Pfizer Acquires Rare-Drug Company Therachon for $810 Million

Pfizer announced it intends to buy Therachon, a rare disease biotech company based in Basel, Switzerland.

Under the terms of the deal, Pfizer will pay $340 million upfront with another $470 million in payments contingent on milestones for the development and commercialization of TA-46 for the treatment of achondroplasia. Achondroplasia is a genetic condition that is the most common form of short-limbed dwarfism. The disorder can also cause serious cardiovascular, neurological and metabolic problems for about 250,000 people worldwide. There are currently no approved treatments for achondroplasia.

TA-46 is a soluble recombinant human fibroblast growth factor receptor 3 (FGFR3) decoy. It is believed it can normalize the overactive FGFR3 signaling pathway that is implicated in bone development abnormalities linked to achondroplasia. Therachon is developing it as a weekly subcutaneous injection for children and adolescents with the disorder.

The drug has finished Phase I trials. The European Medicines Agency (EMA) and the U.S. Food and Drug Administration (FDA) have both granted the drug Orphan Drug Designation.

Before the deal closes, Therachon plans to spin-off its apraglutide development program into an independent company. The drug is a once-weekly GLP-2 analog. It is currently in Phase II trials for short bowel syndrome. Pfizer Ventures, the drug company’s venture capital arm, holds a minority stake currently and will continue to have an equity stake in the spin-off company.

“At Pfizer, our strategy is focused on advancing the most promising science in the world, regardless of whether it is found inside or outside of our labs,” stated Mikael Dolsten, Pfizer’s chief scientific officer and president, Worldwide Research, Development, and Medical. “By acquiring Therachon, we hope to leverage Pfizer’s leading scientific and development capabilities to more rapidly advance this potentially promising therapy for people with achondroplasia.”

Pfizer intends for the acquisition to bolster its rare diseases portfolio, which includes several research programs for pediatric growth disorders.

In addition to headquarters in Switzerland, Therachon has research laboratories in Nice, France and business operations in the New York area.

On March 25, Therachon presented data from the Phase I trial of apraglutide in short bowel syndrome at the ASPEN 2019 Nutrition Science & Practice Conference organized by the American Society for Parenteral and Enteral Nutrition in Phoenix, Ariz. Short bowel syndrome (SBS) is caused by extensive intestinal resection as the result of chronic inflammatory bowel disease (IBD), acute events such as mesenteric infarction or congenital abnormalities. Symptoms include diarrhea, dehydration, malnutrition and weight loss.

Apraglutide is designed to increase the intestine’s ability to absorb nutrients and decrease the need for parenteral support (PS), which is the intravenous delivery of essential nutrients, calories and fluids. The drug is currently being studied in two Phase II clinical trials in SBS patients in Denmark.

For its part, Pfizer’s acquisition of the achondroplasia will set them on a path to compete with BioMarin Pharmaceutical, which is developing vosoritide for the disorder. In 2015, BioMarin announced positive Phase II proof-of-concept data for the drug, with data from 26 children in the trial showing a favorable safety profile and efficacy at the 15 micrograms/kilogram/daily dose.

In June 2018, BioMarin dosed the first patient in a global Phase II trial of the drug. The drug has also been granted Orphan Drug Designation in the U.S. and Europe. The trial will study the drug in about 70 infants and young children with achondroplasia in newborns and children less than 5 years old for 52 weeks. There will then be an open-label extension. The primary objectives of the study are safety, tolerability, and the effect of vosoritide on height Z-scores. It also plans to augment that score with data on proportionality, functionality, quality of life, sleep apnea, and foramen magnum dimension in addition to the beginning of major illnesses and surgeries.

https://www.biospace.com/article/pfizer-buys-theracon-for-810-million/

Esperion reports Q1 EPS $3.07, may not compare to consensus 74c

Reports Q1 revenue $145.4M, consensus $72.22M. “The potential of bempedoic acid is being broadly realized as evidenced by publication and presentation of Phase 3 clinical study results in the New England Journal of Medicine, the American College of Cardiology 2019 Scientific Sessions and other industry leading platforms. This high-profile external validation of the potential of bempedoic acid to meet the LDL-C lowering needs of the millions of patients with high LDL-C despite the use of currently accessible therapies confirms the beliefs of our Lipid Management Team,” said Tim M. Mayleben, president and CEO of Esperion. “We will continue to work closely with regulatory authorities to bring our LDL-C lowering therapies to physicians, payers and their patients.”

https://thefly.com/landingPageNews.php?id=2905563

Horizon Pharma sees FY19 revenue $1.26B-$1.28B, consensus $1.24B

The company now expects full-year 2019 net sales of $1.26 billion to $1.28 billion, an increase from the previous guidance range of $1.23 billion to $1.25 billion. Full-year 2019 adjusted EBITDA is now expected to be $450 million to $465 million, an increase from the previous guidance range of $440 million to $455 million, while also increasing investment in the potential U.S. launch of teprotumumab following Phase 3 trial results.
https://thefly.com/landingPageNews.php?id=2905571

