BioMarin: EC OKs Palynziq (pegvaliase injection) for Phenylketonuria

BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) today announced that the European Commission (EC) has granted marketing authorization for Palynziq® (pegvaliase injection) at doses of up to 60 mg once daily, to reduce blood phenylalanine (Phe) concentrations in patients with phenylketonuria (PKU) aged 16 and older, who have inadequate blood Phe control (blood Phe levels greater than 600 micromol/L) despite prior management with available treatment options. Palynziq, a PEGylated recombinant phenylalanine ammonia lyase enzyme, is the first enzyme substitution therapy approved in Europe to target the underlying cause of PKU by helping the body to break down Phe.  In addition, the EC acknowledged that the Phase 3 trial and extension study is suggestive of an improvement in inattention and mood symptoms.

On March 1, 2019 BioMarin announced that the Committee for Medicinal Products for Human Use (CHMP), the scientific committee of the European Medicines Agency (EMA), adopted a positive opinion for the company’s Marketing Authorization Application (MAA) for Palynziq. This is BioMarin’s second approved treatment for this rare genetic disease.

https://www.marketscreener.com/BIOMARIN-PHARMACEUTICAL-I-8587/news/BioMarin-Pharmaceutical-European-Commission-Approves-Palynziq-pegvaliase-injection-for-Treatmen-28544450/

Bruker price target raised to $46 from $39 at Needham

Needham analyst Stephen Unger raised his price target on Bruker to $46 and kept his Strong Buy rating after its Q1 earnings beat, also raising his FY19 EPS view to $1.60 from $1.56 on the expected contribution of its RAVE LLC acquisition. The analyst adds that he had some reservations around Bruker’s growth in Asia-Pacific heading into the print, but the segment generated 19.9% revenue growth to $152M, above his forecasted $132M.

https://thefly.com/landingPageNews.php?id=2903850

Lundbeck Acquires Abide Therapeutics for $250M, Gains San Diego Discovery Site

With a $250 million cash down payment, Denmark’s H. Lundbeck A/S snapped up San Diego-based Abide Therapeutics to gain a novel discovery platform and a U.S.-based research hub.

Deborah Dunsire, president and chief executive officer of Lundbeck said the acquisition of Abide provides the company with a “differentiated chemo-proteomic platform to discover new classes of drugs for a broad spectrum of brain diseases starting with those that harness the therapeutic potential of the endocannabinoid system.” Dunsire said Abide’s research and development program provides both an immediate and long-term boost to its pipeline. Bringing Abide under the Lundbeck portfolio puts the Danish company in a position to deliver multiple new and transformative treatments for brain diseases, Dunsire said in a statement.

In its announcement, Lundbeck did not provide details regarding the 40 employees at Abide, including its leadership team. Kevin Finney, who joined Abide in January as president and chief operating officer, told Xconomy that the company had actually been in the midst of a financing round and eying an initial public offering when the Lundbeck offer came along. The deal was too good to pass up, Finney said, according to the report.

Abide Chief Executive Officer Alan Ezekowitz noted Lundbeck’s commitment to developing therapies for the brain and said it was that expertise that convinced the company’s board of directors to support the deal. Lundbeck, he said, “was the best way to attain Abide’s goal to develop novel therapeutics that make a fundamental difference in the lives of patients with a range of neurological and mood disorders.” Ezekowitz added that Lundbeck’s support for the La Jolla discovery site means that we can continue to leverage the insights of co-founder Ben Cravatt’s laboratory at Scripps Research and maintain its “outstanding discovery team.”

What attracted Lundbeck to acquiring Abide is that company’s lead molecule, ABX-1431. Abide’s ABX-1431 is a potent selective inhibitor of the serine hydrolase monoacylglycerol lipase (MGLL). ABX-1431 is designed to increase the power of endocannabinoid signaling to restore homeostatic balance in the central nervous system. The company believes it has the potential to address multiple indications in psychiatry and neurology. ABX-1431 is currently in an exploratory Phase IIa trial for the treatment of Tourette syndrome and a Phase I trial for neuropathic pain. In addition to the clinical and pre-clinical programs targeting MGLL, Abide has a rich pipeline of inhibitors targeting other serine hydrolases that may be pursued as future novel treatments.

In addition to the $250 million paid upfront to Abide, Lundbeck will also pay an additional $150 million in future development and sales milestones. After the deal closes, Abide’s laboratory in La Jolla will become a U.S. drug discovery hub for Lundbeck. Lundbeck is covering the costs of the acquisition of Abide from its existing cash reserves. The transaction is expected to close during the second quarter of 2019. Lundbeck said the deal will not have any impact on Lundbeck’s 2019 financial guidance range provided in February 2019.

https://www.biospace.com/article/denmark-s-h-lundbeck-acquires-abide-therapeutics-gains-u-s-discovery-site/

GlycoMimetics completes enrollment for Phase 3 trial evaluating rivipansel

GlycoMimetics  (GLYC) announced that patient enrollment has been completed in the Phase 3 RESET clinical trial, which is evaluating the efficacy and safety of rivipansel for the acute treatment of vaso-occlusive crisis in sickle cell disease. In 2011, GlycoMimetics and Pfizer Inc. (PFE) entered into a worldwide license agreement for the development and, if approved by applicable regulatory authorities, commercialization of rivipansel. Since completion of the Phase 2 clinical trial, Pfizer, has been responsible for clinical development of rivipansel, including the RESET clinical trial. Under the Company’s license agreement, GlycoMimetics is eligible to receive payments of up to $80M upon the achievement of specified development milestones, up to $70 million upon the achievement of specified regulatory milestones, and up to $135M upon the achievement of specified levels of annual net sales of licensed products. GlycoMimetics is also eligible to receive tiered royalties, with percentages ranging from the low double digits to the low teens, based on net sales of rivipansel worldwide
https://thefly.com/landingPageNews.php?id=2903811

