Orna goes into orbit on $3.65B Merck megadeal, $221M series B round
Orna Therapeutics Inc., which is pioneering a novel circular RNA protein expression technology in several therapeutic areas, has achieved lift-off. The Cambridge, Mass.-based company has closed a broadly based alliance in infectious disease and oncology with Merck & Co. Inc., under which it is getting $150 million up front and up to $3.5 billion in development, regulatory and sales-based milestones. In addition, Merck, of Rahway, N.J., is investing another $100 million in Orna’s equity, as part of its $221 million series B round, which the company also disclosed on Aug. 16.

Orna goes into orbit on $3.65B Merck megadeal, $221M series B round
Orna Therapeutics Inc., which is pioneering a novel circular RNA protein expression technology in several therapeutic areas, has achieved lift-off. The Cambridge, Mass.-based company has closed a broadly based alliance in infectious disease and oncology with Merck & Co. Inc., under which it is getting $150 million up front and up to $3.5 billion in development, regulatory and sales-based milestones. In addition, Merck, of Rahway, N.J., is investing another $100 million in Orna’s equity, as part of its $221 million series B round, which the company also disclosed on Aug. 16.
Canwell raises ¥100M in series A+ financing for antitumor immunotherapies
Canwell Biotech Ltd. raised more than ¥100 million (US$14.8 million) in a series A+ financing. The funds will help accelerate trials for its pipeline of anticancer assets, such as the TLR7 agonist CAN-1012, and preclinical development of other projects too, CEO Henry Yu told BioWorld. The State Development and Investment Corporation Venture Capital Co. Ltd. was the round’s sole investor.
US Rx negotiations the law of the land
“It’s now law,” U.S. President Joe Biden said after he signed H.R. 5376 Aug. 16. His signature made Medicare drug pricing negotiations a near-term reality, along with new inflationary rebates, new caps on annual out-of-pocket drug spending and monthly insulin copays for Medicare beneficiaries, and tax changes that could affect the bottom line for several multinational drug and device companies beginning next year.
Today's news in brief
BioWorld briefs for Aug. 16, 2022.
Attempt to hijack shelved weight loss drug ends in guilty plea
Alain Bouaziz, a French citizen and resident of the United Arab Emirates, pleaded guilty Aug. 12 to one count of lying to the U.S. FDA in an attempt to steal a discontinued Novartis AG weight loss drug. The charge carries a maximum prison term of five years and a fine of up to $250,000, or twice the gain or loss caused by the offense, whichever is greatest.
Paradigm raises AU$66M to fund pivotal trials for Zilosul for pain associated with knee osteoarthritis
Paradigm Biopharmaceuticals Ltd. announced a AU$66 million (US$46.39 million) capital raise to fund its phase III pivotal trial for injectable Zilosul (pentosan polysulfate) to treat osteoarthritis of the knee.
US government seeks feedback on measures for competitiveness in artificial intelligence
The field of artificial intelligence (AI) is beginning to mature, but this is also becoming a competitive space, with several governmental entities declaring their intent to develop policies to maintain national competitiveness. The U.S. has now entered the fray with a request for information by the International Trade Administration seeking information on policies that might foster the export of AI products developed by U.S. companies, but also on the policies that might enable small and medium enterprises to remain competitive.

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Analysis and data insight

U.S. government dominates biopharma nonprofit deals and grants
Deals and M&A
Through the second week of August, BioWorld has tracked 439 bio/nonprofit deals worth $6.6 billion, with the bulk of that total coming within the last six weeks through the U.S. government purchasing more vaccines and therapies to fight COVID-19.
Biopharma stocks climb on upcoming filings, strong data and potential mergers
BioWorld’s Biopharmaceutical Index is at its highest point this year, up 7.85%, showing one of its sharpest rises within the last week. Out of 22 component stocks, 13 are trading above where they were at the end of 2021, as of Aug. 8. This is an improvement from June, when only nine were in such a...
Deal values top all recent years; M&As still in a slump
Deals and M&A
While biopharma M&As fail to keep up with prior years, the dealmaking environment remains strong, as deal values continue to grow. Through early August of this year, biopharma companies have completed 955 deals, including licensings, joint ventures and collaborations, valued at $114.5 billion,...
Biopharma financings return to pre-pandemic levels
Financings
The amount of money raised by biopharma companies so far this year is down by 59% in comparison with last year, and there are 42% fewer transactions.

