Ocular Therapeutix to Detail Interim Phase 1 Data at Upcoming Presentation

 Ocular Therapeutix™, Inc. (NASDAQ: OCUL), a biopharmaceutical company focused on the formulation, development, and commercialization of innovative therapies for diseases and conditions of the eye, announced its intention to present data from its Phase 1 clinical trial of OTX-TKI, an axitinib intravitreal implant for the treatment of patients with wet age-related macular degeneration (wet AMD) and other retinal diseases, at the upcoming Angiogenesis, Exudations, and Degeneration 2021 Meeting being held virtually on February 12-13th.

Details of Ocular’s Presentation are as follows:

TITLE: Intravitreal Hydrogel-Based Axitinib Implant (OTX-TKI) for the Treatment of Neovascular AMD: Phase 1 Trial Update
PRESENTER: Andrew A. Moshfeghi, MD, MBA, Associate Professor of Clinical Ophthalmology; Medical Director of the USC Roski Eye Institute; Director of Clinical Trials; Director of the Vitreoretinal Surgery Fellowship Program; and Director of the Medical Retina Fellowship Program
PRESENTATION DATE AND TIME: Saturday, February 13, 2021, 12:30 p.m. ET

"We are pleased to be presenting an update on OTX-TKI at the upcoming Angiogenesis meeting," said Michael Goldstein, MD, MBA, President, Ophthalmology and Chief Medical Officer of Ocular Therapeutix. "OTX-TKI is an intravitral implant of axitinib delivered via injection, leveraging a new administration and mechanism of action for the treatment of patients with wet AMD and other retinal diseases. We are particularly encouraged with the interim data presented to date from the Phase 1 trial. The data support the product’s safety profile and signal its potential biological activity and durability in patients with wet AMD across the first two dose cohorts and a portion of the third dose cohort for up to six months or longer in some cases."

The presentation can be accessed February 13th on the "Events and Presentations" section of the Ocular Therapeutix website.

https://finance.yahoo.com/news/ocular-therapeutix-announces-upcoming-presentation-130000650.html

Inovio: Oppenheimer Says Covid Vax Developer Nears Finish Line

 Shares of coronavirus vaccine developer Inovio Pharmaceuticals Inc (NASDAQ:INO) were surging Friday after a vote of confidence from the Street. 

Bullish Analyst, Fund Stake Prop Up Stock: Oppenheimer analyst Hartaj Singh initiated coverage of Inovio shares with an Outperform rating and $35 price target in a Thursday note. 

Although current mRNA vaccines have set a high efficacy bar in Phase 3 settings, INO-4800 has a differentiated profile, with a better safety and tolerability profile; longer shelf-life and easier storage; and potential flexibility to adjust for mutations and boosting, the analyst said.

Inovio's deep expertise and decades-long history in DNA science, he said, could bring the company to the "finish line" soon, in a fashion similar to mRNA-based vaccines.

Singh is also left impressed with the rest of Inovio's pipeline and its execution over the last 12 months.

The analyst highlighted Inovio's unique cancer vaccine candidate INO-5401.

It has intriguing early-stage efficacy in a hard-to-treat cancer, namely glioblastoma, and an HPV-targeted DNA vaccine candidate, codenamed VGX-3100, has a Phase 3 readout scheduled for the first half of 2021 in high-grade cervical dysplasia, Singh said. 

Inovio's stock is set up well, with value-driving catalysts approaching, according to Oppenheimer. 

"In our view, INO-4800 could potentially make INO break-even in 2022, and its newsflow could 'turbo-charge' the platform value growth." 

Separately, 13G filings done in the the last two days showed that State Street Corp had 8.895 million shares in Inovio, giving it a 5.25% stake.

Vanguard Group held about 9.186 million shares, or a 5.42% stake, and Wasatch Advisors beneficially owned 3.93 million shares, or a 2.3% stake in the company.

The Invoio Stock Trajectory: Inovio shares peaked at $33.79 in late June 2020 amid progress in the company's vaccine program. The company began developing a DNA vaccine codenamed INO-4800 against the novel coronavirus in January 2020, with claims of having successfully developed the vaccine construct in three hours after receiving the genetic sequence from Chinese researchers.

Following successful preclinical testing, the company commenced a Phase 1 study in the U.S. in early April. It was followed up by the initiation of CEPI-funded Phase 1/2 study of the vaccine candidate in South Korea in early June.

The company announced positive interim readout from the U.S. Phase 1 study June 30, and the June 26 high was in anticipation of the readout.

The rally began to lose steam thereafter, and a partial clinical hold imposed by the FDA on the start of the Phase 2/3 study on Sept. 28 pressured the stock further. Inovio received the green light to proceed with the Phase 2 part of the trial in mid-November.

After trading at sub-$10 levels for much of January, the stock began gathering some momentum in February.

https://docoh.com/news/benzinga/19641651/inovio-shares-rally-as

Artelo started at Buy by Ladenburg

 Target $7

https://finviz.com/quote.ashx?t=ARTL

Decibel Therapeutics prices upsized IPO at $18, the high end of the range

 Decibel Therapeutics, which is developing preclinical gene therapies for hearing loss and balance disorders, raised $127 million by offering 7.1 million shares at $18, the high end of the range of $16 to $18. The company had originally filed to sell 5.9 million shares. Decibel Therapeutics plans to list on the Nasdaq under the symbol DBTX. Citi, SVB Leerink, BMO Capital Markets and Barclays acted as lead managers on the deal.

