Roche Hemlibra data show continued benefits in hemophilia A at ISTH 2019

• New analyses from phase III HAVEN studies support Hemlibra’s sustained efficacy, safety and quality of life benefit in people with haemophilia A, with and without factor VIII inhibitors
• First data of phase IIIb STASEY study reinforces safety profile of Hemlibra seen in pivotal HAVEN 1 clinical trial 
• New analysis of pivotal data suggests additional factor treatment may not be needed for people on Hemlibra undergoing certain minor surgery

Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced new data for Hemlibra^® (emicizumab) across multiple pivotal studies in people with haemophilia A with and without factor VIII inhibitors at the International Society on Thrombosis and Haemostasis (ISTH) 2019 Congress on 6-10 July in Melbourne, Australia. In total, Roche presented 21 abstracts from its haemophilia programme, including five oral presentations. Further data from the four pivotal HAVEN clinical trials were presented, demonstrating the long-term safety, efficacy and quality of life benefit of Hemlibra in people with haemophilia A with and without factor VIII inhibitors. Roche also presented the first interim analysis from the phase IIIb STASEY study, reinforcing the safety profile of Hemlibra in adults and adolescents (aged 12 years or older) with haemophilia A with factor VIII inhibitors seen in the HAVEN 1 clinical trial.

“Data presented at ISTH continues to reinforce Hemlibra’s potential to redefine the standard of care for people living with haemophilia A,” said Sandra Horning, MD, Roche’s Chief Medical Officer and Head of Global Product Development. “We are particularly excited to present the first interim analysis of safety data from the STASEY study, which adds to the growing body of evidence supporting Hemlibra as an important treatment option for people with haemophilia A.”

https://www.marketscreener.com/ROCHE-HOLDING-LTD-9364975/news/Roche-presents-a-broad-range-of-data-for-Hemlibra-demonstrating-continued-benefits-for-people-with-h-28869822/

Teva unloads plant, U.S. OTC line to PL Developments

There are only three manufacturing sites in the world approved by the FDA to make nicotine gum and Teva has sold one, along with its U.S. portfolio of OTC products, to a company that is all about store brands.

Westbury, New York-based PL Developments says the nicotine gum operations it bought from Teva will compliment the nicotine lozenges business it already has. The deal also comes with 40 approved and pending generic products and over-the-counter products. Terms of acquisition to the private, family-owned company were not disclosed.

“Adding Teva’s nicotine chewing gum ANDAs to our recently approved nicotine lozenge ANDA, gives PLD one of the most comprehensive portfolios in the high-value NRT (nicotine replacement therapy) space,” the company said in its announcement. “T­he acquisition significantly reinforces PLD’s position as the second largest packager and distributor of U.S. store-brand drugs.”

In addition to several copies of Nicorette products, the sale includes knockoffs of Rogaine products, Claritin D and others.

PLD President Evan Singer told Newsday that the deal includes a 93,000-square-foot manufacturing facility in Copiague, New York with about 80 employees. He said it is one of three the FDA has approved globally to make nicotine gum.

The company, which sells to chains like Walmart and Costco, has moved into other product areas through M&A in the past. In 2013, it expanded beyond its base in solid dose products with the acquisition of Aaron Industries, a liquid dose maker in Clinton, South Carolina.

In 2016, PLD invested about $45 million to build another manufacturing and distribution site in South Carolina. The one in Piedmont, South Carolina, was its sixth site.

Teva, on the other hand, has been aggressively shedding manufacturing facilities for several years. It targeted about 40 facilities and 14,000 jobs as part of a $3 billion cost-cutting endeavor after misguided acquisitions and a softening U.S. generics market left it struggling financially.

https://www.fiercepharma.com/manufacturing/pl-developments-glad-to-get-nicotine-gum-lines-otc-deal-teva

MorphoSys, Vivoryon Agree on Small Molecule Inhibitors in Immuno-Oncology

Vivoryon Therapeutics AG(Euronext Amsterdam: VVY) and MorphoSys AG (FSE: MOR; Prime Standard Segment; MDAX & TecDAX; Nasdaq: MOR) today announced that they have entered into an agreement under the terms of which MorphoSys has obtained an exclusive option to license Vivoryon’s small molecule QPCTL inhibitors in the field of oncology. The option covers worldwide development and commercialization for cancer of Vivoryon’s family of inhibitors of the glutaminyl-peptide cyclotransferase-like (QPCTL) protein, including its lead compound PQ912. In exchange, MorphoSys has committed to investing up to EUR 15 million in a minority stake in Vivoryon Therapeutics as part of a capital raise planned for later this year.