Alder Biopharmaceuticals presents new analysis in Eptinezumab study at AAN

Alder announced a new analysis of patient-reported outcomes data from the PROMISE-2 Phase 3 clinical trial of eptinezumab for the prevention of chronic migraine. Eptinezumab is an investigational monoclonal antibody targeting the calcitonin gene-related peptide administered by quarterly infusion for migraine prevention. The new analysis showed improvements in most bothersome migraine symptoms and patients’ global impression of change in their migraine status by Month 1 after treatment, with improvements sustained or increased through the first and second quarterly infusion. Detailed data will be presented today at the 71st AAN Annual Meeting in Philadelphia, PA. Highlights from the data analysis include: At Month 1 after treatment: 45% of patients treated with 100 mg of eptinezumab and 57% of patients treated with 300 mg of eptinezumab indicated their MBS was much improved or very much improved vs. 29% of patients receiving placebo; 45% of patients treated with 100 mg of eptinezumab and 59% of patients treated with 300 mg of eptinezumab indicated their PGIC was much improved or very much improved vs. 32% of patients receiving placebo. At Month 6, three months after the second quarterly infusion: 57% of patients treated with 100 mg of eptinezumab and 57% of patients treated with 300 mg of eptinezumab indicated their MBS was much improved or very much improved vs. 42% of patients receiving placebo; 60% of patients treated with 100 mg of eptinezumab and 60% of patients treated with 300 mg of eptinezumab indicated their PGIC was much improved or very much improved vs. 41% of patients receiving placebo.

https://thefly.com/landingPageNews.php?id=2905659

CEL-SCI says length of Phase 3 study could be sign of Multikine’s efficacy

CEL-SCI Corporation issued a letter to its shareholders, which read in part, “The first 4 months of this year have already been very positive for us. Our Phase 3 head and neck cancer study is continuing and we are moving forward with our Rheumatoid Arthritis product development. Since January 1, we have received about $9.6 million from the conversion of warrants to finance our ongoing projects. We believe the following developments with regard to the Multikine* Phase 3 trial have contributed to additional investments in our Company: We have not yet hit the primary endpoint of our 928 patient Phase 3 study with our investigational cancer immunotherapy drug Multikine. That endpoint will be reached when 298 events (deaths) have occurred and are recorded in the two main comparator arms of the study. Since the scientific literature does not suggest an improvement in the survival rates for oral head and neck cancer patients receiving standard of care only, we believe a delay in reaching these 298 events could be a good sign for the potential effectiveness of Multikine…At the end of March 2019, we had an official review of the Phase 3 study by the IDMC. The IDMC recommended to “continue the trial until the appropriate number of events has occurred”. The IDMC reviews all the study results at the time of each IDMC meeting. Had the study results indicated that meeting the primary survival endpoint of the study would no longer be possible, the study could have been deemed futile. The IDMC recommended to continue the study. In our Phase 3 trial the primary endpoint is met by reaching a 10% improvement in the overall survival of the group of patients receiving the Multikine treatment regimen plus the Standard of Care versus the overall survival of the group of patients receiving the Standard of Care only. Futility has been on the minds of investors a lot more recently, because finding futility in the case of a Phase 3 Alzheimer study by Biogen in March 2019 resulted in a loss to shareholders of close to $30 billion…We are benefiting from renewed interest in CEL-SCI’s Multikine cancer immunotherapy by meeting with new investors weekly. What is striking is that hardly any of them have ever heard of CEL-SCI, even though we have been running the largest study ever in head and neck cancer. We think that the absence of new clinical data for over 8 years (we are blinded to the Phase 3 results and therefore could not announce clinical data) is probably the reason for CEL-SCI being virtually unknown. But, while that presented a problem in the past, it now represents a fantastic opportunity. New investors are very interested for two reasons: 1) during our Phase 3 study cancer immunotherapy has become accepted as a new treatment modality for cancer; and 2) investors prefer investing near the end of this long trial. It has taken decades to get to this point in time, but we are finally near the end, and we feel good about it. Each of the last few years I have purchased more CEL-SCI stock. And I did so again this week when I purchased $100,000 worth of restricted CEL-SCI stock at $6.86 directly from the Company at the full market price. Several others from our senior management team and one member of the Board of Directors have joined me and purchased another $110,000 worth of CEL-SCI stock on the same terms. ”

https://thefly.com/landingPageNews.php?id=2905667

Biohaven Pharmaceutical price target raised to $70 from $56 at Needham

Needham analyst Alan Carr raised his price target on Biohaven Pharmaceutical to $70 and kept his Buy rating after its Q1 results and updated progress on its glutamate modulating platform. The analyst anticipates approval of the company’s Nurtec for ALS by July 2019 PDUFA date with $24M in peak sales modeled for the program and also cites migraine platform expansion with the start of Phase 2/3 trial of intranasal BHV-3500 in Acute Migraine in April.

https://thefly.com/landingPageNews.php?id=2905711

Sarepta gets exclusive option to CAPN-3 from Nationwide Children’s Hospital

Sarepta announced it has recently signed an agreement with the Research Institute at Nationwide Children’s Hospital giving Sarepta the exclusive option to a Nationwide Children’s gene therapy candidate, calpain 3, or CAPN-3, to treat Limb-girdle muscular dystrophy type 2A, or LGMD2A. LGMDs represent a group of distinct genetic neuromuscular diseases with a generally common set of symptoms, including progressive, debilitating weakness and wasting that begins in muscles around the hips and shoulders before progressing to muscles in the arms and legs. Many LGMD sub-types are life-limiting and often life-ending diseases. Also known as calpainopathy, LGMD2A is caused by mutations in the CAPN-3 gene and is the most common type of LGMD, accounting for almost a third of cases. Like Sarepta’s micro-dystrophin and five other LGMD programs, the LGMD2A program employs the AAVrh74 vector, designed to deliver treatment to skeletal muscle, including the diaphragm, without crossing the blood brain barrier. The CAPN-3 program is currently in pre-clinical trials.

https://thefly.com/landingPageNews.php?id=2905761