Financings

Opthea to advance wet AMD drug with up to $170M in Carlyle backing
Ocular
Opthea Ltd. has secured nondilutive funding to complete phase III development and commence commercialization of its wet age-related macular degeneration (wet AMD) treatment OPT-302, in a $170 million agreement with Launch Therapeutics, an operating company set up earlier this year by the private...

Science

New method developed to produce generic CAR T cells at scale
Cancer
A new method has been devised to produce generic chimeric antigen receptor (CAR) T cells at scale by directing induced pluripotent stem cells to differentiate into mature T cells in vitro. The generic T cells can then be engineered to express a range of different chimeric antigen receptors.

Researchers reprogram stem cells to uncover new genetic signatures of age-related AMD
Ocular
Researchers are closer to better diagnosing and treating age-related macular degeneration (AMD) after discovering new genetic signatures of the disease by reprogramming stem cells to generate high-resolution disease models.
‘3D view of the protein universe’ as Deepmind reveals 200M protein structures
Artificial intelligence
It is now possible to look up the 3D structure of every known protein following the latest release of Alphafold, an open database run in partnership by Deepmind, the London-based artificial intelligence company owned by Google parent Alphabet and the European Molecular Biology Laboratory’s European...

Newco news

Setting cancer cells on the road to ruin is Modifi’s goal
Cancer
Exploiting deficiencies in tumor cells’ inability to repair damage to their DNA has been one of the most successful endeavors of oncology research in recent memory. There are four approved PARP inhibitors to treat certain types of breast, ovarian, pancreatic and prostate cancers. Multiple other...
Replay spins up genomic medicine companies with $55M seed round
Cell therapy
Increased payload capacity for gene therapies, off-the-shelf genome-engineered allogeneic cell therapies, reduced cost of goods and faster bioprocesses, are promised by “big DNA” specialist Replay Holdings LLC. The newco arrived with a $55 million seed round and having assembled a portfolio of...
Korea’s Medipost builds US, Japan presence for umbilical cord blood stem cells
Inflammatory
South Korea’s Medipost Co. Ltd. is gearing up for phase III trials in the U.S. of its stem cell therapy, Cartistem, an allogeneic human umbilical cord blood-derived mesenchymal stem cell therapy for treatment of knee articular damage in patients with osteoarthritis.
Vicebio emerges from stealth with revamped molecular clamp vaccine platform
Coronavirus
The technology behind one of the most high profile COVID-19 vaccine development programs has been re-engineered to correct a design fault and now forms the basis of newco Vicebio Ltd. The London-based company has shown its face for the first time following its founding in 2019 around molecular...
CRISPR co-inventor’s Epic Bio launches with $55M series A
Financings
Controlling the epigenetics of a patient, figuring out what genes are expressed and understanding their level of expression, is at the center of Epic Bio, a new company founded by Stanley Qi, co-inventor on the CRISPR patent held by the University of California.

U.S.

Federal Circuit short-circuits attempt to provide inventorship for AI
Artificial intelligence
The question of whether an artificial intelligence (AI) algorithm should enjoy the status of an inventor has been making the rounds in various nations, but the U.S. Court of Appeals for the Federal Circuit has rejected the notion based on a plain reading of the statute. The court said that the...

Europe

Eurekare moves to spark European synthetic bio, microbiome startups
Financings
Eurekare SA, a technology commercialization and investment firm, has opened the doors to the first of a number of biotech “studios” it is setting up to hothouse synthetic biology and microbiome companies formed around intellectual property sourced from universities across Europe. It’s the most...