While Vivoryon’s lead drug candidate PQ912 has already completed a phase 2a clinical trial in Alzheimer’s disease, recent preclinical data strongly suggest that the compound could represent a novel approach for cancer therapy. Vivoryon’s orally available compounds target the QPCTL enzyme, which has been shown to be a modulator of the CD47-SIRP alpha interaction. Left unchecked, this interaction, known as the “don’t eat me” signal, allows cancer cells to escape the body’s innate immune defense through inhibition of the phagocytic activity of macrophages. During the option period, MorphoSys will conduct preclinical validation experiments on Vivoryon’s family of QPCTL inhibitors, including an assessment of the potential benefits of combining them with MorphoSys’s proprietary program tafasitamab (MOR208), which is currently in late-stage development for the treatment of relapsed/refractory diffuse large B-cell lymphoma (r/r DLBCL).

“This deal gives us access to a unique set of drug candidates with exciting potential in cancer”, said Dr. Simon Moroney, CEO of MorphoSys. “A number of studies suggests that the CD47-SIRP alpha interaction may be of central importance to the activity of some anti-cancer antibodies. In this regard, securing rights to Vivoryon’s estate of compounds in oncology makes strong strategic sense for us. In particular, we are looking forward to exploring the potential for synergy with tafasitamab (MOR208), our most advanced drug candidate. If successful, the use of these orally formulated QPCTL inhibitors may open the way to combinations with other anti-cancer antibodies aiming at boosting their cell killing activity.”

https://www.biospace.com/article/morphosys-and-vivoryon-therapeutics-enter-agreement-on-small-molecule-inhibitors-of-cd47-sirp-alpha-signaling-in-immuno-oncology/

Skyhawk Partners with Merck to Discover and Develop RNA-Binding Molecules

Privately-held Skyhawk Therapeutics struck an agreement with Merck to discover and develop small molecules that modulate RNA splicing as a new modality for the potential treatment of certain neurological diseases and cancer.

Waltham, Mass.-based Skyhawk, which launched last year, said it will use its proprietary SkySTAR technology platform will be employed to discover and develop innovative RNA-binding small molecules. Under terms of the agreement, Skyhawk is eligible to receive approximately $600 million per program target. Bill Hanley, co-founder and chief executive officer of Skyhawk, said the company looks forward to the collaboration as it will demonstrate the ability of SkySTAR to “deliver novel drug candidates for the disease targets Merck has selected and advancing those compounds to address the unmet medical needs of patients.”

Skyhawk is targeting diseases driven by a type of RNA missplicing called exon skipping, which occurs when key regions on the RNA are left out during the RNA splicing process. Skyhawk’s proprietary technology enables the rational design of small molecules that target specific binding pocket regions on RNA, using both sequence and structural specificity, at particular moments in the RNA splicing process. By doing so, they reverse the missplicing and treat the disease, the company said. The SkySTAR (Skyhawk Small molecule Therapeutics for Alternative splicing in RNA) platform integrates information from computational, kinetic and structural models of RNA in order to generate “unique and selective families of chemistry for each target,” the company said.

Under the agreement struck with Merck, Skyhawk will grant the pharma giant the option to exclusively license worldwide intellectual property rights to candidates discovered and developed under the collaboration that are directed to program targets. If Merck exercises that option, the larger company will be responsible for further development and commercialization. Skyhawk will receive an upfront cash payment and, to the extent Merck exercises its option, potential milestone payments and royalties on sales of approved products resulting from the collaboration.

Dean Y. Li, senior vice president, discovery and translational medicine, Merck Research Laboratories, said RNA splicing modification provides a new approach to modulating targets previously considered undruggable. Li said Merck looks forward to its collaboration to “explore the potential of this new modality.”

Merck isn’t the only dance partner for Skyhawk. This morning, the company announced an expansion of its agreement with Biogen. The initial agreement, which include a $74 million upfront payment, was forged in January with the companies using the SkySTAR platform to target neurological diseases. The research is paying off as Skyhawk said the new agreement extends Biogen’s exclusive license to worldwide intellectual property rights beyond the original collaboration’s research-stage therapeutic candidates for the treatment of conditions including multiple sclerosis, spinal muscular atrophy and additional neurological disorders. As part of the expansion, Skyhawk receives an upfront payment from Biogen and may receive potential future milestone payments and royalties.

In May, Skyhawk and Takeda Pharmaceutical also forged an agreement to use the SkySTAR platform to target neurological diseases.

https://www.biospace.com/article/in-twin-deals-skyhawk-therapeutics-forges-collaboration-with-merck-expands-agreement-with-biogen/

AzurRx initiates new mid-stage study of MS1819-SD in cystic fibrosis

Thinly traded nano cap AzurRx BioPharma (NASDAQ:AZRX) is up 12% after hours on light volume in response to the launch of a Phase 2 clinical trialevaluating MS1819-SD, combined with standard porcine enzyme replacement therapy (PERT), in cystic fibrosis patients with severe exocrine pancreatic insufficiency who continue to experience clinical symptoms of fat malabsorption despite maximum PERT therapy. Preliminary data should be available in early 2020. The trial should be completed later that year.

