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Often enough, some biotech stocks propel through expectations and invite investor frenzy. This week, it happened to Longeveron (Nasdaq: LGVN). Owing to the prospective treatment of hypoplastic left heart syndrome (HLHS) in the Phase 2 trial, the clinical-stage firm gained a 234% value boost over the week.

Once again, investors are reminded that despite the high risks inherent in the biotech sector due to complexities, these stocks can mimic the performance of memecoins. It also helps that plenty of biotech stocks are in the penny stock territory, making it easier to move their market caps.

The S&P Biotechnology Select Industry Index shows 5% year-to-date returns, underperforming the broader market benchmark S&P 500 by nearly 3x. But which biotech stocks could briefly and massively jump out of that underperformance like Longeveron?

Arvinas (Nasdaq: ARVN)

The Connecticut-based Arvinas is pushing the envelope with its proprietary PROteolysis TArgeting Chimeras (PROTAC) technology to pave the road to new treatment approaches. In the case of an HIV infection, the tech shows promise to go one step further by depleting the viral reservoir instead of just suppressing HIV with antiretroviral drugs.

Simply put, this pioneering platform degrades proteins linked to many serious and rare diseases. Arvinas uses its Protac Discovery Engine platform to develop a drug pipeline in oncology, immuno-oncology, and pro-inflammatory rare diseases.

Presently, Arvinas cleared Investigational New Drug (IND) applications ARV-393 for non-Hodgkin lymphoma and authorized ARV-102 for neurodegenerative diseases, such as Parkinson’s and progressive supranuclear palsy (PSP). Arvinas’ flagship drug for breast cancer, vepdegestrant (ARV-471) is being developed in partnership with Pfizer as the most likely to gain global commercialization.

Arvinas also showed promising work with ARV-766 for prostate cancer. The company’s protein degradation approach for previously undruggable diseases seems to yield results. 

ARVN stock is down 38% year-to-date, given that the company has yet to yield a profitable quarter. Presently priced at $26.45, ARVN shares are far above the 52-week low of $13.57 and below the 52-week average of $30.83 per share. 

Ocuphire Pharma (Nasdaq: OCUP)

As everyone knows, obesity is a massive problem across the developed world. One of the manifestations of obesity is diabetes. In turn, diabetics face great trouble with diabetic retinopathy (DR) and diabetic macular edema (DME). DR alone accounts for 9.6 million people in the US, with a prevalence above one quarter among diabetics, depending on age.

This gives Ocuphire Pharma a large addressable market. In the form of oral pill, the company’s APX3330 tackles DR, having completed End of Phase 2 (EOP2) and filed for FDA’s Special Protocol Assessment (SPA). Ocuphire is scheduled to start ZETA-2 Phase 2/3 trials for APX3330 in early 2025.

Ocuphire’s retina drug pipeline is in the exploratory stage. The company has APX2009 for geographic atrophy and APX2014 as the second-generation drug for the treatment of retinal diseases such as age-related macular degeneration. Ocuphire will start ZETA-2 Phase 2/3 trials for APX3330 in early 2025.

In other words, Ocuphire’s approach focuses on a specific domain via small-molecule oral inhibitors, but that domain has a large potential market. Year-to-date, OCUP stock is down 45%. At $1.68 per share, OCUP stock is nearly cut in half from its 52-week average of $2.90, which is well under the 52-week high of $4.58 per share.

Rocket Pharmaceuticals (Nasdaq: RCKT)

What AI represents for investors in the tech world, gene therapies represent in the pharma world. Rocket Pharmaceuticals uses a multi-platform approach based in New Jersey, integrating vivo lentiviral (LV) and in vivo adeno-associated viral (AAV) gene therapies.

The company, which is mainly focused on hematology (blood disorders) and cardiovascular (heart diseases), filed a Marketing Authorisation Application (MAA) for its LV RP-L102 to treat Fanconi anemia. On June 30th, the FDA is scheduled to respond to the company’s application for LV KRESLADI for the treatment of severe Leukocyte Adhesion deficiency-I (LAD-I).

RCKT is likely to visit its yearly high if the response is positive. In the meantime, RCKT stock is down 25% year-to-date. At the present price of $22.86, RCKT shares are aligned with their 52-week average, still well under the 52-week high of $32.52 per share.  

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