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Ascendis Pharma files for late-stage study with TransCon in Japan

Ascendis Pharma (NASDAQ:ASND) has filed a Clinical Trial Notification with the Pharmaceuticals and Medical Devices Agency (PMDA) in Japan seeking approval to initiate Phase 3 riGHt Trial of TransCon hGH (lonapegsomatropin), a long-acting prodrug of somatropin (hGH), for the treatment for pediatric growth hormone deficiency (GHD).

The 40-subject trial will evaluate and compare the annualized height velocity in prepubertal treatment naïve children with GHD, treated with weekly TransCon hGH to that of a commercially available daily hGH formulation, at 52 weeks.

Relay Therapeutics rallies 7% on EcoR1 pitch

Relay Therapeutics (RLAY +7.3%) is up, albeit on below-average volume, in apparent response to a presentation on the company by fundamental shop EcoR1 Capital at the Sohn Conference.

The company debuted on July 16 at $20. Shares are currently exchanging hands at $47.13, up 136%.

It develops small molecule therapies leveraging its know-how in protein motion. Its Dynamo platform, which integrates experimental and computational approaches, enables it to more effectively identify previously undruggable protein targets.

Lead candidates are RLY-1971, an inhibitor of a protein called SHP2 (Src homology region 2 domain-containing phosphatase-2), and RLY-4008, an inhibitor of a protein called fibroblast growth factor receptor 2 (FGFR2), both in Phase 1 development for the potential treatment of solid tumors.

GoodRx jumps on IPO, +50% after pricing above range

Telehealth company GoodRX (GDRX, +48.5%) is surging on its first day of trading.

Shares opened at $46, well ahead of the IPO price of $33/share, which was ahead of the expected price range of $24-$28/share.

GoodRx raised about $772M with its offering.

Private equity investor Silver Lake owned about 35.3% of GoodRx before the offering.

“The firm is primarily an advertising revenue company that is seeking to expand into other revenue streams,” Donovan Jones wrote on Seeking Alpha. “While I suspect that GDRX will be successful over the medium term in doing so, it is still subject to the vagaries of advertising rates. The question I have is whether an advertising funded business model should command an EV/Revenue multiple of 24x.”

The stock is trading around 37x EV/revenue at current levels.

Veeva partners with RedHill Bio to advance Opaganib in COVID-19

Veeva Systems (NYSE:VEEV) and Bioforum collaborates with RedHill Biopharma (NASDAQ:RDHL) on a global Phase 2/3 clinical study evaluating opaganib (Yeliva, ABC294640), an orally-administered, sphingosine kinase-2 (SK2) selective inhibitor, in patients hospitalized with severe COVID-19 pneumonia requiring supplemental oxygen.

The opaganib study is set to enroll up to 270 patients. To support the study, RedHill has adopted Veeva Vault CDMS, a modern cloud platform for electronic data capture, coding, data cleaning, and reporting.

Bioforum, a global data-focused contract research organization has been selected to implement and configure Vault CDMS for this COVID-19 study.

“Our immediate goal is to compile clinical data as early as the fourth quarter of 2020 to support potential applications for emergency use authorizations of opaganib,” said Aida Bibliowicz, vice president of clinical affairs at RedHill.

Humanigen in manufacturing deal with Thermo Fisher on lenzilumab

Humanigen (NASDAQ:HGEN) will collaborate with Thermo Fisher Scientific (NYSE:TMO) to expand the manufacturing capacity for lenzilumab, for use in COVID-19, to support a potential Emergency Use Authorization (EUA) application.

“Manufacturing preparation, precision and expertise are critical as we execute on an aggressive strategy, pending a potential EUA from the FDA, to deliver a COVID-19 therapeutic this winter that has the potential to reduce the risk of ventilation or death and send patients home from the hospital earlier,” said Cameron Durrant, MD, MBA, CEO.

Thermo Fisher will begin the technical transfer of the lenzilumab bulk drug substance process and commercial scale production could begin before year-end.

GoodRx Holdings valued at $12.7B after pricing IPO above expected range

California-based telehealth company, GoodRx Holdings (NASDAQ:GDRX) has priced its IPO at $33, well above expected price range of $24 – $28 per share.

The company will raise $772.9M through selling 23.4M shares, and selling stockholders to sell 11.2M.

Private equity investor Silver Lake owned about 35.3% of GoodRx before the offering.

MacroGenics’ MGC018 shows encouraging action in treatment of solid tumors

MacroGenics (MGNXannounces the development of MGC018, an investigational antibody-drug conjugate (ADC) targeting B7-H3 for the treatment of solid tumors in a journal of the American Association for Cancer Research.

