GSK, AZN 1st To Use NVDA’s Supercomputer, IOVA To Delay BLA Filing, YMAB Plunges, GLYC On Watch
Today’s Daily Dose brings you news about Eidos’ merger with BridgeBio, the first pharmaceutical companies that will make use of the most powerful supercomputer, known as “Cambridge-1”, the FDA granting Rare Pediatric Disease Designation to GlycoMimetics’ sickle cell disease drug candidate, the delay in filing of Lifileucel BLA by Iovance, disappointing regulatory update related to Y-mAbs Therapeutics’ investigational drug Omburtamab and Miragen’s decision to discontinue further internal development of cancer drug candidate Cobomarsen.
1. Eidos to merge with BridgeBio
BridgeBio Pharma Inc. (BBIO), which already owns 63.7% of the outstanding shares of Eidos Therapeutics Inc. (EIDX), has agreed to acquire the remaining shares of Eidos for $73.26 per share in cash, which equates to an aggregate of $175 million of cash or 1.85 shares of BridgeBio common stock per Eidos share.
The transaction is expected to close in the first quarter of 2021, and upon closing, Eidos will become a wholly-owned subsidiary of BridgeBio. Eidos’ common stock will cease trading independently on The Nasdaq Global Select Market.
The lead drug candidate of Eidos is AG10, which is under a pivotal global phase III trial in patients with transthyretin (TTR) amyloid cardiomyopathy, dubbed ATTRibute-CM. Topline results from Part A of the trial are expected in late 2021 or early 2022 and from Part B in 2023.
We had alerted readers to EIDX on August 23, 2019, when it was trading around $45. (Report titled “On track to make its mark…”)
EIDX closed Monday’s trading at $73.51, up 41.58%.
2. GlaxoSmithKline, AstraZeneca to Use Supercomputer for Research
GlaxoSmithKline plc (GSK) and AstraZeneca PLC (AZN) will be among the first pharmaceutical companies that will make use of the most powerful supercomputer, known as “Cambridge-1”, for research and for addressing medical challenges, including those presented by COVID-19.
The Cambridge-1 supercomputer, developed by NVIDIA Corp. (NVDA), is ranked No. 29 on the latest TOP500 list of the world’s most powerful supercomputers and is listed among the world’s top 3 most energy-efficient supercomputers on the current Green500 list.
The supercomputer, which is expected to come online by the end of this year, features pre-trained artificial intelligence models and application-specific frameworks to help researchers define the next generation of drug discovery processes, from finding targets to building compounds to developing responses.
GSK closed Monday’s trading at $37.69, up 1.21%.
3. GlycoMimetics’ SCD Candidate Gets Rare Pediatric Disease Designation
Shares of GlycoMimetics Inc. (GLYC) jumped over 15% in after-hours Monday, on news of the FDA granting Rare Pediatric Disease designation to the company’s investigational drug Rivipansel for the treatment of sickle cell disease in patients 18 years old and younger.
The company had a partnership with Pfizer (PFE) for the development of Rivipansel that was inked in 2011. But in February of this year, Pfizer terminated the deal after a pivotal trial of Rivipansel in patients aged six and older with sickle cell disease who were hospitalized for a vaso-occlusive crisis, dubbed RESET, failed to meet its primary and key secondary efficacy endpoints. With Pfizer walking out of the partnership, GlycoMimetics regained rights to Rivipansel.
GlycoMimetics said it is “exploring options to move forward in sickle cell disease, including discussions with the FDA as to whether there is a regulatory path to approval”
The FDA defines a “rare pediatric disease” as a serious or life-threatening rare disease in which the serious or life-threatening manifestations primarily affect individuals aged from birth to 18 years. Under the FDA’s Rare Pediatric Disease Priority Review Voucher program, a company that receives an initial approval for a drug or biologic for a “rare pediatric disease” may qualify for a voucher that can be redeemed to receive a priority review of a subsequent marketing application for a different product.
GLYC closed Monday’s trading at $3.27, up 4.47%. In after-hours, the stock was up 15.90% at $3.79.
4. Iovance Sees Delay in Filing Lifileucel BLA
Shares of Iovance Biotherapeutics Inc. (IOVA) were down over 20% in extended trading Monday, following news that the company now expects to submit its Biologics License Application for Lifileucel as a treatment for metastatic melanoma only in 2021 and not by the end of 2020 as previously expected.
The company was hoping that clinical data from its pivotal trial of Lifileucel in patients with metastatic melanoma, dubbed C-144-01 trial, will be enough to support the Biologics License Application submission.
Lifileuce is a tumor-infiltrating lymphocyte (TIL) therapy, which is a first-in-class, one-time administration cell therapy targeting solid tumors.
In a recent Type B meeting, regarding the requirements and timing of certain information that would be provided as part of the BLA submission, the company and the FDA have not been able to agree on the required potency assays to fully define its TIL therapy.
IOVA closed Monday’s trading at $32.06, up 1.78%. In after-hours, the stock fell 20.62% to $25.45.
5. Miragen Throws In the Towel on Cobomarsen
Shares of Miragen Therapeutics Inc. (MGEN) were down over 20% in extended trading Monday on news that the company is discontinuing further internal development of Cobomarsen, its clinical product candidate for the treatment of certain cancers.
An internal review of preliminary topline data from the company’s phase II clinical trial of Cobomarsen in patients with Cutaneous T-Cell Lymphoma, dubbed SOLAR, suggest that Cobomarsen lacks a compelling result for the study’s primary endpoint, i.e., objective skin response of at least four months duration (ORR4).
The SOLAR study was designed to evaluate the safety and efficacy of Cobomarsen given by intravenous infusion in an active control comparison trial for patients with the mycosis fungoides subtype of CTCL. In December 2019, the company announced it would halt enrollment in the SOLAR trial well short of the intended 126 CTCL patients in order to reduce the time and resource expenditure to evaluate the drug’s potential. The downsized SOLAR study is not statistically powered for superiority or equivalence.
MGEN closed Monday’s trading at $0.76, up 2.72%. In after-hours, the stock was down 22.37% to $0.59.
6. FDA Sends Y-mAbs Back to Drawing Board
Shares of Y-mAbs Therapeutics Inc. (YMAB) plunged more than 18% in extended trading Monday, following a disappointing regulatory update related to its investigational drug Omburtamab.
The company completed the submission of its Biologics License Application for Omburtamab, proposed for the treatment of pediatric patients with CNS/leptomeningeal metastasis from neuroblastoma in August this year.
Upon preliminary review, the FDA has found that certain parts of the Chemistry, Manufacturing, and Control (“CMC”) module and the Clinical module of the BLA require further detail.
So, the FDA has refused to accept for review the Omburtamab BLA and has issued a Refusal to File letter for Y-mAbs Therapeutics.
The company has decided to request a Type A meeting with the FDA as soon as possible and is targeting the resubmission of the amended BLA before the end of 2020.
YMAB closed Monday’s trading at $41.70, up 7.2%. In after-hours, the stock was down 18.59% at $33.95.
7. Stocks That Moved On No News
Enlivex Therapeutics Ltd. (ENLV) closed Monday’s trading at $14.30, up 38.16%.
ProPhase Labs Inc. (PRPH) closed Monday’s trading at $6.89, up 33.79%.
Humanigen Inc. (HGEN) closed Monday’s trading at $12.14, up 23.25%.
AnPac Bio-Medical Science Co., Ltd. (ANPC) closed Monday’s trading at $3.41, down 11.43%.
Repare Therapeutics Inc. (RPTX) closed Monday’s trading at $27.30, down 9%.
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