Ocugen and Its COVID Vaccine

The coronavirus vaccine race didn't include Ocugen (NASDAQ:OCGN) a few months ago. And yet, recently, this biotech company leapt past rivals. The company is in talks with the U.S. Food and Drug Administration (FDA) regarding an Emergency Use Authorization (EUA) pathway for its candidate. All of a sudden, it looks like Ocugen is just steps away from the finish line.

As a result, Ocugen shares have soared more than 350% since the start of the year, and the company's market capitalization has grown to $1.56 billion from about $350 million in that period. So, with the possibility of a new coronavirus vaccine on the market in the U.S. -- should you buy shares of Ocugen?

Sharing U.S. profits

Earlier this month, Ocugen finalized a deal to codevelop and commercialize Covaxin in the U.S. The developer of Covaxin, Bharat Biotech, is now selling the product in its home country of India under emergency authorization. As part of the agreement between the two companies, Ocugen will keep 45% of Covaxin profits in the U.S.; Bharat will pocket the rest.

Vaccines against infectious diseases aren't Ocugen's usual business. The company specializes in gene therapies for eye diseases that cause blindness. The company has three such candidates in the pipeline for eight indications. The four most advanced programs, which are meant to treat genetic-mutation-associated retinal degeneration, will head into phase 1 studies next.

The decision to partner with Bharat on Covaxin is good news because it brings a closer-to-market product into the fold. And Ocugen has taken steps to assure a successful launch. The company put together a vaccine scientific advisory board including six experts from industry and academia. Ocugen also raised $23 million in a direct offering of shares to institutional investors. We can imagine funds from this operation supporting the advancement of Covaxin in the U.S.

It's too early to say whether Covaxin is a strong vaccine candidate; we'll have to see phase 3 data first. Bharat expects to generate results by March. Ocugen points out a positive detail, though: Covaxin has invoked an immune response to various viral proteins -- not just the spike protein that the virus uses to infect host cells. That gives Covaxin a better chance of beating new viral strains. As mutations transform the spike protein, vaccines focused on the spike only may lose efficacy.

The chances of success

Of course, at this point, the biggest risk is a negative or lackluster data report. But let's imagine that Covaxin's data readout is incredibly positive. Then what will Ocugen's chances of success be with this vaccine candidate under its belt?

A recent move by the Biden administration will make it difficult for most market newcomers -- at least this year. The government bought enough doses of the Pfizer (NYSE:PFE) and Moderna (NASDAQ:MRNA) vaccines to fully immunize the entire U.S. population. That leaves little room for new players in that market. The biggest problem right now for Ocugen is its deal with Bharat allows it to eventually sell Covaxin only in the U.S. -- and Pfizer and Moderna have deals in place with the federal government which allow them to supply enough vaccines to inoculate the entire population.

Vaccine developers haven't clearly said how often people will need to be vaccinated against COVID, but Moderna has offered a few clues. It's already said that its vaccine is likely to protect for a year or two. We can imagine that boosters will be necessary. The good news is that Ocugen may have a chance at market share once the population needs to be vaccinated more than once or on a regular basis.

How to steal market share

But here's another point to keep in mind: For Ocugen to take market share from Moderna and Pfizer later on, it will have to offer an advantage. Otherwise, the government may continue to buy from its current suppliers. So Covaxin's performance against new strains will be crucial. Johnson & Johnson (NYSE:JNJ) and Novavax (NASDAQ:NVAX) have reported phase 3 data, and are seeking a share of the U.S. market as well. Covaxin must closely resemble (or beat) their candidates' performances, too.

All of this means it may be difficult for Ocugen to carve out a significant share of the U.S. market -- even down the road. And that means buying shares of Ocugen just for its coronavirus vaccine probably isn't a good idea.

But Covaxin could be positive for Ocugen in another way. If the vaccine candidate is able to gain even a small share of the U.S. market in the coming years, it would offer Ocugen a revenue source. And that revenue would help fund the rest of Ocugen's pipeline programs. If this scenario plays out, it may be worth giving this biotech a second look.

https://www.fool.com/investing/2021/02/26/should-you-buy-ocugen-for-its-covid-vaccine/

Fate Therapeutics started at Buy by B of A

 Target $115

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

Tricida appeal to FDA denied

 Tricida, Inc. (Nasdaq: TCDA), a pharmaceutical company focused on the development and commercialization of its investigational drug candidate, veverimer, a non-absorbed, orally-administered polymer designed to treat metabolic acidosis in patients with chronic kidney disease (CKD), today provided an update on its U.S. Food and Drug Administration (FDA) interactions.

