Insmed Incorporated (NASDAQ:INSM) shares are soaring by over 100% today, after the drug maker reported positive top-line results for its study into the treatment of a rare lung disease caused by bacterial infection. The disease, nontuberculous mycobacteria (NTM), scars and damages the lungs and can take years to clear.
The Phase 3 Convert study showed that incorporating Insmed’s inhaled antibiotic, ALIS, had a meaningful impact to guideline-based therapy (GBT). In particular, adding ALIS eliminated evidence of NTM lung disease caused by MAC in sputum by month 6 in 29% of patients, versus 9% of patients on GBT alone. Insmed will now seek accelerated approval for the drug, which has already been given breakthrough therapy designation by the US’s Food and Drug Administration (FDA).
Following the news, Leerink Swann analyst Joseph Schwartz reiterated his buy rating on the stock. Schwartz has a strong track record on INSM specifically with a 67% success rate and 45% average return per recommendation. He says that patients who culture converted showed a statistical significant improvement, a pre-specified analysis. (To watch Schwartz’s track record, click here)
“We consider these compelling top-line data to be a remarkable accomplishment in a rare disease state with no currently approved therapies,” commented INSM CEO Will Lewis. “We are particularly encouraged by the consistency of these data when compared with our Phase 2 study results, and look forward to additional data as the CONVERT study continues over the next two years.”
Overall the stock has a Strong Buy analyst consensus rating on TipRanks. In fact, in the last three months, INSM has received 6 back-to-back buy ratings from analysts. We can also see that the average analyst price target of $28.25 translates into a further 6.36% upside from the current share price.
BioCryst Pharmaceuticals, Inc. (NASDAQ:BCRX) announced encouraging results for its Phase 2 APeX-1 study assessing BCX7353 as a preventative treatment to reduce the frequency of attacks in patients with hereditary angioedema (HAE). The results showed that the treatment was very successful, reducing HAE attacks by as much as 90% versus the placebo.
Now BCRX will meet with U.S. and European regulators in the fourth quarter to finalize the next step which is the Phase 3 program. The company intends to start pivotal clinical trials including long-term safety studies, in the first quarter of next year.
Jon Stonehouse, CEO and president, commented: “We are delighted to see a robust treatment effect after completing the largest ever Phase 2 trial in HAE patients. We now have the information necessary to select doses for Phase 3. The 125 mg once-daily oral dose of BCX7353 provided a high level of efficacy and excellent tolerability. This product profile will be an extremely attractive treatment option for physicians and patients.”
TipRanks shows that the stock has a Moderate Buy analyst consensus rating. In the last three months, BCRX has received 2 buy and 1 hold rating from analysts. Their average price target of $8 now stands at a 62.6% upside from the current share price.
Cellect Biotechnology Ltd (NASDAQ:APOP) shares are up by 9.53% in Tuesday’s trading after soaring by 53% in the pre-market. The stock is currently trading at $10. The spike comes on the back of news that the FDA has granted Cellect’s ApoGraft technology orphan drug status. ApoGraft is designed to prevent transplant-associated diseases that occur when two immune systems crash into each other- a particular risk in bone marrow transplants.
The new orphan drug status will bring a slew of benefits to APOP under the FDA Orphan Drug Act. The Act provides incentives for companies to develop products for rare diseases affecting fewer than 200,000 people in the US. These can include tax credits for clinical trial expenses, an exemption from the FDA user fee, FDA assistance in clinical trial design and potential market exclusivity for seven years following approval.
Cellect’s CEO, Dr. Shai Yarkoni commented “We may provide the answer to a great unmet clinical need causing severe morbidity and death in thousands of patients worldwide. The Orphan Drug status gives us 7 years exclusivity following approval as well as other advantages for ApoGraft™ commercialization.”