Preclinical studies showed that MGC018 mediated specific in vitro killing across a range of B7-H3-expressing solid tumor cell types. Furthermore, MGC018 mediated bystander in vitro killing of B7-H3-negative tumor cells in the presence of B7-H3-positive tumor cells was observed.

MGC018 displayed potent antitumor activity in preclinical tumor xenograft models of breast, ovarian and lung cancer, as well as melanoma.

Additionally, antitumor activity was observed toward patient-derived tumor xenograft models of breast, prostate and head and neck cancer displaying heterogeneous expression of B7-H3.

Importantly, MGC018 exhibited an acceptable pharmacokinetic and safety profile in cynomolgus monkeys following repeat-dose administration.

“With its overexpression on a wide range of solid cancers but limited presence on normal tissues, B7-H3 is an attractive candidate for an ADC-targeting approach,” said Deryk Loo, Ph.D., Senior Director of Research at MacroGenics and the lead author of the paper.

UK to host first human challenge studies for COVID-19 vaccines

The Financial Times reports that the UK will host the world’s first human COVID-19 challenge trials, in which healthy volunteers are deliberately infected with coronavirus, in order to evaluate the effectiveness of experimental vaccines.

The government-funded studies should launch in January 2021 at a 24-bed secure quarantine facility in east London.

About 2,000 potential UK volunteers have signed up through an advocacy group called 1Day Sooner which campaigns for COVID-19 infection trials aimed at accelerating development which are most useful when the viral infection rate is low. About 37K people globally have enlisted.

The group is launching a campaign this week to petition parliament for funds for a biocontainment facility with sufficient capacity to quarantine up to 200 people.

Volunteers will receive up to £3,750 for participating but may earn more considering the length of isolation, potentially as long as a month.

One key aspect of the studies is to select and purify a strain of the coronavirus that generally represents the SARS-CoV-2 strain currently circulating in the overall population.

Also, investigators will have a rescue remedy on hand to prevent serious illness in subjects. The London trial will initially use Gilead Sciences’ (GILD +0.3%) Veklury (remdesivir).

In the U.S., NIH has awarded a $3.6M contract to Colorado State University to support the manufacture of two coronavirus strains that could be used in challenge studies. NIH is currently investigating the technical and ethical aspects for such trials.

Selected tickers: Johnson & Johnson (JNJ +1.0%), Moderna (MRNA -3.3%), Pfizer (PFE -0.1%), BioNTech SE (BNTX -3.6%), Sanofi (SNY +1.4%), GlaxoSmithKline (GSK +1.0%), AstraZeneca (AZN +1.4%)

Ligand forecasts big jump in Captisol material sales in 2021

Ligand Pharmaceuticals (LGND +5.0%) expects Captisol material sales of ~$200M in 2021 compared to its 2020 full-year expectation of $90M.

The company said “recent new contracting with partners and investments in manufacturing capacity have contributed to its Captisol business operating at the highest levels in the history of the technology and position Captisol for major growth.”

120+ Captisol research use agreements and 8 clinical and/or commercial license agreements this year, is more contracts than any other year, the company says.

LGND facilitated installation of equipment to allow production at significantly higher levels to support anticipated demand, as well as added processing capacity for Captisol in both the U.S. and England.

Its pivotal trial for Captisol-enabled Iohexol (CE- Iohexol) is planned to initiate in December; CE-Iohexol is an iodine-based contrast agent for hospital-based imaging procedures.

Captisol is a chemically modified cyclodextrin with a structure designed to optimize the solubility and stability of drugs.

Dr. Reddy’s says Avigan effective against COVID-19 symptoms

Healthcare solutions provider Global Response Aid (GRA) and Dr. Reddy’s Lab (RDY) say that the anti-viral drug Avigan demonstrated ‘promising’ results against COVID-19, from a Phase 3 study conducted in Japan with the sponsorship of FujiFilm Toyama Chemical.

Analysis from 156 hospitalized patients showed that patients who received Avigan recovered from COVID-19 symptoms 2.8 days earlier, on average, compared with the control group.

Avigan, contains the active ingredient Favipiravir, was developed by FujiFilm Toyama Chemical as an anti-influenza drug. It is undergoing testing in the U.S. in a Phase 2 study involving initially hospitalized COVID-19 patients, sponsored by FujiFilm Toyama Chemical, as well as an investigator-initiated Phase 2 study in mild or asymptomatic COVID-19 subjects conducted by the Stanford University School of Medicine.

Avigan Tablet was approved in Japan in 2014 as an influenza anti-viral drug.

GRA, Dr. Reddy’s, and FujiFilm Toyama recently entered into a licensing agreement for Avigan.