BridgeBio: PDUFA Date Expected In November 2024 Could Shift Momentum (NASDAQ:BBIO)

The last time I spoke about BridgeBio Pharma, Inc. (NASDAQ:BBIO), it was in a Seeking Alpha article entitled “BridgeBio Pharma: FDA Submission Acromidis and Top-Line Results On Deck.” The main thing to note about this article is that I mentioned that the New Drug Application [NDA] of this drug for the treatment of patients with transthyretin amyloid cardiomyopathy [ATTR-CM] was submitted to the FDA. However, at the time, there was to be a review period whereby the agency would decide whether to accept the submission of this application.

I’m happy to state that not only did the FDA accept the NDA submission of Acoramidis for the treatment of this patient population, but has set a PDUFA date of November 29th, 2024. Even better than that, the Marketing Authorization Application [MAA] of Acoramidis for this patient population was also accepted as well, setting up for success in the European territory. Speaking of which, another important event that happened since the last time I spoke about this is that it linked a license deal with Bayer (OTCPK:BAYRY). This big pharma would have licensing rights to sell this drug for this patient population in Europe.

This brought in a lot of cash, along with the fact that BridgeBio now has a huge pharma partner to commercialize the drug in that respective territory. As far as the use of BBP-631 for the treatment of patients with congenital adrenal hyperplasia [CAH] goes, there is a go/no go update planned in August 2024 for it. That is, a program update will be provided at that time, stating whether it intends to continue development with this specific program.

Acoramidis Review Date Set With Potential To Target Large ATTR-CM Market

As I stated above, things are looking very good for BridgeBio because the FDA accepted its NDA of Acoramidis for the treatment of patients with ATTR-CM. Having said that, the PDUFA date established whereby the FDA will review whether this drug should be approved for the treatment of this patient population has been set for November 29th of 2024. What’s even better regarding this established review date is that at the time of acceptance, it was announced that the FDA would not hold an advisory committee to discuss it.

What’s even better is that there might be an opportunity to have this drug approved for the European territory as well. That’s because the European Medicines Agency [EMA] accepted the Marketing Authorization Application [MAA] of Acoramidis for this patient population as well. Both of these applications acceptances for review of this drug shape up a good future for this company. There is even the possibility of expanding this drug to other territories by way of other regulatory submissions which are planned in the upcoming years.

In terms of being able to commercialize Acoramidis for ATTR-CM, BridgeBio is not going it alone. Instead, it has chosen to license European rights to a partner by the name of Bayer. This big pharma will be responsible for marketing this drug in this territory. I believe that this is a good deal for BridgeBio and that is because it accomplishes a few things. The first item being that it brought in a large sum of cash. Matter of fact, it received a $300 million upfront and near-term milestones payment because of it. From there, it is eligible to receive additional undisclosed sales-based milestone payments and tiered royalties in the low thirties-percent. The second item is that it allows Bayer to be responsible for marketing this drug in the European territories. This allows BridgeBio the ability to focus on U.S. commercialization and other markets.

In order for the company to be in a position to file for regulatory applications of Acoramidis, it had to achieve the primary endpoint of the phase 3 ATTRibute-CM study. This late-stage study recruited up to 632 patients with ATTR-CM. Patients were randomized to receive either Acoramidis or placebo twice daily. The primary endpoint of this study was All-Cause mortality, cumulative frequency of CV-related Hospitalization, change from Baseline in NT-proBNP and change from baseline in 6-minute walk test [6MWT]. This primary endpoint was met with statistical significance with a Win Ratio of 1.8 with a p-value of p

The key question is that will this be enough to counter other competitors on the market. For instance, Pfizer (PFE) has its approved drug Vyndamax [Tafamidis], which has been approved to treat these patients with ATTR-CM. The thing is that it is not clear whether cardiologists are going to switch patients from entrenched Vyndamax over to Acoramidis. However, such a switch can happen if BridgeBio can continue to highlight key differences like I stated above, which is greater separation of ACM and CVH at month 3. Not only that, but where it might be able to compete with Pfizer is if it can achieve a lower price for Acoramidis.

A disadvantage for BridgeBio is that this treatment must be taken orally twice-daily, whereas Vyndamax only needs to be taken once-daily. Another competitor would be by the name of Alnylam Pharmaceuticals (ALNY) with its drug Amvuttra [vutrisiran], which has already received FDA approval of this drug for the treatment of patients with transthyretin [ATTR] amyloidosis. However, it is in the process of advancing this drug for the treatment of patients with ATTR-CM as well.