Alnylam’s Amvuttra rare disease drug among positive recommendations from Europe’s CHMP
Regulatory
Alnylam Pharmaceuticals Inc.’s Amvuttra (vutrisiran), a treatment for the rare disease hereditary transthyretin-mediated amyloidosis, was among medicines recommended for approval by regulators from Europe’s Committee for Medicinal Products for Human Use (CHMP) in a busy sitting.
UK competition watchdog fines Pfizer and Flynn Pharma £70M for gouging epilepsy drug price
Regulatory
The U.K.’s competition watchdog has found that a subsidiary of Pfizer Inc. and a generics firm jacked up the price of a life-saving epilepsy drug by up to 2,600%, fining them a total of £70 million (US$83.72 million). The Competition and Markets Authority’s decision is part of a long-running...

Clinical

Kringle Pharma’s phase II ALS study falls short
Musculoskeletal
Kringle Pharma Inc.’s phase II trial evaluating its recombinant human hepatocyte growth factor ligand, oremepermin alfa, failed to meet both primary and secondary endpoints in a study of its potential to help people with amyotrophic lateral...
Novartis canakinumab effort in oncology misses mark in phase III lung cancer study
Cancer
Novartis AG’s attempt to repurpose its immunology drug canakinumab as an oncology therapy was always considered a long shot. Now, the chances of success have receded further after another phase III failure in lung cancer. The phase III Canopy-A...
GSK move puts onus on Ideaya to advance some once-partnered programs
Cancer
News of GSK plc’s decision to decline its option for MAT2A inhibitor IDE-397, the lead compound from a 2020 collaboration with Ideaya Biosciences Inc. sent the latter’s shares (NASDAQ:IDYA) slipping 35% to close Aug. 15 at $10.20. For Ideaya,...
See level: Unity’s phase II in macular edema boosts the stock
Ocular
Positive data from Unity Biotechnology Inc.’s phase II study of UBX-1325, a senolytic BCL-XL inhibitor for treating diabetic macular edema, boosted the stock out of the penny range. Shares of the South San Francisco-based company’s stock...
Stargardt space crowds as Iveric, Apellis face off in GA
Ocular
The phase III miss disclosed Aug. 11 by Kubota Pharmaceutical Holdings Co. Ltd. subsidiary Kubota Vision Inc. in Stargardt disease put more eyes on the rare, inherited, juvenile-onset form of macular degeneration, for which nothing is approved.
Siga leads monkeypox antiviral research, amid lack of interest from big pharma
Infection
After declarations from the World Health Organization and the U.S. government that monkeypox is a public health emergency, attention is turning to the pharma industry’s response to the disease. Vaccines look likely to play a crucial role in...

BioWorld Perspectives

U.S. politicking threatens public trust in the FDA and CDC
BioWorld
The truth that every action has a reaction is being proven again in the public square of the U.S. as the shrill, endless clamor of politicians hoping to...
When it comes to drug quality, is a ‘C’ acceptable?
BioWorld
In releasing its annual report card for drug manufacturing inspections in which the per-country/region grading curve peaked at 77% and bottomed at 68%, the...
A decade of incentives to promote antibiotic development and still no viable route to commercial success
BioWorld
It should be motoring to profitability, but nine months after the U.S. launch of its new antibiotic, Zemdri (plazomicin), Achaogen Inc. has filed for...
BioWorld's Top 10: Biggest newsmakers and trending stories of 2018
BioWorld
In many ways, 2018 resembled the previous year in fast motion. Political turmoil accelerated on both sides of the Atlantic, with Asia swept into the fold,...
Deck the halls, or get decked out: BioWorld’s 12th annual Holiday Gift Guide
BioWorld
The holidays are (already) here again. Much as we try to push back the calendar, those Thanksgiving doorbuster, Black Friday blowout, Small Business...
Price increases - tone deaf or defiant?
BioWorld
Defying the laws of nature, many drug companies seem intent on proving that what goes up doesn’t have to come down. For them, the sky’s the limit when it...
PABNAB – A disappointing distraction at BIO, not associated with BIO
BioWorld
On what planet are the PABNAB (Party at BIO not associated with BIO) organizers living to think it was acceptable to spotlight topless dancers with company...

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