Grant Playter

Biogen announced that it had entered into a definitive agreement to acquire Reata Pharmaceuticals, a pharmaceutical company working in areas of high unmet need, on July 28, 2023. Biogen will acquire Reata for $172.50 per share in cash, an approximate valuation of $7.3 billion. According to a company press release, Reata owns the rights to the first and only FDA-approved treatment for Friedreich’s ataxia, an ultra-rare, genetic, life-shortening, debilitating, and degenerative neuromuscular disorder.

Reata’s Skyclarys (omaveloxolone), is an oral, once-daily medication indicated for the treatment of Friedreich’s ataxia in adults and adolescents aged 16 years and older in the U.S. Omaveloxolone was approved by FDA in February 2023 and is currently under European regulatory review. In addition to omaveloxolone, Reata is developing a portfolio of treatments for various neurological diseases.

“With extensive expertise in rare disease product development and global commercialization… we believe Biogen has the foundation in place to accelerate the delivery of [omaveloxolone] to patients around the world,” said Christopher Viehbacher, president and CEO, Biogen, in the release. “This is a unique opportunity for Biogen to bolster our near-term growth trajectory, and [omaveloxolone] is an excellent complement to our global portfolio of treatments for neuromuscular and rare disease.”

“Biogen’s expertise and commercial footprint make it the optimal choice to help [omaveloxolone] realize its full potential,” said Warren Huff, chairman and ChEO, Reata, in the release. “With its clear understanding of the rare disease patient journey and existing commercial infrastructure, we believe Biogen will establish [omaveloxolone] as the standard of care in the treatment of this devastating genetic disease.”

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Biogen’s acquisition of Reata will grant them access to Reata’s FDA-approved treatment for Friedreich’s ataxia.

Biogen to Acquire Reata Pharmaceuticals for $7.3 Billion 

Alexion Pharmaceuticals, a subsidiary of AstraZeneca focusing on rare disease management, announced a definitive purchase agreement with Pfizer on July 28, 2023. Alexion will acquire a portfolio of preclinical rare disease gene therapies from Pfizer for a total consideration of up to $1 billion.

According to a company press release, the transaction features various novel adeno-associated virus (AAV) capsids, which are used for delivering therapeutic gene cargos for both gene therapy and gene editing. In addition to the portfolio, Alexion will welcome talent from Pfizer associated with the portfolio.

In the release, Alexion indicated that the deal is designed to further the combined capabilities of both it and AstraZeneca in genomic medicines. It follows a move from last October, where the company 

, a genetic medicines company, with the objective of developing new genetic therapies with improved safety and efficacy profiles.

“Today’s announcement represents another major step forward in Alexion and AstraZeneca’s ambition to be an industry leader in genomic medicine, which has potential to be transformative and even curative for patients with devastating diseases,” said Marc Dunoyer, CEO, Alexion, AstraZeneca Rare Disease, in the release. “We look forward to continuing our work to develop enhanced platforms and technologies with broad therapeutic application while integrating best-in-class expertise to accelerate promising therapeutics into the clinic.”

AstraZeneca, via its subsidiary Alexion Pharmaceuticals, will purchase a portfolio of preclinical rare disease gene therapies from Pfizer in a deal worth up to $1 billion.

AstraZeneca and Pfizer Ink $1 Billion Rare Disease Deal

Alnylam Pharmaceuticals, a pharmaceutical company specializing in RNA interference (RNAi) therapeutics, announced a strategic agreement with Roche on July 24, 2023. The focus of this agreement is to develop and commercialize zilebesiran, Alynlam’s investigational RNAi therapeutic intended for the treatment of hypertension. Under the agreement, Alnylam will receive $310 million in upfront payments for the rights to zilebesiran, with a total payment of approximately $2.8 billion contingent on satisfying various development, regulatory, and sales milestones.