Another Phase 2, OPTION, assessing MS1819-SD in a head-to-head comparison with PERT, is ongoing with topline data expected in August/September.

Management will host a conference call at 4:30 pm ET to discuss the study and provide an R&D update.

https://seekingalpha.com/news/3476326-azurrx-initiates-new-mid-stage-study-ms1819-sd-cystic-fibrosis-shares-12-percent-hours

SC Health files for $150M IPO

SC Health (SCHU) has filed a preliminary prospectus for a $150M IPO of 15M units at $10 per Unit, each consisting of one Class A ordinary share and 1/2 of a five-year warrant to purchase one Class A ordinary share at $11.50.

The blank check company is domiciled in the Cayman Islands.

https://seekingalpha.com/news/3476340-sc-health-files-150m-ipo

Cardinal Health Faces Recruitment Challenge as CFO Departs

Pharmaceuticals distributor Cardinal Health Inc. is turning to its chief executive to oversee the books following the departure of its finance chief, indicating a lack of potential successors in an industry facing declining profitability and potential settlement costs associated with the U.S. opioid crisis.

The Dublin, Ohio-based company Monday said Chief Financial Officer Jorge M. Gomez — who has served in the role since Jan. 2018 — will step down to become finance chief at Dentsply Sirona Inc., a maker of dental products. Mr. Gomez leaves Cardinal on Aug. 9, a day after the release of the company’s year-end results for fiscal 2019.

Chief Executive Michael Kaufmann will take over as interim CFO while

Cardinal searches for a successor. Mr. Kaufmann served as the company’s finance chief from 2014 to 2017 and was elevated to the CEO position in Jan. 2018.

The appointment of Mr. Kaufmann as interim finance chief points to Cardinal’s lack of potential candidates to succeed Mr. Gomez, analysts said, and suggests the company has had a challenge attracting the right talent.

“Companies this size normally have deep benches to fill this type of vacancies from below,” said Brian Tanquilut, an analyst at financial services advisory Jefferies LLC. Both Mr. Kaufmann and Mr. Gomez were promoted internally for their most recent jobs.

Cardinal Chairman Gregory Kenny in an email highlighted the strength of the firm’s finance team and praised Mr. Kaufmann’s knowledge of the company.

“As a company with a rich culture that is focused on our people and delivering value to the healthcare industry, we are looking for a person that embodies our values and will promote career development across the organization,” Mr. Kenny said.

The vacancy in a key management position comes at a challenging time for Cardinal.

The company has been battling with falling profitability, specifically in its generic drugs business. Cardinal’s earnings per share slumped to 81 cents in fiscal 2018, down 80% from $4.03 per share in fiscal 2017.

Two recent acquisitions, Cordis and Patient Recovery, so far haven’t yielded forecast returns, adding to the pressure on the company’s management, Mr. Tanquilut said.

“The loss of any executive during a key point in a turnaround is a challenge,” said Eric W. Coldwell, an analyst at financial services firm Robert W. Baird & Co. “Cardinal seemed a bit unprepared for Jorge’s [Mr. Gomez] departure and it won’t sit well with investors that the CFO is transitioning as the company prepares and delivers its fiscal year outlook,” Mr. Coldwell said.

Cardinal has also disclosed it is among several pharmaceutical wholesale distributors that have been named as defendants in more than 2,000 lawsuits related to the distribution of prescription opioid pain medications in the U.S. More than 60 of these lawsuits are purported class actions, according to the company’s latest regulatory filings.

The pressure on profit margins as well as potential settlement costs associated with these lawsuits complicate Cardinal’s efforts to find a new CFO, said John Ransom, an analyst at financial services provider Raymond James Financial Inc. “It will be tough for them to fill the spot, ” Mr. Ransom said.

As interim CFO, one of Mr. Kaufmann’s tasks will be to deal with the costs of a potential settlement, analysts said. Competitor McKesson Corp. in May agreed to pay $37 million to resolve claims that the company helped fuel the opioid epidemic.

“As a pharmaceutical wholesale distributor, we do not control either the supply of, or the demand for, opioids, since we do not manufacture medications or write prescriptions,” Cardinal said on its website.

Other companies in the space, including McKesson and AmerisourceBergen Corp., have also suffered from executive turnover in recent years. “The industry continues to see a lot of departures and struggles to attract new talent,” said Raymond James’ Mr. Ransom.

https://www.marketscreener.com/CARDINAL-HEALTH-11969/news/Cardinal-Health-Drug-Distributor-Cardinal-Health-Faces-Recruitment-Challenge-as-CFO-Departs-28868002/