Ligand Acquires Milestone and Royalty Rights to SB206 from Novan

Ligand Pharmaceuticals Inc. (NASDAQ: LGND) announces the acquisition of economic rights to SB206 from Novan, Inc. SB206 is a Phase 3 topical antiviral gel for the treatment of skin infections, including molluscum contagiosum. Ligand will pay $12 million to Novan and in return will be entitled to receive a tiered royalty of 7% to 10%, as well as up to $20 million in regulatory and commercial milestones. Novan is responsible for all expenses to develop or commercialize SB206, and will use 100% of the proceeds from this transaction in the development and pursuit of regulatory approval for SB206.

“This deal enables Ligand to participate in the potential future revenue of a promising Phase 3 stage asset with a potential commercial launch in 2021. The drug candidate targets a condition mostly affecting children and with a significant unmet medical need,” said John Higgins, Chief Executive Officer of Ligand. “We believe Novan is a well-capitalized company with an experienced management team. This deal adds an attractive late-stage asset to our growing list of partnered programs that could be approved and launch within the next three years.”

Novan is developing SB206 as a nitric-oxide-based topical gel for the treatment of viral skin infections, with a current focus on the treatment of molluscum contagiosum, a contagious skin infection caused by the molluscipoxvirus. There are currently no therapies approved by the U.S. Food and Drug Administration (FDA) for the treatment of molluscum. Nitric oxide has diverse biological activity within the body, affecting the immune, cardio/pulmonary and neurological systems, and depending on dose and release kinetics, nitric oxide can have agonistic or antagonistic effects. The role and mechanics of nitric oxide have been well researched. Novan’s technology platform is the first macromolecular platform to achieve stable, tunable and druggable delivery of nitric oxide. SB206, if approved, could be a topical, at-home, caregiver-applied therapy with a rapid treatment benefit. Novan’s issued U.S. and foreign patents and pending U.S. and foreign patent applications, if issued, relating to SB206 are projected to expire between 2026 and 2034.

https://www.biospace.com/article/releases/ligand-acquires-milestone-and-royalty-rights-to-sb206-from-novan-inc-/

Intermolecular (IMI) to be acquired by Merck KGaA for $1.20/share

Intermolecular, Inc. (NASDAQ: IMI) has signed a definitive agreement pursuant to which a wholly owned subsidiary of Merck KGaA, Darmstadt, Germany, a leading science and technology company, will acquire Intermolecular for $1.20 per share in an all cash transaction, representing an equity value of Intermolecular of approximately $62 million. The acquisition has been unanimously approved by Intermolecular’s Board of Directors and the Executive Board of Merck KGaA, Darmstadt, Germany.

“We are pleased to become an integral part of Merck KGaA, Darmstadt, Germany’s leading electronic materials business and look forward to all of the new and exciting opportunities we see for our customers and employees. We believe our technology expertise is very complementary and creates a unique offering that will continue to shape the innovations of tomorrow,” said Chris Kramer, President and Chief Executive Officer of Intermolecular.

“Intermolecular’s unique capabilities in rapid material screening, in combination with the R&D pipeline of Merck KGaA, Darmstadt, Germany, will allow us to offer our customers faster materials innovation, through parallel composition experiment and full performance testing and characterization,” said Kai Beckmann, member of the Merck KGaA, Darmstadt, Germany, Executive Board and CEO of Performance Materials. “We are excited to join forces with Intermolecular and bring significant advantages to our customers compared to conventional materials R&D.”

The transaction is expected to close in the second half of 2019, subject to the approval of Intermolecular’s stockholders, clearance by the Committee on Foreign Investment in the United States (CFIUS) and the satisfaction of other customary closing conditions.

Merck KGaA, Darmstadt, Germany, will acquire Intermolecular through its wholly owned subsidiary EMD Group Holding II, Inc.

As a result of this transaction, Intermolecular will not be holding its previously scheduled conference call on May 14, 2019.

Cowen served as exclusive financial advisor to Intermolecular and Latham & Watkins LLP served as Intermolecular’s legal advisor. Nixon Peabody LLP served as Merck KGaA, Darmstadt, Germany’s legal advisor.

https://www.streetinsider.com/Corporate+News/Intermolecular+%28IMI%29+to+be+acquired+by+Merck+KGaA+for+%241.20share/15456334.html

Savara receives fast track designation from FDA for Molgradex

Savara announced that the U.S. Food and Drug Administration granted Fast Track designation for Molgradex, an inhaled formulation of recombinant human granulocyte-macrophage colony-stimulating factor. Molgradex, the Company’s lead product candidate, is being investigated in a pivotal Phase 3 study, called IMPALA, for the treatment of autoimmune pulmonary alveolar proteinosis. Topline results from the study are expected in June 2019. Positive results would facilitate the submission of a Biologics License Application in the first half of 2020, with an anticipated commercial launch later in 2020 or early 2021.
https://thefly.com/landingPageNews.php?id=2903653