According to a company press release, in a Phase I study, zilebesiran was associated with dose-dependent reductions in serum angiotensinogen relative to placebo. Patients achieved tonic blood pressure control with consistent and durable blood pressure reduction throughout a 24-hour period, sustained up to six months after single doses of ≥ 200 mg. The treatment is now in Phase II of clinical trial testing.

“We are thrilled to announce this collaboration, as it combines Alnylam’s proven track record in RNAi therapeutics with Roche’s global commercial reach, commitment to innovation, and desire to transform the landscape for patients with severe cardiovascular diseases,” said Yvonne Greenstreet, CEO, Alnylam, in the release. “With this collaboration, we now can develop zilebesiran in a more robust way, allowing us to have cardiovascular outcomes data in hand at launch to ensure results relevant not only for health authorities but also for access and clinical practice in order to ultimately reach as many patients as possible.”

“We are excited to work together with Alnylam and leverage our strong R&D capabilities, our leadership in cardiovascular diagnostics and our global commercial footprint to further develop and provide this promising therapy with best-in-disease potential to patients,” said Teresa Graham, CEO, Roche Pharma, in the release. “... Together with a strong partner like Alnylam, we are looking forward to making a significant impact for patients living with hypertension at high cardiovascular risk and potentially other cardiovascular indications.”

The agreement stipulates that Roche will receive exclusive rights to commercialize zilebeserian outside of the United States in exchange for low double-digit royalties on net sales of the treatment. The companies will co-commercialize the treatment in the US, splitting profits equally.

Roche will pay up to $2.8 billion for the rights to Alnylam’s RNA-based hypertension therapeutic.

Roche and Alnylam Strike $2.8 Billion RNAi Deal

Tarsus Pharmaceuticals, a biopharmaceutical company developing treatments in the fields of eye care, dermatology, and infectious disease prevention, announced FDA approval of Xdemvy (lotilaner ophthalmic solutions) 0.25% for the treatment of Demodex blepharitis on July 25, 2023. According to a company press release, Tarsus’ lotilaner ophthalmic solution is the first and only FDA approved treatment that directly targets Demodex mites, the root cause of Demodex blepharitis.

According to the release, Demodex blepharitis is a common lid margin disease that is characterized by eyelid margin inflammation, redness, and ocular irritation. The disease impacts approximately 25 million adults in the United States. Lotilaner ophthalmic solution treats the condition by selectively inhibiting the Demodex mites’ gamma-aminobutyric acid-gated chlorine (GABA-Cl) channels. Lotilaner ophthalmic solution was evaluated in two pivotal trials of more than 800 patients that the met primary endpoint and all secondary endpoints, with statistical significance and no serious treatment-related adverse events.

“We are thrilled to announce the FDA approval of Xdemvy [lotilaner ophthalmic solution] for the treatment of Demodex blepharitis and look forward to making this product broadly available to the millions of patients who have not had any FDA approved therapeutics for this disease,” said Bobak Azamian, CEO and chairman, Tarsus, in the release. “This tremendous milestone was achieved through a successful collaboration of our talented Tarsus team, countless eye care providers, and the hundreds of patients who participated in our trials. We are grateful and honored for the opportunity to introduce the first and only approved therapeutic for this disease to the eye care community.”

“After years of seeing Demodex blepharitis in my practice without an effective way to target the root cause of the disease, I’m so pleased to have a new treatment to offer my patients,” said Christopher Starr, associate professor of Ophthalmology, director of Refractive Surgery, Ophthalmic Education and the Cornea Fellowship Program, Weill Cornell Medicine, New York Presbyterian Hospital, in the release. “Demodex blepharitis, easily diagnosed by the presence of eyelash collarettes, can result in ocular damage in multiple ways, including irritation, eyelash distention or loss and inflammation, which can be uncomfortable for patients. This new medicine is a positive step forward for the treatment of this disease in many patients who have been struggling for years.”

Tarsus Pharmaceuticals’ Xdemvy (lotilaner ophthalmic solutions) is designed to treat Demodex blepharitis by directly targeting Demodex mites.

