News

Dec 10 2018

Biohaven Announces Positive Results from Ongoing Rimegepant Long-Term Safety Study

Dec 10 2018

NEW HAVEN, Conn., Dec. 10, 2018 /PRNewswire/ — Biohaven Pharmaceutical Holding Company Ltd. (NYSE: BHVN) today announced initial positive results from its ongoing long-term, open-label safety study of its oral calcitonin gene-related peptide (CGRP) receptor antagonist, rimegepant, for the acute treatment of migraine. Based upon this interim analysis (database cutoff of November 21, 2018) of Study BHV3000-201 (NCT03266588 or “Study 201”) it appears that rimegepant may be safe and well tolerated with long-term dosing in patients with migraine. In this study, patients are allowed to treat migraine attacks of all severities (mild to severe) up to once daily for up to a full year.

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Dec 3 2018

Biohaven Delivers Positive Phase 3 Results with Rimegepant Zydis® Orally Dissolving Tablet (ODT): Rapid and Lasting Benefit for the Acute Treatment of Migraine

Dec 3 2018

NEW HAVEN, Conn., Dec. 3, 2018 /PRNewswire/ — Biohaven Pharmaceutical Holding Company Ltd. (NYSE: BHVN) today announced positive topline results from a randomized, controlled Phase 3 clinical trial (BHV3000-303 or Study 303) evaluating the efficacy and safety of its Zydis® orally dissolving tablet (ODT) formulation of rimegepant, an oral calcitonin gene-related peptide (CGRP) receptor antagonist, for the acute treatment of migraine. In Study 303, rimegepant Zydis ODT statistically differentiated from placebo on the two co-primary endpoints as well as the first 21 consecutive primary and secondary outcome measures that were prespecified in hierarchical testing. Consistent with the two previous Phase 3 clinical trials, Study 303 met its co-primary registrational endpoints of pain freedom and freedom from most bothersome symptom (MBS) at 2 hours using a single dose.

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Nov 26 2018

FDA Approves Vitrakvi® (larotrectinib), the First Ever TRK Inhibitor, for Patients with Advanced Solid Tumors Harboring an NTRK Gene Fusion

Nov 26 2018

STAMFORD, Conn. and WHIPPANY, N.J., Nov. 26, 2018 (GLOBE NEWSWIRE) — The U.S. Food and Drug Administration (FDA) today approved Vitrakvi® (larotrectinib), the first ever oral TRK inhibitor, for the treatment of adult and pediatric patients with solid tumors that have a neurotrophic receptor tyrosine kinase (NTRK) gene fusion without a known acquired resistance mutation, are metastatic or where surgical resection is likely to result in severe morbidity, and have no satisfactory alternative treatments or that have progressed following treatment. This indication is approved under accelerated approval based on overall response rate (ORR) and duration of response (DOR). Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials. Vitrakvi is the first treatment to receive a tumor-agnostic indication at the time of initial FDA approval. In clinical trials of patients with TRK fusion cancer, Vitrakvi demonstrated an ORR of 75 percent (N=55) (95% CI, 61%, 85%), including a 22 percent complete response (CR) rate.

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Nov 20 2018

LEO Pharma and PellePharm Announce $760 Million Collaboration to Advance Innovative Therapies for Rare Skin Diseases

Nov 20 2018

SAN FRANCISCO & BALLERUP, Denmark–(BUSINESS WIRE)–Danish dermatology specialists LEO Pharma and California rare disease pioneers PellePharm today announced a strategic development and commercialization collaboration to address unmet medical needs across various skin diseases with no approved treatments, advancing innovation and access to potential therapies for patients with life-altering conditions, such as Gorlin Syndrome and High Frequency Basal Cell Carcinoma (BCC), two distinct and rare forms of skin cancer.

Under the terms of the agreement, LEO Pharma has initially committed $70 million comprised of equity financing and financial R&D support to fund the global Phase 3 trial for patidegib topical gel 2% for the prevention and treatment of Gorlin Syndrome, with LEO Pharma securing an option to acquire all shares in PellePharm. PellePharm and its stockholders could receive up to an additional $690 million including merger consideration, and regulatory and commercial milestone payments. In addition, PellePharm stockholders are eligible to receive a double-digit royalty after achieving certain commercial milestones.

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Nov 18 2018

Landmark PALISADE Trial of AR101 Published in New England Journal of Medicine

Nov 18 2018

BRISBANE, Calif.–(BUSINESS WIRE)–Nov. 18, 2018– Aimmune Therapeutics, Inc. (Nasdaq:AIMT), a biopharmaceutical company developing treatments for potentially life-threatening food allergies, today announced that the New England Journal of Medicine (NEJM) has published the full results of the landmark phase 3 PALISADE clinical trial of AR101, Aimmune’s investigational biologic oral immunotherapy for desensitization of patients with peanut allergy. PALISADE is the largest and first successful phase 3 peanut allergy immunotherapy trial to date.