Tricida has received an Appeal Denied Letter (ADL), from the Office of New Drugs (OND) of the FDA in response to its Formal Dispute Resolution Request (FDRR) submitted in December 2020. While the FDRR was focused on whether the magnitude and durability of serum bicarbonate change seen in the TRCA-301/TRCA-301E trial is reasonably likely to predict clinical benefit in the treatment of metabolic acidosis in patients with CKD, the OND’s decision additionally addressed other deficiencies identified in the Complete Response Letter (CRL), which Tricida received in August 2020. The additional issues addressed included the reliability of the data from the TRCA-301/TRCA-301E trial due to the disproportionate impact of data from a single high-enrolling clinical site on the trial’s results and the applicability of the trial results to the U.S. patient population given that the majority of the subjects in the study were enrolled in sites outside of the United States or were in regions that the FDA does not consider “U.S.-like,” such as Eastern Europe.

In the ADL, the OND acknowledged that the TRCA-301/TRCA-301E trial met its serum bicarbonate endpoints with statistical significance but concluded that the extent of serum bicarbonate increase observed in the TRCA-301/TRCA-301E trial is not reasonably likely to provide a discernible reduction in CKD progression. The OND also concluded that the confirmatory trial, VALOR-CKD, is underpowered to detect the effect size (13%) predicted by the original Tangri model (also known as the Predictive MA Model) based upon the placebo-subtracted mean treatment effect observed in the TRCA-301/TRCA-301E trial.

The OND also provided feedback on other concerns that are particularly relevant in an NDA supported by a single registrational trial. The OND noted concerns around the trial results being strongly influenced by a single site, and the majority of sites for the TRCA-301/TRCA-301E trial being in Eastern Europe, where differences in patient management, including concomitant medications and diet, might affect the treatment response to veverimer and raise a concern of the applicability to a U.S. patient population. The FDA did not raise any concerns related to its completed inspection of the highest-enrolling clinical trial site and there was no FDA Form 483 issued. Also, while the OND did not suggest that there was a specific unblinding issue in the TRCA-301/TRCA-301E trial, the OND noted concerns around adequate blinding and that, while the measures in place to protect the study blind in the TRCA-301/TRCA-301E trial were reasonable, they may not have optimally protected the blind.

Although the ADL provides greater clarity on the potential path for approval of veverimer through the Accelerated Approval Program, Tricida believes the timeline to meet the requirements for accelerated approval as suggested in the ADL may not result in the most rapid development path for veverimer. For example, the OND suggested that Tricida meet with the Division of Cardiology and Nephrology (the Division) to discuss submission of 52-week serum bicarbonate results from the fully randomized VALOR-CKD trial and that such submission should include a substantial proportion of U.S. and “U.S.-like” patients. The OND also indicated that, if the results of this trial were to demonstrate a meaningfully larger treatment effect on serum bicarbonate than seen in the TRCA-301/TRCA-301E trial, results from VALOR-CKD, along with the results from the TRCA-301/TRCA-301E trial, could address the deficiencies identified in the CRL. However, the OND noted that whether these data would support accelerated approval would remain a review issue and therefore would be subject to the Division’s assessment of the adequacy of the magnitude of increase in serum bicarbonate. Moreover, based on the concerns expressed, we believe that the FDA could require an additional trial or trials to confirm the magnitude, durability of effect or applicability to the U.S. population for resubmission of the veverimer NDA through the Accelerated Approval Program.

Given the feedback provided by the FDA in the ADL, Tricida intends to continue the VALOR-CKD trial without further modifications at the present time with consideration of both the accelerated and traditional approval pathways. Tricida’s planned interim analyses in the VALOR-CKD trial could result in early stopping for efficacy and resubmission of the NDA through a traditional approval pathway with a potential indication of treatment of metabolic acidosis to slow CKD progression. Tricida is also evaluating several options with respect to the VALOR-CKD trial that are focused on obtaining additional data prior to the end of 2022 on the effect of veverimer on (1) CKD progression; (2) physical functioning; and (3) serum bicarbonate. These options include the possibility of stopping the trial early for administrative reasons, which would allow analysis of the data using all alpha remaining at that time. In any event, Tricida believes data from VALOR-CKD will be very important in furthering the understanding of the regulatory path for approval of veverimer.

https://finance.yahoo.com/news/tricida-provides-fda-interactions-210500860.html

CDC says trend of decline in US COVID-19 cases may be stalling

 The head of the U.S. Centers for Disease Control and Prevention said on Friday that a recent decline in COVID-19 cases may be stalling, a development she described as concerning while urging that restrictions to fight the virus remain in place.

Dr. Rochelle Walensky told reporters the CDC was watching the concerning data closely.

The White House on Friday also urged companies to join efforts to help fight the pandemic by requiring mask wearing by employees and educating customers.

Andy Slavitt, a senior adviser on the White House’s COVID-19 response team, listed a number of companies that were taking measures to help with the pandemic fight and urged more to join.

Ford and the Gap were producing and donating millions of masks, he said, while Best Buy, Target and Dollar General were giving workers paid time off to get vaccines.

