Stay at the forefront of the week’s champions and runners-up among publicly traded biotech companies and the reasons behind the ups or downs of their stock price fluctuations.

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Bluebird Bio (BLUE) shares flew back in the right direction at the start of this week, taking off with a 71% premarket surge that shrunk into a 14.5% increase (from $3.73 to $4.27) on Monday, the first trading day after the company announced two unanimous recommendations in its favor from an FDA advisory panel.

The FDA’s Cellular, Tissue, and Gene Therapies Advisory Committee (CTGTAC) earlier this month recommended approval by the agency of two Bluebird Bio lentiviral vector (LVV) gene therapy candidates:

  • Betibeglogene autotemcel (beti-cel) for the treatment of people with beta-thalassemia who require regular red blood cell (RBC) transfusions; and
  • Elivaldogene autotemcel (eli-cel) for the treatment of cerebral adrenoleukodystrophy (CALD) in patients less than 18 years of age who do not have an available and willing human leukocyte antigen (HLA)-matched sibling hematopoietic stem cell (HSC) donor.

The recommendations appeared to validate Bluebird’s faith in its LVV platform—a faith maintained by CEO Andrew Obenshain, Speaking with GEN Edge in November, he cited in part the ability of LVVs to carry larger genes than adeno-associated virus (AAV)-based gene therapies.

Mani Foroohar, MD, Senior Managing Director, Genetic Medicines, and a senior research analyst with SVB Securities wrote in research notes last week that both gene therapies represented relatively small market opportunities for Bluebird. SVB’s model projects $64 million in peak annual sales for beti-cel and $38 million for eli-cel. Foroohar maintained SVB’s “Market Perform” rating on Bluebird shares.

“Small but much needed win for BLUE,” Foroohar wrote of eli-cel’s approval on June 10, adding three days later that SVB projected a “high modeled probability of success for both products.”

The FDA has set target decision dates under the Prescription Drug User Fee Act (PDUFA) of August 19 for beti-cel, and September 16 for eli-cel.

“We are pleased that the advisory committee recognized the significant potential of LVV gene therapies to address severe unmet medical needs—demonstrated over more than a decade of research and more than 500 patient-years of experience—as well as the urgent need for improved treatments,” said Anne-Virginie Eggimann, chief regulatory officer, bluebird bio.

Bluebird’s shares rose 14% on June 13, from $3.73 to $4.27. But that momentum faded later in the week, however, as its price fell 16% to $3.58 on Tuesday and dipped another 3% to $3.48 on Wednesday.

Belite Bio (BLTE)

The bear market has dampened investor appetite for biotech stocks, yet some exceptions stand out. One of those is Belite Bio. The San Diego-based drug developer has seen its shares more than quadruple from the $6 price it set for its $36 million initial public offering of American Depositary Shares back in April.

Much of that surge came this past week, starting on Monday when Belite’s shares jumped 23%, from $18.51 to $22.75. Shares climbed another 24% to $28.20 before declining 2.5% on Wednesday to $27.50.

Belite appeared to win over investors after announcing June 9 that it submitted a Clinical Trial Application (CTA) to Belgium’s Federal Agency for Medicinal and Health Products (FAMHP) to launch a Phase III clinical trial of its lead pipeline candidate LBS-008 (Tinlarebant), designed to assess its effectiveness against Stargardt disease 1 (STGD1) in adolescents. Approximately 60 patients are targeted for enrollment in the trial, to be randomized 2:1 LBS-008 to placebo.

LBS-008 is designed to prevent the buildup of toxins in the eye that cause STGD1 and contribute to dry age-related macular degeneration (AMD). LBS-008 reduces and maintains levels of serum retinol binding protein 4 (RBP4), a carrier protein that transports retinol to the eye.

By modulating the amount of retinol entering the eye, Belite says, LBS-008 can reduce the formation of toxins which have been implicated in STGD1 and dry AMD in order to maintain the health of retinal tissues. LBS-008 has been granted Fast Track Designation by the FDA for the treatment of STGD1.

Investors initially responded to the Belgian Phase III announcement by sending Belite shares up 14% on June 10, from $16.29 to $18.51.

Clovis Oncology (CLVS)

Clovis share surged 47% this week after the company on Tuesday reported positive initial Phase I safety data for its targeted radiotherapy candidate FAP-2286 labeled with lutetium-177 (177Lu-FAP-2286).

At the Society of Nuclear Medicine & Molecular Imaging (SNMMI) Annual Meeting 2022, held June 11-14 in Vancouver, BC, Canada, researchers presented findings from the Clovis-sponsored Phase I/II LuMIERE trial (NCT04939610) , in which 177Lu-FAP-2286 showed a manageable safety profile and encouraging evidence of activity against advanced solid tumors in nine patients treated in the first two dose cohorts.

Among encouraging examples cited by Clovis was a confirmed RECIST partial response in one patient in the 3.7 GBq dose cohort with pseudomyxoma peritonei of appendiceal origin who completed six administrations of 177Lu-FAP-2286. A decrease in the level of the serum tumor marker carcinoembryonic antigen (CEA) was also seen in the patient over the course of 177Lu-FAP-2286 administration.

