Amicus announced the dosing of the first patient in a global phase 3 clinical study of AT-GAA in adult patients with late onset Pompe disease. PROPEL is a 52-week, double-blind randomized study designed to assess the efficacy, safety and tolerability of AT-GAA compared to the current standard of care, alglucosidase alfa, an enzyme replacement therapy. All participants randomized to AT-GAA in the PROPEL study will receive drug manufactured at the 1000L scale intended for clinical and commercial supply. Amicus also expects to initiate a smaller, open-label study of AT-GAA in pediatric patients in 2019.
Thursday, December 20, 2018
Spectrum selloff yesterday a buying opportunity, says H.C. Wainwright
H.C. Wainwright analyst Edward White views yesterday’s weakness in shares of Spectrum Pharmaceuticals as a “good buying opportunity.” The analyst points out that Breakthrough Therapy Designation is given to drugs intended to treat a serious condition that may demonstrate substantial improvement over current treatment, with “substantial” being a subjective FDA judgment. While not getting the BTD for poziotinib is disappointing, this should not change the timeline for the Biologics License Application submission or ultimately the possible approval of poziotinib, White tells investors in a research note. He keeps a Buy rating on Spectrum with a $40 price target.
Gilead, Agenus enter into immuno-oncology partnership
Gilead (GILD) and Agenus (AGEN) have entered into an immuno-oncology partnership focused on the development and commercialization of up to five novel immuno-oncology therapies. Agenus will receive $150M upon closing, which includes a $120M upfront cash payment and a $30M equity investment.The agreement also includes approximately $1.7B in potential future fees and milestones. Gilead will receive worldwide exclusive rights to AGEN1423, which has an estimated IND filing by year-end 2018. Gilead will also receive the exclusive option to license two additional programs: AGEN1223 and AGEN2373. Agenus has filed the IND for AGEN1223 and has a planned IND filing for AGEN2373 in 1H19. Agenus will be responsible for developing the option programs up to the option decision points, at which time Gilead may acquire exclusive rights to the programs on option exercise. For one of the option programs, Agenus will have the right to opt-in to shared development and commercialization in the U.S. Gilead will also receive right of first negotiation for two additional, undisclosed preclinical programs.
Apellis expects to restart enrollment in DERBY, OAKS trials in 2Q19
Apellis provided an update on the status of its Phase 3 program for APL-2 in patients with Geographic Atrophy, or GA. Apellis expects to be able to restart enrollment of its two Phase 3 GA trials in Q2 2019 and to have fully enrolled both studies by the end of Q1 2020, within the originally planned timeline for completion. As previously disclosed, the company voluntarily implemented a temporary pause in dosing in the DERBY and OAKS Phase 3 trials due to observed cases of non-infectious inflammation in patients treated from a single manufacturing lot of APL-2 intravitreal drug product. The company believes the source of inflammation in the Phase 3 trials resides in an impurity or contaminant in the active pharmaceutical ingredient, or API. Inflammation in all affected patients has resolved. In the Phase 2 FILLY trial, where more than 1,500 intravitreal injections of APL-2 were given, APL-2 was well-tolerated with only a single case of non-infectious inflammation reported. The source of inflammation has had no known impact on the studies utilizing systemic administration of APL-2. Based on Apellis’ investigation, which included a full manufacturing process review, six non-clinical studies in two species and an ongoing Phase 1b clinical study in patients with GA with low vision, the company does not believe that the following were the root causes for the observed inflammation in the Phase 3 GA program: the pharmacology or chemical composition of APL-2, a formulation change that was implemented between the Phase 2 FILLY trial and the Phase 3 trials, and the fill and finish process for the APL-2 intravitreal drug product. In order to resume the Phase 3 GA program in a safe and expeditious manner, the company has introduced improvements to the manufacturing process in order to eliminate impurities and potential contaminants like those that are believed to have caused inflammation in the Phase 3 trials. A new lot of API incorporating these improvements has been manufactured and is ready for release in a quantity sufficient to complete both the DERBY and OAKS Phase 3 clinical trials. The company believes that the improved manufacturing process can be used to supply API at a scale required for global commercialization. In non-clinical testing, treatment with API manufactured through the improved manufacturing process did not cause inflammation. Finally, prior to introduction in the Phase 3 clinical trials, new lots of API may first be introduced in a small Phase 1b study of patients with GA with low vision.