Relevant Profile: DBTX

https://www.renaissancecapital.com/IPO-Center/News/77586/Decibel-Therapeutics-prices-upsized-IPO-at-$18-the-high-end-of-the-range

Roche's Eylea rival poised for FDA filings with new phase 3 data

 Roche’s bispecific antibody matched Regeneron’s blockbuster Eylea in four phase 3 studies comparing the two in diabetic macular edema (DME) and wet age-related macular degeneration (AMD). The studies found the experimental drug did no worse than the incumbent despite a majority of the patients taking it going longer between injections.

The company pitted the bispecific antibody faricimab, which targets VEGF and Ang2, against Eylea in two studies involving nearly 600 patients with DME and two studies with more than 600 patients who had wet AMD. All four studies met their primary endpoint, with faricimab matching Eylea at improving vision after one year as measured by Best Corrected Visual Acuity (BCVA), or the best vision a person can achieve wearing glasses or contact lenses. Investigators assessed BCVA using a standard vision testing tool featuring rows of progressively smaller letters.

But that’s not all—about half of the patients across all four trials could go four months between doses of faricimab, compared to the two-month dosing schedule for Eylea. That’s an important distinction for patients whose treatment is injected into the eye.

“Faricimab’s potential to extend time between treatments may benefit those patients who struggle to keep up with the regular physician visits and eye injections needed to preserve their vision,” said Jeffrey Heier, M.D., director of retinal research at Ophthalmic Consultants of Boston, in a statement.

The DME trials tested faricimab in two patient groups: those who received it every two months or those who could choose their treatment intervals of every one, two, three or four months. The wet AMD trials dosed patients every two, three or four months, depending on their disease activity.

About half of the DME patients and about 45% of the wet AMD patients could be treated with faricimab every four months, while about one-fifth of DME patients and one-third of the wet AMD patients could be dosed every three months. Their vision improvement was on par with that seen in the Eylea groups, Roche said. However, the company did not separate figures for individual dosing regimens, so the vision improvement seen in the studies also include results from patients being treated at shorter intervals.

Roche will present results from the studies Saturday at Angiogenesis, Exudation, and Degeneration 2021, a medical symposium presented by Bascom Palmer Eye Institute of the University of Miami Miller School of Medicine. It will submit results from all four studies to health authorities around the world, including the U.S. and EU, for approvals in DME and wet AMD.

Despite the promise faricimab showed at longer treatment intervals, it is unclear whether that will be enough to turn it into a major product. One issue for Roche is faricimab will likely need to compete with Eylea and biosimilar copies. Mylan, Samsung Bioepis and Amgen are among the companies with copies of Eylea in development. Filings for FDA approval could start this year, led by Mylan.

https://www.fiercebiotech.com/biotech/roche-s-eylea-rival-poised-for-fda-filings-new-phase-3-data

SPAC to take digital health firm Sharecare public for about $4 bln

  Digital health startup Sharecare Inc said on Friday it has agreed to go public through a merger with a blank-check firm backed by veteran investment banker Alan Mnuchin, in a deal that values the combined company at $3.9 billion.

https://www.reuters.com/article/sharecare-ma-falcon-capital/alan-mnuchin-backed-spac-to-take-digital-health-firm-sharecare-public-for-about-4-bln-idUSL4N2KI1VK

Amicus misses goal in Pompe phase 3 but still pushes for approval

 A phase 3 clinical trial of Amicus Therapeutics’ late-onset Pompe disease prospect has missed its primary endpoint. Yet Amicus looked past the failure of AT-GAA to improve statistically on Sanofi’s market incumbent Lumizyme, zeroing in on details of the data to justify forging ahead with plans to seek approval of the candidate.

Amicus designed the clinical trial to compare AT-GAA—a combination of recombinant human acid alpha-glucosidase and an enzyme stabilizer—to Sanofi’s Lumizyme. The primary endpoint of the trial of 123 adult Pompe patients was the six-minute walk test.

At Week 52, subjects who received AT-GAA could walk 21 meters farther than at baseline, compared to a seven-meter improvement in the Lumizyme group. The numerical difference between the two arms fell short of statistical significance.

On a measure of lung function, Amicus tracked a 1% decline in the AT-GAA group and a 4% drop in the Lumizyme cohort. Amicus said the result was “nominally statistically significant” with a p-value of 0.023. 

The differences between the two cohorts were more pronounced in the subgroup of patients who had previously received enzyme replacement therapy. In that subgroup, patients on AT-GAA walked 17 meters farther than their peers on Lumizyme. The difference in lung function scores was bigger than in the broader population, too. Both subgroup analyses had p-values below 0.05.

As those results imply, the data on patients who had never received enzyme replacement therapy at baseline are unflattering to AT-GAA. Numerically, Lumizyme beat the experimental drug on the lung function and walking endpoints, although it is hard to draw concrete conclusions from the 27-subject subgroup. 

Investors were swift to slam the data, sending shares in Amicus down around 20%. The leadership team at Amicus is publicly much more upbeat, with CEO John Crowley talking up the prospect of the drug quickly becoming the new standard of care and outlining plans to seek regulatory approval in multiple markets. 

If Amicus persuades regulators to approve AT-GAA, it may need to contend with two Sanofi drugs to win share. Sanofi currently markets Lumizyme. Last year, Sanofi filed for approval of a new late-onset Pompe drug, avalglucosidase alfa, on both sides of the Atlantic. Sanofi has priority review in the U.S., teeing it up to win approval by May. 

The Sanofi filing is based on data from a phase 3 trial that read out last summer. Like Amicus, Sanofi compared its investigational drug to Lumizyme, and, like Amicus, Sanofi found its candidate was no better than the incumbent against the primary endpoint. The primary goal showed avalglucosidase alfa was statistically non-inferior to Lumizyme on a measure of lung function. 

https://www.fiercebiotech.com/biotech/amicus-misses-goal-pompe-phase-3-but-still-pushes-for-approval-showdown-sanofi