clear in corneal dystrophy trial
Ocular
Trefoil Therapeutics Inc. has reported promising data from a phase II trial suggesting its engineered version of fibroblast growth factor 1, TTHX-1114, may speed and improve recovery of patients with Fuchs endothelial corneal dystrophy who are...
Clarity in early Alzheimer’s a breakthrough as lecanemab meets primary, secondary endpoints
Neurology/Psychiatric
Analysts were upbeat about positive results from Eisai Co Ltd. and Biogen Inc.’s phase III confirmatory Clarity Alzheimer’s disease (AD) trial of lecanemab that met both primary and secondary endpoints, and most agreed that the data should be...
Avidity searches for cause of SAE as FDA puts partial hold on dystrophy study
Regulatory
A serious adverse event (SAE) in one participant has led the U.S. FDA to place a partial clinical hold on Avidity Biosciences Inc.’s lead program. The action is centered on the phase I/II Marina study of AOC-1001, an antibody oligonucleotide...
Talks with partner over next-gen COVID-19 shot may take months and could fail, Valneva warns
Regulatory
Valneva SE is approaching a crucial point with its troubled efforts to develop a COVID-19 vaccine, having announced Sept. 26 it’s in talks with a potential partner for its next-generation jab. The company cautioned the negotiations could take...

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