Geoff MacKay, Avrobio CEO
An oft-troubled Fabry disease program got the axe Tuesday as its parent company will shut down development after reviewing some new Phase II data. And the news is sending shares tumbling again.
Avrobio will ‘deprioritize’ the Fabry disease candidate AVR-RD-01, the company said in a press release Tuesday morning, citing variable engraftment patterns in newly dosed patients from the Phase II trial, which has been halted. The biotech also said it’s facing tougher than expected competition in the Fabry disease market and could face a tougher regulatory environment.
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Their Staying Power Lies in their Patient-Centricity
Decentralized clinical trials (DCTs) were traditionally utilized in an isolated fashion prior to the COVID-19 pandemic. To continue their research within the constraints of the pandemic, sponsors and clinical investigators pivoted to a decentralized model out of necessity. At the onset, regulatory agencies offered some guidance on the digital approaches that are acceptable to ensure DCT approaches are applied in a way that maintains patient safety, as well as data quality and integrity.
For anyone who’s been following how the US government has been allocating and shipping supplies of its Covid-19 treatments over the past year, the news has shifted so many times that it can be difficult to keep track of what’s still being shipped and where.
More change is coming this week too, as HHS has now decided to re-start shipments of both Eli Lilly (bamlanivimab plus etesevimab) and Regeneron (casirivimab plus imdevimab) monoclonal antibody products after a short pause because neither product works against the new variant Omicron. Lilly’s combo also was halted last June due to the presence of other variants.
Kicking off 2022, hundreds of pharmaceuticals, including some blockbusters, saw their list prices rise by about 5% on average. But overall, net drug prices (cost after rebates) declined for the fourth year in a row, potentially complicating already stalled drug price reform efforts.
Among the drugs seeing new increases as of Jan. 1 are Gilead’s bevy of blockbuster HIV drugs.
Biktarvy, which pulled in more than $7 billion in worldwide sales in 2020, saw a 4.8% price increase in 2021, and now, another 5.6% increase in 2022, according to a new report from the nonprofit 46brooklyn Research.
All the big R&D trends are on display in this new, record-topping list of drug approvals for 2021. Plus one.
Add up everything OK’d from CDER and CBER, and you have 61 new drug approvals for last year, topping the 59 OKs that had tied a record in 2020. The dark days of the early 2000s are a distant memory now, with a host of hungry upstarts promising to make their own entries one day as Big Pharmas double down on innovation.
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Piramal CEO Peter DeYoung (Credit: Piramal Pharma Solutions via Youtube)
During a typically quiet holiday break in biotech, Piramal Pharma made some noise, and it’s starting off 2022 with yet another big announcement.
The CDMO will invest millions into creating a high-throughput screening facility that will enhance the already existing in vitro biology capabilities at its Ahmedabad, India drug discovery site. The new expansion is scheduled to go live in Q3 of this year, and couple its biology services with chemistry capabilities already in operation.
When the FDA lifted a clinical hold on Applied Therapeutics’ lead program in galactosemia last February, the New York biotech signaled that they were then on a smooth road toward an accelerated approval, with plans to file an NDA in the third quarter of 2021.
Regulators, though, apparently changed their mind.
Applied has decided to hold on submitting an NDA for AT-007 as a treatment for galactosemia, the company disclosed, following discussions with the FDA in which the agency indicated that ‘clinical outcomes data will likely be required for approval.’
Still recovering from a setback in its lead program, Ovid Therapeutics has turned to AstraZeneca for a suite of experimental epilepsy drugs.
Ovid is paying the Big Pharma partner $5 million in cash and $7.5 million in stock to access a library of early-stage small molecules targeting the KCC2 transporter, including lead candidate OV350. AstraZeneca stands to receive milestones adding up to more than $200 million and retains an option for a strategic collaboration.
Johan Kördel, Sound Bioventures founding partner
Novo Holdings starts the new year off with a bang — right into a biotech VC’s first fund.
The firm announced its undisclosed investment — a ‘cornerstone’ investment, according to Novo — this morning into Sound Bioventures Fund I, a fund appropriately managed by Sound Bioventures. The venture capital firm aims to invest one-third of its fund in Scandinavian biotechs, another third in US and UK biotechs, and the final third in other European biotechs outside Scandinavia and the UK.
Michel Vounatsos (Credit: World Economic Forum/Ciaran McCrickard)
As Biogen stares down an impending patent cliff for its spinal muscular atrophy blockbuster Spinraza, it’s betting $60 million that a new antisense oligonucleotide from Ionis will help keep the company’s lead in that space.
Building on a yearslong relationship with Ionis, Biogen plumped down $60 million just before the new year to secure commercial and development rights to BIIB115, a preclinical SMA drug looking to one-up Spinraza, the companies announced Tuesday. The latter raked in just over $2 billion last year (a slight decrease from the year before) but faces a patent expiration as early as 2023.
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