Researchers also presented updated data from an investigator-initiated Phase I study of FAP-2286 labelled with gallium-68 (68Ga-FAP-2286) as a novel imaging agent to identify metastatic cancer in patients with solid tumors. FAP-2286 is the first peptide-targeted radionuclide therapy (PTRT) and imaging agent targeting FAP to enter clinical development and is the lead candidate in Clovis’ targeted radionuclide therapy (TRT) development program.

From 66 cents a share on June 10, Clovis shares rose 6% to 70 cents on Monday, then climbed 16% on Tuesday to 81 cents, and jumped another 20% on Wednesday, closing at 97 cents.

Day One Biopharmaceuticals (DAWN)

Shares of Day One more than doubled in price Monday, rocketing 126% from $6.62 to $14.96. The surge occurred during the first trading day after Day One announced positive initial data from the Phase II FIREFLY-1 trial (NCT04775485), assessing the company’s lead pipeline candidate tovorafenib (DAY101) as a once-weekly monotherapy in patients aged 6 months to 25 years old with relapsed or progressive pediatric low-grade glioma (pLGG), the most common brain tumor diagnosed in children.

Day One reported an overall response rate (ORR) of 64% and a clinical benefit rate (CBR) of 91% in the first 22 evaluable patients treated with tovorafenib. The 22 included 14 partial responses (13 confirmed responses and one unconfirmed response), as well as six patients with stable disease. All six showed tumor shrinkage ranging between 19% and 43%. All 14 responding patients remain on tovorafenib and none have ended treatment due to treatment-related adverse events.

“These initial findings underscore the potential of tovorafenib monotherapy to become a significant and transformative new option for relapsed/progressive pLGG, a pediatric brain tumor with no approved treatments today,” said Samuel Blackman, MD, PhD, co-founder and chief medical officer of Day One.

The trial’s primary endpoint is ORR by Response Assessment for Neuro-Oncology (RANO) criteria as assessed by blinded independent central review. FIREFLY-1 is being conducted in collaboration with the Pacific Pediatric Neuro-Oncology Consortium (PNOC).

Day One said it plans to present additional interim trial results from FIREFLY-1 at an upcoming medical conference in the second half of this year, with full topline results expected in the first quarter of 2023. Should the data prove favorable, Day One plans to submit a new drug application (NDA) to the FDA in the first half of 2023.

Before then, Day One plans to launch the 400-patient global Phase III FIREFLY-2/LOGGIC trial, which will be conducted with the Low-Grade Glioma in Children (LOGGIC) consortium and a network of pediatric oncology centers. FIREFLY-2/LOGGIC will be designed to evaluate the efficacy and safety of once-weekly tovorafenib in patients with newly-diagnosed pLGG harboring a known activating BRAF alteration. Tovorafenib will be compared with three standard of care chemotherapy options.

Tovorafenib is an oral, brain-penetrant, highly-selective type II pan-RAF kinase inhibitor designed to target a key enzyme in the MAPK signaling pathway.

Day One shares climbed another 6% on Tuesday, to $15.83, before declining 4% on Wednesday, to $15.25.  after the company capitalized on its new momentum by launching a public offering of $125 million of shares of its common stock. The company also gave Underwriters a 30-day option to purchase up to an additional $18.75 million of shares at the public offering price, less underwriting discounts and commissions.

Day One derives its name from the “Day One” talk physicians have with their patients and their families about an initial cancer diagnosis and treatment plan.

Soleno Therapeutics (SLNO)

Shares of micro-cap Soleno surged 35%—albeit only from 17 to 23 cents—in early trading Tuesday as of 10:15 a.m., before closing the day at 18 cents, a mere 6% gain. The roller-coaster ride occurred a day after the company presented positive clinical data from its ongoing late-stage Diazoxide Choline Extended-Release (DCCR) development program for the treatment of Prader-Willi syndrome (PWS) at ENDO 2022, held June 11-14, 2022, in Atlanta.

Eric Felner, MD, of Emory University School of Medicine presented data from the Phase III C601 trial (NCT03440814) and its ongoing open-label extension C602 (NCT03714373) reported that 82 patients experienced improvements in body composition, as shown by statistically significant changes in lean body mass and the ratio of lean body mass to fat mass based on DEXA (DXA) scanning.

Accompanying these changes were reduced levels of leptin, fasting insulin, and an improvement in insulin sensitivity, likely related to a combination of reduced fat mass and improved leptin resistance. A statistically significant increase of the cardioprotective marker adiponectin was also seen. Similar effects were observed in the 40 obese participants enrolled in both studies, according to Soleno.

“These results further emphasize the potential for DCCR to be a meaningful treatment that improves multiple key symptoms of PWS, which has significant implications for patients and families struggling to manage this devastating disease,” Felner, principal investigator for C601 and C602, said in a statement.

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