AMSTERDAM, The Netherlands—Sanofihas partnered with Audion Therapeutics, a private biopharmaceutical companyfocused on developing treatments for conditions affecting the ear, to developpotential treatments for hearing loss through the optimization of smallmolecules by using a regenerative medicine approach.
The companiesannounced their partnership with little fanfare on June 16, and are notgranting media interviews. However, their collaboration holds significantpromise, as hearing loss is a condition with a significant unmet patient need.According to the World Health Organization (WHO), approximately 500 millionpeople worldwide suffer from some form of it, and there are currently noprescription products for a disease-modifying treatment.
According to theWHO, of people aged 65 to 74, nearly 33 percent have hearing loss and close to50 percent of people over the age of 75 are affected. Of people over the age of85, almost everyone is affected to some extent.
Currentestimates have approximately 900 million people suffering from age-relatedhearing loss by 2050—but younger people are reportedly suffering more as well,due to frequent exposure to excessive noise.And according tothe National Institutes of Health and the Centers for Disease Control andPrevention, the total annual costs of hearing loss are approximately $50billion per year in the United States alone.
That's a tallorder for Audion, whose science and drug discovery platform originate from theMassachusetts Eye and Ear Infirmary (MEEI), a specialty hospital providingpatient care for disorders of the eye, ear, nose, throat, head and neck. TheMEEI also has a leading international reputation in hearing loss research andis a teaching partner of Harvard Medical School.
The company'sfounders include Albert Edge, associate professor at Harvard Medical School,Helmuth van Es (who is also the co-founder of Galapagos NV) and Rolf JanRutten. Audion's technologies originated from Edge's research at the MEEI andare exclusively licensed to Audion.
Audion approacheshearing loss by focusing on the regeneration of sensory hair cells in the innerear, which are lost in the vast majority of hearing loss cases. To achievethis, the company has a validated industrialized screening cascade and acritical path to identify, verify and develop new small-molecule compounds. Thecompany also has a broad patent portfolio that covers its drug discoveryplatform, screening cascades, molecular targets and new compounds.
Under itsagreement with Sanofi, Audion will use the technology developed at the MEEI inthe Eaton-Peabody Laboratory by Edge. Sanofi has an option to licensetechnology rights from Audion related to research conducted under thecollaboration. No further details were made available.
"Thisalliance with Sanofi validates our thinking around developing small-moleculeregenerative drugs for the treatment of hearing loss. We are very excited aboutthis collaboration," said Jan Rutten and van Es in a statement released bythe companies. "Sanofi's interest in the hearing loss field plus its vastexperience and infrastructure in small-molecule drug discovery make them theperfect partner to move this program forward as diligently as possible."
Jan Rutten,however, hinted to ddnthat the company will be making headlines again before year's end: "We arefocusing our attention on the collaboration and the build-up of our pipeline atthis time, and we would rather put ourselves in the media again when we havemore news," he said via e-mail.
Sanofi, Rib-Xink deal to seek antibiotics for multi-drug resistant pathogens
NEW HAVEN,Conn.—In other news from Sanofi last month, the pharma and Rib-XPharmaceuticals Inc. have signed an exclusive worldwide research collaborationagreement and option for license for novel classes of antibiotics that resultfrom Rib-X's RX-04 program for treating resistant Gram-positive andGram-negative pathogens.
Per the terms ofthe collaboration agreement, Rib-X will receive $10 million from Sanofi in anupfront payment, and is also eligible to receive up to $9 million in near-termresearch milestones. Rib-X will also be eligible for further payments, based onthe achievement of certain research, preclinical, regulatory and commercialmilestones.
Sanofi will havethe right to develop multiple products under the agreement. With the exclusionof the assets licensed to Sanofi through the agreement, Rib-X will retain therights to the discovery platform as well as its future programs. Rib-X retainsa co-promotion option within the United States on one of the moleculesresulting from the collaboration, as well. Development and regulatorymilestones could result in up to $86 million on a per product basis, whilecommercial milestones could result in over $100 million on a per product basis.Royalty rates on net sales could reach low double-digit figures.
"We could not bemore excited about partnering with a preeminent global pharmaceutical companysuch as Sanofi," said Mark Leuchtenberger, president and CEO of Rib-X, in apress release regarding the agreement. "This partnership reflects our sharedcommitment to staying ahead of the growing problem of antibiotic resistance bydelivering new standards of care for patients in need."
The proprietaryapproach of Rib-X's RX-04 program is one of rational drug design and createsnew families of compounds, specifically ones with demonstrated efficacy at low,single doses in murine infection models. The program has demonstratedantibacterial activity against several difficult-to-treat, clinicallyimportant, multi-drug resistant Gram-negative and Gram-positive pathogens.Rib-X's RX-04 program specifically targets bacterial ribosomes within thecells, the site of protein synthesis. Recent data confirms that these novelclasses of compounds impact ribosome function directly, with theiranti-bacterial activity working to interfere with the synthesis ofproteins.
"The RX-04program's completely novel classes of antibiotics should lead to truebreakthrough therapies and we look forward to working in partnership withSanofi to advance these treatments into the clinic and eventually bring them tothe global market. Importantly, this agreement will enable Rib-X toaggressively advance our clinical stage candidates, delafloxacin and radezolid,towards pivotal trials and support additional discovery-stage programs likeRX-05 and RX-06," said Leuchtenberger.
Multi-drugresistant bacteria are one of the faster growing crises in public health.Bacteria come in two main classes and are defined by their appearance when theyare stained and viewed under a microscope: Gram-negative, which appear pink,and Gram-positive, which look violet blue and generally lack the outer membranefound in Gram-negative bacteria. According to the World Health Organization, atleast 25,000 people in the European Union alone die from infections caused bymulti-drug resistant bacteria every year.
"We are very enthusiastic about entering into this collaboration withRib-X," said Dr. Elias Zerhouni, president of global R&D at Sanofi, in apress release about the agreement. "The clinical need for new antibiotics isreaching crisis level, yet the antibiotic pipeline is running dry and fewer andfewer companies are working to develop drugs in this space. This partnershipexemplifies Sanofi's commitment to translate novel approaches for treatmentinto patient solutions addressing the global critical need to combat the risingthreat of antibiotic drug resistance."