Aquestive Therapeutics, Indivior filing petition for rehearing on CAFC ruling
Aquestive Therapeutics, Indivior filing petition for rehearing on CAFC ruling Aquestive (AQST) and (INVVY) are filing a petition with the U.S. Court of Appeals for the Federal Circuit for a rehearing by the original panel of judges, as well as a rehearing en banc, on the CAFC’s ruling to vacate the preliminary injunction granted by the U.S. District Court of New Jersey to enjoin Dr. Reddy’s Laboratories (RDY) from launching its generic buprenorphine/naloxone sublingual film product, which utilizes Aquestive’s PharmFilm technology. The PI will remain in place to enjoin DRL’s “at-risk” entry into the U.S. market, until the CAFC rules on the joint petition. This decision will have no impact on the core Aquestive patent portfolio. Aquestive’s Suboxone revenues are driven by order volume from both branded and authorized generic products. Aquestive has a sole and exclusive worldwide manufacturing agreement with Indivior for both branded and authorized generic Suboxone and will continue to fulfill non-U.S. orders, which also continue to grow as a percent of total Suboxone business.
Nabriva Therapeutics submits two NDAs to FDA for lefamulin
Nabriva Therapeutics announced the submission of two new drug applications, or NDAs, to the FDA for the oral and intravenous, or IV, formulations of lefamulin semi-synthetic pleuromutilin antibiotic, for the treatment of community-acquired bacterial pneumonia, or CABP. Both formulations of lefamulin were granted qualified infectious disease product and fast track designation by the FDA, enabling potential priority review of the NDAs by the FDA. Nabriva Therapeutics plans to submit a marketing authorization application for lefamulin in Europe in Q1 of 2019.
Merck KGaA assigns CAR-T rights to Intrexon
Merck KGaA (MKGAY) announced that through its wholly owned subsidiary, Ares Trading, it has evolved its agreement with Intrexon Corporation (XON) for the development of Chimeric Antigen Receptor T-cell therapies, genetically engineered T-cells with synthetic receptors that recognize a specific antigen expressed on tumor cells. The agreement with Intrexon and its wholly-owned subsidiary, Precigen, enables Merck KGaA, Darmstadt, to continue to implement its focused R&D strategy, while maintaining an investment in the future potential of next-generation CAR-T development. Under the terms of the agreement, Merck KGaA will assign its exclusive CAR-T development rights to Intrexon. Merck KGaA will receive shares of Intrexon common stock valued at $150M in exchange for assigning Intrexon its CAR-T rights. Merck KGaA first entered into a collaboration and license agreement with Intrexon in 2015 to develop and commercialize CAR-T cancer therapies utilizing Intrexon’s proprietary RheoSwitch Therapeutic System and the Sleeping Beauty non-viral gene integration technology. The combination of these platforms enables regulation of gene expression and delivery with a non-viral approach and preclinical data indicate the potential to improve therapeutic safety and facilitate shortened manufacturing to improve time-to-treatment. As of December 31, 2017, these rights were considered intangible assets not yet available for use with a carrying amount of EURO$104M. In addition to receiving $150M of Intrexon common stock, this agreement also includes a further $25M investment in Intrexon. In return, Merck KGaA will receive a $25M convertible note, providing the option to receive either Precigen or Intrexon stock. The closing of the transactions contemplated by the agreement is subject to customary closing conditions, including the expiration of the waiting period under the Hart-Scott-Rodino Antitrust Improvements Act.
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