After being in the doldrums for the majority of the year, public biopharmaceutical companies, it appears, have turned the corner and are now on a major upswing. Following a 10% increase in value in October, the BioWorld Biopharmaceutical index repeated the performance in November with the sector’s elite companies posting significant gains in their share prices. Once again, the sector outperformed the general market, with the Dow Jones Industrial average gaining 4% in the period and the Nasdaq Composite index posting a 4.5% gain. Year-to-date, the index is tracking up a respectable 11%. (See BioWorld Biopharmaceutical index.)
Following the repeat of strong stock performances, Cowen and Co. analysts, writing in their monthly Biotech Thermometer noted, "Those who stuck with the sector through the famine of Q3 have feasted on a cornucopia of returns during the first two months of Q4."
Gene therapy hot
Index member Cambridge, Mass.-based Sarepta Therapeutics Inc.’s push into gene therapy with its collaboration and license agreement to develop in vivo AAV-based therapies for up to eight central nervous system and neuromuscular targets with Stridebio Inc., of Research Triangle Park, N.C., catalyzed the value of the company’s shares (NASDAQ:SRPT), which closed the month up almost 36%.
Sarepta gains an exclusive license on selected targets to leverage Stridebio's structure-driven capsid technology, intended to enhance specific tropism to tissues of interest and evade neutralizing antibodies. The companies also plan to focus on strategies intended to address redosing challenges in patients who have received AAV-delivered gene therapy. Stridebio will conduct all IND-enabling research, development and manufacturing for the first four targets, which are MECP2 (Rett syndrome), SCN1A (Dravet syndrome), UBE3A (Angelman syndrome) and NPC1 (Niemann-Pick). Sarepta will have an exclusive option to four additional targets based on Stridebio's capsid technology. Stridebio will receive a $48 million up-front payment in the form of cash and Sarepta stock, in addition to significant future development, regulatory and commercial milestones for the four programs, along with royalties on worldwide net sales of any commercial products developed through the collaboration. Sarepta has also obtained an exclusive option to expand the collaboration to include up to an additional four targets, with an up-front payment of up to $42.5 million along with future downstream milestone payments, while Stridebio has an option to obtain co-development and co-commercial rights to one of the collaboration targets.
Investors have certainly gravitated to companies in the field, a move that will be reinforced by today’s news that Audentes Therapeutics Inc. is being acquired by Astellas Pharma Inc., which is paying $60 per share in cash for an equity value of about $3 billion.
Like many big pharma companies, Tokyo-based Astellas has been attracted by the prospects offered in gene therapy. The deal is expected to finish in the first quarter of next year. Audentes has a lead candidate, AT-132, for the treatment of X-linked myotubular myopathy for which a BLA submission is on track for next year.
Regeneron Pharmaceuticals Inc. posted a 20% gain in its shares (NASDAQ:REGN) as did Dublin-based Jazz Pharmaceuticals plc (NASDAQ:JAZZ). Last month, the latter company announced that the EMA’s Committee for Medicinal Products for Human Use adopted a positive opinion recommending the marketing authorization of solriamfetol, a dual-acting dopamine and norepinephrine reuptake inhibitor, to improve wakefulness and reduce excessive daytime sleepiness (EDS) in adults with narcolepsy or obstructive sleep apnea (OSA) whose EDS has not been satisfactorily treated by primary OSA therapy, such as continuous positive airway pressure.
Drug developers on a tear
In just one month, the BioWorld Drug Developers index jumped a whopping 26% to bring its year-to-date performance to 33%, its highest value since January. (See BioWorld Drug Developers index.)
Pushing the index higher was the dramatic resurgence of investor interest in Clovis Oncology Inc., whose shares (NASDAQ:CLVS) jumped an incredible 371% in November. The Boulder, Colo.-based company reported strong third-quarter revenue for PARP inhibitor Rubraca (rucaparib) totaling $37.6 million, up 65% from the third quarter of 2018 and up 14% over the second quarter of this year. Investors clearly took note of the company increasing its net product revenue guidance for the full year to a range between $141 million and $147 million. The company also plans to file a supplemental NDA seeking use of Rubraca in patients with BRCA1/2-mutant advanced prostate cancer before year-end.
Clovis also reported that the Italian Medicines Agency had approved rucaparib for reimbursement in Italy, and the drug will be available as an option for monotherapy maintenance treatment for adults with relapsed, platinum-sensitive high-grade epithelial ovarian, fallopian tube or primary peritoneal cancer that has responded to platinum-based chemotherapy.
A strong pipeline and a positive third-quarter business update was enough to vault the shares (NASDAQ:ARWR) of Arrowhead Pharmaceuticals Inc. 82%. Of note, the company reported it started dosing patients in an adaptive design phase II/III trial, called SEQUOIA, which has the potential to serve as a pivotal registrational study of ARO-AAT, its second-generation subcutaneously administered RNAi therapeutic being developed as a treatment for a rare genetic liver disease associated with alpha-1 antitrypsin deficiency
Arrowhead also reported its collaborator, Janssen Pharmaceuticals Inc., part of the Janssen Pharmaceutical Cos. of Johnson & Johnson, began dosing patients a phase IIb triple-combination study called REEF-1, designed to enroll up to 450 patients with chronic hepatitis B infection.
The Medicines Co.’s shares (NASDAQ:MDCO) closed the month up almost 61% fueled by the announcement that it will be acquired by Novartis AG for $9.7 billion.
The Basel, Switzerland-based pharma made an $85 per share offer for the company, which has taken inclisiran, an siRNA-based PCSK9 inhibitor, into late-stage development for reducing the risk of a cardiovascular event – heart attack or stroke – in high-risk patients with cardiovascular disease or high levels of low-density lipoprotein cholesterol who are inadequately controlled on current therapies.
The Medicines Co., which will continue to operate as a separate and independent company, plans to submit an FDA filing in the current quarter and a European filing will follow in the first quarter next year.