Speaking of which, just a few months ago on June 24th of 2024, it announced positive topline results from its phase 3 HELIIOS-B study using this drug to treat this patient population. Where this company might have an advantage is that its drug is given as a subcutaneous injection once every three months. This will be a highly competitive area, but if the company can price its drug lower compared to the others, plus show an efficacy advantage over these other drugs, then it will still be poised to obtain market share. The transthyretin amyloidosis treatment market size is expected to reach $11.2 billion by 2032. This market is continuing to grow and currently, Pfizer’s Vyndamax is dominating in it. However, I believe that if some competitive advantages could be established with Acoramidis, then the company should be in a position to have some market share in this space. It believes that, as an estimate, it might be in a position to obtain between 25% to 40% market share for this ATTR-CM market.

Financials

According to the 10-Q SEC Filing, BridgeBio Pharma had cash, cash equivalents, marketable securities and short-term restricted cash of $587.2 million as of June 30th of 2024. The reason for the cash on hand is because of several financial transactions it had made to obtain all of these additional funds. Such financial transactions established to obtain funding were as follows:

• Net proceeds from term loan under the credit facility with Blue Owl of $434 million
• Net proceeds from various equity offerings of financings of $314.8 million
• Proceeds from the sale of investments in equity of securities of $63.2 million
• Special cash dividends received from investments in equity securities of $25.7 million.

The truth is that this biotech is likely to keep enacting financial transactions, which are likely to affect the stock price in some way. Why do I state that? That’s because in its 10-Q SEC filing, it states that it believes it has a cash runway, or enough cash on hand for at least, the next 12 months.

Risks To Business

There are several risks that investors should be aware of before investing in BridgeBio Pharma. The first risk to consider would be regarding the development of Acoramidis for the treatment of patients with ATTR-CM. The PDUFA date for this drug for the treatment of this patient population has been set for November 29th of 2024. The risk here is that there is no assurance that the FDA will approve this drug for marketing in the United States. Plus, for now, there is no advisory committee planned to review this drug. However, while not guaranteed to happen, it is possible that one could be convened later on. Lastly, rejection of the drug for approval by the FDA would mean a loss of an expected $500 million milestone payment. This would really be a considerable loss in terms of financing.

The second risk to consider would be with the MAA filing of Acoramidis for ATTR-CM to the European Medicines Agency. There is no assurance that this drug will be approved for marketing in the European territories. If it is approved, the expected commercial launch of the drug would happen in 2025. Even if this drug is marketed in Europe, there is no guarantee that sales of the drug will go well.

The third risk to consider would be in terms of competing drugs in the ATTR-CM market space. As I have stated above, Pfizer is the lead contender with Vyndamax for these patients. In order for BridgeBio to compete against it and other companies to obtain market share, it is going to have to find competitive advantages. One key area where it might be able to establish such an advantage would be in terms of lower pricing. If it can price its drug lower compared to all others, then it will definitely be in a good position to obtain market share. The risk is that there is no assurance that it will be able to price its drug lower compared to Vyndamax or other competing ATTR-CM treatments.

The fourth and final risk to consider would be regarding the development of BBP-631 for the treatment of patients with congenital adrenal hyperplasia or CAH. There is a program update for this candidate planned at some point during August 2024. Such a program update will dictate whether it has plans to move forward with this candidate or axe it from the pipeline. The risk here is that the removal of this program would lead to a loss of a significant program. One of which, is a considerable market opportunity.

Conclusion

I believe it was important to update the prospects of Bridge Bio. This is especially because it not only had its NDA regulatory submission of Acoramidies for ATTR-CM accepted by the FDA, but the fact that a PDUFA date of November 29th of 2024 had been established for it.

The global transthyretin amyloidosis treatment market is a multibillion-dollar market opportunity. If this company can somehow obtain regulatory approval for it in the United States and several other territories, then it might be in a position to capture a good chunk of it. Despite competition, the conservative estimate is that it could get between 25% to 40% of ATTR-CM market share. Even on the low end of the spectrum of obtaining only 25% market share would still be a win in my eyes.

Editor’s Note: This article discusses one or more securities that do not trade on a major U.S. exchange. Please be aware of the risks associated with these stocks.