FDA Approves Novel Demodex Blepharitis Treatment

Sanofi and Recludix Pharma, a pharma company specializing in inflammatory diseases and cancer, announced a strategic collaboration on July 20, 2023. The partnership focuses on developing and commercializing small-molecule signal transducer and activator of transcription 6 (STAT6) inhibitors for patients with immunological and inflammatory (I&I) diseases. Under the agreement, Sanofi will pay Recludix $125 million in near-term payments and $1.2 billion in additional payments contingent on various development, regulatory, and sales-based milestones.

According to a company press release, Recludix will advance various STAT6 inhibitors from preclinical research and development until the start of Phase II clinical trials, after which Sanofi will handle further development and commercialization of the candidate. Sanofi will retain global rights to any small-molecule STAT6 inhibitors produced from the partnership, although Recludixhas the opportunity to participate in profit/loss share and certain co-promotion opportunities in the United States. Recludix will also be eligible for double-digit royalties on future product sales.

“This collaboration for the advancement of a preclinical oral STAT6 inhibitor speaks to our ability to successfully solve the challenge of drugging this attractive, yet elusive, therapeutic target,” said Nancy Whiting, CEO, Recludix, in the press release. “We are proud of the breakthroughs we have made in drugging SH2 [Src homology 2] domains as demonstrated by the STAT6 program, and we are excited to partner with Sanofi … who shares our vision for advancing an oral STAT6 inhibitor forward quickly for the benefit of patients. This strategic collaboration empowers the program while ensuring meaningful development participation and economic value for Recludix.”

“Recludix’s approach to targeting STAT6 has significant potential for a number of I&I diseases, especially where a precisely tailored oral therapy could best fit within the patient’s needs at various stages of disease,” said Frank Nestle, global head, Research, and chief science officer, Sanofi, in the release. “We are excited to partner with the Recludix team to continue to advance innovative strategies for targeting this previously considered undruggable target, aligned with our mission to best serve patients with inflammatory disease.”

Sanofi and Recludix Pharma’s deal centers on developing treatments for various immunological and inflammatory diseases.

Sanofi and Recludix Enter STAT6 Inhibitor Partnership Worth More Than $1.3 Billion 

WuXi STA, a contract research development and manufacturing organization (CRDMO) that functions as a subsidiary of WuXi AppTech, announced the launch of its first high potency (HP) fully automated sterile injectable manufacturing line at its drug product site in Wuxi City, China on July 17, 2023. According to a company press release, the new HP injectable line has an annual capacity of 12 million units, and will enhance the company’s manufacturing capabilities and capacity for injectable dosage forms.

The line uses fully enclosed isolation systems and automated filling machines, which is intended to minimize human intervention, prevent cross-contamination, and ensure quality assurance for sterile products; according to the release, the new line meets Occupational Exposure Limits as low as 10 nanograms per cubic meter. Additionally, the line is equipped with two 20 meters squared lyophilizers with a filling rate of up to 200 units per minute, which are designed to support liquid and lyophilized vials of various specifications.

"I'm thrilled that our HP injectable R&D and manufacturing platform has achieved yet another significant milestone," said Minzhang Chen, co-CEO, WuXi AppTec, and CEO, WuXi STA, in the release. "In addressing the growing needs of high potency drugs, WuXi STA... will continue to strengthen our CRDMO platform capabilities. With our proven track record of consistent quality and [environment, health, and safety] systems across all sites globally, we are committed to accelerating pharmaceutical development and bringing new therapeutics for patients worldwide."

According to the release, WuXi STA plans to add two more injectable manufacturing lines at the same site by 2024. In that timeframe, additional lines are also set to be added to the Couvet site in Neuchâtel, Switzerland and the upcoming Middletown site in Delaware, USA.

WuXi STA’s new high-potency injectable line has an annual capacity of 12 million units.