The White House is working on a broad campaign to educate Americans about the vaccine as it seeks to bring the pandemic that has killed more than 500,000 people in the United States under control.

President Joe Biden on Thursday noted concerns that later this spring supply of the vaccines would outstrip demand because of vaccine hesitancy.

https://www.reuters.com/article/us-health-coronavirus-whitehouse/u-s-cdc-says-trend-of-decline-in-covid-19-cases-may-be-stalling-idUSKBN2AQ2GG

Puma regains China rights to Nerlynx, amends deal with Fabre

 Puma Biotechnology (Nasdaq: PBYI) has agreed to terminate its 2018 license agreement with China-based CANbridge Pharmaceutical, in which the latter was granted exclusive rights to develop and commercialize Nerlynx (neratinib) in Greater China, and to settle their arbitration related to the license agreement.

Simultaneous, Puma – whose shares dipped 2.7% to $10.26 by mid-morning - has agreed with privately-held French drugmaker Pierre Fabre to amend the terms of their 2019 license agreement, which grants Pierre Fabre exclusive rights to develop and commercialize Nerlynx within Europe, Turkey, Middle East and Africa, to also include Greater China, which includes mainland China, Taiwan, Hong Kong and Macau.

At the same time, CANbridge and Pierre Fabre have entered into agreements under which CANbridge will provide Pierre Fabre certain transition services in Greater China and distribute and market Nerlynx for Pierre Fabre in Hong Kong, Macau, and Taiwan until year end 2022, with an option to renew.

Financial terms

Under the terms of the various agreements implementing this transaction among the three companies, Puma will receive an upfront payment of $50 million from Pierre Fabre in consideration for the amendment to their 2019 license agreement, and CANbridge will receive a one-time $20 million termination fee from Puma to return all rights to neratinib in Greater China back to Puma.

Finally, Puma has agreed to dismiss the arbitration demand it filed on July 28, 2020 against CANbridge related to the parties’ 2018 license agreement, and as part of the settlement, CANbridge has agreed to dismiss its counterclaims against Puma.

James Xue, founder, chairman and chief executive of CANbridge said: “We are pleased to have reached a mutually beneficial agreement that also serves patients, as CANbridge shifts its focus to rare disease and rare oncology. We look forward to working closely with Pierre Fabre to continue to bring this important medicine to patients in Hong Kong, Taiwan and Macau.”

Alan Auerbach, CEO president of Puma, said: “We are pleased to extend our collaboration with Pierre Fabre into the Greater China region. Pierre Fabre is well equipped with existing infrastructure to make Nerlynx a success in mainland China and Pierre Fabre plans to make Nerlynx available to breast cancer patients in mainland China in the second quarter of this year.”

Neratinib is approved in the USA for both the extended adjuvant treatment of adult patients with early stage HER2-positive breast cancer following adjuvant trastuzumab-based therapy and HER2-positive metastatic breast cancer.

https://www.thepharmaletter.com/article/puma-regains-china-rights-to-nerlynx-and-amends-deal-with-fabre

Editas cut to Hold from Buy by Truist

 Target $45

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

Moderna Shares Extend Gains as Cramer Talks About Potential Cancer Vaccine

 Moderna Inc.  (MRNA) - Get Report shares jumped higher Friday after Jim Cramer said the drugmaker is nearing the development of a vaccine that could protect against certain forms of cancer.

Cramer, speaking on CNBC's Squawk on the Street program, said the developments were "pre-clinical" but nonetheless remarkable and are based on the drugmaker's messenger-RNA delivery techniques, which it used to develop its mRNA-1273 coronavirus vaccine. 

"This is not an RNA messenger vaccine company for COVID," Cramer said. "This is a new way to be able to stop diseases and people I know that are working with this company know that they may have a vaccine against certain forms of cancer."

"That's how powerful RNA messenger is," he added "I've been reluctant to say that but I have it from more than one source. I have it from two sources, both of whom are involved ... and it's just incredible. We're taking about a Noble Prize. It works."

Moderna shares were marked 5.2% higher in early trading Friday and changing hands at $156.00 each. 

Alongside a similar messenger-RNA vaccine developed by Pfizer  (PFE) - Get Report and its German partner BioNTech  (BNTX) - Get Report, Moderna has taken the lead role in the government's accelerating rollout.

Late Thursday, Moderna said its mRNA-1273 coronavirus vaccine, which earned Emergency Use Authorization from the U.S. Food & Drug Administration in late December, should generate around $18.4 billion in sales this year and deliver the drugmaker's first annual profit since 2011.

CEO Stephane Bancel said Moderna plans to produce as many as 1 billion doses of mRNA-1273 this year, adding that it's working on boosters that would address the growing spread of coronavirus variants first identified in the United Kingdom and South Africa. 

https://www.thestreet.com/investing/moderna-extends-gains-as-jim-cramer-talks-of-cancer-vaccine