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Nov 12 2018

Aimmune Therapeutics Announces Additional Equity Investment by Nestlé Health Science

Nov 12 2018

BRISBANE, Calif.–(BUSINESS WIRE)–Nov. 12, 2018– Aimmune Therapeutics, Inc. (Nasdaq:AIMT), a biopharmaceutical company developing treatments for potentially life-threatening food allergies, today announced that Nestlé Health Science will make an additional equity investment in Aimmune of $98 million, increasing Nestlé Health Science’s ownership of Aimmune to approximately 19 percent.

Aimmune and Nestlé Health Science also entered into a two-year extension of their original two-year strategic collaboration agreement, focused on offering innovative food allergy therapies. The agreement does not contain any partnership, collaboration, or negotiation restrictions on Aimmune. Aimmune retains all rights to its current and future pipeline assets, and Aimmune and Nestlé Health Science will collaborate towards successful development of such assets.

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Nov 10 2018

Eidos Therapeutics Announces Positive Phase 2 Data For AG10 In Symptomatic Patients With Mutant Or Wild-Type TTR Amyloid Cardiomyopathy

Nov 10 2018

SAN FRANCISCO, Nov. 10, 2018 (GLOBE NEWSWIRE) — Eidos Therapeutics, Inc. (Eidos) (Nasdaq:EIDX), today announced positive results of its Phase 2 clinical trial studying AG10 in subjects with symptomatic transthyretin (TTR) amyloidosis cardiomyopathy (ATTR-CM). The data were presented in a late-breaking Featured Science oral presentation at the American Heart Association (AHA) Scientific Sessions. AG10 was well tolerated, demonstrated >90% TTR average stabilization at day 28, and increased serum TTR concentrations, a prognostic indicator of survival in ATTR-CM, in a dose-dependent manner. Subject to discussions with regulatory agencies, these data support the advancement of AG10 into Phase 3 pivotal trials planned to be initiated in the first half of 2019.

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Oct 18 2018

TapImmune and Marker Therapeutics Announce Successful Closing of Previously Announced Merger and Financing

Oct 18 2018

Jacksonville, FL, October 17, 2018 —TapImmune Inc. (NASDAQ: TPIV), today announced the closing of the previously announced merger with privately-held Marker Therapeutics, Inc. In connection with the merger, TapImmune Inc. changed its name to Marker Therapeutics, Inc., and reincorporated from Nevada into Delaware. The combined company will focus on the continued development and commercialization of T cell therapies. Beginning Thursday, October 18, 2018, the Company’s stock will begin trading under the new ticker symbol “MRKR” on the Nasdaq Capital Market and will have a new CUSIP number, 57055L 107.

“The closing of this merger marks a significant milestone, since the combined company is well-positioned to become a leader in cancer immunotherapy, with potentially transformative therapies,” said Peter Hoang, CEO of Marker Therapeutics, Inc. “The combined company will have exponentially superior capabilities and resources than either company had alone. With the transaction completed, we can now push our clinical trials forward more efficiently with the full resources available to the combined company. We are confident that our therapies can fundamentally improve therapeutic outcomes for patients with life-threatening diseases, and drive life-changing results for patients suffering from a variety of terrible cancers.”

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Oct 17 2018

Aeglea BioTherapeutics Announces New Positive Interim Clinical Data and Improvements in Disease Manifestations for Ongoing Phase 1/2 Trial of Pegzilarginase in Patients with Arginase 1 Deficiency

Oct 17 2018

AUSTIN, Texas, Oct. 17, 2018 (GLOBE NEWSWIRE) — Aeglea BioTherapeutics, Inc. (NASDAQ: AGLE), a clinical-stage biotechnology company that designs and develops innovative human enzyme therapeutics for patients with rare genetic diseases and cancer, today announced new positive interim clinical data at the 2018 American Society of Human Genetics (ASHG) Conference from its ongoing Phase 1/2 trial of pegzilarginase, its lead investigational therapy, in patients with the rare genetic disease Arginase 1 Deficiency (ARG1D).

“Our clinical experience with ARG1D and pegzilarginase has advanced rapidly given the accelerated enrollment over the last few months. The developing efficacy profile is compelling, particularly given the short period of repeat dosing,” said Anthony G. Quinn, M.B Ch.B, Ph.D., president and chief executive officer of Aeglea. “We are pleased with the recently granted rare pediatric disease designation and we will continue to collaborate closely with the FDA and EMA on the design of our pivotal trial.”

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